AskBio Co-Founder R. Jude Samulski and Team of AskBio Scientists Author Paper Published in Prestigious Journal 

— Leading research journal, Molecular Therapy, published a manuscript that explores the improvement and expansive use of AAV vectors in gene therapy — 

Research Triangle Park, N.C.– January 26, 2023 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, is pleased to announce the recent publication of “AAV vectors: The Rubik’s cube of human gene therapy” in Molecular Therapy. The manuscript, which reviews the different approaches to improve adeno-associated virus (AAV) vectors for gene therapy, was authored by a team of AskBio scientists; a scientist from AskBio subsidiary Viralgen Vector Core; and AskBio Co-Founder, President and Chief Scientific Officer, R. Jude Samulski, PhD, a world-renowned gene therapy pioneer. 

The article focuses on AAV capsid selection and engineering, strategies to overcome anti-AAV immune response and the design of vector genomes. Also included is a look at the expanded use of AAV vectors, beyond treating monogenic genetic diseases, to enable gene transfer approaches for a wide range of neurodegenerative, heart, blood, muscular and retinal disorders, as well as complex cancers, such as glioblastoma. The manuscript ends with a glimpse at vector production methods and the current state of recombinant AAV (rAAV) at the clinical level.

“As we see the potential efficacy of rAAV and the assembly and packaging of high yielding vectors for gene transfer, we have opened the door to changing the course of a broader range of monogenic and pathway diseases,” remarked Dr. Samulski. “This article further reveals the possibilities of rAAV and how we can improve current applications of AAV vectors used in gene therapy today.”

The manuscript’s lead author was AskBio scientist Amaury Pupo, PhD. Other AskBio contributing authors were Audry Fernández, PhD, SiewHui Low, PhD, and Lester Suárez, DVM, PhD, along with Viralgen Vector Core Director of Molecular Development, Achille François, PhD. This work was conducted under the guidance of Dr. Samulski, who is also a professor of Pharmacology at The University of North Carolina at Chapel Hill, and the manuscript was featured on Molecular Therapy’s December cover.

Dr. Samulski was recently awarded the inaugural European Society of Gene and Cell Therapy (ESGCT) “Founders Award” in Edinburgh, Scotland, during the ESGCT 29th Congress. Dr. Samulski developed the foundational technology for making AAV-based gene therapy vectors and self-complementary AAV vectors over 40 years ago. These contributions are believed to be key components of every FDA approved AAV therapeutic today and will remain central to future advances in gene therapy.

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 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, Scotland, 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 www.askbio.com 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, AskBio
E: pmcnamara@askbio.com
T: +1 984.389.1797