AskBio secures IND acceptance for late-onset Pompe disease gene therapy

AskBio Inc., an RTP-based gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, has received U.S. Food and Drug Administration (FDA) acceptance of its Investigational New Drug (IND) application for AB-1009, an adeno-associated virus gene therapy in development for late-onset Pompe disease (LOPD).

IND approval allows the AB-1009 program to move into Phase 1/Phase 2 studies, with patient recruitment for a U.S. clinical trial expected to begin in early 2026. The therapy was also granted FDA Fast Track and Orphan Drug designations.

“These advancements in the AB-1009 program, particularly the recently granted regulatory designations, highlight the recognized need for late-onset Pompe treatments,” said Mansuo Shannon, chief scientific officer of AskBio.

Pompe disease is a rare, inherited condition that prevents the body from breaking down stored sugars properly, leading to progressive muscle weakness and serious health complications if not diagnosed and treated early. The LOPD form of the disease is characterized by progressive muscle weakness and increasing difficulty breathing over time.

AB-1009 is being studied for its potential to address the underlying genetic defect of LOPD and to explore whether it can increase production of the acid alpha-glucosidase (GAA) enzyme, which is deficient in patients with the disease.

Success through partnership

The development of the AB-1009 gene therapy was a collaborative effort involving Genethon, Belief BioMed Inc. and Duke University.  

“AskBio and Duke University have been collaborating for years in Pompe, and the expertise of both organizations has contributed greatly to Pompe’s existing body of scientific knowledge,” said Canwen Jiang, chief development officer and chief medical officer of AskBio. “Technology licensed from Duke is incorporated into the AB 1009 program, and Duke is an AB-1009 program clinical trial site.”

Researchers led by Giuseppe Ronzitti at Genethon developed and demonstrated the preclinical efficacy of a gene therapy approach using a transgene encoding a form of GAA. This component of AB-1009 was recently licensed to AskBio for the treatment of Pompe disease through an exclusive worldwide agreement.

In September 2024, Belief BioMed and AskBio entered a strategic collaboration to explore the potential for new gene therapies. BBM granted AskBio a sublicense to use the proprietary adeno-associated virus capsid in the AB-1009 program and provided adeno-associated virus vector production services.

"We are delighted to learn that our partner AskBio has received IND acceptance for its gene therapy,” said Xiao Xiao, co-founder, chairman and chief science officer of Belief BioMed. “This marks a significant milestone in our strategic collaboration and represents a practical achievement in globalized innovation within the gene therapy field.”

AB 1009 is an investigational gene therapy that has not been approved by any regulatory authority, the company said, and safety and efficacy have not been established.