AskBio Inc., a gene therapy subsidiary of Bayer AG, has completed enrollment in its Phase 1 clinical trial evaluating AB-1005 gene therapy for multiple system atrophy-parkinsonian type (MSA-P), marking a significant milestone in developing treatments for this rare and devastating neurodegenerative disease.
The REGENERATE MSA-101 trial enrolled 11 participants aged 35 to 75 years with clinically diagnosed MSA-P across five leading medical centers, including Ohio State University Wexner Medical Center, University of California Irvine, Quest Research Institute, Vanderbilt University, and New York University Langone.
Addressing Critical Unmet Medical Need
MSA-P affects approximately 400,000 people worldwide and represents a particularly challenging neurodegenerative condition. The disease can initially be difficult to distinguish from Parkinson's disease but is marked by slow movement, lack of coordination, imbalance, dizziness, and fainting. Patients experience progressive loss of nerve cells in the brain and spinal cord, leading to increasing difficulty with movement and autonomic dysfunction.
"There is currently no disease-modifying treatment to stop or slow the progression of MSA, which makes the completion of enrollment in REGENERATE MSA-101 an important step in AskBio's efforts to advance this clinical program and potentially bring a safe treatment to patients," said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson's and MSA at AskBio.
The disease typically develops in people over 50 years of age and follows a rapid progression within 5-10 years, making the development of effective treatments particularly urgent.
Novel Gene Therapy Approach
AB-1005 represents an innovative approach to treating neurodegenerative diseases through gene therapy. The investigational treatment uses an adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene.
GDNF is a homodimer and distantly related member of the transforming growth factor-β superfamily that has shown promise in experimental models. In midbrain cell culture studies, recombinant human GDNF promoted the survival and morphological differentiation of dopaminergic neurons and increased their high-affinity dopamine uptake.
The therapy allows for stable and continuous expression of GDNF in localized brain regions after direct neurosurgical injection using MRI-monitored convection-enhanced delivery to the putamen.
Trial Design and Timeline
REGENERATE MSA-101 is designed as a randomized, double-blind, placebo-controlled Phase 1 trial focused on determining the safety of AB-1005. Participants were randomized on the day of surgery to receive either AB-1005 or a non-invasive control surgery without any infusion to the putamen.
The trial includes a three-year follow-up period, with initial results expected after all participants complete one year of blind clinical assessments and the trial is unblinded. Brain Neurotherapy Bio, Inc., an AskBio subsidiary, serves as the sponsor for the study.
Broader Development Program
The completion of MSA-P enrollment follows significant progress in AskBio's broader AB-1005 development program. In February 2025, the company received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for AB-1005 in Parkinson's disease, following presentation of Phase 1b 36-month data that demonstrated a favorable safety profile and continued positive trends in clinical outcome measures with no product-related serious adverse events.
AskBio is simultaneously advancing AB-1005 in the REGENERATE-PD trial, a randomized, double-blind, surgically controlled Phase 2 clinical trial evaluating safety and efficacy in adults aged 45-75 years with moderate-stage Parkinson's disease. The first participants in that trial were randomized in January 2025.
The company's comprehensive gene therapy platform includes over 900 employees across five countries and holds more than 600 patents and patent applications in areas such as AAV production and chimeric capsids, positioning it as a leader in the gene therapy field for neurodegenerative diseases.
