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# AskBio Gets Fast Track for Parkinson’s Gene Therapy
- URL: https://www.fdaweb.com/askbio-gets-fast-track-for-parkinsons-gene-therapy/
- Published: 2024-07-12T12:00:00.000Z
- Updated: 2026-09-14T14:35:18.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5157338

FDA has granted Bayer-owned Asklepios BioPharmaceutical (AskBio) a fast track designation for AB-1005, a gene therapy candidate for treating moderate Parkinson’s disease. The investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy is designed for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with MRI-monitored convection enhanced delivery, according to the company.

“GDNF is a homodimer that is a distantly related member of the transforming growth factor-β superfamily,” AskBio explains. “In midbrain neuronal cell cultures, recombinant human GDNF promoted the survival and morphological differentiation of dopaminergic neurons and increased their high-affinity dopamine uptake. GDNF has long been evaluated as a potential disease modifying neurorestorative treatment for diseases, such as Parkinson’s, marked by progressive degeneration of midbrain dopaminergic neurons.”

Currently, the company says it is enrolling patients in the Phase 2 REGENERATE-PD clinical trial. In the randomized, double-blind, surgery-controlled trial, 87 subjects will receive either bilateral image-guided infusion of AB-1005 into the putamen, a single dose (active treatment arm), or bilateral partial burr/twist holes (control arm).