AskBio Publishes Full Phase 1b Trial Results of AB-1005 Gene Therapy for Parkinson's in Movement Disorders

In Research Triangle Park, N.C., AskBio Inc., a gene therapy company under Bayer AG, has announced the publication of promising results from its Phase 1b trial of AB-1005, a gene therapy designed for Parkinson’s disease (PD). The study, published in the peer-reviewed journal Movement Disorders, highlights the potential of AB-1005 in treating PD through gene therapy involving glial cell line-derived neurotrophic factor (GDNF).

The Phase 1b trial involved 11 participants with mild to moderate PD and assessed the safety and efficacy of AB-1005 over an 18-month period following gene transfer. The results indicate that the therapy was well tolerated, with no serious adverse events attributed to the gene therapy, and clinical assessments showed stability in symptoms for both mild and moderate cohorts. These findings suggest that AB-1005 may have a positive impact on disease progression, warranting further investigation through a randomized trial.

Dr. Chad Christine, Professor of Neurology at the University of California, San Francisco, commented on the significance of exploring GDNF's neurorestorative and neuroprotective potential, emphasizing that AB-1005 could become a groundbreaking treatment for slowing PD progression. The study's encouraging results underscore the need for continued evaluation to fully understand the therapy's impact.

Lila Collins, PhD, Associate Director at the California Institute for Regenerative Medicine, expressed optimism about the ongoing research. She highlighted the importance of AB-1005's study in potentially modifying disease progression and looks forward to the upcoming Phase 2 REGENERATE-PD trial results, which will provide further insights into the therapy's durability and disease-modifying effects.

In February 2025, the United States Food and Drug Administration (FDA) granted AB-1005 a Regenerative Medicine Advanced Therapy (RMAT) designation, recognizing its potential in addressing unmet medical needs for PD. This designation underscores the importance of developing new therapies, as current treatment options for PD focus primarily on symptom management without altering the disease's course.

Parkinson’s disease is a progressive neurodegenerative disorder characterized by the death of dopamine-producing neurons, leading to motor function loss. Symptoms include tremors, muscle rigidity, and slow movement, along with non-motor issues like cognitive challenges and depression. The prevalence of PD has significantly increased, affecting over 10 million people globally, and is now the second most prevalent neurodegenerative disease.

The ongoing REGENERATE-PD trial, a Phase 2 study, aims to further investigate AB-1005's efficacy and safety in adults aged 45-75 with moderate-stage PD. This trial employs a randomized, double-blind, sham-controlled design and seeks to enroll 87 participants across sites in Germany, Poland, the United Kingdom, and the United States.

AB-1005 utilizes an adeno-associated viral vector (AAV2) containing the human GDNF transgene, facilitating stable GDNF expression within localized brain regions after direct neurosurgical injection. Nonclinical studies have demonstrated GDNF's ability to support dopaminergic neuron survival and differentiation, and its potential therapeutic benefits in PD are being explored through innovative gene therapy and neurosurgical delivery methods.

AskBio Inc., located in Research Triangle Park, North Carolina, is a pioneering gene therapy company with a diverse portfolio of clinical programs targeting various diseases. The company leverages its proprietary gene therapy platform, Pro10™, and has developed numerous capsids and promoters, some of which are undergoing pre-clinical and clinical evaluation. As a subsidiary of Bayer AG, AskBio is committed to developing transformative therapies that improve lives through innovation and collaboration in the field of gene therapy.

Through its groundbreaking research, AskBio continues to advance the understanding and treatment of Parkinson’s disease, striving to offer hope for more effective management and potential modification of this challenging condition.