Policy & Regulation
Asklepios BioPharmaceutical randomises first subject in AB-1005 Phase one REGENERATE MSA-101 clinical trial
20 November 2023 -

Asklepios BioPharmaceutical, Inc. (AskBio), a US-based gene therapy company wholly owned and independently operated as a subsidiary of Germany-based Bayer AG, announced on Friday that the first subject has been randomised in the Phase one REGENERATE MSA-101 clinical trial of AB-1005, a gene therapy being developed as a treatment for multiple system atrophy-parkinsonian type (MSA-P), at the Ohio State University Wexner Medical Center.

This development is claimed to represent an important milestone in the development of AB-1005 gene therapy, an adeno-associated viral vector encoding for glial cell line-derived neurotrophic factor (AAV2-GDNF) that is delivered to the putamen, and brings this therapeutic one step closer to potentially reaching patients.

The product is also currently being assessed for the treatment of mild to moderate Parkinson's disease with the enrolment of the Phase 1b study having now been completed.

The Phase one REGENERATE MSA-101 trial, a randomised, double-blind, placebo-controlled trial, is aimed at assessing the safety of AB-1005. The trial is to include adults aged 35 to 75 years, with a clinical diagnosis of MSA-P (as defined by current consensus criteria), who are suitable surgical candidates. The trial is expected to enrol up to nine subjects who will be randomised on the day of surgery to receive either the AAV2-GDNF investigational medicinal product or a control surgery.