AskBio Inc., a gene therapy company wholly owned by Bayer AG, has received US FDA acceptance for its Investigational New Drug application for AB-1009.
This milestone allows the AB-1009 program to advance into Phase 1/2 clinical development in the United States.
What the FDA IND Acceptance Means?
The FDA clearance enables AskBio to begin a first-in-human clinical trial evaluating the safety of AB-1009 in patients with late-onset Pompe disease.
Key highlights include:
- AB-1009 is an investigational AAV-based gene therapy
- The Phase 1/2 trial will be conducted in the US
- First patient enrollment is expected in early 2026
This marks a critical transition from preclinical research to clinical evaluation.
Strategic Collaboration with Belief BioMed
The AB-1009 program is supported by a strategic collaboration between AskBio and Belief BioMed (BBM), announced in September 2024.
Under this collaboration:
- BBM sublicensed its proprietary AAV capsid to AskBio
- BBM provided AAV vector production services
- Both teams worked closely during IND-enabling activities
BBM’s technical and manufacturing contributions played a key role in achieving IND acceptance.
Leadership Perspective on the Milestone
Dr. Xiao Xiao, Co-founder and Chief Science Officer of Belief BioMed, highlighted the significance of the achievement.
He emphasized the strength of the partnership, AskBio’s R&D execution, and the value of global collaboration in advancing gene therapies.
Understanding Late-Onset Pompe Disease
Pompe disease is a rare, inherited lysosomal storage disorder caused by deficiency of acid alpha-glucosidase (GAA).
This deficiency leads to glycogen accumulation in cells, resulting in progressive muscle damage.
Key disease characteristics include:
- Severe skeletal muscle weakness
- Diaphragmatic involvement and respiratory insufficiency
- A wide clinical spectrum from infantile to late-onset forms
Late-onset Pompe disease primarily affects skeletal muscles and progresses more slowly than the infantile form.
Globally, Pompe disease is estimated to affect between 5,000 and 10,000 people.
About AB-1009
AB-1009 is designed as a one-time AAV gene therapy intended to address the underlying genetic cause of Pompe disease.
By enabling sustained GAA expression, the therapy aims to reduce glycogen accumulation and slow disease progression.
About Belief BioMed
Belief BioMed is a global biotech company focused on gene therapy research, manufacturing, and clinical development.
The company has built:
- Proprietary AAV capsid and vector platforms
- Commercial-scale viral vector manufacturing capabilities
- A broad pipeline across rare and chronic genetic diseases
Several BBM programs are already in clinical development, with regulatory approvals achieved in China.
Why This Development Matters?
The IND acceptance for AB-1009 reflects continued momentum in gene therapy innovation for rare neuromuscular diseases.
It also highlights the growing role of strategic partnerships in accelerating clinical translation for complex genetic disorders.
