AskBio secures FDA Fast Track designation for AB-1005 to treat PD

AB-1005 is a gene therapy developed based on adeno-associated viral vector serotype 2 containing the human glial cell line-derived neurotrophic factor (AAV2-GDNF) transgene, evaluated as a potential treatment for diseases like PD

AskBio, a gene therapy subsidiary of Bayer has received the US Food and Drug Administration (FDA) Fast Track Designation for its investigational Parkinson’s disease (PD) drug, AB-1005.

The experimental drug also received the Innovation Passport, the UK Medicines and Healthcare Products Regulatory Agency (MHRA’s) innovative medicine designation, to treat PD.

AB-1005 is a gene therapy developed based on adeno-associated viral vector serotype 2 containing the human glial cell line-derived neurotrophic factor (AAV2-GDNF) transgene.

It works by enabling a stable and continuous expression of GDNF in the regions of the brain after direct neurosurgical injection with MRI-monitored convection-enhanced delivery.

GDNF is a homodimer that is a remotely related member of the growth factor-β superfamily, which has been evaluated as a potential treatment for diseases like PD.

Earlier this year, AskBio reported positive results for AB-1005, from the 18-month Phase 1b clinical trial, which met its primary objective of evaluating the safety of the drug.

July 17, 2024