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Gene therapy is making a world where cancer and AIDS can be cured, and a person can alter their genetic makeup, changing the direction of their own and their offsprings evolution in the process. However, the effects of gene therapy are long-lasting, and may impact both your and the health of your future children.

What Is Gene Therapy?

Utilizing a gene or genes to treat or cure a disease or medical condition is known as gene therapy. Gene therapy frequently involves replacing a damaged gene in a patients cells with a healthy copy or inserting new copies of a damaged gene.

Instead of drugs or surgery, gene therapy procedures allow doctors to treat a problem by changing a persons genetic composition. The first gene therapy technique, also known as gene transfer or gene addition, was created to:

Add a new gene to cells to aid in treating a disease

Introduce a healthy gene copy to replace the disease-causing altered copy

Later research helped to advance gene therapy methods. A more recent method, genome editing, takes a different tack in addressing genetic discrepancies. Genome editing uses molecular tools to alter the DNA already present in cells rather than introducing new genetic material. Research on genome editing aims to:

Restore a genes normal function

Correct the genetic change that underlies an illness

Activate a gene that isnt working correctly

Eliminate a section of DNA that interferes with gene activity and causes illness

However, gene therapy is still a field that primarily exists in research labs, and its application is still being tested. The majority of trials take place in Europe, US, and Australia. The treatment is extensive and is being tested to be used to treat acquired genetic diseases like cancer and some viral infections like AIDS, as well as diseases brought on by recessive gene defects, including cystic fibrosis, hemophilia, muscular dystrophy, and sickle cell anemia.

Types of Gene Therapy

Gene therapy comes in various forms, including:

Gene editing: Gene editing aims to remove undesirable genes or fix mutated genes

Cellular gene therapy: The patients cells are taken out, genetically altered (typically via a viral vector), and then put back in

Plasmid DNA: It is possible to genetically modify circular DNA molecules to deliver healing genes into human cells

Viral vectors: Certain gene therapy items are made from viruses because they naturally possess the potential to introduce genetic material into cells. These altered viruses can be employed as vectors to transport therapeutic genes into cells once viruses have been altered to reduce their capacity to spread infectious diseases

Ethical Issues in Gene Therapy

The idea of genetically altering genes has long been the subject of contentious debate in the scientific community. When new techniques are developed, bioethics is always present to evaluate the procedures hazards and moral ramifications. Genetic therapy in somatic cells is widely accepted in the scientific community, particularly in severe diseases like cystic fibrosis and Duchenne muscular dystrophy.

For instance, the first experiment for modifying healthy human embryos was allowed in the United Kingdom. On the other hand, American research organizations remained conservative, restating their stance that they do not support this kind of trial and saying that they must wait for advancements in both the methods and the definitions of ethical issues.

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What to Know About Gene Therapy - Biotech - HIT Consultant

New York, July 15, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and Key Geographical Regions : Industry Trends and Global Forecasts, 2022-2035" - https://www.reportlinker.com/p06292885/?utm_source=GNW To provide more context, the treatment regimen of such therapies, encompassing gene replacement and gene-editing modalities, is aimed at correction of the mutated gene in patients using molecular carriers (viral and non-viral vectors). Further, post the onset of the COVID-19 pandemic, there has been a steady increase in the investigational new drug (IND) applications filed for cell and gene therapies. In fact, in 2021, more than 200 gene therapies were being evaluated in phase II and III studies. Moreover, in 2022, six gene therapies are expected to receive the USFDA market approval. Promising results from ongoing clinical research initiatives have encouraged government and private firms to make investments to support therapy product development initiatives in this domain. In 2021 alone, gene therapy developers raised around USD 9.5 billion in capital investments. Taking into consideration the continuous progress in this domain, gene therapies are anticipated to be used for the treatment of 1.1 million patients suffering from a myriad of disease indications, by 2035.

Presently, more than 250 companies are engaged in the development of various early and late-stage gene therapies, worldwide. In recent years, there has been a significant increase in the integration of novel technologies, such as gene modification, gene-editing, genome sequencing and manipulation technologies (molecular switches), in conjugation with gene delivery methods. For instance, the CRISPR-Cas9 based gene-editing tool is one of the remarkable technological advancements, which enables the precise alteration of the transgene. It is worth mentioning that the new generation delivery platforms, including nanoparticles and hybrid vector systems, have been demonstrated to be capable of enabling effective and safe delivery of gene based therapeutics. Further, a variety of consolidation efforts are currently ongoing in this industry. Such initiatives are primarily focused on expanding and strengthening the existing development efforts; this can be validated from the fact that 56% of the total acquisitions reported in the domain were focused on drug class consolidation. Driven by the collective and consistent efforts of developers and the growing demand for a single dose of effective therapeutic, the gene therapy market is anticipated to witness significant growth in the foreseen future.

SCOPE OF THE REPORTThe Gene Therapy Market (5th Edition) by Type of Therapy (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Delivery Method Used (Ex vivo and In vivo), Type of Vector Used (Adeno-associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Non-Viral Vectors, Retrovirus and Others), Target Therapeutic Areas (Cardiovascular Diseases, Dermatological Diseases, Genetic Diseases, Hematological Diseases, Infectious Diseases, Metabolic Diseases, Muscle-related Diseases, Oncological Diseases, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebral, Intracoronary, Intradermal, Intralesional, Intramuscular, Intrapleural, Intrathecal, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal, Topical and Others), and Key Geographical Regions (US, Europe, Asia-Pacific and rest of the world): Industry Trends and Global Forecasts, 2022-2035 report features an extensive study of the current market landscape and the likely future potential associated with the gene therapy market, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies.

Amongst other elements, the report features:A detailed overview of the overall market landscape of gene therapies, including information on their phase of development (marketed, clinical, preclinical and discovery), key therapeutic areas (autoimmune diseases, cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, hepatic diseases, immunological diseases, infectious diseases, inflammatory diseases, metabolic diseases, muscle-related diseases, neurological diseases, oncological diseases, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene / molecule targeted, type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), type of gene delivery method used (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any).A detailed overview of the current market landscape of players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain.An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors.A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies.An elaborate discussion on the various commercialization strategies that have been adopted by drug developers engaged in this domain across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing stage.Detailed profiles of marketed and late stage (phase II / III and above) gene therapies, along with information on the development timeline of the therapy, current development status, mechanism of action, affiliated technology, patent portfolio strength, dosage and manufacturing details, as well as details related to the developer company.A review of the various emerging technologies and therapy development platforms that are being used to manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates.An in-depth analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2017, based on several relevant parameters, such as type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation.A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2022, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product, target therapeutic area and deal multiples.An analysis of the investments made at various stages, such as seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings, by companies that are engaged in this domain.An analysis of completed, ongoing and planned clinical studies, based on several relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies.An analysis of the startup companies engaged in this domain (established between 2017-2022) based on year of experience.A detailed review of the various gene therapy-based initiatives undertaken by big pharma players, highlighting trend across parameters, such as number of gene therapies under development, funding information, partnership activity and patent portfolio strength.An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors.A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, over the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2022-2035. Our year-wise projections of the current and future opportunity have further been segmented on the basis of [A] type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), [B] type of gene delivery method used (ex vivo and in vivo), [C] type of vector used (adeno-associated virus, adenovirus, herpes simplex virus, lentivirus, non-viral vectors, retrovirus and others), [D] target therapeutic areas (cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, infectious diseases, metabolic diseases, muscle-related diseases, oncological diseases, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebral, intracoronary, intradermal, intralesional, intramuscular, intrapleural, intrathecal, intratumoral, intravenous, intravesical, intravitreal, subretinal, topical and others), and [F] key geographical regions (US, Europe, Asia-Pacific and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industrys growth.

The opinions and insights presented in this study were Influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals:Buel Dan Rodgers (Founder and CEO, AAVogen)Sue Washer (President and CEO, AGTC)Patricia Zilliox (President and CEO, Eyevensys)Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics))Adam Rogers (CEO, Hemera Biosciences)Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela))Al Hawkins (CEO, Milo Biotechnology)Jean-Phillipe Combal (CEO, Vivet Therapeutics)Robert Jan Lamers (former CEO, Arthrogen)Tom Wilton (former CBO, LogicBio Therapeutics)Michael Triplett (former CEO, Myonexus Therapeutics)Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics)Cedric Szpirer (Executive and Scientific Director, Delphi Genetics)Marco Schmeer (Project Manager) and Tatjana Buchholz (former Marketing Manager, PlasmidFactory)Jeffrey Hung (CCO, Vigene Biosciences)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGYThe data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

Th secondary sources of information include:Annual reportsInvestor presentationsSEC filingsIndustry databasesNews releases from company websitesGovernment policy documentsIndustry analysts views

While the focus has been on forecasting the market till 2035, the report also provides our independent view on various emerging trends in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market, gathered from various secondary and primary sources of information.

KEY QUESTIONS ANSWEREDWho are the key industry players engaged in the development of gene therapies?How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products?Which types of vectors are most commonly used for effective delivery of gene therapies?What are the key regulatory requirements for gene therapy approval, across various geographies?Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development?What are the different pricing models and reimbursement strategies currently being adopted for gene therapies?What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies?Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development?What are the key value drivers of the merger and acquisition activity in the gene therapy industry?Who are the key stakeholders that have actively made investments in the gene therapy domain?Which are the most active trial sites (in terms of number of clinical studies being conducted) in this domain?How is the current and future market opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES

Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and ex vivo and in vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the various advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.

Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

Chapter 6 includes information on over 1150 gene therapies that are currently approved or are in different stages of development. It features a detailed analysis of the therapies, based on several relevant parameters, such as key therapeutic areas (autoimmune diseases, cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, hepatic diseases, immunological diseases, infectious diseases, inflammatory diseases, metabolic diseases, muscle-related diseases, neurological diseases, oncological diseases, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene / molecule targeted, type of gene delivery method used (ex vivo and in vivo), type of therapy (gene augmentation, oncolytic viral therapy, immunotherapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and type of therapy.

Chapter 7 provides a detailed overview of the current market landscape of players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe, Asia-Pacific, and Middle East and North Africa region on the basis of company size.

Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the companys patent portfolio and collaborations focused on its gene therapy product / technology.

Chapter 9 features an elaborate discussion on the various strategies that have been adopted by therapy developers engaged in this domain across key commercialization stages, including prior to drug launch, during drug launch and post-launch stage. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2022.

Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II / III and above). Each drug profile provides information on the development timeline of the therapy, current developmental status, route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, affiliated technology, patent portfolio strength, clinical trials and collaborations (if any).

Chapter 11 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile features details on the various pipeline candidates that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic diseases, metabolic diseases, neurological diseases, oncological diseases and ophthalmic diseases.

Chapter 13 provides an overview of the various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2017, based on several relevant parameters, such as type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed / granted). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation.

Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2022, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product, target therapeutic area and deal multiples.

Chapter 15 presents details on various funding instances, investments and grants reported within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and secondary offerings) received by the companies between 2015 and 2022, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 16 presents an analysis of completed, ongoing and planned clinical studies, based on several relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.

Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.

Chapter 18 presents a detailed analysis of the start-up companies engaged in the field of gene therapy, established between 2017-2022, based on year of experience.

Chapter 19 provides a detailed review of the various gene therapy-based initiatives undertaken by big pharma players, highlighting trend across parameters, such as number of gene therapies under development, funding information, partnership activity and patent portfolio strength. In addition, it also a detailed analysis of the big pharma players based on several parameters, such as therapeutic area, type of vector used, type of therapy and type of gene delivery method used.

Chapter 20 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.

Chapter 21 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2035. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II / III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] type of therapy (gene augmentation, immunotherapy, oncolytic viral therapy and others), [B] type of gene delivery method used (ex vivo and in vivo), [C] type of vector used (adeno-associated virus, adenovirus, herpes simplex virus, lentivirus, non-viral vectors, retrovirus and others), [D] target therapeutic area (cardiovascular diseases, dermatological diseases, genetic diseases, hematological diseases, infectious diseases, metabolic diseases, muscle-related diseases, oncological diseases, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebral, intracoronary, intradermal, intralesional, intramuscular, intrapleural, intrathecal, intratumoral, intravenous, intravesical, intravitreal, subretinal, topical and others), and [F] key geographical regions (US, Europe, Asia-Pacific and rest of the world).

Chapter 22 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 23 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain.

Chapter 24 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.

Chapter 25 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Buel Dan Rodgers (Founder and CEO, AAVogen), Sue Washer (President and CEO, AGTC), Patricia Zilliox (President and CEO, Eyevensys), Christopher Reinhard (CEO and Chairman, Gene Biotherapeutics (previously known as Cardium Therapeutics)), Adam Rogers (CEO, Hemera Biosciences), Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela)), Al Hawkins (CEO, Milo Biotechnology), Jean-Phillipe Combal (CEO, Vivet Therapeutics), Robert Jan Lamers (former CEO, Arthrogen), Tom Wilton (former CBO, LogicBio Therapeutics), Michael Triplett (former CEO, Myonexus Therapeutics), Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics), Cedric Szpirer (Executive and Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) and Tatjana Buchholz (former Marketing Manager, PlasmidFactory), and Jeffrey Hung (CCO, Vigene Biosciences). In addition, a brief profile of each company has been provided.

Chapter 26 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 27 is an appendix, which contains a list of companies and organizations mentioned in this report.Read the full report: https://www.reportlinker.com/p06292885/?utm_source=GNW

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Original post:
Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and...

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Link:
Systemic gene therapy with thymosin 4 alleviates glomerular injury in mice | Scientific Reports - Nature.com

Recently, a patient centricity concept has spread across all corners of the healthcare community with a straightforward goal: to improve and save patients lives through innovative and effective drug therapies adapted to patients' individual needs. However, for this concept to truly come to life, it is necessary to count on temperature-controlled supply chain logisticslike CRYOPDP to turn what could be a complex operation into a highly efficient process. This is extremely vital, because a patients life could literally be on the line with every shipment.Patient centricity in the clinical trial community involves meeting the patients where they are.The clinical trial industry is globalising and demands international healthcare logistics partners that can serve its growing and continuously evolving needs. For decades, temperature-controlled logistics experts have operated behind the scenes with patients barely aware of the hard work that went into their medicines journey. Thanks to the Covid-19 vaccine distributions impressive performance and its universal effect on people's health across the globe, it has become apparent to the world just how critical and important a logistics providers mission is for both patients and the industry.The endless pandemic lockdowns meant that patients could not travel to hospital sites for clinical trials due to travel and access restrictions. This led to a seismic shift where sponsors quickly moved to a direct-to-patient model to try to continue trials and keep patient treatments on schedule. As part of our response to this unique situation, CRYOPDP as a specialist in temperature-controlled logistics, with the mission to improve people's healthcare options, had to go further and dive deeper to better understand customers perceptions of the impact of the pandemic and work with them to provide those options.With the patient always at the centre of its business and considering the huge development of decentralised clinical trials, the development and implementation of a new service such as Direct-to-Patientseems to be the most natural evolution for CRYOPDP. We have been offering this turnkey solution completely adapted to patients needs, with the same efficiency and total peace of mind, to the benefit of many customers.This model makes life a lot easier for patients and their families, as they dont have to make multiple trips to a clinic or hospital that is potentially a great distance from their home. It is also beneficial for patients who may be too ill to travel, as well as saves patients time and money.Because of these benefits, the direct-to-patient model has increased clinical trial recruitment by up to 60 percent and helped to maintain patient retention by over 95 percent. Drug developers can also gain access to a larger patient population by onboarding those who are not located near participating hospitals or clinics.Patient centricity becomes more evident with the new generation of cell and gene therapies.From day one, CRYOPDP temperature-controlled logistics solutions have always been essential to improve and save patients lives. But, when speaking about cell and gene therapies in particular, this gains an even more significant meaning.The increase in personalised medicine, advanced therapies, and improved access to healthcare in the developing world are influencing future supply chain solutions development. Cell and gene therapies are enabling the healthcare community to shift the arrow and think about patients in a whole new way.Cell and gene therapies demand rigorous and precise temperature control to ensure that the therapies maintain their viability. And maintaining temperature control calls for flawless implementation and execution.From designing the best transportation route to selecting the correct packaging, every detail is critical to keep product integrity high under all conditions, and for this to happen, the healthcare community can count on CRYOPDP specialists who can meticulously handle the entire supply chain process. We've been supporting the life sciences and healthcare communities and focused on improving patient centricity with innovative temperature-controlled logistics solutions because thinking about the patient and the outcome of our work is what moves and inspires us to be better every day.

In all the geographies of the world that we cover, around 150 countries, we produce an operational performance of 99.96%. And to deliver such operational performance, we count on our employees, the specialists around the globe, to follow our quality standards and protocols in detail, so we can deliver a quality service.

When dealing with patients lives, there is no room for errors. Its all about quality of service making sure that lifesaving samples are distributed on time, within the correct specifications and at the right temperature to ensure the patient's health is never compromised.This commitment has helped CRYOPDP to win numerous industry awards, including Best Clinical Trial Logistics Provider in APAC at the Bioprocessing Excellence Awards 2021 and Most Advanced Healthcare Solution Providers from Europe 2021 by Healthcare Insights Magazine.Our achievements are being recognised across the industry, and as we continue to improve our services for healthcare communities, it will be the end patient that benefits the most.

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The Importance of Patient Centricity in Clinical Research and Cell / Gene Therapy Development - Contract Pharma

Purespring Therapeutics

Purespring Therapeutics and SwanBio Therapeutics announce licensing agreement for use of FunSel screening platform

FunSel is Puresprings proprietary in-vivo gene search engine, which allows functional selection of therapeutic factors unconstrained by previous drug discovery bias

SwanBio will use FunSel to advance AAV-based gene therapies for use in neurological diseases

London, UK & Philadelphia, USA 18 July 2022Purespring Therapeutics, a pioneering gene therapy company focused on transforming the treatment of kidney diseases, has entered into a collaborative licensing agreement with SwanBio Therapeutics, a gene therapy company advancing adeno-associated virus (AAV)-based therapies for inherited neurological conditions, for the use of FunSel, Puresprings proprietary gene therapy search engine.

FunSel enables a completely new way of discovering effective biological drugs, removing the narrow, target-focused bias built into traditional drug discovery. FunSel allows functional selection of the most effective factor for disease phenotype correction, starting from a library of thousands of AAV vectors that are selected directly in vivo. All factors in the FunSel library are secreted outside the cells, maximising therapeutic benefit. FunSel is gene and mechanism of action agnostic and is based on the physiology of the organ and disease in question.

Under the terms of the agreement, SwanBio will license the FunSel screening platform from Purespring to explore new targets for early-stage pipeline programs. SwanBio will have an option to develop any targets identified and to own and commercialise any molecules developed. Purespring will be entitled to milestones and royalties upon successful commercialisation. Additional financial terms are not being disclosed.

The FunSel platform has already been instrumental in the creation of Puresprings sister company Forcefield Therapeutics, a pioneer of best-in-class therapeutics to protect heart function, where FunSel was used to discover naturally occurring proteins that retain heart function and help existing cardiomyocytes protect themselves after myocardial infarction.

Story continues

Richard Francis, Chief Executive Officer of Purespring, commented: FunSel brings unique and pioneering screening capabilities and can help deliver the promise of gene therapy to non-monogenic disorders, allowing far broader patient populations to be treated than is the case with most gene therapies. This is the second example of how this gene search engine is helping to underpin a companys development strategy and demonstrates our ability to maximise the value of this platform by sharing this unique capability. SwanBio has a clear mission to develop AAV therapies for neurological disorders and its great to be able to support them and potentially others exploring new targets.

Tom Anderson, Chief Executive Officer of SwanBio, added: FunSel has the potential to enable acceleration and greater precision of candidate selection, and SwanBios application of FunSel has the potential to expand and confirm our early pipeline candidates. SwanBio is committed to advancing AAV-based therapies for the most debilitating neurological disorders and this technology promises to significantly support our ability to refine and further develop potential candidates for development.

- ENDS -

For further information, contact:

Purespring:

Richard Francis, CEOcontact@purespringtx.com+44 (0)20 3855 6324LinkedIn

Consilium Strategic Communications:

Amber Fennell, Jessica Hodgson, Genevieve Wilsonpurespring@consilium-comms.com

SwanBio Therapeutics:

Lara Furstlfurst@swanbiotx.com+1 703 946 0183

Notes to Editors

About Purespring

Purespring is the first company to treat kidney diseases by directly targeting the podocyte, a specialised kidney cell implicated in many kidney diseases, through AAV gene therapy.

Headed by former Sandoz CEO, Richard Francis, Purespring was founded on the work of Professor Moin Saleem, Professor of Paediatric Renal Medicine at the University of Bristol, where he heads a world leading group researching glomerular diseases. Purespring seeks to advance gene therapies for the treatment of both monogenic and non-monogenic chronic renal diseases that are currently poorly addressed with existing treatments.

The company also has a proprietary in-vivo pipeline engine, FunSel, which is a library of all biological factors that could be candidates for gene therapy, combined with a screening method to evaluate these factors in disease models. FunSel allows Purespring to discover new gene therapy candidates across all indications, unconstrained by genetics, to find the right candidate to make the best therapy.

An initial 45 million commitment to Purespring from Syncona Ltd is enabling Purespring to progress its assets to the clinic. Synconas Chief Investment Officer, Chris Hollowood, serves as Chairman. For more information please visit: purespringtx.com and follow us on LinkedIn.

About FunSel

FunSel is a proprietary in vivo gene therapy search engine developed by Purespring Therapeutics co-founder Professor Mauro Giacca, Professor of Cardiovascular Sciences at Kings College, London.

FunSel is gene agnostic: unconstrained by genetics and therefore does not rely on knowledge of which gene is causing a disease. It allows functional selection of therapeutic factors for disease phenotype correction. FunSel contains a library of thousands of AAV vectors encoding secreted proteins.

Because it is gene agnostic, FunSel offers potential to allow the application of gene therapy to non-monogenic disorders and treat much broader patient populations.

The platform has already been instrumental in the creation of Purespring Therapeutics and sister company Forcefield Therapeutics, a pioneer of best-in-class therapeutics to protect heart function and has broad potential for researchers developing gene therapy in many disease areas. It provides Purespring with opportunities to support industry peers through partnering and potential collaborations.

About SwanBio Therapeutics

SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions. SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications spastic paraplegias, monogenic neuropathies, and polygenic neuropathies. SwanBios lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). For more information, visit SwanBioTx.com.

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Purespring Therapeutics and SwanBio Therapeutics announce licensing agreement for use of FunSel screening platform - Yahoo Finance

Cergentis is a cornerstone acquisition that expands Solvias' solutions supporting complex and emerging therapies.

According to pharmaceutical market intelligence provider, Evaluate, global sales of cell and gene therapies are projected to accelerate at a 63% compound annual growth rate through 2026. As more researchers leverage emerging genetic engineering techniques to develop complex, novel medicines, they require sophisticated solutions to analyze their safety and effectiveness.

With the addition of Cergentis, Solvias supports the increasing number of global pharmaceutical, biotech, and contract development and manufacturing organizations developing genetically engineered therapies with an expanded platform of testing services highlighted by:

Archie Cullen, CEO, Solvias, stated:"We are relentlessly focused on ensuring the safety of new therapies in development. Cergentis is a cornerstone acquisition that expands our solutions supporting complex and emerging therapies. We will continue to pursue strategic acquisitions that add specialized capabilities to our offering and advance our goal of being a forerunner in our industry."

Joris Schuurmans, CEO, Cergentis, added:"We are excited to become part of a global leader that complements our scientific expertise, innovation and customer service. Solvias and Cergentis share a deep commitment to providing our customers with the highest quality solutions and support to safely get their products into the hands of patients who need them."

Effective immediately, Mr. Schuurmans will join Solvias' leadership team and continue to lead operations for Cergentis.

Cergentis marks Solvias' second acquisition since partnering with health care investors Water Street Healthcare Partners and JLL Partners in 2020. The company has recruited industry leaders to join its board and commenced a program to significantly upgrade and expand its information technology platform and infrastructure to support its plans for global expansion.

Financial terms of the acquisition are not being disclosed. Achelous Partners served as the advisor to Cergentis on the transaction.

About CergentisCergentis is a trusted genomics-focused biotechnology company providing services and in-house solutions based on its proprietary genomic analysis platform to all leading biopharmaceutical companies and renowned research institutes. With widely published and recognized genetic analyses, Cergentis supports a global customer base in the characterization and QC of genetically engineered models, biopharmaceutical cell line development, and cell- and gene therapy products. By helping to de-risk R&D program decisions, minimizing time-to-clinic, providing objective genomic evidence for regulators, and supporting patient safety, Cergentis aims to support biopharmaceutical medicine development programs worldwide. For more information, visit cergentis.com.

About Solvias AGSolvias is a worldwide leader in contract research, development and manufacturing for the pharmaceutical, biotech, material science and cosmetic industries. Drawing on 20 years of scientific excellence, the company provides flexible and scalable analytical and manufacturing solutions that ensure the integrity of pharmaceutical and medical device products across their life cycle. Headquartered in Kaiseraugst near Basel, Switzerland, Solvias and its laboratories operate to the highest standards and in accordance with ISO, GMP, GLP and FDA regulations. For more information, visit solvias.com.

SOURCE Solvias

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Solvias Acquires Cergentis to Bolster Biologics and Cell & Gene Therapy Capabilities - PR Newswire

Sekar Kathiresan, MD, Verve Therapeutics co-founder and CEO

Verve Therapeutics has dosed its first patient with what it said today was the first in vivo base editing therapy to reach the clinic, a potential treatment for Heterozygous Familial Hypercholesterolemia (HeFH).

Verve, which specializes in gene editing therapies for cardiovascular disease, said that its VERVE-101 is a single-course gene editing treatment designed to reduce the low-density lipoprotein cholesterol (LDL-C) that drives HeFH.

VERVE-101 consists of an adenine base editor messenger RNA that Verve has licensed from another base editing therapy developer, Beam Therapeutics, as well as an optimized guide RNA targeting thePCSK9gene packaged in an engineered lipid nanoparticle.

By making a single A-to-G change in the DNA genetic sequence ofPCSK9, VERVE-101 aims to inactivate that target gene. Verve reasons that inactivation of thePCSK9gene has previously been shown to up-regulate LDLR expression, leading to lower LDL-C levels and thus reducing the risk for atherosclerotic cardiovascular disease (ASCVD)of which HeFH is a subtype.

Base editing is a pinpoint method for engineering base substitutions without cleaving the DNA double helix backbone. The underlying technology was developed in the lab of Harvard University chemistDavid Liu, PhDwho co-founded Beam with Feng Zhang, PhD, and Keith Joung, MDwith research led by two postdocs,Alexis Komor, PhD, and Nicole Gaudelli, PhD.

Beam is also expected to enroll its first patient later this year in its first clinical trial for one of its base editing therapies, BEAM-101 for the treatment of sickle cell disease (SCD). Beam also plans two IND applications this yearone for its second SCD candidate BEAM-102, and the other for BEAM-201, a treatment for relapsed/refractory T cell acute lymphoblastic leukemia/T cell lymphoblastic lymphoma.

The dosing of the first human with such an investigational base editing medicine represents a significant achievement by our team and for the field of gene editing, Sekar Kathiresan, MD, Verves co-founder and CEO, said in a statement.

Preclinical data suggest thatVERVE-101 has the potential to offer people with HeFH a game-changing treatment option, transforming the traditional chronic care model to a single-course, life-long treatment solution, Kathiresan added.

Andrew Bellinger, MD, PhD, Verves chief scientific and medical officer, added that VERVE-101 is intended to improve upon current standard of care treatment for HeFH. He stated that less than 20% of patients achieve LDL-C goal levels due to the limitations of the chronic model, which include requirements for rigorous patient adherence, regular health care access, and extensive health care infrastructure.

Our ultimate goal withVERVE-101 is to bring a new option to the millions of people with ASCVD around the world, and dosing participants in the Phase I study for this first indication, HeFH, is a key inflection point to achieving that goal, Bellinger said.

Publicly traded Verves stock rose 8% on Tuesday, closing at $22.20.

VERVE-101 is under study in the Phase Ib heart-1 trial (NCT05398029), designed to assess the safety and tolerability ofVERVE-101 with additional analyses for pharmacokinetics and reductions in blood PCSK9 protein and LDL-C.

The trial is designed to enroll approximately 40 adults and includes three parts: A single ascending dose portion, followed by an expansion single-dose cohort where additional participants will receive the selected potentially therapeutic dose, and an optional second-dose cohort, in which eligible participants in lower dose cohorts in Part A have the option to receive a second treatment to reach the selected potentially therapeutic dose.

Verve is conducting the trial at a pair of clinical sites in New Zealand, according to ClinicalTrials.gov.

The company has said it plans to submit both a clinical trial application (CTA) in the United Kingdom and an IND application in the U.S., both in the second half of this year.

Interim clinical data for the heart-1 trialwhich will include safety parameters, blood PCSK9 level and blood LDL-C levelare expected in 2023, Verve said.

Given the once-and-done treatment potential for very large CV [cardiovascular] market opportunities, we seefirst human proof-of-concept data in 2023 for leadVERVE-101 as a key inflection point for the shares, Eun Yang, PhD, an analyst with Jefferies, wrote July 7 in a research note.

In January during a presentation at the 40th Annual J.P Morgan Healthcare Conference, Verve described its market opportunity in CV as potentially hundreds of millions of patients worldwide, including 31 million with a genetic form of ASCVD. Verve presented figures also cited by the National Organization for Rare Disorders (NORD) showing that HeFH affects 1 in 250 people, while HoFH is much rarer, affecting 1 in 250,000, the company said.

VERVE-101 has the potential to change the way cardiovascular disease is cared for by lowering LDL-C as low as possible for as long as possible after a single treatment, Bellinger added.

Given the once-and-done treatment potential for very large CV [cardiovascular] market opportunities, we seefirst human proof-of-concept data in 2023 for leadVERVE-101 as a key inflection point for the shares, Eun Yang, PhD, an analyst with Jefferies, wrote July 7 in a research note.

Verve is also developing a second program designed to lower blood lipids such as LDL-C by permanently turning off theANGPTL3gene in the liver, in order to treat homozygous familial hypercholesterolemia (HoFH), a rare genetic subtype of ASCVD characterized by extremely high blood LDL-C, as well as for patients with ASCVD who have not achieved goal LDL-C lowering with oral therapy and a PCSK9 inhibitor.

The company expects to identify its base editing development candidate targeting ANGPTL3 and begin IND-enabling studies later this year.

Since 2019, Verve had licensed Beams technology for human therapeutics against PCSK9 and three other liver-mediated, cardiovascular disease targets, including ANGPTL3, plus two undisclosed gene targets.

Just last week, Verve and Beam amended their agreement to develop and commercialize products targeting another liver-mediated, cardiovascular disease target, though that target and development timeline have not been disclosed.

Verve agreed to oversee development and commercialization of products targeting that gene, with Beam holding options to co-develop them after the final dosing of a patient in a Phase I clinical trial. If Beam opts in, it would share 35% of worldwide development expenses for the product, as well as jointly commercialize and share 35% of the profits and expenses of commercializing that product worldwide.

But if Beam doesnt opt in, it would be entitled to receive milestones and royalties as called for in the companies original agreement.

The amended agreement does not change Beams co-development option for Verves programs targeting PCSK9 and ANGPTL3. BEAM has the option to co-develop those programs at the end of Phase I. If Beam exercises that option, it would fund 33% of major market development costs and 50% of U.S. commercialization costsbut in return would receive 50% of U.S. profits, whileVerve wouldretain ex-US rights. For those ex-U.S. opt-in products, on aproduct-by-productbasis, Verve agreed to pay Beam clinical and regulatory milestones of up to $5.6million and sales-based milestones of up to $7.5million.

If Beam declines to opt in, it would be eligible instead for $11.3 million per product in payments tied to achieving development milestones, $15 million per product in sales milestone payments and royalties in the low single digits.

Verve finished the first quarter with cash, cash equivalents and marketable securities of $323.3 million as of March 31, 2022, down 10% from $360.4 million as of December 31, 2021.

Based on current operating plans, Verve expects its existing cash, cash equivalents and marketable securities will enable the company to fund its operating expenses and capital expenditure requirements into 2024, Verve said in May when it reported Q1 results.

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Verve Therapeutics Doses First Patient with In Vivo Base Editing Therapy - Genetic Engineering & Biotechnology News

Plain Language Summary

Hemophilia is a bleeding disorder where blood does not clot normally because of a genetic mutation that causes missing or defective clotting protein. People with severe hemophilia have painful spontaneous bleeding in their joints, which can lead to chronic pain and disability. Gene therapy, which introduces the instructions to make the missing/defective clotting protein and prevent spontaneous bleeding, is being investigated as a potential treatment for severe hemophilia. Gene therapy for severe hemophilia A is a new treatment that is much different from standard, intensive hemophilia treatments. The Haemo-QOL-A questionnaire is used to evaluate health-related quality of lifein people with hemophilia on standard treatments. In this manuscript, we determine whether the Haemo-QOL-A questionnaire can be used to evaluate change in quality of lifefor people with severe hemophilia A after receiving gene therapy. Using data from previous clinical trials of the gene therapy valoctocogene roxaparvovec (AAV5-hFVIII-SQ), we show that the Haemo-QOL-A did measure a change in quality of life after gene therapy. We also estimate how much change in Haemo-QOL-A scores from before to after gene therapy represents a clinically meaningful improvement in quality of life. This threshold can be used in future research evaluating the effects of gene therapy on quality of life for people with hemophilia. While the results of this study are important, only 23 people were included in the analysis. To confirm our results, future analyses with larger numbers of participants are needed.

Hemophilia is a genetic bleeding disorder caused by deficiency or inactivity of factor VIII (FVIII; hemophilia A) or factor IX (hemophilia B) protein. Hemophilia A accounts for approximately 80% of hemophilia cases worldwide.1 Severe hemophilia A (FVIII <1 IU/dL) is characterized by recurrent and spontaneous musculoskeletal bleeding episodes, resulting in joint damage, mobility issues, and early mortality.1 People with severe hemophilia A (PWSHA) experience significant health-related quality of life (HRQOL) impairment, including negative effects on emotional and cognitive health, joint pain, poor functioning in school, and difficulties securing and maintaining employment.27

Treatment options for hemophilia A are rapidly evolving. Standard of care for PWSHA is prophylactic factor replacement therapy using exogenous FVIII or bypassing agents such as emicizumab-kxwh.1,8 Factor replacement therapy must be administered frequently through intravenous infusions (14 infusions/week), resulting in substantial treatment burden and potential risk of breakthrough bleeding following poor treatment adherence.9 Despite prophylactic treatment, most PWSHA still require on-demand treatment with FVIII or emicizumab to treat bleeding episodes.10

Gene therapies using recombinant adeno-associated viral (AAV) vectors carrying human FVIII gene may offer a novel approach to hemophilia A treatment.1113 In phase 1/2 and phase 3 studies, a 61013 vg/kg infusion of valoctocogene roxaparvovec, an investigational gene therapy utilizing a codon-optimized AAV serotype 5 vector encoding a B-domaindeleted human FVIII (AAV5-hFVIII-SQ), resulted in clinically relevant reductions in annualized treated bleed rate and exogenous FVIII replacement up to 5 and 1 years of follow-up, respectively.11,12,14,15 If long-term efficacy of viral vector gene transfer is established, it could represent a paradigm shift in hemophilia A treatment.

The general health questionnaire EuroQOL-5D-5L (EQ-5D-5L) is used to evaluate overall HRQOL.16 However, people with hemophilia completing the EQ-5D-5L reported higher health states than the general population, indicating the presence of a disability paradox in PWSHA.17 Thus, HRQOL assessment with the EQ-5D-5L scores may not accurately reflect the hemophilia-related burden they experience.18 To address the need for a reliable HRQOL assessment, the hemophilia-specific HRQOL questionnaire for adults (Haemo-QOL-A) was developed.19 It consistently performs well among adults relative to other hemophilia-specific questionnaires, demonstrating robust validity in people undergoing standard treatment regimens.2023

Gene therapy is a novel, potentially one-time intervention. To date, HRQOL has been evaluated only in a small number of PWSHA receiving gene therapy.14 It is unclear how gene therapy will incrementally benefit the HRQOL for PWSHA compared with standard of care or whether the Haemo-QOL-A will be an appropriate measure to detect HRQOL changes after gene therapy.24 Thus, in addition to estimating Haemo-QOL-A clinically important differences (CID) specifically for gene therapy recipients, this post hoc analysis of phase 1/2 and phase 3 clinical trial data aimed to evaluate the content validity, construct validity, and reliability of the Haemo-QOL-A for measuring HRQOL in adult PWSHA treated with gene therapy.

A post hoc psychometric analysis to determine the content validity, construct validity and reliability of the Haemo-QOL-A was conducted using data from a phase 1/2 open-label dose-escalation study (Clinicaltrials.gov, NCT02576795; EudraCT, 2014-003880-38)12 and phase 3 open-label, single-arm study (NCT03370913, EudraCT, 2017-003215-19) to evaluate the efficacy and safety of valoctocogene roxaparvovec in adult PWSHA.15 Full study design details have been published previously.11,12,14,15 All protocols were reviewed and approved by local institutional review boards or ethics panels and conducted according to the Declaration of Helsinki; all participants provided informed consent.

All participants were male and 18 years old with severe hemophilia A (FVIII levels 1 IU/dL). The phase 1/2 dose-escalation study included participants on FVIII prophylaxis or on-demand therapy who had 12 bleeding episodes within 12 months prior to enrollment; participants received an infusion of AAV5-hFVIII-SQ at 61012 vg/kg, 21013 vg/kg, 41013 vg/kg, or 61013 vg/kg. The ongoing phase 3 study included participants from 48 sites worldwide who were on prophylactic FVIII replacement therapy for 12 months prior to enrollment; all participants received an infusion of 61013 vg/kg AAV5-hFVIII-SQ.

Data from up to 3 years of follow-up for participants in the phase 1/2 study who received 61013 vg/kg dose of AAV5-hFVIII-SQ were used. The phase 3 study intention-to-treat (ITT) population was defined as all enrolled and treated participants, and the modified ITT (mITT) population was defined as all treated participants who were human immunodeficiency virus (HIV)-negative and who completed the week 26 visit. Data up to 26 weeks from both populations were used in these analyses.

Patient HRQOL was assessed using the Haemo-QOL-A questionnaire in both study populations, administered at baseline and at weeks 1, 2, 3, 4, 16, 28, 52, 78, 104, 130, and 156 in the phase 1/2 study population and at baseline and weeks 4, 12, and 26 in the phase 3 study population. The EQ-5D-5L was administered in the phase 3 study at baseline and weeks 4, 12, and 26.

The 41 items on Haemo-QOL-A include the domains of Consequences of Bleeding (7 items), Emotional Impact (6 items), Physical Functioning (9 items), Role Functioning (11 items), Treatment Concern (3 items), and Worry (5 items). Items were scored on a 6-point Likert-type scale with higher scores indicating better HRQOL or less impairment. Subscale scores for each disease domain range from 0 to 5 and the Total Score ranges from 0 to 30. Both domain and total raw scores are transformed to a 0 to 100 scale using the formula:

Domain scores were imputed using mean domain scores if <50% of items were missing. Total Score was not calculated, and no imputations were performed if >50% of items were missing. Change in scores at specific time points were calculated using the formula:

EQ-5D-5L was used to assess general health status for the 5 domains of Mobility, Self-care, Usual Activities, Anxiety and Depression, and Pain and Discomfort. Items on EQ-5D-5L were scored on a scale of 1 to 5 with higher scores representing increased impairment. The EQ-5D-5L vertical visual analog scale (VAS) of current health status scores were assessed on a scale ranging from 0 to 100, with higher scores indicative of better HRQOL.

Statistical analyses were performed using IBM SPSS Statistics 25 (IBM Corporation, Armonk, NY). Data normality was assessed graphically using frequency distribution histograms and statistically using skewness, kurtosis, and standard error. Categorical variables were described using frequencies and percentages. Continuous variables with normal distribution were assessed using descriptive statistics of mean standard deviation (SD) or range. Variables with non-normal distribution were assessed using mean SD and median (interquartile range [IQR]). Significance was assessed at <0.05 (2-tailed) with 95% confidence intervals (CIs).

Item facility, which assesses the possible presence of floor or ceiling effects, was calculated using data from the phase 1/2 study and the phase 3 ITT study populations. An item was considered to have floor or ceiling effects and poor item facility if >50% of reported responses for items were either the minimum/maximum option.25

Convergent validity, which assesses the degree of correlation with existing measures for a condition, was measured by correlating Haemo-QOL-A scores with EQ-5D-5L scores in participants treated in the phase 3 mITT study population. Spearmans Rank correlation coefficients were calculated between Haemo-QOL-A Total and domain scores vs EQ-5D-5L domain scores at baseline and week 26. Spearmans Rank correlation coefficients were calculated to assess the relationship between Haemo-QOL-A Total and domain score change and score change vs EQ-5D-5L domain score changes from baseline to week 26 in the phase 3 study. Pearsons Rank correlation coefficient was calculated between Haemo-QOL-A Total and domain score changes vs EQ-5D-5L VAS score changes. Regression analyses were also conducted to evaluate the relationship between the 2 instruments when assessing Haemo-QOL-A vs EQ-5D-5L score change from baseline to week 26 (change on change).

Discriminant validity is the ability of a scale to distinguish between different patient subgroups and was assessed by comparing groups of participants from the phase 3 mITT study population based on baseline EQ-5D-5L VAS scores. Independent sample t-tests were used to compare participants with baseline EQ-5D-5L VAS scores >87.15 vs <87.15 (the UK population norm for males aged 2535).26 Data from baseline and week 26 were pooled in order to compare mean Haemo-QOL-A Total and domain scores for participants with baseline EQ-5D-5L VAS scores >87.15 vs those with baseline EQ-5D-5L VAS scores <87.15.

Item discrimination and internal consistency, which assess the ability of an item to discriminate against others on its subscale, were calculated using data from the phase 1/2 study population and the phase 3 ITT study population. Item discrimination was predicted using Spearmans Rank correlation coefficient between individual item scores and Total Score. Cronbachs alpha and squared multiple correlation were used to calculate internal consistency. Items with Cronbachs alpha >0.7 were considered to have properties of internal consistency.

For QOL evaluation in chronic disease, half SD of baseline mean is used to determine a distribution-based estimate of the CID threshold.27 We estimated the CID via distribution methods; the half SD rule (of baseline) was applied for Haemo-QOL-A Total and domain scores.

Given the small sample size and large range of changes in EQ-5D-5L VAS score (ie, 25 to +25), different anchor methods were sequentially deployed to provide the most accurate CID estimate. Data from the phase 1/2 study population were not used since the EQ-5D-5L was not administered to study participants. The mean difference in Haemo-QOL-A Total Score change from baseline to week 26 was calculated between the following categories of participants based on their EQ-5D-5L VAS score change from baseline to week 26 in the phase 3 mITT study population:

a. Participants with VAS score change +3.

b. Participants with a VAS score change between +3 and 3.

c. Participants with VAS score change 3.

Next, results from regression analyses were extrapolated to predict change in Haemo-QOL-A Total Score (dependent variable) vs an EQ-5D-5L VAS score increase of 3. Then, the mean difference in Haemo-QOL-A Total and domain score changes from baseline to week 26 were calculated between categories of participants based on their EQ-5D-5LVAS score change from baseline to week 26 shown in Table 1. Between those categories, the relative magnitude of EQ-5D-5LVAS score change was mapped against relative magnitude of Haemo-QOL-A score change to derive the mean change in Haemo-QOL-A score vs EQ-5D-5L VAS score change of 1 and 3, respectively.

Table 1 EQ-5D-5L VAS Score Change Categories Used in Anchor-Based Estimation of Clinically Important Difference

Haemo-QOL-A data were analyzed from 7 participants in the phase 1/2 study through 156 weeks and 16 participants in the phase 3 study through 26 weeks (mITT = 16). Mean (SD) age was 30.4 (5.8) years and 29.7 (6.2) years for participants in the phase 1/2 and 3 trials, respectively.11,12,15

Baseline Haemo-QOL-A scores were similar in the phase 1/2 and phase 3 studies (Table 2). In the phase 3 mITT study population, both EQ-5D-5L VAS and Haemo-QOL-A Total Scores at week 26 were higher than at baseline, suggesting an improvement in HRQOL of participants following AAV5-hFVIII-SQ infusion (Table 3; Supplemental Figure 1).

Table 2 Mean Change from Baseline in Haemo-QOL-A Total and Domain Scores in the Phase 1/2 and 3 Studies

Table 3 Mean Transformed Haemo-QOL-A Total Score and EQ-5D-5L VAS Scores at Baseline and Week 26 by EQ-5D-5L VAS Score Change Subgroups

Items where >50% of responses were reported as the minimum/maximum response were considered to have poor items facility (ie, floor or ceiling effects). Out of 41 items, 13 had ceiling effects (Supplemental Table 1). The domains with the highest percentage of items with ceiling effects were Treatment Concern (2/3 items) and Emotional Impact (3/6 items). No floor effects were observed.

Our results indicate good convergent validity between the Haemo-QOL-A and EQ-5D-5L. Overall, Haemo-QOL-A Total and domain scores were inversely correlated with EQ-5D domain scores demonstrating that as degree of impairment in Mobility, Self-care, Usual Activities, Anxiety and Depression, and Pain and Discomfort decrease (demonstrating improvement), Haemo-QOL-A scores increase (demonstrating improvement), and vice versa.

The strongest correlations were seen when comparing week 26 and change vs change scores. In both cases, the Pain and Discomfort domain of the EQ-5D-5L was strongly and significantly correlated with the Haemo-QOL-A. At week 26, the strongest correlations were seen between Role Functioning and Pain and Discomfort (0.87, P <0.01), Emotional Impact and Pain and Discomfort (0.85, P <0.01), Physical Functioning and Pain and Discomfort (0.79, P <0.01), and Total Score and Pain and Discomfort (0.87, P <0.01) (Supplemental Table 2). When comparing change in Haemo-QOL-A to EQ-5D-5L scores, the strongest correlations were seen between Emotional Impact and Anxiety and Depression (0.88, P <0.01), Emotional Impact and Pain and Discomfort (0.7, P <0.01), Physical Functioning and Pain and Discomfort (0.73, P <0.01), Total Score and Anxiety and Depression (0.82, P <0.01), and Total and VAS scores (0.77, P <0.01), implying that improvement in Physical Functioning and Emotional Impact are being driven by a decrease in pain (Table 4). At baseline, the strongest correlations were between Physical Functioning domain of the Haemo-QOL-A and the Usual Activities (0.75, P <0.01) and Pain and Discomfort (0.64, P <0.01) domains of EQ-5D-5L (Supplemental Table 3). Regression analyses showed a linear correlation between change in Haemo-QOL-A Total Scores and EQ-5D-5L VAS scores (Figure 1), with a predicted change of 0.63 in Haemo-QOL-A Total Score for every EQ-5D-5L VAS score change of 1 (R2 = 0.5, adjusted R2 = 0.46).

Table 4 Correlations Between Change from Baseline to Week 26 in Haemo-QOL-A Transformed Scores and Corresponding Change in EQ-5D-5L Scores, Modified Intent-to-Treat Population, Phase 3 Study

Figure 1 Linear regression plot of change in Haemo-QOL-A Total Score vs change in EQ-5D-5L VAS scores, from baseline to week 26, modified intent-to-treat population, phase 3 study.

Abbreviations: EQ-5D-5L, EuroQOL-5D-5L; Haemo-QOL-A, hemophiliaspecific healthrelated quality of life questionnaire for adults; TS, total score; VAS, visual analog scale.

There was a substantial difference between mean Haemo-QOL-A scores reported by participants with an EQ-5D-5L VAS score change of magnitude <3 vs those with an EQ-5D-5L score change of magnitude 3 (Table 3). Good discriminant validity was demonstrated by a mean difference between participants with EQ-5D-5L scores above and below the UK population norm ranging from 6.4617.31. The Haemo-QOL-A Total Score (mean [CI] difference 11.81 [20.2, 3.4], P = 0.01) and domains scores for Emotional Impact (17.3 [26.4, 8.2], P = 0.001) and Role Functioning (15.4 [24.6, 6.2], P = 0.002) were most sensitive to discriminate differences in impairment based on EQ-5D-5L scores (Figure 2).

Figure 2 Difference between mean Haemo-QOL-A Total and domain scores in EQ-5D-5L VAS participant subgroups (UK population norm [n = 9]), modified intent-to-treat population, phase 3 study. an = 23 for VAS score

Abbreviations: EQ-5D-5L, EuroQOL-5D-5L; Haemo-QOL-A, hemophiliaspecific healthrelated quality of life questionnaire for adults; VAS, visual analog scale.

Additionally, review of participant-level data indicated a relationship between clinical outcomes (eg, ongoing arthropathy, bleeds and comorbidities) and Haemo-QOL-A scores, suggesting that the tool can discriminate between different disease states. In the phase 3 study population, 3/15 participants reported HRQOL score decreases greater in magnitude than the instrument CID estimate. All 3 participants had degenerative joint damage alongside other comorbidities. Of the 4/15 participants who reported Haemo-QOL-A score changes lower in magnitude than the CID estimate, 3 had ongoing arthropathy. Of the 8 participants that reported score increase >CID, only 3 had arthropathy.

Of the 41 items in Haemo-QOL-A, 38 had corrected item-total correlation scores >0.4. Three items were identified as non-discriminatory in their domain (corrected item-total correlation scores <0.4). The item I am able to complete household tasks in the Physical Functioning domain had a corrected item-total correlation of 0.03. However, the domain showed good internal consistency overall, and although deletion of this item improved Cronbachs alpha, it improved from an acceptable level regardless (from 0.71 to 0.76). In addition, 2 of 3 items in the Treatment Concern domain, I worry about the safety of my treatment and I worry about the availability of hemophilia products, had poor discrimination (item correlation-total scores 0.09 and 0.26, respectively). Overall, the domain showed poor internal consistency and deletion of each item only improved the Cronbachs alpha for this domain from 0.31 to 0.42 and 0.317, respectively.

Results of the distribution-based method to estimate CID in Haemo-QOL-A are shown in Supplemental Table 4. A variety of anchor-based methods were also tested. For participants with EQ-5D VAS score change categories of +3, between +3 and 3, and 3, the mean changes in Haemo-QOL-A Total Scores were 16.3, 1.8, and 2.8, respectively (Table 3). Comparison of groups indicated a score change 6.31 would represent a clinically meaningful change in the Haemo-QOL-A Total Score. However, as the mean VAS score change in the groups experiencing a score change 3 (ie a clinically meaningful change in VAS28) is far greater than the CID threshold for the VAS (mean score change of 13), it is likely that the true CID is <6.31; thus, further analyses were conducted and detailed below. First, results from regression analyses were extrapolated to predicted change in Haemo-QOL-A Total Score (dependent variable) vs EQ-5D VAS score increase of 3. Analyses predicted that for each 1-point increase in VAS score, Haemo-QOL-A Total Score would increase by 0.63. Therefore, a 3-point VAS score change should equate to a Haemo-QOL-A Total Score change of 1.89. Next, given the distribution of the available data, mean change in Haemo-QOL-A Total and domain scores were mapped against reported VAS change categories shown in Table 1 that were greater in magnitude than the accepted CID of 3.28 An average of all domain CID estimates was calculated to give an overall estimate for what would constitute a CID score change for the domains of the Haemo-QOL-A (Supplemental Table 5). The resultant Haemo-QOL-A Total Score CID estimate (score change 5.5 in magnitude) was lower than the estimated domain score (score change 6.0 in magnitude) due to lack of relevance of 2/3 items in the Treatment Concern domain in patients treated with gene therapy. Given the distribution of the data and small sample size, the latter anchor analyses in Supplemental Table 5 were deemed most reliable. This is further corroborated by the fact that the Haemo-QOL-A Total Score CID estimate derived from this method matches others that use a distribution-based method.

Participants in the phase 1/2 and 3 studies had higher mean Haemo-QOL-A Total Score change from baseline than the CID estimate at weeks 28, 52, 104, and 156 post-infusion and at week 26 post-infusion, respectively (Table 2). Similarly, participants reported clinically meaningful improvements in the domain scores for Consequences of Bleeding, Physical Functioning, Role Functioning, and Worry at all time points (Table 2). Changes in mean domain scores for Emotional Impact and Treatment Concern were not consistently above the CID threshold in the phase 1/2 study population (Table 5).

Table 5 Mean Change in Transformed Haemo-QOL-A Scores for Domains of Emotional Impact and Treatment Concern, Phase 1/2 Study

This is the first study to psychometrically validate the Haemo-QOL-A in PWSHA undergoing gene therapy. Validation of Haemo-QOL-A is important to inform clinical analysis of the effect of gene therapy on HRQOL. Our preliminary results demonstrate good psychometric validity of the Haemo-QOL-A when measured in participants undergoing gene therapy, consistent with the US Food and Drug Administration guidelines for patient-reported outcome instrument validation, though all results should be interpreted with caution and confirmed in a larger sample size.29

In this study, the Haemo-QOL-A had good preliminary construct validity, including convergent validity, as there was a high degree of correlation between the Haemo-QOL-A and EQ-5D-5L. Despite the limited sample size, mean Haemo-QOL-A Total and domain scores were capable of detecting differences in participant populations with vs without a high burden of disease, suggesting the instrument had good discriminant validity. The scale also showed good content validity with very few items displaying floor and ceiling effects, except in the Treatment Concern domain, where 2/3 questions are not relevant to an individual undergoing gene therapy in a clinical trial. Our results also establish CIDs in Haemo-QOL-A scores after gene therapy and demonstrate likely applicability in ongoing clinical studies.

Convergent validity preliminarily identified significant correlations between Haemo-QOL-A and EQ-5D-5L scores at both baseline and week 26. Correlations at baseline, although significant, were not as strong as at week 26, highlighting the disability paradox experienced by PWSHA.17 Improved outcomes on the Haemo-QOL-A Total Scores and domains of Emotional Impact, Physical Functioning, and Treatment Concern at week 26 post-infusion were inversely correlated with EQ-5D-5L domain scores for Usual Activities, Anxiety and Depression, and Pain and Discomfort. There were no significant correlations between the EQ-5D-5L domain of Mobility and the Haemo-QOL-A domain of Physical Functioning, suggesting that improvements in physical QOL of participants treated with gene therapy may be driven largely by a decrease in pain.

Mean Haemo-QOL-A Total Scores discriminated well among our limited sample of study participants with differing degrees of HRQOL impairment based on EQ-5D-5L VAS scores. Previous studies show similar discrimination between hemophilia A populations differing in disease severity, HIV status, and type of treatment (on-demand vs prophylaxis) using Haemo-QOL-A.19 The current analysis used general population scores based on UK norms.26 It will be interesting to evaluate the discriminant characteristics of Haemo-QOL-A in studies conducted in different geographic regions. Additionally, the tool discriminated between participants with and without ongoing arthropathy in our limited sample of study participants, suggesting that joint damage and subsequent pain heavily impact HRQOL in PWSHA; therefore, treatment should aim to prevent joint damage before it occurs.

Of the 6 Haemo-QOL-A domains, only Treatment Concern had poor internal consistency. Two items relating to treatment safety and availability lacked discrimination. The third item on concerns regarding clinician inexperience performed well. Thus, two-thirds of this domain was insensitive to the psychological HRQOL of participants pertaining to their treatment, which lowered the Haemo-QOL-A Total Score. Since a one-time infusion of gene therapy may circumvent the need for repeated factor infusions, participants may not be as concerned about treatment availability. Further, since gene therapy is provided through specialist treatment centers with thorough follow-up, participants may not be as concerned about the safety of their treatment. In the future, the Treatment Concern domain could be revised to be more appropriate for evaluating change post-gene therapy by, for example, using the remaining item I worry about the safety of my treatment to score the domain or adding additional questions that better reflect the current treatment landscape.

A total of 13 items showed ceiling effects. These were most prevalent in the Treatment Concern and Emotional Impact domains. Floor and ceiling effects diminish sensitivity of psychometric instruments and can result in underestimation of treatment effectiveness. Scores pertaining to these items must therefore be interpreted cautiously in future applications of Haemo-QOL-A in gene therapy.

It is important to benchmark HRQOL improvements in participants undergoing gene therapy for hemophilia A against CID estimates generated using both distribution- and anchor-based approaches. The current study used both methods to establish CID estimates that are in line with those previously published following FVIII prophylaxis, where the distribution-based CID estimate for Haemo-QOL-A Total Score ranged from 5 to 7 and that of the Physical Functioning domain ranged from 6 to 9.30 However, given the small number of participants receiving gene therapy in the current analysis and the vastly different nature of the HRQOL burden experienced by participants receiving gene therapy vs those receiving standard of care, clinicians should be cautious in comparing the magnitude of CID scores across these 2 studies.

Small sample size was a limitation in both populations, which precluded using several anchor-based estimation approaches.28 A low anchor magnitude of 3-point EQ-5D-5L VAS score change was adopted given the EQ-5D-5Ls limitation in determining differences in disease state in hemophilia.17 Of all participants evaluated across both populations, 4 reported baseline Total Score >85%, indicating possible ceiling effects. Here, some participants reported VAS scores higher than population norms; other research using the EQ-5D-5L in PWSHA found similar results, suggesting a disability paradox.17 People with chronic conditions often report higher or similar health state valuations than the general population,31,32 in part due to processes of adjustment and coping. A larger sample size may provide more meaningful anchor scores and allow more accurate estimation of CID.

Future availability of novel gene therapy interventions will likely shift the paradigm of hemophilia A treatment. This small study is the first to validate the Haemo-QOL-A for detecting clinically meaningful improvement in the HRQOL of PWSHA receiving gene therapy. Our analyses establish initial CID estimates and support the validity and reliability of the Haemo-QOL-A for measuring changes in HRQOL. These preliminary findings suggest that the Haemo-QOL-A is likely fit for evaluating HRQOL and provides a guide for future applications for measuring HRQOL outcomes in PWSHA following gene therapy. Prospective studies with larger sample sizes are needed to evaluate this instrument in a broader severe hemophilia A population.

De-identified individual participant data underlying these results (including text, tables, figures, and appendices) will be made available, together with the clinical protocol and data dictionaries, for non-commercial, academic purposes. Additional supporting documents may be available upon request. Investigators will be able to request access to these data and supporting documents via the Publication Data Request page at http://www.BioMarin.com beginning 6 months and ending 2 years after publication. Data associated with any ongoing development program will be made available within 6 months after approval of the relevant product. Requests must include a research proposal clarifying how the data will be used, including proposed analysis methodology. Research proposals will be evaluated relative to publicly available criteria available at http://www.BioMarin.com to determine if access will be given, contingent upon execution of a data access agreement with BioMarin Pharmaceutical Inc.

The protocol for the 201 trial was reviewed and approved by the Imperial College Research Ethics Committee at Imperial College London and the ethics committees of all other participating sites. The protocol for the 301 trial was reviewed and approved by the Research Ethics Committee at the State University of Campinas and the institutional review boards or ethics committees of all other participating sites. Both studies were conducted in accordance with the guidelines outlined in the Declaration of Helsinki. All participants in both trials provided written informed consent prior to participating in any protocols.

We thank the study participants, study site personnel, and investigators who were involved in these clinical trials. We thank Nina Mitchell of BioMarin Pharmaceutical Inc., for her contributions to this project. Project management support was provided by Sara Hawley of BioMarin Pharmaceutical Inc. Medical writing support was provided by Kathleen Pieper, PhD, and Atreju Lackey, PhD, of AlphaBioCom, LLC, and funded by BioMarin Pharmaceutical Inc.

All authors made a significant contribution to the work reported, whether that is in the conception, study design, execution, acquisition of data, analysis and interpretation, or in all these areas; took part in drafting, revising, or critically reviewing the article; gave final approval of the version to be published; have agreed on the journal to which the article has been submitted; and agree to be accountable for all aspects of the work.

Funding for this research was provided by BioMarin Pharmaceutical Inc.

Jennifer Quinn was an employee and stockholder of BioMarin Pharmaceutical Inc., London, UK at the time of the study. Wing Yen Wong and Kathleen A Delaney are employees of BioMarin Pharmaceutical Inc., Novato, CA, USA. Wolfgang Miesbach has received speaker honoraria and project grants from Bayer, BioMarin Pharmaceutical Inc., Biotest, CSL Behring, Chugai, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Sobi, Takeda/Shire, and uniQure. Monika Bullinger has received speaker honoraria and project grants from Bayer, BioMarin Pharmaceutical Inc., Janssen Cilag, Otsuka Lundbeck, and Pfizer. The authors report no other conflicts of interest in this work.

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Psychometric validation of the Haemo-QOL-A | PROM - Dove Medical Press

CAMBRIDGE, Mass., July 18, 2022 (GLOBE NEWSWIRE) -- Ring Therapeutics, a life sciences company founded by Flagship Pioneering to revolutionize gene therapy with its commensal virome platform, today announced three upcoming presentations at the 41st Annual Meeting of the American Society for Virology to be held from July 16-20, 2022 in Madison, Wisconsin. These presentations showcase Rings significant progress building out the Anellogy platform to engineer novel precision medicines through harnessing the unique biology of anelloviruses.

Oral Presentations:

Title: In vitro production of recombinant human anellovirus particles using synthetic viral genomesSession Title: Replication and Gene ExpressionAbstract Number: 3733463Board number: W49-10Presenter: Dr. Dhananjay NawandarPresentation Date and Time: Tuesday, July 19, 2022, 4:15 - 4:30 p.m. CST

This talk will present data from a study demonstrating an in vitro system to effectively produce anelloviruses and recapitulate tissue specific delivery in vivo. These results amplify basic understanding of anelloviruses and offer new insights into harnessing their unique biology for development of novel precision medicines.

Title: The First Structure of an Anellovirus Particle Reveals a Mechanism for Immune EvasionSession Title: W41: StructureAbstract Number: 3733650Presenter: Dr. Kurt SwansonPresentation Date and Time: Monday, July 18, 2022, 7:30 7:45 p.m. CST

This talk will showcase the first high resolution structure of an anellovirus and regions of the genome that are required for capsid assembly. The structure also provides a potential mechanism for anellovirus immune evasion, a hallmark of this viral family that could make them an ideal viral-based therapy.

Poster Presentations:

Title:Comprehensive profiling of antibody responses to anelloviruses within the commensal human virome using programmable phage display Session Title: Oncolytics, Gene Therapy and Viral VectorsAbstract Number: 3731105Board number: P28-1Presenter: Dr. Harish SwaminathanPresentation Date and Time: Monday, July 18, 2022, 8:30 - 10:00 p.m. CST

Data from this poster will illustrate that most anellovirus peptides are not associated with an antibody response in humans when compared to other known human viruses. These results further the understanding of anellovirus immune favorability.

About Ring TherapeuticsRing Therapeutics is revolutionizing the gene therapy and nucleic acid medicine space by harnessing the most abundant and diverse member of the human commensal virome, anelloviruses. The company developed the Anellogy platform which focuses on anelloviruses to potentially treat a broad range of diseases. Through harnessing the unique properties of these commensal viruses, the Anellogy platform generates diverse vectors that exhibit both tissue-specific tropism and the potential to be re-dosed. Ring Therapeutics, founded by Flagship Pioneering in 2017, aims to develop and further expand its portfolio by leveraging its platform to unlock the full potential of gene therapy and nucleic acid medicines, enabling a variety of mechanisms that successfully deliver therapeutic cargo to unreachable organs and tissues. To learn more, visit https://www.ringtx.com/ or follow us on Twitter at @Ring_tx.

Ring Therapeutics Media:Brittany Leigh, Ph.D.LifeSci Communicationsbleigh@lifescicomms.com+1-813-767-7801

Link:
Ring Therapeutics Announces Three Presentations at the 41st Annual Meeting of the American Society for Virology (ASV) - GlobeNewswire