Monthly Archives: August 2021

After Bluebird Bio (BLUE) announced plans this week to end commercial operations in Europe, the move is raising fresh questions about the extent to which selling such pricey treatments is viable for small biotechs in one of the worlds biggest markets.

In explaining the move, the biotech complained that it has become a losing proposition trying to convince European Union member states to make large upfront payments for therapies that can save health care systems much higher costs later. So instead, the company is shifting its focus to the U.S., where it is more likely to get reimbursed at the desired prices.

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As Bluebird backpedals from Europe, gene therapy players face hurdles - STAT

- Founder and CEO Lindsay Androski previously served as a federal prosecutor and an executive at Roivant Sciences

- MIT spinout Sunflower Therapeutics, focused on improving access to medicines through improvements in complex manufacturing, is Roivant Social Ventures' first investment, building upon prior funding from the Bill and Melinda Gates Foundation

- Nature Gene Therapy publication spotlights efforts to expand gene therapy access in India and Africa

NEW YORK, Aug. 17, 2021 /PRNewswire/ -- Roivant Social Ventures (RSV), a not-for-profit social impact organization launched out of Roivant Sciences, today unveiled its executive team and announced its inaugural partnership with Sunflower Therapeutics, a biopharmaceutical company developing simplified manufacturing methods for therapeutic proteins. RSV also announced the publication in Nature Gene Therapy of the Global Gene Therapy Working Group's initiative to expand gene therapy access in Africa and India.

Executive Team

Lindsay Androski, JD, MBA, CFA is the Founder, President and CEO of Roivant Social Ventures. Ms. Androski sits on the MIT board of trustees, the Visiting Committee for the MIT Department of Biology, and the Visiting Committee for MIT Sponsored Research. She also serves as President of Incubate, an organization which educates policymakers on the role of venture capital in the biopharma industry. Ms. Androski joined the founding team at Roivant Sciences in order to build and lead the transaction team primarily responsible for in-licensing and acquiring over 40 therapeutics programs. Earlier in her career she served as an Assistant U.S. Attorney in the Eastern District of Virginia, where she investigated and prosecuted high-profile cybercrime and national security cases.

Rachel Rubin, who serves as Vice President of Programs at RSV, also joins from Roivant Sciences, where she oversaw launch and business operations for several healthcare technology Vants. Prior to Roivant, Ms. Rubin worked at the Michael J. Fox Foundation for Parkinson's Research and spent several years as an investor.

Story continues

Saranna Biel-Cohen joins RSV as Vice President of Strategic Development. Ms. Biel-Cohen previously served as Executive Director of the Hermitage Museum Foundation, where she facilitated major funding and programming partnerships, including with the United States federal government and leading corporations.

Partnership with Sunflower Therapeutics

RSV's first partnership, which includes a financial investment and company incubation assistance, is with Sunflower Therapeutics, a public benefit corporation operationalized in 2019 by CEO Dr. Kerry Love and a team of engineers previously involved in the development of the InSCyT (Integrated and Scalable Cytotechnology) Program in the lab of J. Christopher Love at the Koch Institute for Integrative Cancer Research at MIT.

Sunflower's Daisy System is a multi-product manufacturing platform for distributed production of protein biologics, monoclonal antibodies, and subunit vaccines, which enables efficient production of multiple medicines tailored to a region's specific needs. RSV's funding will be used to deploy the Daisy System for the first time and builds upon prior funding from the Bill & Melinda Gates Foundation in 2019 to develop the Daisy System. The system will be deployed to a non-profit partner serving low-or-middle-income countries (LMICs) and used to bolster local capacity to provide advanced therapeutics to underserved populations and reduce reliance on the global supply chain.

"Roivant Social Ventures is truly a one-of-a-kind impact investor, creating productive partnerships with companies like Sunflower that are focused on increasing global health access and equity," said Dr. Kerry Love, CEO of Sunflower Therapeutics. "I am excited to further development of Sunflower's technologies with the goal of ensuring advanced therapeutics are accessible to all members of the global community."

"We are thrilled to play a role in solving supply chain issues that disadvantage LMICs and inhibit their ability to deliver needed therapeutics to their local populations," said Ms. Androski. "Sunflower Therapeutics embodies the core values of Roivant Social Ventures and is a phenomenal first partner in our mission to use advances in science and technology as vehicles to change the ethical norms of healthcare delivery."

Publication in Nature Gene Therapy

RSV is also a founding member of the Global Gene Therapy Initiative (GGTI), a cross-collaborative group with expertise spanning manufacturing, drug development, patient advocacy, clinical practice, regulatory, and diplomacy, focused on a common goal: to bring gene therapy treatments to patients worldwide. Among other operational pillars, the group is using scaled-down and modular manufacturing systems to deliver gene therapies to patients in Uganda and India by 2024, with an initial focus on HIV and sickle cell therapies.

A publication regarding the group's progress, titled "Towards Access for All: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)", is in press in Nature Gene Therapy, with Lindsay Androski and Alex Popovski as Contributing Authors.

"Gene therapy, and other advanced therapies, offer patients hope far beyond most current treatment methods," said Ms. Androski. "We cannot accept as normal a world where advanced therapies are only available to a small number of patients in the wealthiest countries, and we must prevent this from becoming a reality by investing now in ways to bring these treatments to patients worldwide. We are thrilled to help advance the important work of GGTI."

About Roivant Social VenturesRoivant Social Ventures (RSV) is a not-for-profit social impact organization founded by Roivant Sciences focused on improving healthcare access and outcomes for underserved groups. RSV invests in initiatives to expand worldwide access to cell and gene therapies through simplified manufacturing techniques, facilitates research and development on promising deprioritized programs across the biopharma industry, and encourages continued focus on challenging therapeutic areas where significant unmet medical needs remain. RSV applies Roivant Sciences' unique approach to incubating biotech and healthcare technology companies, including providing non-financial assistance to companies and health initiatives, and seeks to maximize impact through strategic collaborations.

About Sunflower TherapeuticsSunflower Therapeutics, PBC is a unique biotech company with a mission to enable more medicines to reach patients worldwide. Sunflower's goal is to transform access to biologic medicines for patients worldwide by creating novel technologies for development and manufacturing with the whole global community in mind. Using its core technologiesan efficient host, data-driven methods for process development, and novel manufacturing facilitiesthe team aims to create efficient, fast and less costly cycles of development and production for many new innovative patient-focused products. Sunflower's research labs are currently located at LabCentral in Cambridge, Massachusetts.

Cision

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Social Impact Nonprofit Roivant Social Ventures Announces Leadership Team, Inaugural Partnership, and Publication in Nature Gene Therapy - Yahoo...

CALGARY, Alberta, Aug. 17, 2021 (GLOBE NEWSWIRE) Zenith Capital Corp. (Zenith or the Company), a clinical stage biotechnology company focused on the development of novel epigenetic combination therapies for the treatment of cancers, announces a publication in the high-impact Nature Publishing Group journal of Cancer Gene Therapy, titled: Combination of ZEN-3694 with CDK4/6 inhibitors reverses acquired resistance to CDK4/6 inhibitors in ER-positive breast cancer.

This important study highlights the potential for ZEN-3694 in combination with CDK4/6 inhibitors as a new therapeutic option for patients with advanced ER+ breast cancer who developed resistance to current therapies. The study shows that the combination can reverse acquired CDK4/6i resistance by targeting key oncogenic pathways involved in cell cycle regulation, cellular growth, proliferation, apoptosis, inammation, and cellular immune response. Additionally, ZEN-3694 down-regulated markers involved in CDK4/6 inhibitor resistance including CDK6, CCND1, CDK4, MYC, ESR1 and others. Notably, the combination led to the synergistic increase of apoptosis and inhibition of proliferation in the resistant ER+ breast cancer models.

The publication can be viewed using the following LINK.

This compelling data highlights the role of ZEN-3694 in the potential treatment of patients with advanced ER+ breast cancer, leading to the reversal of the acquired resistance to the current standard of care therapies, said Donald McCaffrey, President and Chief Executive Officer of Zenith. As there is a significant unmet need in this population, this study reveals the huge potential in this indication and rationale for a future clinical trial.

About ER+ Breast Cancer

According to Cancer Statistics, 2021, Estrogen receptor-positive breast cancer (ER+) is a prevalent disease with over two million global new cases diagnosed in 2020. Approximately 7080% of women diagnosed with breast cancer are ER+. The standard of care for metastatic ER+ breast cancer patients include selective estrogen receptor modulators, estrogen receptor degraders, aromatase inhibitors, as well as a combination of endocrine therapy with inhibitors of cyclin-dependent kinases (CDK4/6 inhibitors, CDK4/6i). Three approved CDK4/6 inhibitors (palbociclib, ribociclib, and abemaciclib) have been shown to signicantly improve progression-free survival in combination with endocrine therapy for the treatment of metastatic breast cancer patients. Although significant advances have been made in treating metastatic ER+ breast cancer, resistance to therapies invariably occurs over time, making it essential to develop new therapies that address resistance.

About Zenith

Zenith Capital Corp. is a biotechnology investment company originally spun out of Resverlogix Corp. (TSX: RVX) in 2013. Zenith Epigenetics Ltd., a wholly-owned subsidiary of Zenith Capital Corp., is a clinical stage biotechnology company focused on the discovery and development of novel therapeutics for the treatment of cancer and other disorders with significant unmet medical need. Zenith Epigenetics is developing various novel combinations of BET inhibitors with other targeted agents. The lead compound, ZEN-3694, is in clinical development for:

For further information, please contact:

Investor Relations & Communications

Zenith EpigeneticsPhone: 587-390-7865Email: info@zenithepigenetics.com Website: http://www.zenithepigenetics.com

This news release may contain certain forward-looking information as defined under applicable Canadian securities legislation, that are not based on historical fact, including without limitation statements containing the words believes, anticipates, plans, intends, will, should, expects, continue, estimate, forecasts and other similar expressions. In particular, this news release includes forward looking information related to the potential role of ZEN-3694 in combination with CDK4/6 inhibitors in the treatment of advanced ER+ breast cancer and a future clinical trial. Our actual results, events or developments could be materially different from those expressed or implied by these forward-looking statements. We can give no assurance that any of the events or expectations will occur or be realized. By their nature, forward-looking statements are subject to numerous assumptions and risk factors including those discussed in our most recent MD&A which are incorporated herein by reference and are available through SEDAR at http://www.sedar.com. The forward-looking statements contained in this news release are expressly qualified by this cautionary statement and are made as of the date hereof. Zenith disclaims any intention and has no obligation or responsibility, except as required by law, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

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Zenith Epigenetics Announces a Publication in the Journal of Cancer Gene Therapy with Compelling Data for the Treatment of ER-Positive Breast Cancer...

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TSHA - Market Data & News

Taysha Gene Therapies Inc (NASDAQ: TSHA) shares fell 8.98%, or $1.56 per share, to close Monday at $15.82. After opening the day at $17.66, shares of Taysha Gene Therapies fluctuated between $17.66 and $15.75. 102,676 shares traded hands an increase from their 30 day average of 84,431. Monday's activity brought Taysha Gene Therapiess market cap to $599,854,423.

Taysha Gene Therapies is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, Taysha aims to rapidly translate our treatments from bench to bedside. Taysha Gene Therapies has combined its teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, Taysha Gene Therapies leverages its fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives.

Visit Taysha Gene Therapies Incs profile for more information.

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Taysha Gene Therapies (TSHA) falls 8.98% on Strong Volume August 16 - Equities.com

Dublin, Aug. 13, 2021 (GLOBE NEWSWIRE) -- The "Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market by Scale of Operation, Type of Vector, Application Area, Therapeutic Area, and Geographical Regions: Industry Trends and Global Forecasts, 2021-2030" report has been added to ResearchAndMarkets.com's offering.

This 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. The study presents an in-depth analysis of the various firms / organizations that are engaged in this domain, across different regions of the globe.

Over the past few years, a number of advanced therapy medicinal products, including cell and gene therapies, have been developed and approved for the treatment of a variety of disease indications. In fact, as of 2020, close to 15 such therapeutics have received marketing approval across different regions worldwide. Further, over 1,000 clinical trials focused on the evaluation of cell and gene therapies have been registered globally. It is worth noting that the clinical success of these therapies heavily 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 developing / producing viral and / or non-viral vectors for gene therapies. In this context, it is worth mentioning that multiple viral and non-viral vector based vaccine candidates are being developed against the novel coronavirus (SARS-CoV-2). As of January 2021, the WHO revealed that more than 55 such vaccines are under evaluation, while two viral vector based vaccines (AZD1222 and Sputnik V), being developed by AstraZeneca / Oxford University and Gamaleya Research Institute / Acellena Contract Drug Research and Development, have been approved. This is indicative of the lucrative opportunities for companies that have the required capabilities to manufacture vectors and gene therapies.

Vaccine production is a challenging process and dealing with vectors (viral and non-viral) further adds to the complexity. Therefore, outsourcing is a common practice among biopharmaceutical companies when it comes to vector development and / or manufacturing. Several players have developed / are developing versatile technology platforms for designing and manufacturing different types of gene delivery vehicles. Innovation in this segment of the pharmaceutical industry is presently focused on the enhancement of transduction efficiency and improving gene delivery efficiencies. In fact, some vector-related technology providers claim that their proprietary solutions have the ability to enable further improvements in existing genetically modified therapeutic products, and / or optimize affiliated manufacturing processes. The viral / non-viral vectors and gene therapy manufacturing market has also witnessed significant partnership activity in the recent past, especially now that COVID-19 vaccine developers are actively approaching such companies for their services. 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.

Key Questions Answered

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION3.1. Chapter Overview3.2. Viral and Non-Viral Gene Transfer Techniques3.3. Viral Vectors Used in Genetically Modified Therapies3.4. Types of Viral Vectors3.5. Types of Non-Viral Vectors3.6. Gene Delivery using Non-Viral Vectors3.7. Applications of Viral and Non-Viral Vectors3.8. Current / Ongoing Trends in Vector Development / Manufacturing3.9. Vector Manufacturing3.10. Future Perspectives

4. VIRAL VECTOR AND GENE THERAPY MANUFACTURERS (INDUSTRY PLAYERS): MARKET LANDSCAPE4.1. Chapter Overview4.2. Viral Vector and Gene Therapy Manufacturers: Overall Market Landscape4.2.1. Analysis by Year of Establishment4.2.2. Analysis by Company Size4.2.3. Analysis by Location of Headquarters4.2.4. Analysis by Type of Product Manufactured4.2.5. Analysis by Location of Vector Manufacturing Facilities4.2.6. Analysis by Purpose of Production4.2.7. Analysis by Scale of Production4.2.8. Analysis by Location of Headquarters and Scale of Production4.2.9. Analysis by Type of Vector Manufactured4.2.10. Analysis by Scale of Production and Type of Vector Manufactured4.2.11. Analysis by Application Area4.2.12. Information on Production Capacity

5. PLASMID DNA AND GENE THERAPY MANUFACTURERS (INDUSTRY PLAYERS): MARKET LANDSCAPE5.1. Chapter Overview5.2. Plasmid DNA and Gene Therapy Manufacturers: Overall Market Landscape5.2.1. Analysis by Year of Establishment5.2.2. Analysis by Company Size5.2.3. Analysis by Location of Headquarters5.2.4. Heat Map: Analysis by Company Size and Location of Headquarters5.2.5. Analysis by Type of Product Manufactured5.2.6. Analysis by Location of Plasmid DNA Manufacturing Facilities5.2.7. Analysis by Purpose of Production5.2.8. Analysis by Scale of Production5.2.9. Analysis by Application Area5.2.10. Information on Production Capacity

6. VECTOR AND GENE THERAPY MANUFACTURERS (NON-INDUSTRY PLAYERS): MARKET LANDSCAPE6.1. Chapter Overview6.2. Vector and Gene Therapy Manufacturers: Overall Market Landscape6.2.1. Analysis by Year of Establishment6.2.2. Analysis by Location of Vector Manufacturing Facilities6.2.3. Analysis by Purpose of Production6.2.4. Analysis by Scale of Production6.2.5. Analysis by Type of Vector Manufactured6.2.6. Analysis by Scale of Production and Type of Vector Manufactured6.2.7. Analysis by Application Area

7. VECTOR AND GENE THERAPY MANUFACTURING TECHNOLOGIES: MARKET LANDSCAPE7.1. Chapter Overview7.2. Vector and Gene Therapy Manufacturing Technologies7.2.1. Analysis by Type of Technology7.2.2. Analysis by Purpose of Technology7.2.3. Analysis by Scale of Production7.2.4. Analysis by Type of Vector7.2.5. Analysis by Application Area7.2.6. Most Active Players: Analysis by Type of Technology7.3. Concluding Remarks

8. COMPANY COMPETITIVENESS ANALYSIS8.1. Chapter Overview8.2. Methodology and Key Parameters8.3. Vector and Gene Therapy: In-House Manufacturers8.3.1. Players based in North America8.3.2. Players based in Europe8.3.3. Players based in Asia-Pacific and Rest of the World8.4. Vector and Gene Therapy: Contract Manufacturing Organizations8.4.1. Players based in North America8.4.2. Players based in Europe8.4.3. Players based in Asia-Pacific and Rest of the World8.5.. Vector and Gene Therapy: Players Engaged in In-house and Contract Manufacturing8.5.1. Players based in North America8.5.2. Players based in Europe8.5.3. Players based in Asia-Pacific and Rest of the World

9. VECTOR AND GENE THERAPY MANUFACTURERS IN NORTH AMERICA9.1. Chapter Overview9.2. Advanced BioScience Laboratories9.3. Aldevron9.4. Audentes Therapeutics9.5. BioReliance / SAFC Commercial (Merck KGaA)9.6. bluebird bio9.7. Brammer Bio9.8. Emergent BioSolutions9.9. FUJIFILM Diosynth Biotechnologies9.10 MeiraGTx9.11. Other Companies9.11.1. MassBiologics9.11.2. Spark Therapeutics9.11.3. Vigene Biosciences

10. VECTOR AND GENE THERAPY MANUFACTURERS IN EUROPE10.1. Chapter Overview10.2. Biovian10.3. Centre for Process Innovation10.4. Cobra Biologics10.5. FinVector10.6. Kaneka Eurogentec10.7. Lonza10.8. MolMed10.9. Novasep10.10. Orchard Therapeutics10.11. Oxford BioMedica10.12. Richter-Helm10.13. Sanofi (CEPiA, Sanofi Pasteur, Genzyme)10.14. uniQure10.15. Vibalogics10.16. VIVEbiotech10.17. Other Companies

11. VECTOR AND GENE THERAPY MANUFACTURERS IN ASIA-PACIFIC11.1. Chapter Overview11.2. Wuxi AppTec11.2.1. Company Overview11.2.2. Financial Information11.2.3. Manufacturing Facilities11.2.4. Manufacturing Experience11.2.5. Recent Developments and Future Outlook11.3. Other Key Players

12. RECENT PARTNERSHIPS12.1. Chapter Overview12.2. Partnership Models12.3. Vector and Gene Therapy Manufacturing: Recent Partnerships12.3.1. Analysis by Year of Partnership12.3.2. Analysis by Type of Partnership12.3.3. Analysis by Scale of Production12.3.4. Analysis by Type of Vector12.3.5. Analysis by Therapeutic Area12.3.6. Most Active Players: Analysis by Number of Partnerships12.3.7. Geographical Analysis12.3.7.1. Intercontinental and Intracontinental Agreements12.4. Other Collaborations

13. RECENT EXPANSIONS13.1. Chapter Overview13.2. Expansions Models13.3. Vector and Gene Therapy Manufacturing: Recent Expansions13.3.1. Analysis by Year of Expansion13.3.2. Analysis by Type of Expansion13.3.3. Analysis by Amount Invested by Key Players13.3.4. Analysis by Scale of Production13.3.5. Analysis by Type of Vector13.3.6. Analysis by Application Area13.3.7. Most Active Players: Analysis by Number of Expansions13.3.8. Geographical Analysis13.3.8.1. Analysis by Location of Expansion Project

14. STRATEGIC PARTNER ANALYSIS14.1. Chapter Overview14.2. Strategic Partner Analysis: Viral Vector based Therapy Developers14.3. Methodology and Key Parameters

15. EMERGING VECTORS15.1. Chapter Overview15.1.1. Alphavirus based Vectors15.1.2. Anc80 based Vectors15.1.3. Bifidobacterium longum based Vectors15.1.4. Cytomegalovirus based Vectors15.1.5. Listeria monocytogenes based Vectors15.1.6. Minicircle DNA based Vectors15.1.7. Myxoma Virus based Vectors15.1.8. Self-Complementary Vectors15.1.9. Sendai Virus based Vectors15.1.10. Sleeping Beauty Transposons15.1.11. Vaccinia Virus and Modified Vaccinia Ankara based Vectors

16. KEY INSIGHTS16.1. Chapter Overview16.2. Vector and Gene Therapy Manufacturers: Analysis by Purpose of Manufacturing, Type of Vector Manufactured and Scale of Operation16.3. Vector and Gene Therapy Manufacturers: Analysis by Company Size and Type of Vector Manufactured16.4. Vector and Gene Therapy Manufacturers: Prominent Geographical Hubs by Type of Organization16.4.1. Contract Manufacturing Organizations16.4.2. In-House Manufacturers16.5. Vector and Gene Therapy Manufacturers: Analysis by Location of Manufacturing Facilities and Type of Vector Manufactured16.5.1. AAV Vector Manufacturers16.5.2. Adenoviral Vector Manufacturers16.5.3. Lentiviral Vector Manufacturers16.5.4. Retroviral Vector Manufacturers16.5.5. Plasmid DNA Manufacturers

17. COST PRICE ANALYSIS17.1. Chapter Overview17.2. Factors Contributing to High Price of Viral Vector and Plasmid DNA based Therapies17.3. Viral Vector and Plasmid DNA based Therapies: Pricing Models17.3.1. On the Basis of Expert Opinions17.3.2. On the Basis of Manufacturing Cost17.3.2.1. On the Basis of Technology Used17.3.2.2. On the Basis of Scale of Manufacturing17.3.2.3. On the Basis of Type of Client17.3.3. Prices of Different Types of Vectors17.4. Concluding Remarks

18. CAPACITY ANALYSIS

19. DEMAND ANALYSIS

20. MARKET SIZING AND OPPORTUNITY ANALYSIS

21. IMPACT OF COVID-19 PANDEMIC ON THE VECTOR AND GENE THERAPY MANUFACTURING MARKET

22. KEY DRIVERS AND CHALLENGES

23. SURVEY ANALYSIS

24. CONCLUDING REMARKS

25. EXECUTIVE INSIGHTS

26. APPENDIX I: TABULATED DATA

27. APPENDIX II: LIST OF COMPANIES AND ORGANIZATIONS

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

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Insights on the Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Global Market to 2030 - GlobeNewswire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Be Biopharma (Be Bio), whose mission is to pioneer the emerging new class of engineered B cells as medicines, today announced that Krishnan Viswanadhan, Pharm.D., MBA, has been appointed President and Chief Operating Officer and Brad Hartman has joined as Chief People Officer. They join a rapidly growing team of scientists, drug developers, manufacturing experts, and business leaders whose track records have led to the creation and development of some of the industrys most impactful gene and cell therapies.

I am thrilled to welcome both Krishnan and Brad to Be Bio. Krishnan brings an exceptional track record building, rapidly scaling, and effectively leading operations that have delivered multiple highly impactful cell therapy products to patients, said Joanne Smith-Farrell, PhD, Chief Executive Officer at Be Bio. At Be Bio, we believe people are our most important asset, and Brads track record of building cell and gene therapy enterprises with innovative, inclusive cultures will help us push the boundaries of whats possible to create an unparalleled work experience. added Dr. Smith-Farrell. Both of these industry leaders have come to Be Bio to build something truly unique a fantastic team and a bespoke operating environment designed especially to unleash the breadth and depth of our leading engineered B cell medicines platform on behalf of patients who need options.

Prior to joining Be Bio, Krishnan served as Senior Vice President, Global Cell Therapy Franchise Lead at Bristol Myers-Squibb (BMS). In this role, he was responsible for setting the vision and developing the integrated, enterprise-wide cell therapy franchise strategy, building core capabilities, and driving key investments to support long-term growth. He oversaw the program team leaders responsible for the cross-functional development, registration, and lifecycle management of Breyanzi (liso-cel), a CD19 CAR T in large B cell lymphoma, and Abecma (ide-cel), the first BCMA CAR T in relapsed/refractory multiple myeloma. Prior to BMS, Krishnan held senior roles in Business Development & Global Alliances as well as Global Project Leadership at Celgene Corporation. He co-founded Advyzom, a boutique consulting company, and held multiple roles in leading development teams and global regulatory strategy at Hoffman-La Roche. Krishnan is a registered pharmacist and received his Pharm.D. from Rutgers University and holds a MBA from Cornell University. He currently serves as a non-executive director on the board of JW Therapeutics, a leading cell therapy company in China.

What is remarkable about Be Bio is the unprecedented opportunity afforded by this novel B cell therapy platform to tackle a broad set of therapeutic applications that cannot be addressed by other modalities, said Dr. Viswanadhan. The possibilities for transforming patient lives in such a wide range of serious and life-threatening diseases, the passion of the team, and the focus on patients are truly inspiring. I am honored to join this incredible team to build a portfolio of disruptive therapies for patients and to create a truly special place to come to work each day.

Brad joins Be Bio from FerGene where he served as the Chief People Officer leading the rapid expansion of the company in support of a first product commercialization effort for a novel gene therapy. He has spent the past 15 years building small to mid-sized biotech companies from early research stages through commercialization and has played a significant role in launching multiple innovative therapies, including Kalydeco (cystic fibrosis), Kalbitor (hereditary angioedema), and Incivek (hepatitis C), which was one of the fastest blockbuster drug launches of all time. He also founded and built out his own Executive Search firm, ConnectedSearch. Brad received his BS in Neuroscience from the University of Rochester and worked at both the National Cancer Institute and the Graduate School of Pharmacology at the University of Rochester in cellular pharmacology.

There is a deeply held belief at Be Bio in the paramount importance of people, culture, and service to patients, to each other, and to the community, said Mr. Hartman. I am equally awestruck by the enormous potential of this B cell platform to transform patient lives across a wide variety of serious, life-threatening diseases as I am by this teams character, heart, and values. I am incredibly honored to join this distinguished team in building a remarkable place to work together and endeavoring to reshape the lives of so many patients and their families.

About Be Biopharma

Be Biopharma is a leader in developing B cells as medicines, treating disease with the human bodys native protein factories. We precisely engineer B cells to harness their intrinsic drug-like properties remarkable protein production, selective tissue targeting, and fine control of their cellular environment to forge a new category of cell therapy. These medicines are designed to be durable, allogeneic, re-dosable, and administered without toxic conditioning, creating new avenues to halt or reverse severe diseases like cancer, autoimmune conditions, and enzyme deficiency. Founded by Longwood Fund and B cell engineering pioneers David Rawlings, M.D., and Richard James, Ph.D., Be Biopharma is re-imagining medicine based on the power of B cell therapy. Be Bio was founded in October 2020 by Longwood Fund with a $52 million Series A investment led by Atlas Ventures and RA Capital, joined by Alta Partners and Takeda Ventures. For more information, please visit Be Biopharma.

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Be Bio Appoints Leading Cell & Gene Therapy Executives, Krishnan Viswanadhan as President & Chief Operating Officer, and Brad Hartman as Chief...

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SIOX - Market Data & News

Sio Gene Therapies Inc (NASDAQ: SIOX), a 11 12 St Jamess Square, London, company, fell to close at $1.95 Monday after losing $0.08 (3.94%) on volume of 431,354 shares. The stock ranged from a high of $2.02 to a low of $1.94 while Sio Gene Therapiess market cap now stands at $142,235,939.

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Its current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. The company is also expanding the reach of gene therapy to highly prevalent conditions such as Parkinson's disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies.

Visit Sio Gene Therapies Incs profile for more information.

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Sio Gene Therapies (SIOX) falls 3.94% for August 16 - Equities.com

PHILADELPHIAToo many exhausted T cells left in the wake of aggressive chemotherapy regimens for patients with advanced chronic lymphocytic leukemia (CLL) make it more challenging for chimeric antigen receptor (CAR) T cell therapy to do its job. Now, a new study from researchers in the Perelman School of Medicine at the University of Pennsylvania shows how to overcome this type of resistance and reinvigorate these T cells with an experimental small molecule inhibitor.

Reporting online today in the Journal of Clinical Investigation, the team shows how the drug, known as JQ1, improved CAR T cell function by inhibiting what is known as the bromodomain and extra terminal (BET) proteins. BET, the researchers showed, can disrupt CAR expression and key acetylated histone functions in T cells in CLL.

The findings demonstrate, for the first time, this mechanism of resistance and present a much-needed target for CLL when treating patients with cellular therapies like CAR. Only a small subset of patients with advanced CLL respond to CAR T cell therapycompared to 80 percent of acute lymphocytic leukemia patients with advanced disease.

Why CAR T cells fail to fully attack cancer cells in so many CLL patients is an important question that needs to be answered in order to expand the use of these immunotherapies in CLL and other cancers, said senior author Joseph A. Fraietta, PhD, an assistant professor of Microbiology at Penn, and member of the Center for Cellular Immunotherapies. Treating these war weary T cells during the CAR T cell engineering process has the potential to boost responses, weve shown here. Its setting the stage for a very promising set of next steps that rationalize further studies, including clinical trials, to prove this approach is safe and feasible.

Using the small molecule inhibitor and the T cells and CD19 CAR T cells from multiple previously treated patients, the researchers demonstrated that the BET protein plays a role in downregulating CAR expression, and that, if blocked, can diminish CAR cell T cell exhaustion and increase the production of CAR T cells from CLL patients with poor lymphocytes.

Treatment with JQ1 also increased levels of various immunoregulatory cytokines and chemokines previously reported to be produced by CAR T cells in CLL during successful therapy. The array of native immune and CAR cells mirrored those found more typically in patients who do respond.

Given this observed reinvigoration of dysfunctional CLL patient CAR T cells by BET inhibition, the authors suggest that incorporating JQ1 into cellular engineering and expansion processes could lead to a generation of less defective and more potent final CAR T cells for patients.

To what extent the above pathways contribute to the effects of JQ1 on CAR T cells is a focus of ongoing investigations for the research group.

This work shows us that T cells can be taught new tricks, said Bruce Levine, PhD, the Barbara and Edward Netter Professor in Cancer Gene Therapy in Penns Perelman School of Medicine, and co-author on the study.That is to say that the methods of manufacturing can be adapted to improve CAR T cell function, so that what would have been exhausted or dysfunctional cells can now be reinvigorated, and potentially lead to better clinical responses in more patients than before.

This work was supported by the Bob Levis Funding Group, along with the National Institute of Allergy and Infectious Diseases (T32 AI007632), National Cancer Institute (P01 CA214278 575, R01 CA241762 U54 CA244711 576, P30 CA016520-44S3, and P30 CA016520-44S4), National Institute on Aging (U01 AG066100), the National Institute of General Medical Sciences (R01 GM118501), an Emerging Cancer Informatics Center of Excellence award from the Penn Institute for Biomedical Informatics and Abramson Cancer Center,Gabrielles Angel Foundation, an Alliance for Cancer Gene Therapy Investigator Award in Cell and Gene Therapy for Cancer, and Novartis.

Editors note: Fraietta is a co-founder of DeCART Therapeutics, Inc. and Levine is a co-founder of Tmunity Therapeutics, Inc. The University of Pennsylvania has licensed certain study-related technologies to Novartis. Penn and the inventors of these technologies receive significant financial benefits as a result of this licensing relationship with Novartis.

Penn Medicineis one of the worlds leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of theRaymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nations first medical school) and theUniversity of Pennsylvania Health System, which together form a $8.9 billion enterprise.

The Perelman School of Medicine has been ranked among the top medical schools in the United States for more than 20 years, according toU.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $496 million awarded in the 2020 fiscal year.

The University of Pennsylvania Health Systems patient care facilities include: the Hospital of the University of Pennsylvania and Penn Presbyterian Medical Centerwhich are recognized as one of the nations top Honor Roll hospitals byU.S. News & World ReportChester County Hospital; Lancaster General Health; Penn Medicine Princeton Health; and Pennsylvania Hospital, the nations first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.

Penn Medicine is powered by a talented and dedicated workforce of more than 44,000 people. The organization also has alliances with top community health systems across both Southeastern Pennsylvania and Southern New Jersey, creating more options for patients no matter where they live.

Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2020, Penn Medicine provided more than $563 million to benefit our community.

Link:
Existing Drug May Help Improve Responses to Cellular Therapies in Advanced Leukemias - pennmedicine.org

On track for three programs in the clinic by year end; trial site initiation for FLT180a for Hemophilia B and FLT201 for Gaucher disease Type 1 expected by year end; program for FLT190 for Fabry disease progressing

Completed first dose cohort for FLT190 Phase 1/2 dose-finding trial and advancing to next dose level; data presented by year end

Updated durability data from FLT180a Phase1/2 dose-finding trial presented by year end

Defined Company strategic priorities to accelerate value creation; near-term efficiencies extend cash runway into first quarter of 2023

LONDON, Aug. 16, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the Company or Freeline), a clinical-stage biotechnology company developing transformative AAV-mediated gene therapies for patients suffering from inherited systemic debilitating diseases, today announced that Michael J. Parini has succeeded Theresa Heggie as Chief Executive Officer (CEO). The Company also reported financial results for the second quarter of 2021 and provided updates on its lead program FLT180a for Hemophilia B, FLT190 for Fabry disease, FLT201 for Gaucher disease Type 1 and FLT210 for Hemophilia A.

Today we are pleased to announce that Freelines Board of Directors has appointed Michael J. Parini as CEO and Executive Director of the Company, effective immediately, said Dr. Chris Hollowood, Chairman of Freeline. Michael has a long track record of building strong executive leadership teams and has had a significant positive impact on Freeline since he joined the Company from Vertex Pharmaceuticals. In a short period of time, he has mobilized program execution across our pipeline, increased focus on our core scientific and platform technology capabilities, and driven efficiencies across the organization that have extended our cash runway. In doing so, Michael has demonstrated his ability to unlock value at Freeline and deliver on the promise of its therapies and science to patients.

Dr. Hollowood continued, I wish to thank Theresa for leading the Company through the successful completion of its initial public offering last year and the extraordinary challenges of the COVID-19 pandemic. We wish her well in her future endeavors.

Freeline is at the forefront of gene therapy, leveraging a platform innovation engine that holds the potential to deliver functional cures to patients who suffer from debilitating diseases, said Mr. Parini, CEO of Freeline. I joined Freeline to deliver on the differentiated promise of our pipeline and technology, and am honored and excited to take the helm at this critical time for the Company.

Mr. Parini continued, I see three immediate strategic priorities as we turn the page on a new chapter for the Company. First and foremost is our commitment to advancing our pipeline with urgency for patients. Our recent execution in the clinic is a testament to this commitment, and we remain on track to deliver all expected data and developmental milestones across our four clinical and pre-clinical stage gene therapy programs before year end.

Our second strategic priority is to drive increased focus, financial discipline and operational efficiency in our business. Based on a thorough review of our operations, we have identified initial savings to extend our cash runway by nearly two quarters into the first quarter of 2023, funding operations through multiple value-generating milestones. We will continue this work to optimize our business, operational and platform strategy.

Finally, our third priority is to unlock the full value in our proprietary scientific and platform technology. We are refreshing our scientific strategy to leverage our unique combination of manufacturing quality, capsid potency and protein engineering capabilities, which we believe can drive lower and safer doses for high therapeutic effect, into the next wave of pipeline programs. We plan to focus our research and platform efforts where our differentiated capabilities allow us to address unmet needs and create transformative treatments for patients.

Mr. Parini added, We believe gene therapy holds tremendous promise. With our three strategic priorities in place, I look forward to building a world-class team and organization to realize the full potential of Freeline.

Key Pipeline and Operational Updates

Hemophilia B

Fabry Disease

Gaucher Disease (Type 1)

Hemophilia A

Platform Technology

Corporate

Q2 2021 Financial Highlights

Cash Position: Cash and cash equivalents were $164.7million as of June 30, 2021, as compared to $230.0million as of December 31, 2020. Based on the Companys revised operating plan, Freeline expects that its current level of cash and cash equivalents will enable the Company to fund its operating expenses into the first quarter of 2023.

R&D Expenses: Research and development (R&D) expenses for the six months ended June 30, 2021 were $48.1million, as compared to $29.4million for the same period in 2020. The increase of $18.7million was driven by increased investment in activities related to the current and proposed clinical trials for FLT201 and FLT210 and overall research and development, which includes earlier pipeline programs and further development of the Freeline platform.

G&A Expenses: General and administrative (G&A) expenses for the six months ended June 30, 2021 were $24.6million, as compared to $11.7million for the same period in 2020. The increase of $12.9million was driven primarily by an increase in personnel expenses, primarily due to an increase in headcount in corporate, legal, general and administrative functions to support the Companys growth initiatives and public company requirements, as well as an increase in non-cash share-based compensation expense, primarily due to equity grants to employees related to the completion of the Series C financing and the IPO.

As of June 30, 2021, the Company had 35,802,840 ordinary shares outstanding.

About Michael J. Parini

Michael Parini joined Freeline in March of 2021 to serve as the Companys President and Chief Operating Officer. Before coming to Freeline, Mr. Parini was the Executive Vice President and Chief Administrative, Legal and Business Development Officer at Vertex Pharmaceuticals, Inc. from March 2020 until February 2021, among other roles he held in his over five years with the company. Prior to Vertex, Michael Parini spent over a decade at Pfizer Inc. serving in multiple leadership roles within the company's global legal team, including Senior Vice President and Associate General Counsel.

About Freeline Therapeutics

Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (AAV) vector-mediated systemic gene therapies. The Company is dedicated to improving patient lives through innovative, one-time treatments that provide functional cures for inherited systemic debilitating diseases. Freeline uses its proprietary, rationally-designed AAV vector, along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing protein into the patients bloodstream. The Companys integrated gene therapy platform includes in-house capabilities in research, clinical development, manufacturing and commercialization. The Company has clinical programs in HemophiliaB and Fabry disease, as well as preclinical programs in Gaucher disease Type 1 and HemophiliaA. Freeline is headquartered in the UK and has operations in Germany and the US.

Forward-Looking Statements

This press release contains statements that constitute forward looking statements as that term is defined in the United States Private Securities Litigation Reform Act of 1995, including statements that express the Companys opinions, expectations, beliefs, plans, objectives, assumptions or projections regarding future events or future results, in contrast with statements that reflect historical facts. Examples include, among other topics, discussion of the Companys strategies, anticipated operating and financial performance and financial condition, including its review of strategy and operations; the Companys expectations regarding its use of cash and cash runway; statements regarding the initiation, timing, progress and results of the Companys preclinical studies and clinical trials, including the initiation and full enrollment of the Phase1/2 dose confirmation trial for FLT180a and data readouts from that trial, progress with respect to the dose-escalation for the Phase1/2 dose-finding clinical trial of FLT190 and data readouts from that trial, commencement of the Phase 1/2 dose-finding clinical trial of FLT201, and completion of pre-clinical IND/CTA-enabling studies of FLT210; and manufacturing, research, pipeline, and clinical trial plans, including anticipated clinical development milestones for the Companys product candidates. In some cases, you can identify such forward-looking statements by terminology such as anticipate, intend, believe, estimate, plan, seek, project or expect, may, will, would, could or should, the negative of these terms or similar expressions. Forward looking statements are based on managements current beliefs and assumptions and on information currently available to the Company, and you should not place undue reliance on such statements. Forward-looking statements are subject to many risks and uncertainties, including the Companys recurring losses from operations; the uncertainties inherent in research and development of the Companys product candidates, including statements regarding the timing of initiation, completion and the outcome of clinical studies or trials and related preparatory work and regulatory review, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with preclinical and clinical data, including the possibility of unfavorable new preclinical, clinical or safety data and further analyses of existing preclinical, clinical or safety data; the Companys ability to design and implement successful clinical trials for its product candidates; the recent departures of a number of executive officers of the Company, and the Companys ability to fill open positions, implement an orderly transition process and retain key talent; whether the Companys cash resources will be sufficient to fund the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements for the Companys expected timeline; the potential for a pandemic, epidemic or outbreak of infectious diseases in the US, UK or EU, including the COVID-19 pandemic, to disrupt and delay the Companys clinical trial pipeline; the Companys failure to demonstrate the safety and efficacy of its product candidates; the fact that results obtained in earlier stage clinical testing may not be indicative of results in future clinical trials; the Companys ability to enroll patients in clinical trials for its product candidates; the possibility that one or more of the Companys product candidates may cause serious adverse, undesirable or unacceptable side effects or have other properties that could delay or prevent their regulatory approval or limit their commercial potential; the Companys ability to obtain and maintain regulatory approval of its product candidates; the Companys limited manufacturing experience which could result in delays in the development, regulatory approval or commercialization of its product candidates; and the Companys ability to identify or discover additional product candidates, or failure to capitalize on programs or product candidates. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. We cannot guarantee that any forward-looking statement will be realized. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in the Companys Annual Report on Form 20-F for the fiscal year ended December 31, 2020 and in subsequent reports on Form 6-K, in each case including in the sections thereof captioned Cautionary Statement Regarding Forward-Looking Statements and Item 3.D. Risk factors. Many of these risks are outside of the Companys control and could cause its actual results to differ materially from those it thought would occur. The forward-looking statements included in this press release are made only as of the date hereof. The Company does not undertake, and specifically declines, any obligation to update any such statements or to publicly announce the results of any revisions to any such statements to reflect future events or developments, except as required by law. For further information, please reference the Companys reports and documents filed with theU.S. Securities and Exchange Commission (the SEC). You may review these documents by visiting EDGAR on theSECwebsite atwww.sec.gov.

Contact

David S. ArringtonVice President Investor Relations & Corporate CommunicationsFreeline Therapeuticsdavid.arrington@freeline.life+1 (646) 668 6947

Freeline Therapeutics Holdings plcUnaudited Condensed Consolidated Statements of Operations Data(in thousands of U.S. dollars, except per share data)

Freeline Therapeutics Holdings plcUnaudited Condensed Consolidated Balance Sheet Data(in thousands of U.S. dollars, except per share data)

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Freeline Appoints Michael J. Parini as Chief Executive Officer and Reports Second Quarter 2021 Financial Results - GlobeNewswire