# 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.

[View original record](https://reporter.nih.gov/search/IgE1H2ioLUOz0qmA4MDg0Q/project-details/11627834)