SHANGHAI and CLINTON, N.J., May 3, 2023 /PRNewswire/ -- HuidaGene Therapeutics (????; HuidaGene), a global clinical-stage genome-editing company focused on developing CRISPR-based programmable genomic medicine, today announced nineteen (19) presentations on Company's various gene replacement and gene-editing platforms to unlock the full potential of genome medicines at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting taking place in Los Angeles from May 16-20, 2023.
"This is the first time that HuidaGene has participated in ASGCT since the Company was founded 4 years ago. With the 100% acceptance rate for the 19 abstracts including 4 oral presentations, our presence at ASGCT showcases our leadership of technology advancement in gene replacement and genome-editing therapies to address their challenges and limitations. These presentations underscore our commitment to the unmet needs for patients and families living with devastating diseases around the world," said Xuan Yao, Ph.D., Co-Founder and Chief Executive Officer of HuidaGene.
"Based on our unique HG-PRECISE® platform, our team has developed various new genome editing tools (hfCas13X/Y, hfCas12Max, and eRBE etc), which not only have independent IP rights but also have advantages such as smaller size, higher editing efficiency and higher specificity. Therefore, our independently-developed genetic tools demonstrate improved editing efficiency and reduced off-target effects while making gene-editing therapy more effective, safer, and accessible to broader populations living with devastating diseases worldwide," stated Hui Yang, Ph.D., Co-Founder and Chief Scientific Advisor of HuidaGene. "I look forward to attending this international forum with my team to share the newest gene-editing advancement."
HuidaGene's presentations at 26th ASGCT Annual Meeting include:
Genetic Tools | Abstract # | Title | Date/Time | Format |
Ophthalmology | ||||
Gene | 713 | Restoration of retinal function and structure in an RPE65- deficient murine model via. gene replacement therapy | 5/17/2023 12:00-19:00 | Poster |
Cas12f | 712 | Compact CRISPR/Cas12f-based mutation independent therapy strategy for rhodopsin-associated autosomal dominant retinitis pigmentosa via single AAV | 5/17/2023 12:00-19:00 | Poster |
Cas13 | 1663 | Development of a Cas13-based RNA targeting therapy for neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME) | 5/19/2023 12:00-19:00 | Poster |
Gene | 313 | Preclinical safety assessment of an investigational gene replacement therapy for the treatment of RPE65-mediated inherited retinal dystrophies | 5/20/2023 08:15-08:30 Petree Hall C | Oral |
Neurology | ||||
Cas12i | 694 | Gene editing therapy in a humanized SOD1G93A mouse model of Amyotrophic Lateral Sclerosis | 5/17/2023 12:00-19:00 | Poster |
Cas13 | 34 | RNA editing therapy in a humanized mouse model of MECP2 duplication syndrome and non-human primates | 5/17/2023 16:15-16:30 Petree Hall D | Oral |
Cas13 | 1624 | A high-fidelity RNA-targeting Cas13 restores paternal Ube3a expression and improves motor functions in Angelman syndrome mice | 5/19/2023 12:00-19:00 | Poster |
Cas12i | 1285 | Gene editing therapy in a humanized mouse model of Amyotrophic Lateral Sclerosis | 5/19/2023 12:00-19:00 | Poster |
Otology | ||||
RNA base- | 342 | Rescue of OTOF Q829X mutation-induced hearing loss by in-vivo delivery of mini-dCas13X-derived RNA base editor | 5/20/2023 08:30-08:45 Room 502 AB | Oral |
Myology | ||||
DNA base- | 667 | Precise correction of Duchenne Muscular Dystrophy with exon 51 deletion by adenine base editing-induced exon skipping in a humanized mouse model | 5/17/2023 12:00-19:00 | Poster |
RNA base- | 841 | Efficient mini dCas13-mediated base editing for personalized treatment of Duchenne Muscular Dystrophy | 5/18/2023 12:00-19:00 | Poster |
Cas12i | 951 | Single-cut gene editing therapy for Duchenne Muscular Dystrophy via a single AAV vector | 5/18/2023 12:00-19:00 | Poster |
Hepatology | ||||
LNP-based | 1128 | Targeting the hepatitis B cccDNA with the hfCas12Max nuclease to eliminate hepatitis B virus in-vitro and in-vivo | 5/18/2023 12:00-19:00 | Poster |
Technology | ||||
Cas12i | 495 | An engineered xCas12i with high activity, high specificity, and broad PAM range | 5/17/2023 12:00-19:00 | Poster |
RNA base- | 978 | Development of a compact and efficient RNA base editor (ceRBE) by fusing ADAR with engineered EcCas63 | 5/18/2023 12:00-19:00 | Poster |
Cas12f | 1555 | Engineering the novel type V-F CRISPR-Cas system as versatile and efficient genome editing tools delivered by a single AAV | 5/19/2023 12:00-19:00 | Poster |
Cas13 | 1419 | Engineered Cas13 with minimal collateral effect and improved editing efficiency for targeted RNA degradation | 5/19/2023 12:00-19:00 | Poster |
AAV | 1464 | A comprehensive AAV tropism profile in mice, crab-eating macaques, and marmosets | 5/19/2023 12:00-19:00 | Poster |
DNA base- | 257 | Programmable A-to-Y base editing by fusing an adenine base editor with an N-methylpurine DNA glycosylase | 5/19/2023 16:00-16:15 Concourse Hall 152&153 | Oral |
Accepted abstracts and full program is available at ASGCT website (https://annualmeeting.asgct.org/abstracts).
"This is the first time that HuidaGene has presented information on the progress of the scientific research in gene replacement and genome-editing therapies at the ASGCT annual meeting. Having 19 abstracts accepted for presentations with 4 oral presentations reflect the significant progress made by the teams and the strength of the R&D team's innovation and drug development in this field," commented by Alvin Luk, Ph.D, MBA, CCRA, Scientific Advisory Board Member of HuidaGene. "I'm very proud to be part of the groundbreaking work by the teams. I'll attend this ASGCT annual meeting with Prof. Yang and Dr. Yao. We're happy to have deep discussions with experts or companies wishing to advance gene therapy and gene editing technology. Together with Drs. Yang and Yao, we'll continue to advance the therapeutic pipeline and genome-editing research from discovery to bedside and bring more treatment options to patients globally."
About HuidaGene - ????
HuidaGene Therapeutics (????) is a global clinical-stage biotechnology company focusing on discovering, engineering, and developing CRISPR-based genetic medicine 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, otology, myology, and neurology. We are currently advancing clinical programs in RPE65 mutation-associated inherited retinal dystrophies and our preclinical pipeline, including programs in neovascular age-related macular degeneration, retinitis pigmentosa, hereditary hearing loss, Duchenne muscular dystrophy, and MECP2 duplication syndrome. 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.
For more information, please visit http://www.HuidaGene.com
or follow us on LinkedIn at http://www.linkedin.com/company/HuidaGene
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