First participants randomized in AskBio Phase II gene therapy trial for Parkinson’s disease

The REGENERATE-PD trial, launched by AskBio, is testing an experimental treatment called AB-1005 (also known as AAV2-GDNF), a gene therapy aimed at slowing or stopping the progression of PD symptoms. AB-1005 delivers a protein, called glial cell line-derived neurotrophic factor (GDNF), directly into specific regions of the brain. GDNF has shown promise in lab studies for helping the survival and function of brain cells that produce dopamine, a crucial chemical in movement control that is lost in Parkinson’s disease. The REGENERATE-PD trial is in its Phase II, and it involves 87 participants between the ages of 45 and 75, who are in the moderate stages of Parkinson's. This randomized, double-blind study will compare the effects of AB-1005 to a placebo, assessing how well it can help with symptoms and improve quality of life. In an earlier trial, called Phase Ib, AB-1005 showed promising results. At the 36-month mark, patients who had received the therapy experienced fewer side effects and some improvements in movement. Most importantly, the treatment was well-tolerated with no serious adverse events. These findings offer hope for people with Parkinson's who currently don’t have enough relief from existing treatments. Dr. Rajesh Pahwa, a leading expert in Parkinson’s treatment and the principal investigator of the REGENERATE-PD trial, emphasized the need for more effective therapies. “There is a significant need for neurorestorative therapies in Parkinson’s,” he said, adding that the advancement of gene therapy is a hopeful sign for both patients and physicians. Currently, the trial is recruiting participants in the United States, Germany, Poland, and the United Kingdom, with results expected in the coming years. However, as the trial progresses, researchers will be working to determine if this gene therapy could become a treatment option for all Parkinson’s patients, or if it’s more effective for certain groups. Although AB-1005 has not yet been approved for use, its innovative approach is opening up new possibilities in the search for treatments that address the underlying causes of Parkinson’s disease, rather than just masking its symptoms. If successful, gene therapy could become a game-changer for people living with Parkinson’s disease, offering a potential new path to slowing or even halting the condition’s progression. For more information about the REGENERATE-PD trial, or to see if you qualify for participation, visit the clinical trial website or askbio.com.