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 at the American Society of Gene and Cell Therapy (ASGCT) 26^th Annual Meeting taking place May 16–20, 2023, in Los Angeles, USA.

AskBio presentations cover pre-clinical research into immune responses to AAV, data on methods to improve the efficacy of AAV-gene therapy and research into improved analytics and bioassays. A symposium on a platform approach to AAV manufacturing will also be presented by AskBio’s subsidiary Viralgen.  

"Our breadth of scientific presence at ASGCT reflects the type of cutting edge pre-clinical research our teams undertake, which we believe is essential for the development of successful gene therapies in the future," said Jude Samulski, Chief Scientific Officer and Co-Founder of AskBio. "We look forward to presenting updates on pioneering work that spans the subtlety of AAV vector design, quantifiable transgene expression and preclinical efficacy, with the aim of furthering AAV therapy for additional unmet clinical needs."

With an ambitious portfolio of 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, including congestive heart failure, Huntington’s disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. By targeting these therapy areas, AskBio aims to deliver breakthrough treatments that could benefit more than 35 million patients worldwide.^1–7

AskBio’s presentations at ASGCT include:

• A Platform Approach for Adeno-Associated Virus Manufacturing to Support Gene Therapy Products. Presented by Cesar Trigueros, Chief Scientific Officer, Viralgen. [Exhibitor Symposium: 08:15 May 19 (Room 511)]
• Inhibition of AAV-specific adaptive immune response by co-stimulation blockade with CTLA4-Ig fusion protein. Presented by Audry Fernandez, R&D, Asklepios BioPharmaceutical, Inc. [Oral presentation: 16:00 May 18 (Room 411)]
• Impact of Genetic Diversity on Gene Therapy Efficacy. Presented by Joerg Votteler, Associate Director, Asklepios BioPharmaceutical, Inc. [Oral presentation: 16:15 May 18 (Room 403 AB)]
• Characterization of promoter and intron interactions affecting transgene expression in AAV cassettes. Presented by Ferzin Sethna, Senior Scientist, Asklepios BioPharmaceutical, Inc. [Oral presentation: 16:15 May 18 (Room: Concourse Hall 150 & 151)]
• AA, BB, CC, D deciphering the alphabet of the AAV ITR. Presented by Liujiang Song, Principal Scientist, Asklepios BioPharmaceutical, Inc. [Oral symposium presentation: 09:00 May 19 (Room 502 AB)]
• Utilizing AAV entry factors & small molecules to synergistically enhance vector transduction and transgene expression to facilitate the development of in vitro cell-based assays for AAV gene therapy. Presented by Kayla Shumate, Translational Scientist, Asklepios BioPharmaceutical, Inc. [Poster presentation: 12:00 May 17 (Poster #773)]
• Diversity Analyses in AAV Libraries Containing Peptide Insertion Using Short- and Long-Read Next-Generation Sequencing Approaches. Presented by Kathleen Boerner, Managing Director, Asklepios BioPharmaceutical, Inc. [Poster presentations: 12:00 May 17 (Poster #460)]
• Kinetics of Innate and Adaptive Immune Responses to Adeno-Associated Virus in a Preclinical Model. Presented by Alan Curtis, Cellular & Immunology Scientist, Asklepios BioPharmaceutical, Inc. [Poster presentation: 12:00 May 18 (Poster #1039)]
• Characterization of the muscular and cardiac diseases of the DMSXL mouse model, a transgenic mouse model for Myotonic Dystrophy type 1. Presented by Caroline Le Guiner, Senior Scientist, Asklepios BioPharmaceutical, Inc. [Poster presentation: 12:00 May 18 (Poster #930)]
• Rationally designed cardiotropic AAV capsid demonstrates targeted cellular distribution in cardiomyocytes. Presented by Youjun Chen, Histotechnology Scientist, Asklepios BioPharmaceutical, Inc.  [Poster presentation: 12:00 May 19 (Poster #1319)]
• A quantitative method for evaluating AAV-mediated FKRP expression and function in differentiated myotubes. Presented by Jenny Herndon, Scientist, Asklepios BioPharmaceutical, Inc. [Poster presentation: 12:00 May 19 (Poster #1670)]
• Restoration of brain cholesterol metabolism as gene therapy strategy in Huntington’s disease: unilateral injections are insufficient to elicit a treatment effect in HD mice. Presented by Sandro Alves, Head preclinical (France), Asklepios BioPharmaceutical, Inc. [Poster presentation: 12:00 May 19 (Poster #1261)]

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 programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes 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 clinical testing. An early innovator in the gene therapy field, with over 800 employees in five countries, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.askbio.com or follow us on LinkedIn.

About Viralgen
Viralgen, an independently operated Contract Development and Manufacturing Organization (CDMO), was founded in 2017 as a joint venture between AskBio and Columbus Venture Partners (a venture capital firm based in Spain). As one of the world’s leading manufacturers of cGMP-certified AAV, Viralgen uses the Pro10^TM based suspension manufacturing platform, a technology licensed from AskBio and developed by CTO Josh Grieger and co-founder R. Jude Samulski, PhD, at University of North Carolina. It is believed that Pro10™ increases scalability, performance and precision of AAV therapies. Located in Spain, in the Gipuzkoa Science and Technology Park, Viralgen produces AAV gene therapy treatments for pharmaceutical and biotech companies with the aim of accelerating the delivery of new treatments that can improve patients’ lives.

The company’s clinical facilities have four cGMP manufacturing suites, with 250-liter and 500-liter bioreactors. In 2020, Viralgen expanded within the scientific park by constructing a new building for large-scale commercial manufacturing. The new state-of-the-art facility includes three additional cGMP suites with a manufacturing capacity of 2,000 liters each, as well as a suite dedicated to fully automated fill and finish operations. The new facility has received cGMP certification by the Spanish Agency for Medicines and Medical Devices (AEMPS) as part of the EMA network.

For more information, visit viralgenvc.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/…. Accessed May 2023.
3.      Medina A, et al. Prevalence and Incidence of Huntington’s Disease: An Updated Systematic Review and Meta-Analysis. Mov Disord. 2022;37(12):2327-2335.
4.      Malik A, et al. Congestive Heart Failure. In: StatPearls. Treasure Island (FL): StatPearls Publishing; November 7, 2022.
5.      NIH. Limb-girdle muscular dystrophy: MedlinePlus Genetics. Available at: https://medlineplus.gov/genetics/condition/limb-girdle-muscular-dystrophy/#frequency. Accessed May 2023.
6.      Goh Y, et al. Multiple system atrophy [published online ahead of print, 2023 Mar 16]. Pract Neurol. 2023; practneurol-2020-002797.
7.      Stevens D, et al. Pompe Disease: a Clinical, Diagnostic, and Therapeutic Overview. Curr Treat Options Neurol. 2022;24(11):573-588.