AskBio wins FDA RMAT designation for Parkinson’s gene therapy

Germany-based Bayer’s (BAYN: DE) wholly-owned gene therapy company AskBio today announced that its investigational gene therapy AB-1005 for the treatment of Parkinson’s disease (PD) has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the US Food and Drug Administration (FDA).

AskBio said it met the criteria for RMAT designation after a review of information and data provided by the company, including clinical evidence from an open label, uncontrolled study Phase Ib trial of AB-1005. The first participants in the AB-1005 Phase II REGENERATE-PD clinical trial have been randomized in the USA, and additional study sites in the USA, Germany, Poland, and the UK are expected to be open for enrollment in the first half of 2025.

AB-1005 has not been approved by any regulatory authority, and its efficacy and safety have not been fully established or evaluated. However, the candidate has previously been granted FDA Fast Track designation for AB-1005, and awarded the Innovation Passport, the UK Medicines and Healthcare products Regulatory Agency (MHRA) innovative medicine designation, for the treatment of Parkinson’s disease.