Category Archives: Gene therapy

Two years ago, a mother received a phone call with a devastating diagnosis. That mother shares her son's experience with spinal muscular atrophy (SMA), a rare genetic disease, and how early diagnosis and treatment transformed his life.

BANNOCKBURN, Ill., Oct.12, 2022 /PRNewswire/ --

BACKGROUND:

Hannah Weaver did not think much about her son Payne's newborn screening test until she received a phone call five days after bringing him home from the hospital. The results showed Payne tested positive for spinal muscular atrophy (SMA), a rare, progressive neuromuscular disease and a leading genetic cause of infant death when left untreated that affects one in every 11,000 babies born in the U.S.1,2 SMA causes irreversible loss of motor neurons, which can rob infants of their ability to walk, swallow and even breathe.1,2 If left untreated in its most severe forms, 90% of children require permanent feeding and breathing support or pass away by their second birthday.3,4 SMA can progress quickly, making early diagnosis and treatment crucial.

Experience the full interactive Multichannel News Release here: https://www.multivu.com/players/English/9064751-novartis-spinal-muscular-atrophy-health-alert/

Knowing it was imperative to act fast, the Weaver family worked quickly to schedule appointments with specialists to discuss treatment options. Payne's care team went over the available treatment options for SMA, discussing the route of administration and available efficacy and safety data of each. Together, they decided to treat Payne when he was just a few weeks old with a gene therapy called ZOLGENSMA (onasemnogene abeparvovec-xioi), the only SMA treatment designed to directly address the genetic root cause of the disease by replacing the function of the missing or non-working SMN1 gene with a single, one-time dose.

Zolgensma has a boxed warning for acute serious liver injury and acute liver failure. In clinical trials, the most common side effects were elevated liver enzymes and vomiting. Please keep reading for additional important safety information and please see accompanying Full Prescribing Information.

Payne's parents are now able to plan for his future something that would not be possible without early intervention and treatment. They look forward to what he will accomplish next and celebrate every milestone along the way. On the heels of SMA Awareness Month, we kick off Newborn Screening Awareness Month this September, and Hannah is advocating for parents and physicians to recognize the early signs of SMA to avoid a delayed diagnosis.

Dr. Sandra Reyna (Vice President of Global Medical Affairs at Novartis Gene Therapies) and Hannah Weaver (SMA parent advocate) shared information on the signs of SMA, the importance of an early diagnosis, and how Zolgensma has the potential to transform the lives of babies born with this disease.

Results and outcomes vary among children based on several factors, including how far their SMA symptoms progressed prior to receiving treatment.

Please continue reading for Indication and Important Safety Information, and please see accompanying Full Prescribing Information including Boxed Warning.

For more information, please visit: http://www.Zolgensma.com

Interview opportunities are courtesy of Novartis Gene Therapies.

About Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular disease and a leading genetic cause of infant death.3,5Caused by the lack of a functional SMN1 gene, the most severe forms of SMA result in the rapid and irreversible loss of motor neurons, affecting muscle functions including breathing, swallowing and basic movement.1Severity varies across a spectrum of types corresponding to the number of copies of the back-up SMN2 gene.6The majority of patients with two copies of SMN2 develop Type 1, the most common form accounting for 60 percent of cases.4,7,8Type 1 is severe and, left untreated, leads to death or the need for permanent ventilation by the age of two in more than 90 percent of cases.3,5Most patients with three copies of SMN2 develop Type 2, accounting for about 30 percent of cases.4,8 Loss of motor neurons cannot be reversed, so it is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression.9,10

IndicationandImportant Safety InformationforZOLGENSMA(onasemnogeneabeparvovec-xioi)

Whatis ZOLGENSMA?

ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into a vein. ZOLGENSMA was not evaluated in patients with advanced SMA.

WhatisthemostimportantinformationIshouldknowaboutZOLGENSMA?

Whatshould Iwatchforbeforeandafterinfusion withZOLGENSMA?

WhatdoIneedtoknowaboutvaccinationsandZOLGENSMA?

DoIneedtotakeprecautionswiththepatient'sbodilywaste?

Temporarily, small amounts of ZOLGENSMA may be found in the patient's stool. Use good hand hygiene when coming into direct contact with bodily waste for 1 month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.

Whatarethepossible orlikelysideeffectsof ZOLGENSMA?

The most common side effects that occurred in patients treated with ZOLGENSMA were elevated liverenzymesandvomiting.

The safety information provided here is not comprehensive. Talk to the patient's doctor about anysideeffects thatbotherthepatientorthatdon'tgoaway.

You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 orwww.fda.gov/medwatch,orNovartis GeneTherapiesat833-828-3947.

Please see the Full Prescribing Information.

2022 Novartis Gene Therapies, Inc.

US-ZOL-22-0136

References

MEDIA CONTACT: Katie Lesch, [emailprotected]

SOURCE Novartis Gene Therapies

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Health Alert for Parents: How one boy is thriving following treatment with a gene therapy after receiving an early diagnosis - PR Newswire

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces data presentations at the 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Edinburgh, United Kingdom, taking place October 11-14, 2022. Presentations will include clinical data from Rockets lentiviral vector (LV)-based gene therapy programs for Leukocyte Adhesion Deficiency-I (LAD-I), Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD). Donald B. Kohn, MD, Distinguished Professor of Microbiology, Immunology & Molecular Genetics, Pediatrics, and Molecular & Medical Pharmacology at University of California, Los Angeles (UCLA) and Director of the UCLA Human Gene and Cell Therapy Program, will also give an Invited Talk incorporating previously disclosed data from the RP-L201 trial for LAD-I.

Positive Updated Safety and Efficacy Data from Phase 2 Pivotal Trial for Fanconi Anemia (FA)

The poster and presentation include updated safety and efficacy data from the Phase 2 pivotal trial of RP-L102, Rockets ex-vivo lentiviral gene therapy candidate for the treatment of FA.

Positive Top-line Clinical Data from Phase 2 Pivotal Trial for Severe Leukocyte Adhesion Deficiency-I (LAD-I)

The oral presentation includes previously disclosed efficacy and safety data at three to 24 months of follow-up after RP-L201 infusion for all patients and overall survival data for seven patients at 12 months or longer after infusion. RP-L201 is Rockets ex-vivo lentiviral gene therapy candidate for the treatment of severe LAD-I.

Interim Data from Ongoing Phase 1 Trial for Pyruvate Kinase Deficiency (PKD)

The poster and presentation include previously disclosed safety and efficacy data from the Phase 1 trial of RP-L301, Rockets ex-vivo lentiviral gene therapy candidate for the treatment of PKD.

Details for Rockets Invited Talk and poster presentations are as follows:

Title: Interim Results from an ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)Session: Clinical Trials (Plenary 2)Presenter: Donald B. Kohn, MD - University of California, Los Angeles, Distinguished Professor of Microbiology, Immunology & Molecular Genetics (MIMG), Pediatrics, and Molecular & Medical Pharmacology; Director of the UCLA Human Gene and Cell Therapy ProgramSession date and time: Wednesday, 12 October at 11:10-13:15 BSTLocation: Edinburgh International Conference Centre (EICC)Presentation Number: INV20

Title: Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Results from Global RP-L102 Clinical TrialsSession: Poster Session 1Presenter: Julin Sevilla MD, PhD - Fundacin para la Investigacin Biomdica, Hospital Infantil Universitario Nio JessSession date and time: Wednesday, 12 October at 19:30-21:00 BSTLocation: Edinburgh International Conference Centre (EICC)Poster Number: P139

Title: Preliminary Conclusions of the Phase I/II Gene therapy Trial in Patients with Fanconi Anemia-ASession: Blood Diseases: Haematopoietic Cell DisordersPresenter: Juan Bueren, PhD - Unidad de Innovacin Biomdica, Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT)Session date and time: Thursday, 13 October at 15:30-17:30 BSTLocation: Edinburgh International Conference Centre (EICC)Presentation Number: INV41

Title: Interim Results from an Ongoing Global Phase 1 Study of Lentiviral-Mediated Gene Therapy for Pyruvate Kinase DeficiencySession: Poster Session 2Presenter: Jos Luis Lpez Lorenzo, MD, Hospital Universitario Fundacin Jimnez DazSession date and time: Thursday, 13 October at 17:30-19:15 BSTLocation: Edinburgh International Conference Centre (EICC)Poster Number: P128

Abstracts for the presentations can be found online at: https://www.esgct.eu/.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Pyruvate Kinase Deficiency

Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 4,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was in-licensed from the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT), Centro de Investigacin Biomdica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacin Sanitaria de la Fundacin Jimnez Daz (IIS-FJD).

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rockets future expectations, plans and prospects, including without limitation, Rockets expectations regarding its guidance for 2022 in light of COVID-19, the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Danon Disease, the expected timing and data readouts of Rockets ongoing and planned clinical trials, the expected timing and outcome of Rockets regulatory interactions and planned submissions, Rockets plans for the advancement of its Danon Disease program and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rockets ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rockets dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rockets Annual Report on Form 10-K for the year ended December 31, 2021, filed February 28, 2022 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 29th Annual Congress of the European Society of Gene...

ST. LOUIS--(BUSINESS WIRE)--M6P Therapeutics (M6PT or the Company), a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage disorders (LSDs), today announced a poster presentation titled, A novel AAV9 gene therapy for producing -glucocerebrosidase enzyme with high mannose 6-phosphate content to treat Gaucher disease, by Lin Liu, Ph.D., Director, R&D of M6P Therapeutics, at the 29th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). The Congress takes place in-person and virtually in Edinburgh, Scotland from Oct. 11-14, 2022 at the Edinburgh International Conference Centre.

The data from this preclinical study illustrate the potential of our AAV-GBA-S1S3 gene therapy candidate for the treatment of Gaucher disease, including neuronopathic phenotypes, said Pawel Krysiak, President and CEO of M6P Therapeutics. M012 is part of our pipeline of candidates leveraging a first-in-class co-expression S1S3 platform technology for the treatment of lysosomal storage disorders. Using our technology platform, we enhance mannose 6-phosphate content on lysosomal enzymes for both enzyme replacement and gene therapies, leading to improved enzyme uptake across target tissues.

Gaucher disease is a rare inherited metabolic disorder of defective lipid catabolism caused by deficient -glucocerebrosidase (GCase) encoding by the GBA1 gene resulting in accumulation of glycosphingolipids in the periphery and central nervous system (CNS). Multiple recombinant human GCase enzyme replacement therapies have been approved to treat Gaucher disease type 1, but there are no effective treatments to address the neuronopathic manifestations for Gaucher disease types 2 and 3. Gene therapy could be a potential therapeutic approach to treat all three types of Gaucher disease.

"In this study, gene copy and transcriptome analysis showed that the dosed novel M012 AAV vector enabled transduction of peripheral tissues and various regions of brain in wild-type mice post-intravenous injection, said Dr. Liu. We observed increased GCase enzyme activity with enhanced CI-MPR affinity in peripheral tissues of treated animals and noted strong GCase protein signal with broad distribution detected in M012 AAV vector-treated brain samples. These results strongly suggest that M012 AAV enables effective GCase cross-correction in the CNS. We look forward to sharing additional updates and data as the program progresses.

Presentation highlights from the poster include:

About M6P TherapeuticsM6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted enzyme replacement and gene therapies for lysosomal storage disorders (LSDs). M6P Therapeutics proprietary co-expression S1S3 platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both enzyme replacement and gene therapies, leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product. This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases. M6P Therapeutics team, proven in rare diseases drug development and commercialization, is dedicated to fulfilling the promise of enzyme replacement and gene therapies by harnessing the power of protein phosphorylation using its co-expression S1S3 platform. M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community. For more information, please visit: http://www.m6ptherapeutics.com.

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M6P Therapeutics Presents Novel AAV Gene Therapy Approach for the Treatment of Gaucher Disease at the ESGCT 29th Annual Congress - Business Wire

Breaking News

Approvals of Zynteglo and Skysona mean that Lonzas Houston site now supports three commercial CGT products.

10.11.22

Lonza Houston is dedicated to contract cell and gene therapy development and manufacturing. The facility successfully passed Food and Drug Administration (FDA) pre-Licensing inspections for the viral vector manufacturing or for the cell therapy manufacturing of three commercial cell and gene therapy products that were subsequently approved by the FDA for commercial use in 2021 and 2022.

Lonzas Cell & Gene Technologies (CGT) business, the contract development and manufacturing business unit of its Cell & Gene division, has developed an NPI process to support customers on their journey from early-stage development to commercialization. It provides a roadmap and a systematic approach to development and manufacturing, ensuring necessary quality standards are met for tech transfers, cGMP manufacturing and pre-approval inspection readiness. This NPI process has been designed to support more CGT customers in reaching commercialization with their new and innovative products.

"The two new approvals represent an important milestone for bluebird bio and the patients who can now benefit from these therapies, said Alberto Santagostino, senior, head of cell and gene technologies. The successful approvals reflect our colleagues dedication to supporting our customers in bringing these cell and gene therapies to market. Opened less than five years ago, our Houston site is now manufacturing three commercial cell and gene therapies. We look forward to supporting more customers on their path to commercialization as we continue on our journey towards wider cell and gene therapy adoption."

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Two Cell and Gene Therapies Manufactured at Lonza Houston Reach FDA Approval - Contract Pharma

Exopharms technology has put the company in a leadership-position in both nave exosomes and engineered exosomes as it progresses to a product-first company.

Its been a challenging yet productive two years for the Exopharm (ASX:EX1) team.

The company has made significant progress over this period and now owns a tool chest of 7 important exosome manufacturing technologies, as well as well identifying two new in-house GM (genetic medicine) product programs.

At the centre of Exopharms is its proprietary manufacturing technologies, which enable the company to develop its own exosome-medicine products and harness their unique drug-delivery capabilities.

Preliminary results from a recently completed study detected no immunogenicity or toxicity of exosomes manufactured by Exopharm, which means they are suitable for clinical pursuit.

The study also helped to validate Exopharms exosomes as a safe drug-delivery chassis meaning they could be used as safe delivery vehicles inside a persons body to deliver drug doses at high frequencies.

Separate earlier clinical trials with the company Plexaris product also demonstrated safety and efficacy in wound healing using Exopharm-produced exosomes.

These results have now put Exopharm in a leadership-position in both nave exosomes and engineered exosomes.

Exosomes are naturally occurring nano-vesicles that are secreted in huge numbers by cells throughout the human body, and are a natural and powerful way that cells communicate and coordinate.

Within the last 7 years, researchers have envisaged using exosomes as delivery vehicles inside a persons body like programmable, targeted and safe nano-sized drones.

Drugs that had delivery problems, like mRNA, DNA and some small molecules, could be packaged into exosomes at a bioprocessing facility to produce modern medicines which were safer, more tolerable, and more specific to the target tissue/destination than their cargo alone.

Over the last few years, Exopharm had worked on developing the technology to manufacture these exosomes, and that led the market to value it as a platform technology company.

But now, the technology has developed significantly and Exopharm believes that market perception is outdated.

Exopharm emphasised that it is now a product-first company after announcing two GM programs under development.

The Exopharm team has assessed hundreds of potential exosome products over the past 15 months using strict assessment criteria and detailed commercial scoping.

From that work now comes two new programs with several potential product variants in each program.

The first lead program is treating Cystic Fibrosis (CF) using exosome-based additive CFTR mRNA gene therapy and nebuliser delivery to lungs.

CF is the most common autosomal recessive disease with >100,000 CF patients worldwide (around 40,000 in US) and the median age of death is 32 years. Lung disorder is the main cause of morbidity and mortality.

There is no cure for cystic fibrosis, but treatment can ease symptoms, reduce complications and improve quality of life.

Current treatment options include antibiotics, anti-inflammatory medications, mucus-thinners, bronchodilators, oral pancreatic enzymes and CFTR modulators.

Exopharm however believes that additive gene therapy is ideal for CF compared to some other genetic diseases.

The Exo-RNA approach, which is gene therapy delivered via exosomes, has several advantages which includes avoiding the use of viral vectors.

The Exo-RNA gene therapy approach also poses no risk of potential insertion into the host genome as the exosomes loaded with CFTR mRNA are formulated to be administered via a nebuliser (inhaler).

This approach is expected to be more efficacious and cost-effective, with no direct competitors identified.

The CF market is predicted to reach US$31 billion by 2027 with 24% compound annual growth rate (CAGR), up from US$5 billion in 2019.

Elastin is a natural molecule in our bodies produced from the ELN gene. Elastin imparts elasticity in various tissues such as skin, lungs, blood vessels and fascia.

With age and exposure to smoke, levels of elastin decrease and medical and aesthetic issues arise from that elastic deficiency.

An additive gene therapy using ELN mRNA would produce additional Elastin in the body and could bring the elastic tissue structure back to normal.

Exopharm has identified several main product opportunities in this program including Chronic obstructive pulmonary disease (COPD) such as pulmonary emphysema and chronic bronchitis.

Destruction of elastin or abnormalities in elastic fibre assembly are major factors in emphysema and COPD, and an estimated 3.1 million Americans have been diagnosed with emphysema and around 11.2 million people in the country have COPD.

Tobacco use is the number one factor in the progression of COPD, along with pollution, infections and genetic factors.

Other potential treatments in this program also include skin conditions and aesthetic dermatology.

Exopharm will also explore hypertension and arterial stiffness, scar prevention, and photoaging in its Elastin program.

Exopharm says these two programs have been selected after considering their potential future commercial value, and as a way to showcase exosomes as a non-viral nanoparticle chassis to overcome drug-delivery problems that GMs face.

With these two lead programs, the company believes its strategy and future-direction is clear and promising.

Exopharm believes there is an emerging opportunity to treat many medical problems with Genetic Medicines to use exosomes as the drug-delivery chassis for potentially hundreds of new products.

Exopharm can also profit from this opportunity by making its exosome technologies available to others on commercial terms.

The company can also generate revenue by developing a small number of its own products in order to build shareholder-value along the way.

This article was developed in collaboration with Exopharm, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.

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Exopharm identifies two new lead programs as exosomes gain prominence in gene therapy - Stockhead

DUBLIN--(BUSINESS WIRE)--The "Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market by Scale of Operation, Type of Vector, Application Area, Therapeutic Area, and Geographical Regions: Industry Trends and Global Forecasts, 2022-2035" report has been added to ResearchAndMarkets.com's offering.

With the increasing number of cell and gene therapies being developed and launched for a wide range of therapeutic areas, these modalities are on their way to become one of the highest valued markets in the biopharmaceutical domain. In fact, in 2021, cell and gene therapy developers raised capital worth more than USD 20 billion, registering an increase of 19% from the amount raised in 2020 (~USD 17 billion).

It is worth highlighting that, in February 2022, the USFDA approved second CAR-T therapy, CARVYKTIT, developed by Johnson and Johnson, which can be used for the treatment of relapsed or refractory multiple myeloma. Additionally, close to 1,500 clinical trials are being conducted, globally, for the evaluation of cell and gene therapies. Over time, it has been observed that the clinical success of these therapies relies on the design and type of gene delivery vector used (in therapy development and / or administration). At present, several innovator companies are actively engaged in the development / production of viral vectors and / or non-viral vectors for cell and gene therapies.

In this context, it is worth mentioning that, over the past few years, multiple viral vector and non-viral vector based vaccine candidates have been developed against COVID-19 (caused by novel coronavirus, SARS-CoV-2) and oncological disorders; this is indicative of lucrative opportunities for companies that have the required capabilities for viral vector manufacturing and gene therapy manufacturing.

The viral vector manufacturing and non-viral vector manufacturing landscape features a mix of industry players (well-established companies, mid-sized firms and start-ups / small companies), as well as several academic institutes. It is worth highlighting that several companies that have the required capabilities and facilities to manufacture vectors for both in-house requirements and offer contract services (primarily to ensure the optimum use of their resources and open up additional revenue generation opportunities) have emerged in this domain.

Further, in order to produce more effective and affordable vectors, several stakeholders are integrating various novel technologies; these technologies are likely to improve the scalability and quality of the resultant therapy. In addition, this industry has also witnessed a significant increase in the partnership and expansion activities over the past few years, with several companies having been acquired by the larger firms. Given the growing demand for interventions that require genetic modification, the vector and gene therapy manufacturing market is poised to witness substantial growth in the foreseen future.

Scope of the Report

The "Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market (5th Edition) by Scale of Operation (Preclinical, Clinical and Commercial), Type of Vector (AAV Vector, Adenoviral Vector, Lentiviral Vector, Retroviral Vector, Plasmid DNA and Others), Application Area (Gene Therapy, Cell Therapy and Vaccine), Therapeutic Area (Oncological Disorders, Rare Disorders, Neurological Disorders, Sensory Disorders, Metabolic Disorders, Musco-skeletal Disorders, Blood Disorders, Immunological Diseases, and Others), and Geographical Regions (North America, Europe, Asia Pacific, MENA, Latin America and Rest of the World): Industry Trends and Global Forecasts, 2022-2035" report features an extensive study of the rapidly growing market of vector and gene therapy manufacturing, focusing on contract manufacturers, as well as companies having in-house manufacturing facilities.

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

4. VIRAL VECTOR AND GENE THERAPY MANUFACTURERS (INDUSTRY PLAYERS): MARKET LANDSCAPE

5. PLASMID DNA AND GENE THERAPY MANUFACTURERS (INDUSTRY PLAYERS): MARKET LANDSCAPE

6. VECTOR AND GENE THERAPY MANUFACTURERS (NON-INDUSTRY PLAYERS): MARKET LANDSCAPE

7. VECTOR AND GENE THERAPY MANUFACTURING TECHNOLOGIES: MARKET LANDSCAPE

8. COMPANY COMPETITIVENESS ANALYSIS

9. VECTOR AND GENE THERAPY MANUFACTURERS IN NORTH AMERICA

10. VECTOR AND GENE THERAPY MANUFACTURERS IN EUROPE

11. VECTOR AND GENE THERAPY MANUFACTURERS IN ASIA-PACIFIC

12. OTHER KEY PLAYERS

13. RECENT PARTNERSHIPS

14. RECENT EXPANSIONS

15. STRATEGIC PARTNER ANALYSIS

16. EMERGING VECTORS

17. KEY INSIGHTS

18. COST PRICE ANALYSIS

19. CAPACITY ANALYSIS

20. DEMAND ANALYSIS

21. MARKET SIZING AND OPPORTUNITY ANALYSIS

22. PORTER'S FIVE FORCES ANALYSIS

23. KEY DRIVERS AND CHALLENGES

24. SURVEY ANALYSIS

25. CONCLUDING REMARKS

26. EXECUTIVE INSIGHTS

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/wg8tme

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Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market Report 2022 - ResearchAndMarkets.com - Business Wire

A biotechnology company launched Wednesday by life sciences venture firm ATP is the latest startup to debut with a new twist on genetic editing.

With $50 million in funding, Boston-based Ascidian Therapeutics claims its RNA exon editing approach could match the durability of gene therapy while avoiding some of the risks that come with editing DNA.

Its platform is designed to correct for mutations in exons the regions of DNA that contain information needed to make proteins. Ascidian aims to do this by replacing mutated exons with functional RNA copies as DNA is being converted into its chemical cousin.

The company will first target a genetic eye condition called Stargardt disease, which is the most common form of inherited macular degeneration and results in vision loss.

According to Ascidian, its technology can fix genetic errors that other editing approaches cant, and can be applied to widely varied genes. Its lead program can replace more than 20 exons at a time, said Romesh Subramanian, Ascidians CEO.

We are changing chapters in a book rather than whiting-out one letter at a time, Subramanian, said in an interview with BioPharma Dive. Subramanian came to Ascidian from Dyne Therapeutics, a biotech he founded and led as CEO. He previously founded RNA specialist Translate Bio, which was bought by Sanofi last year.

Subramanian claims that Ascidians approach, by focusing on RNA, maintains genome integrity and thereby sidesteps concerns around off-target edits. His company also doesnt rely on foreign enzymes to work, potentially easing immunogenicity risks, he added.

Along with Stargardt disease, Ascidian is looking at other eye conditions, neurological disorders and rare diseases. Subramanian declined to disclose how many drug research programs Ascidian plans to roll out.

Ascidians name is derived from a class of ocean-dwelling invertebrate creatures, which are sometimes known as sea squirts. These creatures use RNA trans-splicing to alter the RNA messengers used by their cells, a process that Ascidian plans to leverage to rewrite RNA for treating disease.

Ascidian is not ATPs first foray into genetic medicine. Last year, Ascidian co-founder and ATP venture partner Michael Ehlers, a former Biogen executive,launched a startup called Intergalactic Therapeuticsthat focuses on non-viral gene therapy. ATP has also built a company called Replicate, which is developing another kind of RNA medicine.

We think the RNA space is a big way of manipulating biology and treating disease across the board, and this approach we've taken to Ascidian defines a new class of RNA therapeutics, Ehlers said.

The company expects to spend the rest of 2022 and 2023 on pre-clinical studies for its lead program, along with developing proof of concept for other candidates targeting neurological and neuromuscular diseases.

Gene editing research was catalyzed by the discovery of CRISPR, which has now been extended and adapted to support several different gene editing technologies. But biotech companies are also exploring RNA editing, which in part appeals to scientists because it doesnt change the underlying DNA.

It has drawn in larger drugmakers, too: Roche and Eli Lilly have recently formed partnerships with Shape Therapeutics and ProQR Therapeutics, respectively, to develop treatments for a wide variety of diseases.

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Ascidian starts up with $50M and a twist on RNA editing - BioPharma Dive

Cystic fibrosis (CF) is the most common, fatal genetic disease in the United States. About 30,000 people in the United States have the disease. CF causes the body to produce thick, sticky mucus that clogs the lungs, leads to infection, and blocks the pancreas, which stops digestive enzymes from reaching the intestine where they are required in order to digest food.

Mutations in a single genethe Cystic Fibrosis Transmembrane Regulator (CFTR) genecause CF. The gene was discovered in 1989. Since then, more than 900 mutations of this single gene have been identified.

In normal cells, the CFTR protein acts as a channel that allows cells to release chloride and other ions. But in people with CF, this protein is defective and the cells do not release the chloride. The result is an improper salt balance in the cells and thick, sticky mucus. Researchers are focusing on ways to cure CF by correcting the defective gene or correcting the defective protein.

Gene therapy offers great promise for life-saving treatment for CF patients since it targets the cause of CF rather than just treating symptoms. Gene therapy for CF had its start in 1990 when scientists successfully corrected faulty CFTR genes by adding normal copies of the gene to laboratory cell cultures.

In 1993, the first experimental gene therapy treatment was given to a patient with CF. Researchers modified a common cold virus to act as a delivery vehicleor vectorcarrying the normal genes to the CFTR cells in the airways of the lung.

Subsequent studies have tested other methods of gene delivery, such as fat capsules, synthetic vectors, nose drops, or drizzling cells down a flexible tube to CFTR cells lining the airways of the lungs. Researchers are now testing aerosol delivery using nebulizers.

But finding the best delivery system for transporting normal CFTR genes is only one problem that scientists must solve to develop an effective treatment for CF. Scientists must also determine the life span of affected lung cells, identify the parent cells that produce CFTR cells, and find out how long treatment should last and how often it needs to be repeated.

The first cystic fibrosis gene therapy experiments involved lung cells because these cells are readily accessible and because lung damage is the most common, life-threatening problem in CF patients. But scientists hope that the technologies being developed for lung cells will be adapted to treat other organs affected by CF.

Another research breakthrough offers a promising approach to treating cystic fibrosis. Researchers at the University of Washingtons Genome Center and at PathoGenesis Corporation have completed a genetic map for thePseudomonas aeruginosabacterium. This bacterium is the most common cause of chronic and fatal lung infections for people with CF. Scientists hope to use their knowledge of this bacteriums genetic sequence to develop innovative drugs for treating infections caused byP. aeruginosa.

As the amount of information about CF grows, scientists have recognized the need to share their research findings. To facilitate this sharing of information, the Cystic Fibrosis Foundation fundsCystic Fibrosis Foundation Therapeutics(CFFT) located at The University of North Carolina Chapel Hill. The center is becoming a repository for data derived from gene expression studies. By pooling information, researchers hope to accelerate the process of finding a cure for CF.

What Do We Know About Gene Therapy and Cystic Fibrosis? was originally published by the National Institutes of Health.

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What Do We Know About Gene Therapy and Cystic Fibrosis? - The Epoch Times

CSafe joins BioLife's global partner network of cold chain solution providers using the evo cold chain management platform

MONROE, Ohio and BOTHELL, Wash., Oct. 11, 2022 /PRNewswire/ -- CSafe, one of the largest active and passive temperature-controlled shipping solutions providers for pharmaceuticals, and BioLife Solutions, Inc. (NASDAQ: BLFS), a leading supplier of class-defining bioproduction tools and services for the cell and gene therapy ("CGT") and broader biopharma markets, announced today a new partnership to provide a combined global service network to support CGT products, with a focus on enhanced reliability, security and quality.

CSafe joins BioLife's global partner network of cold chain solution providers serving the CGT market. BioLife expects to support 10,000-12,000 evo shipments of CGT starting materials and manufactured doses over the next 12 months. The new partnership will provide expanded supply chain options for any CGT product, at any stage of development, from early clinical-phase trials to global commercial scale.

CSafe will bring even more of its highly reliable global reach to the CGT sector through this new alliance. Operating from 50+ service centers worldwide, the company is a proven, trusted partner for biopharmaceutical manufacturers seeking to navigate supply chain complexity, with a track record that includes shipping solutions for six billion doses of a COVID-19 vaccine.

"CSafe is honored to partner with the team at BioLife Solutions, whose range of CGT bioproduction tools and services is peerless in the market. We are excited to merge these exceptional tools and services with our expertise in global, reliable scaled delivery," said CSafe CEO Patrick Schafer. "CGT is hitting its stride and needs global support. We know how important these therapies are to patients everywhere, and it's our mission at CSafe to protect every shipment."

"We're excited to work with a global partner with a strong history of reliability and performance and a deep dedication to innovative therapies," said BioLife Solutions CEO Mike Rice. "We have the best LN2 technology and cGMP storage facilities in the market, in addition to our other world-class CGT solutions, and we are confident our collaboration with CSafe will extend our reach and result in even more reliability and real-time service for CGT partners."

Media Contacts:April Lynch+1 937-245-6479alynch@csafeglobal.comFor BioLife Solutions:

At the CompanyTroy WichtermanChief Financial Officer(425) 402-1400twichterman@biolifesolutions.com

InvestorsLHA Investor RelationsJody Cain(310) 691-7100jcain@lhai.com

About CSafe

CSafe offers the most comprehensive suite of thermal shipping solutions for pharmaceutical cold chain shipping needs around the world. With a "patient-first" focus, deep industry expertise and commitment to innovation, CSafe continues to deliver industry-leading products in both the active and passive segments. CSafe is the only provider with an end-to-end portfolio including active and passive bulk air cargo, parcel, cell and gene and specialty last-mile use cases with the ability to meet the complete range of pharma cold-chain shipping requirements with industry-leading quality and reliability. Any size, any duration and any temperature CSafe is At the Heart of Your Cold Chain.csafeglobal.com

About BioLife Solutions

BioLife Solutions is a leading supplier of class-defining bioproduction tools and services for the cell and gene therapy and broader biopharma markets. Our tools portfolio includes our proprietary CryoStor and HypoThermosol biopreservation media for shipping and storage, the ThawSTAR family of automated, water-free thawing products, evo cold chain management system, high capacity cryogenic storage freezers, Stirling Ultracold mechanical freezers, SciSafe biologic storage services, and Sexton Biotechnologies cell processing tools. For more information, please visit http://www.biolifesolutions.com, http://www.scisafe.com, http://www.stirlingultracold.com, or http://www.sextonbio.com and follow BioLife on Twitter.

Caution Regarding Forward Looking Statements

Statements in this press release that are not historical facts are "forward-looking statements" that may involve material risks and uncertainties. The company wishes to caution readers not to place undue reliance on such forward-looking statements. All such statements speak only as of the date made.

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CSafe and BioLife Solutions, Inc. announce partnership to expand supply chain solutions for the cell and gene therapy market - PR Newswire APAC - PR...

Covina, Oct. 11, 2022 (GLOBE NEWSWIRE) -- Genomics is the study of all of persons gene. Genomics play role in health and disease. Genomics are widely used in cancer care treatment for diagnosing and treating cancer disease. Structural Genomics and Functional Genomics are two types of Genomics.Gene Therapy, Gene Discovery, Personalized Medicine, Pharmacogenetics & Targeted Therapy, Metagenomics, Mitochondrial Genomics, Pharmacogenomics are variety of applications in genomics. Metagenomics has become the important application in genomics. The newer technique genome editing is used in gene therapy. Genome editing help to introduce gene-editing tools which can able to change existing DNA in cell. Genomics are used in drug discovery due to their properties like high-throughput sequencing & characterization of expressed human genes. Genomics has allowed effective preventive measures, change in drug research strategy and development process in drug discovery due to knowledge about human genes and their functions. A complete human genome contains about 3 billion base pairs of DNA. Pharmacogenomics is the study of genes and their functions to develop safe medications which are effective and can be prescribed based on persons genetic makeup. Pharmacogenomics choose the drug and drug doses that are effective for that particular person by using genetic information about that person. Pharmacogenomics helps in improving patient safety, health care costs and drug efficiency. Single nucleotide variant (SNV) panels are used in pharmacogenetics. Genomics helps to reveal the abnormalities in genes which has drived the development and growth of different types of cancer.Study of cancer genome has improved in understanding the biology of cancer which has enabled to discover new methods for diagnosing & treating the disease. The importance of Genomics in cancer care has provided to discover new drug development and effective treatment in diagnosing and treating the disease which has driven positive impact on target market growth.

The reportGlobal Genomics in Cancer Care Market, By Type (Structural Genomics, Functional Genomics), By Application (Gene Therapy, Gene Discovery, Personalized Medicine, Pharmacogenetics & Targeted Therapy, Metagenomics, Mitochondrial Genomics, Pharmacogenomics, and Others), By End-User (Research Institute, Hospitals, Academic Research Institutes, Diagnostic Centers, and Others) andBy Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa) - Trends, Analysis and Forecast till 2032

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Increase in cancer disease, rising emergence of clinical relievance in genomic medicine, recent advancement in genomics, newly developed technology like next-generation sequencing has given rise in use ofGenomics in Cancer Care. Wide variety of applications in Gene Therapy, Gene Discovery, Personalized Medicine, Pharmacogenetics & Targeted Therapy, Metagenomics, Mitochondrial Genomics, Pharmacogenomics has fueled the target market growth. Rising awareness in individual who are pertaining to cancer genomics, rapid growth in biotechnology industries, research institutes, diagnostic centers is expected to have positive impact on Genomics in Cancer Care market. Importance of Genomics in cancer care has enabled to provide effective treatment, new drug development, diagnosing and treating disease which has enhanced the target market growth.As a result, market competition is intensifying, and both big international corporations and start-ups are vying to establish position in the market.

Browse 60 market data tables* and 35figures* through 140 slides and in-depth TOC onGlobal Genomics in Cancer Care Market, By Type (Structural Genomics, Functional Genomics), By Application (Gene Therapy, Gene Discovery, Personalized Medicine, Pharmacogenetics & Targeted Therapy, Metagenomics, Mitochondrial Genomics, Pharmacogenomics, and Others), By End-User (Research Institute, Hospitals, Academic Research Institutes, Diagnostic Centers, and Others) andBy Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa) - Trends, Analysis and Forecast till 2032

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Key Market Insights from the report:

GlobalGenomics in Cancer CareMarketaccounted for US$ 16.1 Bn in 2022 and is estimated to be US$ 72.61 Bn by 2032 and is anticipated to register a CAGR of 16.3%.TheGlobalGenomics in Cancer CareMarketis segmented based on Type, Application, End-User and Region.

Competitive Landscape & their strategies ofGlobalGenomics in Cancer Care Market:

The prominent players operating in theGlobalGenomics in Cancer CareMarketincludes,Pacific Biosciences Inc., Abbott Molecular Oxford Gene Technology, Roche Diagnostics, Bio-Rad Labs, Illumina Inc., Quest Diagnostics, Beckman Coulter Inc., Intrexon Bioinformatics Germany GmbH, Agilent Technologies, PerkinElmer, Danaher Corporation, Cancer Genetics Inc., Thermo Fisher Scientific Inc., and others.

The market provides detailed information regarding the industrial base, productivity, strengths, manufacturers, and recent trends which will help companies enlarge the businesses and promote financial growth. Furthermore, the report exhibits dynamic factors including segments, sub-segments, regional marketplaces, competition, dominant key players, and market forecasts. In addition, the market includes recent collaborations, mergers, acquisitions, and partnerships along with regulatory frameworks across different regions impacting the market trajectory. Recent technological advances and innovations influencing the global market are included in the report.

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Genomics in Cancer Care Market is estimated to be US$ 72.61 billion by 2032 with a CAGR of 16.3% during the forecast period 2032 - By PMI -...