Gene therapy AB-1005 found safe in Phase 1b Parkinson’s trial

Treatment with AB-1005 (AAV2-GDNF), AskBio’s investigational gene therapy, was safe and well tolerated among patients with mild to moderate Parkinson’s disease, according to top-line results from a Phase 1b clinical trial. The company, wholly owned by Bayer, has also completed an 18-month data collection which demonstrated the…

Denali’s DNL201 Safely Engages Target Enzyme: Multiple Studies

Denali Therapeutics‘ DNL201, an investigational therapy for Parkinson’s disease, was well tolerated in healthy volunteers and patients, and showed evidence of engaging its target protein, according to multiple preclinical and clinical studies. LRRK2, a protein associated with Parkinson’s, is also the target of BIIB122 (formerly DNL151), which is…

Voyager’s Gene Therapy Improves Parkinson’s Patients’ Movement Over Long Term, Trial Shows

A single administration of Voyager Therapeutics gene therapy improves advanced Parkinson’s patients’ movement up to three years later, a Phase 1b trial indicates. Voyager designed VY-AADC01 to deliver the aromatic L-amino acid decarboxylase (AADC) gene to a specific brain region, the putamen. The gene provides instructions for the production of the AADC…

Phase 1b Trial of VY-AADC01 Gene Therapy for Advanced Parkinson’s Disease Shows Positive Results

An ongoing Phase 1b trial of VY-AADC01, a gene therapy for the treatment of advanced Parkinson’s disease (PD), is showing positive results, including improvement of motor function and activities of daily living, durable clinical effects, and good tolerability. The announcement was made by Voyager Therapeutics, the gene therapy company specialized…