AskBio to showcase cutting-edge research on AAV therapies and manufacturing advancements at ESGCT 2024

Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will deliver 11 presentations offering insights into the research and development of adeno-associated virus (AAV) therapies for a range of diseases as well as advancements in manufacturing technologies, at the European Society of Gene and Cell Therapy (ESGCT) 31^st Annual Meeting taking place October 22–25, 2024, in Rome, Italy.

“Our presence at ESCGT this year demonstrates our commitment to strengthening our end-to-end capabilities, successfully advancing our early pre-clinical pipeline and continuing to innovate in the field of manufacturing technology”, said Gustavo Pesquin, Chief Executive Officer, AskBio. “This year’s presentations complement notable recent progress in our clinical pipeline, with the first patient randomized in our Phase II GenePHIT trial for AB-1002, an investigational gene therapy in congestive heart failure, and the initiation of recruitment for REGENERATE-PD, our Phase 2 Parkinson’s disease trial for investigational gene therapy AB-1005, both announced earlier this year.”

AskBio’s presentations include (all times CEST):

Orals

• In Vivo Gene Therapy Strategies – Focus on CNS (EDU07). Tue., Oct. 22, 09:00–10:45 (Session 1b: CNS Gene Therapy, Gene Therapy for Monogenic Diseases: Concepts – Applications – Challenges and Possible Solutions, Meeting Room 2). Nathalie Cartier-Lacave, MD, Senior Vice President, Sector Lead, Neurobiology, AskBio.
• Clinical Progress and Plans for AB-1005 (AAV2GDNF) Gene Therapy for Parkinson’s Disease (INV43). Thu., Oct. 24, 09:00–11:00 (Session 7d: CNS Gene Therapy, Auditorium PARALLEL). Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. Session co-chaired by Nathalie Cartier-Lacave, MD, Senior Vice President, Sector Lead, Neurobiology, AskBio.

Posters

• Evaluation of Mutant Huntingtin Knockdown after Intra-Striatal Administration of AAV-miHTT Constructs in the YAC128 Mouse Model of Huntington’s Disease. Tue., Oct. 22, 19:30–21:00 (Poster #P0283). Enejda Subashi, AskBio.
• Enhancing the Upstream Performance of Adeno-Associated Virus (AAV) Vector Manufacturing via Multivariate Data Analysis. Tue., Oct. 22, 19:30–21:00 (Poster #P0209). Ainara Apezleguía, Viralgen.
• Metabolomic Profiling Approach to Identify Peripheral Blood Changes after AAV-CYP46A1 Intrastriatal Injection in HD Mice. Tue., Oct. 22, 19:30–21:00 (Poster #P0343). Sandro Alves, AskBio.
• Design-of-Experiment (DoE) Approach Suggests Transfection Viable-Cell Density as Key Parameter for Optimized rAAV Productivity and Total DNA Amount for rAAV Packaging. Tue., Oct. 22, 19:30–21:00 (Poster #P0071). Nerea Arranz, Viralgen.
• Leveraging HTS Technologies to Develop Methods for In-Depth Characterization of rAAV Products. Wed., Oct. 23, 13:30–15:00 (Poster #P0170). Emilie Lecomte, Viralgen.
• Analysis of Genomic Configurations In Vivo after rAAV Delivery. Wed., Oct. 23, 13:30–15:00 (Poster #P0122). Madhuvanthi Vijayan, AskBio.
• Modular Assembly of Capsids Allows Generation of AAV Variants Passing the Blood-Brain-Barrier in Mice. Wed., Oct. 23, 13:30–15:00 (Poster #P0142). Marco Zahn, AskBio.
• Leveraging Platform and Process Characterization Data to Accelerate CGT Validation and Commercialization. Wed., Oct. 23, 14:00–15:30 (Poster #P0841). Ane Quesada, Viralgen.
• Addressing the Complex Regulatory Environment for Gene Therapies. Wed., Oct. 23, 14:00–15:30 (Poster #P1031). Maria Orio, Viralgen.