At the upcoming annual meeting of the American Society of Cell and Gene Therapy, scientists from Huidagene Therapeutics will share an update on the development of their CRISPR-high-fidelity(hf)Cas12Max-based gene editing therapy for Duchenne muscular dystrophy, one of the most severe forms of the inherited muscular dystrophies that affects primarily boys.
Huidagene Therapeutics and its collaborators recently began testing the treatment in patients. And their early data from one patient indicate that a single dose successfully improves symptoms associated with the disease with no ill effects. Alvin Luk, PhD, CEO and co-founder of HuidaGene, will present details of the therapy and clinical trial as part of the Presidential Symposium at the meeting, which will be held from May 13-17 in New Orleans.
As explained in their conference abstract, HG302 is a one-time single-cut CRISPR-Cas12 therapy that is designed to induce exon skipping and restore dystrophin expression with minimal off-target effects. It utilizes HuidaGene’s hfCas12Max, a CRISPR nuclease developed using its HG-PRECISE platform. For the treatment, a single adeno-associated virus is used to deliver the hfCas12Max and a guide RNA to the splice donor site of human exon 51.
Scientists have previously completed preclinical studies in DMD mice and monkeys. They report that in mice, HG302 restored dystrophin protein levels up to 70% and improved motor function. Safety evaluations showed no abnormal liver enzymes or kidney function in both the mice and monkeys.
HG302’s developers have moved on to testing the treatment in humans as part of a multidose, dose-escalation study to understand the safety of CRISPR gene-editing therapy and its long-lasting effects in DMD patients (MUSCLE) trial. The treatment has been granted orphan drug designation and rare pediatric disease designation by the FDA. For the trial, the company is enrolling ambulant boys with DMD between the ages of four and eight with impaired muscle function.
Late last year, HuidaGene announced that it had dosed its first patient with the treatment. According to the conference abstract, the patient has demonstrated favorable safety and tolerability with adverse liver and kidney effects. He also has improved motor function post treatment, the researchers say.
Besides DMD, HuidaGene is also applying its CRISPR-hfCas12Max editing technology to amyotrophic lateral sclerosis cases. The company is also working on a CRISPR-based RNA editing treatment for neovascular age-related macular degeneration, among other projects.
The post ASGCT 2025: CRISPR-Cas12 Editing Shows Early Clinical Benefit in Duchenne Muscular Dystrophy appeared first on GEN - Genetic Engineering and Biotechnology News.
Huidagene Therapeutics and its collaborators recently began testing the treatment in patients. And their early data from one patient indicate that a single dose successfully improves symptoms associated with the disease with no ill effects. Alvin Luk, PhD, CEO and co-founder of HuidaGene, will present details of the therapy and clinical trial as part of the Presidential Symposium at the meeting, which will be held from May 13-17 in New Orleans.
As explained in their conference abstract, HG302 is a one-time single-cut CRISPR-Cas12 therapy that is designed to induce exon skipping and restore dystrophin expression with minimal off-target effects. It utilizes HuidaGene’s hfCas12Max, a CRISPR nuclease developed using its HG-PRECISE platform. For the treatment, a single adeno-associated virus is used to deliver the hfCas12Max and a guide RNA to the splice donor site of human exon 51.
Scientists have previously completed preclinical studies in DMD mice and monkeys. They report that in mice, HG302 restored dystrophin protein levels up to 70% and improved motor function. Safety evaluations showed no abnormal liver enzymes or kidney function in both the mice and monkeys.
HG302’s developers have moved on to testing the treatment in humans as part of a multidose, dose-escalation study to understand the safety of CRISPR gene-editing therapy and its long-lasting effects in DMD patients (MUSCLE) trial. The treatment has been granted orphan drug designation and rare pediatric disease designation by the FDA. For the trial, the company is enrolling ambulant boys with DMD between the ages of four and eight with impaired muscle function.
Late last year, HuidaGene announced that it had dosed its first patient with the treatment. According to the conference abstract, the patient has demonstrated favorable safety and tolerability with adverse liver and kidney effects. He also has improved motor function post treatment, the researchers say.
Besides DMD, HuidaGene is also applying its CRISPR-hfCas12Max editing technology to amyotrophic lateral sclerosis cases. The company is also working on a CRISPR-based RNA editing treatment for neovascular age-related macular degeneration, among other projects.
The post ASGCT 2025: CRISPR-Cas12 Editing Shows Early Clinical Benefit in Duchenne Muscular Dystrophy appeared first on GEN - Genetic Engineering and Biotechnology News.