AskBio Announces First Patient Randomized in Phase 1 Trial of Gene Therapy for Multiple System Atrophy-Parkinsonian

“Enrolling the first patient in our REGENERATE MSA-101 trial is an important step in AskBio’s ongoing work to advance GDNF gene therapy,” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “The clinical advancement of AB-1005 for the treatment of MSA-P comes as we are gathering the results of our Phase 1 programs in Parkinson’s. Taken together we expect the outcomes of these trials to meaningfully contribute to the body of scientific knowledge related to the potential of GDNF gene therapy in the treatment of these debilitating central nervous system diseases. We believe these data, combined with our exploration of AAV-based intracranial gene delivery and our close collaborations with clinical neurosurgical centers of excellence, will support our goal of successfully developing and eventually delivering to patients these much-needed treatments.”

Krystof Bankiewicz, PhD, is a principal investigator from UCSF and one of the founders of this technology.

More