Genethon licenses core AB-1009 technology to AskBio after IND clearance

AB-1009 is an investigational adeno-associated virus (AAV)–based gene therapy being developed by AskBio to address the underlying genetic cause of Pompe disease, which results from a deficiency of the acid alpha-glucosidase (GAA) enzyme.

On the other hand, Genethon’s scientists have previously demonstrated the preclinical efficacy of a gene therapy strategy using a transgene encoding a truncated form of the GAA enzyme. Those findings were published in Science Translational Medicine in 2017.

According to Genethon’s January 14 announcement, AskBio’s AB-1009 incorporates technology that was originally studied in the 2017 research article and subsequently patented by the French research institute. “This component of AB-1009, protected by patents held by Genethon and other French institutions, is now licensed to AskBio for the development of a gene therapy product to treat Pompe disease,” states the press release.

AskBio at a glance

AskBio has been active in the gene therapy field since 2001, when AAV pioneer Jude Samulski co-founded the company alongside life sciences attorney Sheila Mikhail and gene therapy scientist Xiao Xiao, PhD.

Headquartered in Durham, North Carolina, AskBio expanded its European footprint in 2019 through the acquisition of Synpromics Ltd., an Edinburgh-based company specializing in gene control synthetic promoter technology, bioinformatics, and data-driven design. Since then, the US and UK sites have operated in parallel.

In 2020, Bayer acquired AskBio for $2 billion (approximately €1.7 billion) upfront. The deal also included up to an additional $2 billion in milestone payments and gave Bayer control of AskBio’s AAV gene therapy platform and a pipeline led by clinical-stage programs for Parkinson’s disease, congestive heart failure, and Pompe disease. AskBio continues to operate independently as a Bayer subsidiary.

An agreement that came a little late?

Genethon’s announcement came just one week after AskBio reported that the US Food and Drug Administration (FDA) had accepted its Investigational New Drug (IND) application for AB-1009, clearing the therapy to enter a phase 1/2 clinical trial in Pompe disease.

The timing raises questions about why AskBio had already advanced AB-1009 into preclinical and regulatory stages if the program relied on patents held by Genethon and other French institutions. Neither Genethon nor AskBio immediately responded to inquiries from European Biotechnology Magazine seeking clarification.

AskBio also initiated a clinical trial of another asset, ACTUS-101 (NCT03533673), in participants with Pompe disease in 2018. The company said the trial will remain active, but it is no longer recruiting new participants. It will continue to completion with the patients already enrolled.