BayerAsklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, 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 neDNATM 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)
  • neDNATM 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)
  • neDNATM, 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 Pro10TM 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.

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.

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.

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 neDNATM 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 neDNATM 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.

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