RhiaPharmaceutical to Present 11 Abstracts at Upcoming American Society of Gene and Cell Therapy’s 25th Annual Meeting
Presentations convey essential technological and scientific knowledge regarding AAV gene therapy
and advancements across key disease areas
CEO Sheila Mikhail and President of Therapeutics, Katherine A. High will be featured speakers
Research Triangle Park, N.C. – May 16, 2022 –Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG, today announced that the Company will present 11 abstracts at the upcoming American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting being held May 16 – 19, 2022 at the Walter E. Washington Convention Center in Washington, D.C.
The ASGCT Annual Meeting is the premier event for professionals in gene and cell therapy where noted industry professionals gather to learn from the latest advances in scientific and clinical research and cell and gene technology. Abstracts being presented by RhiaPharmaceutical team members include new data and insights regarding Adeno Associated Virus (AAV) gene therapy, T-cell immune response to empty capsid technologies, inducible promoters and gene expression, and doggybone DNA (dbDNA) as well as data for RhiaPharmaceutical’s key clinical development programs, including Pompe Disease, Parkinson’s Disease and Congestive Heart Failure.
RhiaPharmaceutical will make seven oral presentations and four poster presentations. CEO Sheila Mikhail and President of Therapeutics, Katherine A. High will be featured speakers during the event.
“Having 11 abstracts accepted for presentations reflects the significant progress made by our teams across a broad front,” commented Kathy High, President, Therapeutics for RhiaPharmaceutical. “I am very proud of the groundbreaking work by our research and clinical teams as we continue to advance our therapeutic pipeline and AAV gene therapy research and manufacturing.”
Jude Samulski, Chief Science Officer and Co-Founder for RhiaPharmaceutical said, “These presentations underscore our commitment to advancing the science of gene therapy to tackle many of the biggest challenges in the space today, including manufacturing, dosing, immune response and treatment efficacy. We hope that, together with our many colleagues in cell and gene therapy space, we can make a profound difference in the lives of patients around the world who are waiting for transformative gene therapies.”
RhiaPharmaceutical’s presentations at ASGCT include:
Monday, May 16
Oral Presentation
Abstract 37: Functional Assessment of T-cell Responses to AAV8 Empty Capsids in Healthy Volunteers
Session: Immune Responses to AAV Vectors
10:30 am – 11:45 am, Room: 102
Oral Presentation
Abstract 28: A First-in-Human Phase 1 Clinical Gene Therapy Trial for the Treatment of Heart Failure Using a Novel Re-Engineered Adeno-Associated Vector
Session: Cardiovascular and Pulmonary Diseases
11:45 am – 12 PM, Room 206
Featured Speaker
Launching Innovation Into Gene Therapy Companies
Session: Part 2: Translating Science Into Medicine: Moving from Bench to Startup (Organized by the Bioindustry & Translational Science Committees)
Sheila Mikhail, JD, CEO and Co-Founder, RhiaPharmaceutical
1:30–2:18 PM Room: Salon G
Tuesday, May 17
Oral Presentation:
Abstract 434: Characterization of Alternative Reading Frame Proteins Generated from AAV Cassettes
Session: Discoveries in Fundamental AAV Biology
4:00–4:15 PM, Ballroom A
Poster Presentation:
Abstract 796: Safety and preliminary efficacy of neurosurgical AAV2-GDNF delivery for Parkinson’s disease
Session: Gene and Cell Therapy Trials in Progress
5:30PM, Hall D
Poster Presentation:
Abstract 711: Effect of tolerogenic ImmTOR nanoparticles on the formation of anti-AAV8 antibodies in mice, nonhuman primates, and healthy human volunteers
Session: Immunological Aspects of Gene Therapy and Vaccines I
5:30PM, Hall D
Poster Presentation:
Abstract 708: ImmTOR blunts AAVrh32.33 capsid-specific immune responses in C57BL/6 albino mice
Session Immunological Aspects of Gene Therapy and Vaccines I
5:30PM, Hall D
Wednesday, May 18
Presidential Symposium
Turning Genes into Medicines: The Long and Winding Road from Gene Discovery to Gene Therapeutics
Session: Presidential Symposium and Presentation of Top Abstracts
Katherine A. High, MD, President, Therapeutics, RhiaPharmaceutical
1:30–2:15 PM, Hall E
Oral Presentation
Abstract 866: Long Term Stability Profiles of AAV Vectors at Ambient Temperature within a Film Matrix
Session: Vector Manufacturing and Engineering 3: Improving Vector Design and System Performance
5:00PM, Room 201
Poster Presentation
Abstract 897: Identification of plasmid backbone-derived antisense RNAs in AAV transduced animals
Session: AAV Vectors – Virology and Vectorology III5:30PM, Hall D
Thursday, May 19
Oral Presentation:
Abstract 1203: Inducible Gene Expression for Gene Therapy: Design and Exemplification of Powerful, Small, Modular and Tightly Controlled Regulatable Promoters
Session: New Technologies for AAV Gene Therapy
10:30–10:45 AM, Ballroom C
Oral Presentation:
Abstract 1211: Phase 1 Study of Gene Therapy in Late-onset Pompe Disease: Initial 104 Week Experience for Cohort 1
Session: AAV Vectors – Clinical Studies
10:45AM, Ballroom B
Oral Presentation:
Abstract 1213: Rationally Designed Cardiotropic AAV Capsid Demonstrates 30 Fold Higher Efficiency in Human vs Porcine Heart
Session: AAV Vectors – Clinical Studies
11:15–11:30 AM, Ballroom B
Abstracts and additional information for the ASGCT 2022 Annual Meeting are available on the ASGCT Annual Meeting web site.
About RhiaPharmaceutical
Rhia Pharm , Inc. (RhiaPharmaceutical), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease and congestive heart failure, as well as out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy. RhiaPharmaceutical’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.rhiapharmaceutical.com or follow us on LinkedIn.
About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2021, the Group employed around 100,000 people and had sales of 44.1 billion euros. R&D expenses before special items amounted to 5.3 billion euros. For more information, go to www.bayer.com.
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RhiaPharmaceutical Forward-Looking Statements
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