The startup develops gene therapy products targeting neurogenetic diseases in children, specifically focusing on Mucopolysaccharidoses such as MPS I, II, IIIA, IIIB, and IIIC. Their therapies aim to provide effective treatment options for these rare genetic disorders, improving patient outcomes and quality of life.
Funding
$250K raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Children suffering from rare neurogenetic diseases like Mucopolysaccharidoses (MPS) I, II, IIIA, IIIB, and IIIC currently have limited effective treatment options. These diseases, affecting the central nervous system and critical metabolic pathways, lead to significant morbidity and reduced quality of life for patients and their families.
Solution
NeuroGT is developing advanced gene therapy products designed to target the underlying genetic causes of rare neurogenetic diseases, offering a potential new treatment paradigm. Their AAV9 gene replacement therapy platform precisely targets the nervous system, delivering life-changing genes across the blood-brain barrier. The Antibody Cleaver (AbC) Technology enables AAV dosing and re-dosing by transiently depleting pre-existing anti-AAV antibodies, broadening patient eligibility. NeuroGT is also developing AAV-mediated EV-mRNA Cargo Technology, amplifying therapeutic outcomes by facilitating cross-correction using AAV-mediated extracellular vesicles (EV) technology to disperse functional protein into bystander cells.
Target Audience
The primary target audience includes children affected by rare neurogenetic diseases, their families, and the clinicians who treat them.
Features
- AAV9 Gene Replacement Therapy Platform for targeted delivery to the nervous system
- Antibody Cleaver (AbC) Technology to enable re-dosing of AAV therapies
- AAV-mediated EV-mRNA Cargo Technology for enhanced therapeutic outcomes
- Focus on Mucopolysaccharidoses (MPS) including MPS I (Hurler Syndrome), MPS II (Hunter Syndrome), and MPS III (Sanfilippo Syndrome)
- State-of-the-art AAV manufacturing using novel suspension cell line to support in-house development and external partnerships