AskBio Contribution to Advancing Gene Therapy Highlighted at ESGCT 30th Congress

• 2023 congress represents company's most extensive ESGCT presence to date
• Includes data from AskBio wholly owned manufacturing subsidiaries, TAAV and Viralgen

RESEARCH TRIANGLE PARK, N.C., Oct. 23, 2023 /PRNewswire/ -- Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that 20 company abstracts will be presented at the European Society for Gene and Cell Therapy (ESGCT) 30th Congress, which is being held in Brussels, Belgium, from October 24 to 27, 2023. 

The full company presence highlights the breadth of the AskBio contribution to advancing gene therapy and includes an oral presentation of the company's work on immune modulation and the role of genetic diversity in translational animal models of AAV gene therapy, as well as a poster presentation on rational design of regulatable expression cassettes in non-human primates.

"With 20 presentations at this year's ESGCT, including data from our wholly owned subsidiaries, TAAV and Viralgen, AskBio's ambition is clear when it comes to advancing the science and driving innovation in gene therapy," said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. "ESGCT provides us with an important platform to share our work with the broader scientific community and demonstrate the transformative power of AAV technology and gene therapy."

AskBio's presentations include (all times CEST):

Oral

• Immune modulation and the role of genetic diversity in translational animal models of AAV gene therapy: Fri., Oct. 27, from 11:00 to 13:00 (Session 11c, Maison de la Poste PARRALLEL)

Posters

• Leveraging a spectral cytometry immuno-surveillance assay for orthogonal detection of AAV-reactive donors in the general population and beyond: Wed., Oct. 25, from 17:00 to 18:15 and Thurs., Oct. 26, from 20:30 to 21:30 (Poster #P047)
• Achieving successful therapeutic levels of FVIII at low vector doses via enhancing AAV capsid design: Wed., Oct. 25, from 17:00 to 18:15 and Thurs., Oct. 26, from 20:30 to 21:30 (Poster #P057)
• Identification of B-cell receptor sequences against adeno-associated virus (AAV) 8 empty capsids from whole blood of healthy clinical trial participants: Wed., Oct. 25, from 17:00 to 18:15 and Thurs., Oct. 26, from 20:30 to 21:30 (Poster #P769)
• A machine learning model to design manufacturable AAV peptide insertion capsid libraries: Wed., Oct. 25, from 17:00 to 18:15 and Thurs., Oct. 26, from 20:30 to 21:30 (Poster #P153)
• Restoration of brain cholesterol metabolism as gene therapy approach in Huntington's disease (HD): bilateral striatal administration is needed to elicit therapeutic benefit in HD mice: Wed., Oct. 25, from 17:00 to 18:15 and Thurs., Oct. 26, from 20:30 to 21:30 (Poster #P407)
• Comparative analysis of neDNA™ and plasmid DNA for recombinant adeno-associated virus (rAAV) production: Wed., Oct. 25, from 17:00 to 18:15 and Thurs., Oct. 26, from 20:30 to 21:30 (Poster #P143)
• Rationally designed cardiotropic AAV2i8.I-1c demonstrates targeted cardiomyocyte distribution and a promising safety and efficacy profile in an ongoing Phase 1 clinical gene therapy trial in patients with advanced heart failure: Wed., Oct. 25, from 17:00 to 18:15 and Thurs., Oct. 26, from 20:30 to 21:30 (Poster #P019)
• Quantification and characterization of helper plasmid residuals in rAAV drug products: Wed., Oct. 25, from 17:00 to 18:15 and Thurs., Oct. 26, from 20:30 to 21:30 (Poster #P137)
• Rational design of regulatable expression cassettes; predicable, robust, and tuneable expression in non-human primates: Wed., Oct. 25, from 18:15 to 19:30 and Thurs., Oct. 26, from 19:30 to 20:30 (Poster #P024)
• neDNA™ is a robust alternative to plasmid DNA for AAV production: Wed., Oct. 25, from 18:15 to 19:30 and Thurs., Oct. 26, from 19:30 to 20:30 (Poster #P336)
• neDNA™, a robust and high-quality DNA supply for rAAV manufacturing: Wed., Oct. 25, from 18:15 to 19:30 and Thurs., Oct. 26, from 19:30 to 20:30 (Poster #P340)
• Screening out the noise: removing contamination tridents from capsid screens: Wed., Oct. 25, from 18:15 to 19:30 and Thurs., Oct. 26, from 19:30 to 20:30 (Poster #P184)
• PromPT® - Promoter Precision Technology, a platform for design, screening, and characterisation of cell type specific promoters: Wed., Oct. 25, from 18:15 to 19:30 and Thurs., Oct. 26, from 19:30 to 20:30 (Poster #P440)
• Identification of tissue specific AAV variants by highly diverse peptide insertion library screening in mouse and NHP models: Wed., Oct. 25, from 18:15 to 19:30 and Thurs., Oct. 26, from 19:30 to 20:30 (Poster #P088)
• Optimizing scale-up of AAV gene therapy in upstream processing: Wed., Oct. 25, from 18:15 to 19:30 and Thurs., Oct. 26, from 19:30 to 20:30 (Poster #P338)
• Enhancing AAV6 vector production for cell therapies: Harnessing the potential of Viralgen's Pro10™ platform for scalable manufacturing: Wed., Oct. 25, from 18:15 to 19:30 and Thurs., Oct. 26, from 19:30 to 20:30 (Poster #P128)

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 900 employees in five countries, the company holds more than 800 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 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 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. R&D expenses before special items amounted to 6.2 billion euros. For more information, go to www.bayer.com.

About Viralgen

Viralgen is a Contract Development and Manufacturing Organization (CDMO) founded in 2017 and exists as an independently operated subsidiary of AskBio, which is wholly owned and independently operated as a subsidiary of Bayer AG. As a manufacturer of Current Good Manufacturing Practice (cGMP) certified AAV, Viralgen offers the Pro10™ based suspension manufacturing platform, a technology licensed from AskBio and developed by Chief Technical Officer Josh Grieger, PhD, and Co-Founder R. Jude Samulski, PhD, at University of North Carolina. It has been demonstrated that  Pro10™ increases scalability, performance, and yield of AAV therapies¹  at Viralgen and previous partners of AskBio. 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 may 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 with three modules for large-scale commercial manufacturing. Each module of the state-of-the-art space includes three cGMP suites with a manufacturing capacity of >2,000 liters. The first module, which includes a suite dedicated to fully automated fill and finish operations, 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.

About TAAV

TAAV Biomanufacturing Solutions, SLU (TAAV), a wholly owned subsidiary of AskBio and Bayer AG, is a cGMP manufacturer of an enzymatic DNA material used for adeno associated virus (AAV) gene therapies. TAAV-manufactured neDNA™ is produced using a manufacturing process that eliminates the use of fermenters and bacteria, which are required in more common plasmid DNA manufacturing. Enzymatic manufacturing removes significant levels of bacterial contaminants, including plasmid backbone sequences, resulting in the production of enzymatic "no end" DNA product with what we believe is an improved safety profile for the manufacture of recombinant adeno-associated virus (rAAV) gene therapy vectors. The TAAV manufacturing process consistently produces large amounts of neDNA™ using technology licensed from Touchlight IP Ltd.^† in reduced times, which can be measured in days versus the months that are typical when producing plasmid DNA. TAAV was founded in 2019 and became 100% wholly owned by AskBio in 2022. Company headquarters, manufacturing facilities and labs are in San Sebastian, Spain. For more information, go to taav.com.

^†Technology for making dbDNA™, a trademark of Touchlight Genetics Ltd., is licensed from Touchlight IP Ltd.

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.

¹Mol Ther. 2016 Feb;24(2):287-297. doi: 10.1038/mt.2015.187. Epub 2015 Oct 6.

SOURCE AskBio