Alia Therapeutics RHO gene therapy

Alia Therapeutics is developing a mutation-independent gene editing therapy for the treatment of Autosomal Dominant Retinitis Pigmentosa (adRP). The program utilizes a single Adeno-Associated Virus (AAV) vector to deliver proprietary CRISPR nucleases, including Cas9 and Cas12 variants, which are optimized through the company's proprietary PAM Prediction Algorithm and EVOlution platform. The therapeutic strategy involves the mutation-independent inactivation or editing of the rhodopsin (RHO) gene, aiming to provide a treatment option for adRP patients regardless of their specific genetic mutation. This approach is designed to address the high genetic heterogeneity of adRP by targeting the RHO gene in a manner that is not specific to any single disease-causing mutation.

Other names

Alia Therapeutics-RHO (rhodopsin gene, mutation-independent)-AAV vector-gene editing (CRISPR)-gene therapy-Autosomal Dominant Retinitis Pigmentosa (adRP)RHO mutation-independent CRISPR therapy