The startup specializes in developing gene therapy treatments targeting the RHO rhodopsin gene mutation, a leading cause of inherited retinitis pigmentosa. This approach provides patients with a targeted therapeutic option to address vision loss associated with this genetic condition.
Funding
Funding not disclosed
Founders
Product
Problem
Inherited retinal dystrophies, such as retinitis pigmentosa, lead to progressive vision loss due to the degeneration of photoreceptor cells and the retinal pigment epithelium. Current treatments offer limited efficacy, primarily focusing on slowing disease progression rather than providing a curative solution. A significant challenge lies in addressing the diverse genetic mutations, particularly in the RHO gene, that cause these conditions.
Solution
VC Gene Therapy is developing gene-editing therapeutics targeting inherited retinal dystrophies, with a primary focus on retinitis pigmentosa caused by mutations in the RHO gene. Their approach utilizes genome editing and HITI (Homology-Independent Targeted Integration) gene insertion to correct the underlying genetic defects. By employing ZFN (Zinc Finger Nuclease) technology, they aim to achieve efficient and precise gene editing in adult retinal cells, comparable to CRISPR-Cas9 systems. This strategy involves inserting a functional copy of the RHO gene while suppressing the expression of the mutated gene, addressing both dominant-negative effects and restoring normal protein function.
Target Audience
The primary target audience includes patients with autosomal dominant retinitis pigmentosa caused by mutations in the RHO gene, as well as ophthalmologists and retinal specialists seeking advanced therapeutic options for inherited retinal diseases.
Features
- Utilizes ZFN-based genome editing for targeted gene correction in retinal cells.
- Employs HITI gene insertion to integrate a functional RHO gene into the genome.
- Aims to correct dominant-negative mutations and restore normal RHO gene expression.
- Collaborates with Hiroshima University for high-throughput development of highly specific ZFNs.
- Partners with Simprogen Corp. for the development of high-quality adeno-associated viral vectors for gene delivery.
- Works with Kobe Eye Center Hospital to refine gene insertion techniques.
- Focuses on developing a single treatment to address all mutations in the rhodopsin gene locus.