Award
NIH Reporter #5R44NS141514-02
Treatment of GM1 gangliosidosis CNS manifestations by AAV systemic expression of lectin-enhanced enzyme
Recipient
BIOSTRATEGIES, LC
Award Amount
$1,500,000.00
Ceiling
$1,500,000.00
Awarded
September 14, 2026
Identifier
5R44NS141514-02
This award funds the development of BIOS-GM1, a gene therapy for GM1 gangliosidosis that uses AAV vectors to deliver enzyme replacement systemically, crossing the blood-brain barrier to treat CNS manifestations, aiming to provide the first effective treatment for this neurodegenerative disease.
Description
BioStrategies LC proposes to complete IND-enabling studies to develop BIOS-GM1, a novel gene therapy for GM1 gangliosidosis. The therapy combines delivery-enhanced enzyme replacement with gene therapy to treat CNS and other organ pathologies. The approach involves using AAV-based vectors to deliver genes to produce functional acid β-galactosidase, crossing the blood-brain barrier without intrathecal or intraventricular administration. Successful development could provide the first effective treatment for GM1, addressing a critical unmet medical need.