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HuidaGene Showcases Gene-Editing Based Medicine for Neurological Disease at Japan Society of Gene and Cell Therapy 2023 Annual Meeting

2023.09.14 12:00
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·     HG302 CRISPR/Cas12 DNA-editing therapy sufficiently edits and induces exon51 reframing/skipping in DMD gene

·     hfCas12Max-gHTT efficiently knocks down mHTT protein level and improves motor function in HD mouse model

·     HG204 CRISPR/Cas13 RNA-editing significantly rescues motor functions and prolongs lifespan in MDS mouse model and effectively knocks down MECP2 mRNA level in non-human primates


SHANGHAI and CLINTON (NJ), Sep. 14, 2023 – HuidaGene Therapeutics (辉大基因,“HuidaGene”), a global clinical-stage biotechnology company focused on developing CRISPR-based programmable genomic medicine, today announced the oral presentation at the 29th Annual Meeting of Japan Society of Gene and Cell Therapy (JSGCT) in Osaka, Japan, from September 11-13, 2023. The presentation showcased HuidaGene’s robust pipeline of next-generation neurological disease therapeutics, including neuromuscular disease of Duchenne muscular dystrophy (DMD), neurodegenerative of Huntington’s disease (HD), and neurodevelopmental MECP2 duplication syndrome (MDS) leveraging Company’s proprietary CRISPR DNA-targeting (hfCas12Max) and RNA-targeting (hfCas13Y) platforms. For more information of the oral presentation titling “Advances and Challenges of Next-Generation CRISPR 4.0 Gene-Editing Technology for Neurological Diseases”, please visit:


“Neurological diseases affect about 1 in 7 people worldwide, according to the American Brain Foundation,” said Alvin Luk, Ph.D., M.B.A., C.C.R.A., Co-founder and CEO of HuidaGene. “Our presentation at JSGCT highlights the depth of evidence we are generating in support of the clinical applications of novel CRISPR-based genomic medicine to address those fatal diseases. We’re particularly excited about development and advances across the full spectrum of our independently-developed and wholly-owned high-fidelity Cas12Max for DNA editing and high-fidelity Cas13Y for RNA editing platforms demonstrating high editing efficiency and low off-target activity.  As we advance clinical development of our hfCas13Y in patients, we remain focused on continuing to evolve our understanding of our novel DNA- and RNA-editing tools to enable the development of safe and effective genome-editing therapies.”


“In 2022, my team published research papers focusing on the engineering of xCas12i with high activity, high specificity and board PAM range as well as Cas13 variants for targeted RNA degradation with minimal collateral effects in Protein & Cell and Nature Biotechnology, respectively,” said Hui Yang, Ph.D., Co-Founder, Chief Scientific Advisor, and Chairman of the SAB at HuidaGene. “Using AI and deep ML of DNA sequencing and assembly prediction from metagenomic database through HGPRECISE® (HuidaGene – Platform for Rational Engineering of CRISPR-Cas Identification by Synergic Expertise) platform, we identified and engineered novel Cas proteins which are small in size for packaging and delivering in vivo through a single AAV vector and have the highest editing efficiency in mammalian cells.”


About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe, fatal neuromuscular genetic disease caused by changes or mutations in the DMD gene encoding the instructions for dystrophin that abolish the production of dystrophin in muscle, with an incidence of 1 in 3,500 to 5,000 male newborns worldwide. Boys with this X-linked condition often progress on a neurotypical track for the first few years of their lives, but the lack of dystrophin begins to cause noticeable symptoms by the age of 4. Symptoms of DMD usually manifest in early childhood with progressive muscular weakness associated with motor-milestone delays leading to increasing difficulty in walking, climbing stairs, or standing from a sitting position. As the disease progresses, muscle weakness in the lower limbs spreads to the arms, shoulders, and other areas, resulting in loss of ambulation, respiratory impairment, cardiomyopathy, and ultimately premature death. No curative treatment exists for DMD.


About Huntington’s Disease

Huntington’s disease (HD) is a rare, inherited neurodegenerative disease that leads to motor symptoms including chorea, behavioral abnormalities, and cognitive decline resulting in progressive physical and mental deterioration which typically presents in adults aged between 30 and 50, with a prevalence of 2.71 per 100,000 worldwide. HD is caused by a mutation in one of the alleles of the huntingtin (HTT) gene, leaving only one functional and healthy copy of HTT in the cell. The mutated HTT causes disease-causing CAG repeat expansion in the first exon of the HTT that produces and aggregates the mutant HTT protein in the brain, leading to profound neuronal loss and progressive deterioration of motor, psychiatric, and cognitive abilities. There are currently no disease-modifying therapies available for HD.


About MECP2 Duplication Syndrome

Methyl-CpG binding protein 2 (MeCP2) duplication syndrome (MDS) is a rare, fatal childhood neurodevelopmental disorder with an estimated live birth prevalence in males of 1 per 150,000. MDS is one of the most common sub-telomeric genomic rearrangements in males, accounting for about 1-2% of X-linked cases of intellectual disability. MDS which is caused by the duplication or triplication of the MECP2 gene manifests predominantly in boys and is characterized by infantile hypotonia, development delay, intellectual disability, anxiety, loss of motor skills and speech, epilepsy, recurrent respiratory tract infections and premature death. There are no approved disease-modifying therapies for MDS and current treatments for MDS include symptom management and supportive care.

About HuidaGene -辉大基 

HuidaGene Therapeutics (辉大基因) is a global clinical-stage biotechnology company focusing on discovering, engineering, and developing novel gene-editing tools and gene therapies to rewrite the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene is committed to addressing patients’ needs globally with various preclinical therapeutic programs covering ophthalmology and neurology. We are currently advancing clinical programs of HG004 in inherited retinal disease caused by RPE65 mutations (which has been granted both ODD and RPDD by U.S. FDA), HG202 CRISPR/Cas13Y RNA-editing in neovascular age-related macular degeneration (nAMD), and our preclinical pipeline, including programs in retinitis pigmentosa, neuro-developmental disease of MECP2 duplication syndrome, neuromuscular diseases of Duchenne muscular dystrophy, and neuro-otology of hereditary sensorineural hearing loss. Company’s CRISPR-based therapeutics offer the potential to cure patients with life-threatening conditions by repairing the cause of their disease. HuidaGene is committed to transforming the future of genome-editing medicine.

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