Forge Biologics Announces Publication in Human Gene Therapy on a Novel Finding That Changes a Fundamental Understanding of AAV Production

13.12.2023 - 06:01:48

Business Wire India

Research findings indicate a path to the development of potentially safer adeno-associated virus (AAV) gene therapies and provide additional avenues for improving AAV manufacturability for increasing yields
The discovery was published as a peer-reviewed scientific article in a leading research journal, Human Gene Therapy

Forge Biologics (Forge), a leading manufacturer of genetic medicines, announced today the peer-reviewed publication of, “A novel role for the adenovirus L4 region 22K and 33K proteins in adeno-associated virus production,” in the research journal Human Gene Therapy. The article was authored by a team of molecular development scientists at Forge led by David Dismuke, Ph.D., Chief Technical Officer, Linas Padegimas, Ph.D., Molecular Development Senior Director, and Angela Adsero, Ph.D., Molecular Development Scientist II.

Specific adenoviral genes are required for AAV production. However, a novel, undiscovered gene in particular has been overlooked because of its shared DNA sequence with a gene regulatory region that determines when or how much protein is made from a gene. By using molecular techniques to decouple the dual-purpose nature of this sequence, the Forge scientific team demonstrated that the L4 region 22K protein is an additional requirement for AAV vector production that had previously gone unnoticed. The study also suggests that the L4 region 33K protein is important for increasing AAV production.

“Forge is committed to improving gene therapy manufacturing through scientific innovation. The discovery of these vital AAV production requirements provide the potential for a more targeted and efficient production strategy, while also strengthening our IP portfolio,” said David Dismuke, Ph.D., Chief Technical Officer at Forge. “I am exceptionally proud of the Forge team for their hard work in contributing to our understanding of the essential elements of AAV production so that we can keep advancing the field of gene therapy.”

Contributing authors include the following Forge molecular development scientists: Brendan Chestnut, M.Sc., Sara Shahnejat-Bushehri, Ph.D., LalitaSasnoor, Ph.D., Travis McMurphy, Ph.D., Michael Swenor, Ryan Pasquino, Arun Pradhan, Ph.D., Victor Hernandez, Ph.D., Linas Padegimas, Ph.D., and David Dismuke, Ph.D. The full research article can be accessed and will be available through open accesshere: https://www.liebertpub.com/doi/10.1089/hum.2023.146.

“This interesting finding may significantly impact gene therapy manufacturing and that translates to improved and potentially life-transforming genetic medicines for millions of patients suffering from genetic diseases worldwide,” said Robert Kotin, Ph.D., a leading voice in AAV and gene therapy manufacturing and a member of Forge’s Scientific and Manufacturing Advisory Board. “The Forge team has deep roots in developing genetic medicines and in vector manufacturing, as evidenced by these findings.”

About Forge Biologics

Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company focused on enabling access to life-changing gene therapies. Forge was founded in 2020 and is headquartered in the cell and gene therapy hub of Columbus, Ohio. Its 200,000 square foot facility, the Hearth, is dedicated to AAV manufacturing with 20 custom-designed cGMP suites. Offerings include scalable, end-to-end manufacturing services including process and analytical development, cGMP viral vector manufacturing, final fill, plasmid DNA manufacturing, as well as regulatory consulting support to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing. Forge aims to accelerate the timelines of transformative medicines for patients with genetic diseases. To learn more, visit www.forgebiologics.com.