January 26, 2023

— 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 ...read the news