AskBio Initiates Phase 2 Trial of AB-1005 Gene Therapy for Parkinson's Disease
核心洞察
AskBio's REGENERATE-PD, a Phase 2 clinical trial, has begun recruitment to evaluate AB-1005 for moderate-stage Parkinson's disease (搜索).
AB-1005 is an investigational adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF (搜索)) gene therapy.
The Phase 2 trial follows encouraging Phase 1b results, which demonstrated that AB-1005 was well-tolerated with no related serious adverse events.
Asklepios BioPharmaceutical, Inc. (AskBio), a subsidiary of Bayer AG, has commenced recruitment for the REGENERATE-PD, a Phase 2 clinical trial designed to assess the efficacy and safety of AB-1005, an investigational adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF (搜索)) gene therapy, for the treatment of moderate-stage Parkinson’s disease. The trial aims to enhance levels of GDNF, a naturally occurring growth factor, to promote the survival and functioning of vulnerable brain cells that degenerate in Parkinson’s disease.
REGENERATE-PD Trial Details
The REGENERATE-PD trial is a Phase 2, randomized, double-blind, surgery-controlled study. It will evaluate the efficacy and safety of intraputaminal AAV2-GDNF (搜索) in adults aged 45-75 years with moderate-stage Parkinson's disease (搜索). Participants will receive either a single dose of bilateral image-guided infusion of AAV2-GDNF into the putamen or undergo bilateral partial burr/twist holes as a control. The trial anticipates enrolling approximately 87 subjects across study sites in the United States, UK, and Europe.
AB-1005: A Novel Gene Therapy Approach
AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF (搜索)) transgene. This allows for stable and continuous expression of GDNF in localized regions of the brain following direct neurosurgical injection with MRI-monitored convection enhanced delivery. GDNF promotes the survival and morphological differentiation of dopaminergic neurons and increases their high-affinity dopamine uptake.
Previous Clinical Data
The initiation of the Phase 2 trial follows encouraging results from the Phase 1b study of AB-1005. The Phase 1b trial met its primary endpoint, evaluating the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. The gene therapy was well-tolerated, with no serious adverse events related to AB-1005 observed in all 11 patients at 18 months.
Parkinson's Disease: An Unmet Need
Parkinson’s disease is a progressive neurodegenerative disorder affecting more than 10 million people worldwide. It is characterized by the death of nerve cells in the brain, leading to decreased dopamine levels. At diagnosis, patients are estimated to have already lost 50-80% of their dopaminergic neurons. This neuronal loss results in motor function decline and symptoms such as tremors, muscle rigidity, and slowed movement. Current treatments often decrease in effectiveness over time, highlighting the need for new therapeutic options.
Expert Commentary
"By enhancing levels of a naturally occurring growth factor, glial cell line-derived neurotrophic factor (GDNF (搜索)) gene therapy is intended to promote the survival and functioning of vulnerable brain cells that degenerate in Parkinson’s disease," said Alan Whone, MD, PhD, Consultant Senior Lecturer in Movement Disorder, Bristol Medical School, and Honorary Consultant Neurologist at North Bristol Trust, UK. "The advancement of AB-1005 is a significant milestone in the development of a gene therapy for Parkinson’s disease and has the potential to bring an effective treatment one step closer to patients."
Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio, added, "Following the encouraging results from the Phase 1b study and the presentation of 18-month data at the American Association of Neurology (AAN) meeting in April, we are excited to be progressing AB-1005 to this larger, Phase 2 study. This latest advancement highlights our confidence in the potential of AB-1005 to provide a transformative impact for patients with Parkinson’s disease."