The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to HuidaGene Therapeutics’ HG004 gene replacement therapy.
The gene therapy is used for the treatment of inherited retinal disease (IRDs) caused by RPE65 mutations.
Retina is impacted in patients who suffer from RPE65 mutation-associated IRDs and this condition is passed on to children.
IRDs are a group of rare blinding conditions caused by biallelic RPE65 mutations.
HuidaGene co-founder and CEO Xuan Yao said, “We are pleased to have received this significant regulatory feedback from the US FDA. Receiving ODD is an important milestone as we are advancing our HG004 gene replacement therapy program to clinical trial designed to provide safe, durable, and high-quality treatment to children and adults suffering from RPE65 mutation-associated inherited retinal diseases.
“It also underscores the importance of bringing this novel therapy to patients with severe visual impairment or blindness, and strongly motivates us to expedite the clinical development of HG004.”
Using the recombinant non-adeno-associated virus serotype 2 (non-AAV2) vector, HG004 therapy delivers a functional human RPE65 gene to the retina.
It helps to restore, treat, and prevent blindness in children and adults with RPE65 mutation-associated IRDs.
During the extensive preclinical studies, superior transduction efficiency was seen of the RPE layer and the recovery of the retinal functions under HG004 when compared with AAV2-mediated gene replacement therapy via the company’s independently developed Rpe65 gene knockout (KO) murine disease model leveraging the clustered regularly interspaced palindromic repeats (CRISPR) genome-editing system.