HuidaGene: Moving towards clinical trials

In February 2024, David Covini, President of DupMECP2, visited the innovative biotech company HuidaGene Therapeutics in Shanghai. The visit provided an exciting glimpse into the future for a treatment for MECP2 duplication syndrome (MDS).

Led by Dr. Alvin Luk, CEO at HuidaGene, the Company is at the forefront of developing a groundbreaking CRISPR technology that could revolutionize the lives of children affected by MDS. 

The visit was an incredible opportunity to witness firsthand the cutting-edge facilities and the creative environment that fosters such innovative research. The office space even includes a climbing wall, promoting a dynamic and creative work atmosphere.

Alvin and his Medical Director of MDS, Dr. Gloria Tang, presented the details of the CRISPR-Cas13 RNA-editing technology for MECP2 Duplication Syndrome, named HG204. The promising data and preliminary results from non-human primate toxicity studies have led to an exciting development. The Company’s MDS clinical trial, initially planned to initiate in the second half of 2024, has already received ethics committee approval from two hospitals with the plan to enroll MDS patients before the end of the year.

This is fantastic news for the MDS community. The upcoming HG204 MDS clinical trial, named “HERO,” will initially treat a minimum of 4 patients who will be closely monitored for a year. The first phase of this trial will be conducted in China. Further patient enrollment, in China, will depend on the first preliminary results of the interventional study. 

HuidaGene has already made significant strides in regulatory approval, securing two FDA designations (Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD)) to HG204 for the treatment of MECP2 duplication syndrome. They have also initiated contact with both the FDA and EMA for further studies in the US and Europe.

The opportunity to engage in person with Alvin and Gloria was a privilege. We are thrilled to be part of this journey and look forward to keeping our community updated on the progress of this potentially life-changing technology.