AskBio Advances Gene Therapy Trial for Pompe Disease with First Patient Dosed in AB-1009 Study

AskBio Inc., a wholly owned subsidiary of Bayer AG, has announced that the first participant has been dosed in its Phase 1/2 clinical trial of AB-1009, an investigational gene therapy for late-onset Pompe disease (LOPD).

The milestone marks the start of clinical evaluation for a treatment designed to address the underlying genetic cause of a rare and progressive neuromuscular disorder that currently has limited long-term therapeutic options.

What Is AB-1009?

AB-1009 is an investigational adeno-associated virus (AAV)-based gene therapy designed to treat the root cause of Pompe disease by restoring enzyme production.

Mechanism of Action

Pompe disease is caused by deficiency of the enzyme acid alpha-glucosidase (GAA), leading to glycogen buildup in cells.

AB-1009 aims to:

• Deliver a functional gene encoding GAA
• Enable sustained enzyme production
• Reduce glycogen accumulation in muscle cells
• Address the underlying genetic defect rather than symptoms alone

PROGRESS-GT LOPD Clinical Trial Begins

The first-in-human study, named PROGRESS-GT LOPD (NCT07282847), is a Phase 1/2 clinical trial evaluating AB-1009 in adults with late-onset Pompe disease.

Trial Design

• Type: Non-randomized, open-label study
• Phase: 1/2 dose-escalation trial
• Enrollment: Approximately 12 participants
• Location: United States

Key Objectives

The study will assess:

• Safety
• Tolerability
• Early signs of efficacy

Understanding Late-Onset Pompe Disease

Pompe disease is a rare genetic lysosomal storage disorder affecting an estimated:

• 5,000 to 10,000 people worldwide

It results from mutations in the GAA gene, leading to:

• Progressive muscle weakness
• Respiratory insufficiency
• Loss of motor function over time

Disease Forms

• Infantile-onset Pompe disease
  • Rapid progression
  • Severe cardiac involvement
• Late-onset Pompe disease (LOPD)
  • Slower progression
  • Primarily affects skeletal and respiratory muscles

Despite existing enzyme replacement therapies, patients often experience declining response over time, highlighting a persistent unmet medical need.

Why Gene Therapy Matters in Pompe Disease?

Current standard treatments include enzyme replacement therapies using recombinant human GAA. While these can help manage symptoms, they require ongoing administration and may lose effectiveness in some patients.

Gene therapy approaches like AB-1009 aim to:

• Provide long-term enzyme production
• Reduce treatment burden
• Improve disease stability over time
• Potentially modify disease progression

Regulatory Designations Support Development

AB-1009 has received multiple regulatory designations from the U.S. Food and Drug Administration (FDA), including:

• Fast Track Designation
• Orphan Drug Designation
• Investigational New Drug (IND) clearance

What These Mean

• Fast Track: Enables closer regulatory interaction and potential expedited review
• Orphan Drug Status: Supports development for rare diseases and may provide market exclusivity benefits
• IND clearance: Allows initiation of clinical trials in the United States

Expert Perspective on the Trial

Dr. Tahseen Mozaffar, Principal Investigator of the trial at UCI Health ALS & Neuromuscular Center, noted that current therapies may not fully meet long-term patient needs.

He emphasized the importance of continued research to improve outcomes for people living with Pompe disease and advance new treatment options.

AskBio’s Strategic Focus on Rare Disease Gene Therapy

AskBio Inc. is developing a pipeline of gene therapies targeting both rare and more common diseases.

The company aims to:

• Expand access to gene therapy platforms
• Develop one-time or long-duration treatments
• Address diseases with high unmet medical need

AskBio operates as a fully integrated subsidiary of Bayer AG, which focuses on healthcare, agriculture, and nutrition under its “Health for all, Hunger for none” mission.

Broader Impact for Pompe Disease Research

The initiation of this trial reflects growing momentum in gene therapy approaches for lysosomal storage disorders.

If successful, AB-1009 could potentially:

• Reduce dependence on lifelong enzyme infusions
• Improve respiratory and muscle function
• Offer a more durable treatment option for LOPD patients

However, the therapy remains in early clinical stages, and its safety and efficacy are still under evaluation.

Final Thoughts

The first dosing in the AB-1009 trial represents an important early step in advancing gene therapy for late-onset Pompe disease.

While still experimental, this approach reflects a broader shift in rare disease treatment strategy, moving from chronic enzyme replacement toward potentially one-time genetic correction therapies that target disease at its source.

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