MSA typically affects individuals over 50, with symptoms progressing rapidly over 5 to 10 years due to nerve cell loss in the brain and spinal cord.

AskBio has completed enrollment for its Phase 1 clinical trial of AB-1005, a gene therapy targeting multiple system atrophy-parkinsonian type (MSA-P).

This trial, named REGENERATE MSA-101, involves delivering AB-1005 directly to the putamen in the brain to evaluate its safety.

Currently, there are no disease-modifying treatments for MSA, making this trial a significant step forward in developing potential therapies.

MSA-P is a rare neurodegenerative disorder affecting about 400,000 people worldwide, with symptoms like slow movement, imbalance, and fainting.

AB-1005 is based on an adeno-associated viral vector containing the human GDNF transgene, designed to slow or halt disease progression.

The progress of AB-1005 underscores AskBio’s broader efforts to develop gene therapies for neurodegenerative diseases with unmet medical needs.

In addition to MSA-P, AskBio is exploring AB-1005 in Parkinson’s disease through the REGENERATE-PD Phase 2 trial, which assesses safety and efficacy in patients aged 45-75 with moderate-stage Parkinson’s.

AskBio considers the completion of enrollment a key milestone, especially following recent positive safety data from its Parkinson’s disease program.