AskBio Contribution to Advancing Gene Therapy Highlighted at ESGCT 29th Congress

— Company presence includes 2 oral and 12 poster presentations — 

Research Triangle Park, N.C.– OCTOBER 10, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that 14 company abstracts will be presented at the European Society for Gene and Cell Therapy (ESGCT) 29th Congress, which is being held in Edinburgh, Scotland, from October 11 to 14, 2022. 

The full company presence highlights the breadth of the AskBio contribution to advancing gene therapy and includes an oral presentation on the role of empty capsids in overall AAV immunogenicity and toxicity risks, 12-month data from ongoing Parkinson’s and congestive heart failure studies and data on synthetic DNA material from wholly owned subsidiary TAAV Biomanufacturing Solutions, SL. Additional activities include company experts participating in educational sessions and parallel symposia, as well as a symposium sponsored by wholly owned subsidiary Viralgen. Topics include data-driven vector design for precise control of gene expression and manufacturing to support gene therapy.

“AskBio is relentless in its effort to serve as a catalyst for breakthrough innovation in gene therapy,” said Sheila Mikhail, CEO and Co-Founder of AskBio. “ESGCT gives us an opportunity to show how we are doing that in several important ways: driving the science related to capsids and the use of synthetic DNA material, advancing our pre-clinical and clinical stage pipeline and creating opportunities for industry colleagues to exchange knowledge.” 

Data highlights include:

ProgramAbstract TitlePresentation Details/Date
Preclinical/R&DUsing a systems biology approach to unravel the immunogenicity of AAV8 empty capsids in healthy volunteersOral presentation (OR06) / October 11 (17:00 – 19:15 BST)
Congestive Heart FailureAnalysis of vector performance in patient heart biopsy for congestive heart failure provides support for rationally designed capsidsPoster P671 / October 12
Preclinical/R&DIn vivo evaluation of novel synthetic promoters for CNS gene therapyPoster P037 / October 12
Preclinical/R&DTranscriptomics of suspension 293 cells during AAV vector productionPoster P345 / October 12
Huntington’s Disease Pathway gene therapy for Huntington’s disease: A Phase 1/2 dose-finding study to evaluate BV-101 striatal administration in adults with early manifest Huntington’s diseaseInvited oral presentation (INV33) / October 13 (8:30-10:45 BST)
Preclinical/R&DmiRNA coexpression during vector production to increase vector yield and transduction efficiencyPoster P064 / October 13
Congestive Heart FailureA first-in-human Phase 1 clinical gene therapy trial for the treatment of non-ischemic heart failure using a novel rationally designed cardiotropic adeno-associated vector targeting calcium cyclingPoster P670 / October 13
Parkinson’s DiseaseSafety and clinical findings 12-months following bilateral putaminal convection enhanced delivery of AAV2-GDNF in early and moderate stages of Parkinson’s diseasePoster P170 / October 13
Alzheimer’s DiseaseNon-invasive AAV-CYP46A1 gene therapy mitigates disease progression in Alzheimer’s disease micePoster P220 / October 13
Huntington’s DiseaseAstrocytes are key players in cholesterol gene therapy for Huntington’s diseasePoster P182 / October 13
Preclinical/R&DResults from using AskBio capsid screening (ABCs) platformPoster P002 / October 13
TAAVAdvanced characterization of enzymatically amplified doggyboneTM DNA for rAAV manufacturingPoster P300 / October 13
TAAVUHPLC and CE for in-process controls in the manufacturing of linear covalently closed doggyboneTM DNA for rAAV productionPoster P324 / October 13
ManufacturingA universal formulation for several adeno-associated virus serotypes protects against thermal degradationPoster P590 / October 13

About AskBio

Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, 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 Pompe disease, Parkinson’s disease, and congestive heart failure. 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, UK, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at or follow us on LinkedIn

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 pipeline of development candidates, manufacturing technology and process. 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 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 the ongoing COVID-19 pandemic, 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. 

Media Contact:

Phil McNamara
Vice President, Corporate Communications