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

Huidagene Therapeutics is a biotechnology company based in Shanghai and New Jersey, focusing on discovering, engineering, and developing novel CRISPR-based medicines. The company focuses on muscular, ophthalmic and neurological disorders.

Huidagene has developed HG204, an adeno-associated viral (AAV) vector delivering a novel CRISPR-Cas13 complex into cells. Designed for MECP2 duplication syndrome, HG204 aims to regulate the overproduction of the MeCP2 protein levels by inducing the degradation of overexpressed MECP2 RNA. This innovative drug candidate holds the potential to cure MDS through a single administration.

Our organization has had productive discussions with Huidagene’s leaders to learn more about this innovative technology and what it means for the future. Preclinical studies using HG204 in a humanized MDS mouse model demonstrated a significant reduction of MeCP2 protein and reversal of the symptoms. While these are encouraging results, this is also the second proof of the reversibility of MDS symptoms after the regulation of MECP2 protein levels (as previously demonstrated by the administration of the ASO by Prof. H. Zoghbi & Team).

Huidagene is in the process of finalizing pre-clinical studies with HG204 and getting ready for a clinical trial for patients in China, scheduled to begin in mid-2024. If this trial is successful, Huidagene will plan clinical trials in other countries such as the USA and Europe in 2025-2026.

On the 31st of October, the FDA (Food and Drug Administration, the health authority of the US) granted both Rare Pediatric Disease and Orphan Drug Designations to HG204. This regulatory designation is a first step towards a clinical trial outside of China and shall enable an accelerated review of the dossier upon registration of the drug. While the news was totally unexpected, it brings additional hope to the community.

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

To press release