Huidagene: A promising CRISPR-CAS13 technology from China (Shanghai)

Huidagene Therapeutics is a biotechnology company based in Shanghai and New Jersey, focused on the discovery, engineering and development of novel drugs based on CRISPR technology. The company focuses on muscular, ophthalmic and neurological disorders.

Huidagene has developed HG204, an adeno-associated virus (AAV) vector delivering an innovative CRISPR-Cas13 complex into cells. Specially designed for MECP2 gene duplication syndrome, HG204 aims to regulate the overproduction of MeCP2 protein by inducing the degradation of excess MECP2 RNA. This innovative drug candidate has the potential to cure MECP2 gene duplication syndrome with a single administration.

Our organisation has had productive discussions with Huidagene management to find out more about this innovative technology and what it represents for the future. Preclinical studies using HG204 in a humanised mouse model have demonstrated a significant reduction in MeCP2 protein and a reversal of symptoms. In addition to the fact that these results are encouraging, this is also the second proof of the reversibility of the syndrome's symptoms following the regulation of MECP2 protein levels (as previously demonstrated by the administration of ASO by Prof. H. Zoghbi).

Huidagene is currently finalising preclinical studies on HG204 and preparing for a clinical trial for patients in China, which is due to start in summer 2024. If this study proves conclusive, Huidagene plans to conduct clinical trials in other countries, such as the United States and Europe, in 2025-2026.

On 31 October, the US Food and Drug Administration (FDA) granted HG204 rare paediatric disease and orphan drug designations. This regulatory designation is a first step towards a clinical trial outside China, and will enable an accelerated review of the dossier when the drug is registered. Although the news is totally unexpected, it brings additional hope to the community.

Our team will stay in touch with Huidagene and keep our community informed of any new developments.