- Two oral presentations and nine poster presentations will highlight exciting progress in AskBio’s clinical and pre-clinical pipeline and advancements in the company’s manufacturing technologies
- Alongside recent clinical program milestones, presentations demonstrate AskBio’s commitment to advancing end-to-end capabilities and goal of delivering breakthrough therapies
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) 31st 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 2 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.
With an ambitious portfolio of investigational gene therapies at various stages of research and development, AskBio continues to develop AAV-based therapies to treat some of the world’s most debilitating diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications and aims to deliver breakthrough treatments that could potentially benefit tens of millions of patients worldwide.1–6
About AskBio
Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical stage programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications with a clinical-stage pipeline that includes investigational therapeutics for congestive heart failure, Huntington’s disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. AskBio’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, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing. An early innovator in the gene therapy field, with over 900 employees in five countries, the company holds more than 600 patents and patent applications in areas such as AAV production and chimeric capsids. Learn more at www.askbio.com or follow us on LinkedIn.
About Viralgen
Viralgen is a fully integrated contract development and manufacturing organization (CDMO) founded in 2017 as an independently operated subsidiary of Asklepios Biopharmaceutical, Inc. (AskBio), part of the Bayer AG group. Viralgen was created to support development through large-scale commercial production of certified good manufacturing practices (cGMP) AAV (adeno-associated virus) for cell and gene therapies. Through the AskBio licensed proprietary Pro10™ suspension manufacturing platform, Viralgen delivers industry-leading titers for all AAV serotypes, optimizing speed to market and cost-of-goods to accelerate clinical development and commercialization.
The company’s state-of-the-art facilities, based in San Sebastian, Spain, include a commercial facility consisting of three modules, each having three cGMP suites with capacities of >2,000 L, one of which has received the cGMP certification by the Spanish Agency for Medicines and Medical Devices (AEMPS) as part of the European Medicines Agency (EMA) for the manufacture of human medicinal products, investigational medicinal products, and sterile or biological active substances. The recently inaugurated manufacturing science and technology (MSAT) laboratory provides advanced services for transitioning programs from clinical to commercial manufacturing. Leveraging its proven expertise in AAVs, Viralgen has recently launched several development service offerings focused on construct optimization, process optimization, and right-sized scaling for customers needing support, in addition to the established research, process development, formulation, and filling activities.
For more information, visit www.viralgenvc.com.
About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s 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 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to www.bayer.com.
AskBio Forward-Looking Statements
This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding AskBio’s clinical trials. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio’s control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned clinical and regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect AskBio’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.
References
[1] Balestrino R, Schapira AHV. Parkinson disease. Eur J Neurol. 2020;27(1):27-42.
[2] World Health Organization. Parkinson Disease. Available at: https://www.who.int/news-room/fact-sheets/detail/parkinson-disease Last accessed: October 2024.
[3] Malik A, et al. Congestive Heart Failure. In: StatPearls. Treasure Island (FL): StatPearls Publishing; November 7, 2022.
[4] NIH. Limb-girdle muscular dystrophy: MedlinePlus Genetics. Available at: https://medlineplus.gov/genetics/condition/limb-girdle-muscular-dystrophy/#frequency Last accessed: October 2024.
[5] Goh Y, et al. Multiple system atrophy [published online ahead of print, 2023 Mar 16]. Pract Neurol. 2023; practneurol-2020-002797.
[6] Stevens D, et al. Pompe Disease: a Clinical, Diagnostic, and Therapeutic Overview. Curr Treat Options Neurol. 2022;24(11):573-588.
