The HERO clinical trial is a pioneering study evaluating HG204, a novel CRISPR Cas13 RNA-editing therapy, for MECP2 Duplication Syndrome (MDS). Sponsored by HuidaGene, this trial marks an important step in the search for a treatment for MDS. In November 2024, the first patient was successfully administered HG204, offering renewed hope to the MDS community.
Trial Overview
The HERO trial involves a single injection of HG204 via intracerebroventricular delivery, administering the therapy directly into the brain. Recruitment for the trial began in October 2024, and the study aims to enrol six male participants, aged 2 to 18 years, who have a confirmed diagnosis of MECP2 Duplication Syndrome. Each participant will take part in the 60-week trial, consisting of an 8-week screening period, treatment administration, and a 52-week follow-up phase. The primary objectives are to monitor adverse events and assess developmental progress after treatment. Currently, the trial is being conducted at Peking University Hospital in Beijing, China. Further patient enrolment in China will depend on the results of the first patients treated in the study.
For more detailed information, we invite you to read the study protocol.
First Patient Dosed & Preliminary Findings
In November, HuidaGene announced the successful administration of HG204 to the first patient. Six weeks after the injection, the patient showed no major side effects, and preliminary signs of efficacy were observed. Over the next weeks, the patient will be closely monitored to further evaluate the treatment’s safety and efficacy.
Next steps and goals
HuidaGene has made significant progress in regulatory approvals, securing two important FDA designations: Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) for HG204 in the treatment of MECP2 Duplication Syndrome. While HERO is conducted in China, the biotech company has also initiated discussions with the FDA and EMA to explore further studies in the US and Europe.
Published Research
Meanwhile, HuidaGene published details of preclinical experimental findings on the CRISPR-Cas13 technology, including HG204, in Nature Neuroscience. The studies demonstrated that HG204 effectively reduces MECP2 mRNA levels, alleviates disease symptoms, and extends lifespan in mouse and non-human primate models without causing adverse effects. These results strengthen confidence in the ongoing clinical trial and HG204’s potential as a therapeutic option for MDS.
The HERO clinical trial represents a significant step in the quest for a treatment for MECP2 Duplication Syndrome. The combination of cutting-edge CRISPR Cas13 technology and promising preliminary outcomes gives hope to patients and families affected by this syndrome. In the coming months, continued monitoring and research will be critical to fully ascertain the safety and efficacy of HG204 in treating MDS.
