May 27, 2025

Research Triangle Park, N.C.– MAY 27, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced today the recent publication of the complete results of its Phase 1b trial of AB-1005, a glial cell line-derived neurotrophic factor (GDNF) investigational gene therapy for the treatment of Parkinson’s disease (PD), in Movement Disorders, an official peer-reviewed journal of the International Parkinson and Movement Disorder Society.1 “The exploration of the neurorestorative and neuroprotective potential of GDNF gene transfer is critical to advancing our understanding of AB-1005, which may one day be a groundbreaking treatment to slow the progression of Parkinson’s disease,” said Chad Christine, MD, Professor of Neurology at the University of California, San Francisco, and publication author. “These encouraging results support further evaluation of this therapy in a randomized trial.” The publication, which is available online, states that treatment with AB-1005 was well tolerated with no serious adverse events related to GDNF gene therapy in all 11 participants and is associated with numerical stability in clinical assessment readouts (mild cohort) and improvement (moderate cohort) in clinical assessments of motor function at 18 months post-gene transfer. In addition to these data, which were first presented in April 2024 at the American Academy of Neurology Annual Meeting, the publication notes that non-motor assessments remained numerically stable throughout the 18-month follow-up in the mild and moderate cohorts. Stability findings may be an indication of the potential for AB-1005 to positively affect disease progression.1,2 “These results support the continued study of AB-1005 as a potential gene therapy to slow the progression of Parkinson’s disease,” said Lila Collins, PhD, Associate Director of Portfolio Development & Review at the California Institute for Regenerative Medicine. “We are pleased to have supported this important trial and look forward to AskBio’s Phase 2 REGENERATE-PD results, which will provide additional insights into the durability of the clinical response and potential disease-modifying effect of AB-1005.” In February 2025, AB-1005 was granted Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration.3 AB-1005 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been fully established or evaluated. About Parkinson’s disease  Parkinson’s disease (PD) is a progressive neurodegenerative disease.4 It has a significant impact on a person’s daily life.4  In PD, the death of dopamine-producing nerve cells in the brain leads to the continuous loss of motor function.5 Symptoms include tremors, muscle rigidity, and slowness of movement.6 Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression.6 Symptoms typically intensify over time and make everyday tasks increasingly demanding.6 The prevalence of PD has doubled over the past 25 years.4 Today, more than 10 million people worldwide are estimated to be living with PD.7 This makes it the world’s second most prevalent neurodegenerative disease.8 It is also the most frequent movement disorder.4,9  At present there is no cure, and current treatment options lack the holistic management of symptoms, ...read the news