China-based HuidaGene (Shanghai) Biotechnology Co., Ltd has announced receiving Investigational New Drug (IND) approval from the US FDA for its ophthalmology gene therapy HG004. The company plans to initiate a global, multi-center clinical study for the drug, a single-administered, non-adeno-associated virus serotype 2 (non-AAV2) gene replacement therapy, targeting retinopathy associated with RPE65 gene mutation. The study will be launched in multiple countries imminently.
Preclinical Study Results
A preclinical comparative study based on the same dose of HG004 and AAV2 demonstrated that retinal function recovery in the Rpe65 gene knockout mouse model increased by 67.6% with HG004 and 35.8% with AAV2 at the 17th week after a single injection. Compared to AAV2, HG004 showed better retinal pigment epithelium (RPE) transduction efficiency. This suggests that HG004 may reduce the total dose of the carrier, thereby lowering the risk of AAV vector-related immunogenicity or ocular adverse events.
Clinical Trial Progress
An investigator-initiated trial (ITT) for HG004 was launched at the end of last year. Preliminary results indicated that within 7 days of a single injection of HG004, a low dose (with a carrier dose about 1/25th of the AAV2-hRPE65 gene therapy product already on the market) significantly and substantially improved vision in patients who were on the verge of complete blindness.-Fineline Info & Tech
