Huidagene Therapeutics
SHANGHAI and CLINTON, N.J., April 20 /PRNewswire/
HuidaGene Therapeutics (辉大基因; HuidaGene), a global genome editing clinical trials company focused on the development of CRISPR-based programmable genomic medicine, today made nineteen (19) presentations on the various gene replacement and the Company’s gene editing platforms to unlock the full potential of genomic drugs at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, to be held May 16-20, 2023 in Los Angeles.
“This is the first time that HuidaGene has attended the ASGCT since the company was founded 4 years ago. With an acceptance rate of 100% for the 19 abstracts, including 4 oral presentations, our presence at the ASGCT meeting underscores our leadership in advancing the technology of gene replacement and genome editing therapies to address their challenges and limitations. These presentations underscore our commitment to addressing the unmet needs of patients and families living with devastating diseases around the world,” said Dr. Xuan Yao, 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.) that not only have independent intellectual property rights, but also advantages such as smaller size , higher editing efficiency and higher specificity. Therefore, our independently developed genetic tools demonstrate improved editing efficiency, fewer unwanted effects, and make gene editing therapy more effective, safer, and accessible to broader populations living with devastating diseases worldwide,” said Dr. Hui Yang, Co-Founder and Chief Scientific Advisor of HuidaGene. “I look forward to attending this international forum with my team to present the latest advances in gene editing.”
HuidaGene’s presentations at the 26th Annual ASGCT Meeting include:
|
Genetic Tools |
Abstract Nr. |
title |
Date / time |
Format |
|
ophthalmology |
||||
|
gene replacement |
713 |
Restoration of retinal function and structure in a mouse model RPE65-Deficiency caused by gene replacement therapy |
17.05.2023, 12:00 bis 19:00 |
Poster |
|
Cas12f |
712 |
Compact CRISPR/Cas12f-based mutation-independent therapy strategy for rhodopsin-associated autosomal dominant retinitis pigmentosa using a single AAV |
17.05.2023, 12:00 bis 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) |
19.05.2023, 12:00 bis 19:00 |
Poster |
|
gene replacement |
313 |
Preclinical safety assessment of a gene replacement therapy to be tested for the treatment of RPE65-mediated inherited retinal dystrophies |
20.05.2023, 08:15 bis 08:30 Petree Hall C |
Oral |
|
Neurology |
||||
|
Cas12i |
694 |
Gene editing therapy in a humanized SOD1 G93A -Mouse model of amyotrophic lateral sclerosis |
17.05.2023, 12:00 bis 19:00 |
Poster |
|
Cas13 |
34 |
RNA editing therapy in a humanized mouse model of MECP2 duplication syndrome and in non-human primates |
17.05.2023, 16:15 bis 16:30 Petree Hall D. |
Oral |
|
Cas13 |
1624 |
A highly specific RNA-targeting Cas13 restores paternal Ube3a expression and improves motor functions in Angelman syndrome mice |
19.05.2023, 12:00 bis 19:00 |
Poster |
|
Cas12i |
1285 |
Gene editing therapy in a humanized mouse model of amyotrophic lateral sclerosis. |
05/19/2023, 12:00 p.m. to 7:00 p.m |
Poster |
|
ear medicine |
||||
|
RNA-Basis- Editor |
342 |
help with OTOF Q829X mutation-induced hearing loss In-vivo -Providing the RNA base editor derived from mini-dCas13X |
05/20/2023, 8:30 a.m. to 8:45 a.m Room 502 AB |
Oral |
|
muscle theory |
||||
|
DNA-Basis- Editor |
667 |
Precise correction of Duchenne muscular dystrophy with exon 51 deletion by adenine-based edit-induced exon skipping in a humanized mouse model |
05/17/2023, 12:00 p.m. to 7:00 p.m |
Poster |
|
RNA-Basis- Editor |
841 |
Efficient mini-dCas13-mediated baseline editing for a personalized treatment of Duchenne muscular dystrophy |
05/18/2023 12:00 p.m. to 7:00 p.m |
Poster |
|
Cas12i |
951 |
Single-cut gene editing therapy for Duchenne muscular dystrophy via a single AAV vector |
05/18/2023, 12:00 p.m. to 7:00 p.m |
Poster |
|
Hepatology |
||||
|
LNP based Cas12i |
1128 |
Targeting hepatitis B cccDNA with hfCas12Max nuclease to eliminate hepatitis B virus in vitro and live |
05/18/2023 12:00 p.m. to 7:00 p.m |
Poster |
|
Technology |
||||
|
Cas12i |
495 |
A developed xCas12i with high activity, high specificity and a wide PAM range |
05/17/2023 12:00 p.m. to 7:00 p.m |
Poster |
|
RNA-Basis- Editor |
978 |
Development of a compact and efficient RNA basic editor (ceRBE) by merging ADAR with the developed EcCas63 |
05/18/2023 12:00 p.m. to 7:00 p.m |
Poster |
|
Cas12f |
1555 |
Development of the novel VF CRISPR-Cas-type system as a versatile and efficient genome editing tool powered by a single AAV |
05/19/2023, 12:00 p.m. to 7:00 p.m |
Poster |
|
Cas13 |
1419 |
Engineered Cas13 with minimal side effect and improved editing efficiency for targeted RNA depletion |
05/19/2023, 12:00 p.m. to 7:00 p.m |
Poster |
|
AAV |
1464 |
A comprehensive AAV tropism profile in mice, crabbing macaques and marmosets |
05/19/2023, 12:00 p.m. to 7:00 p.m |
Poster |
|
DNA-Basis- Editor |
257 |
Programmable A-to-Y basis editing by fusing an adenine-based editor with an N-methylpurine DNA glycosylase |
05/19/2023, 4:00 p.m. to 4:15 p.m Concourse Hall 152 und 153 |
Oral |
Accepted abstracts and the full program can be found on the ASGCT website ( https://annualmeeting.asgct.org/abstracts).
“This is the first time that HuidaGene has presented information on the progress of scientific research in the field of gene replacement and genome editing therapies at the ASGCT Annual Meeting. The acceptance of 19 abstracts for presentations with 4 oral presentations reflects the significant progress made by the teams and the strength of the R&D team’s innovation and drug development in this area,” commented Dr. Alvin Luk, MBA, CCRA, Scientific Advisory Board Member of HuidaGene “I am very proud to be part of the groundbreaking work of the teams. I will be attending this ASGCT Annual Meeting with Prof. Yang and Dr. Yao. We look forward to in-depth discussions with experts or companies involved in gene therapy and the… Together with Dr. Yang and Dr. Yao, we will continue to advance therapeutic development and research on genome editing from discovery to the bedside, bringing more treatment options to patients worldwide.”
About HuidaGene – Huida Gene
HuidaGene Therapeutics is a global clinical development biotechnology company focused on the discovery, design and development of CRISPR-based genetic drugs to reshape the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene is committed to serving the needs of patients around the world with a variety of preclinical therapy programs in the fields of ophthalmology, otolology, myology and neurology. We are currently driving clinical programs for RPE65 -mutation-associated inherited retinal dystrophies and advancing our preclinical pipeline, including programs in neovascular age-related macular degeneration, retinitis pigmentosa, hereditary hearing loss, Duchenne muscular dystrophy and MECP2 duplication syndrome. The Company’s CRISPR-based therapeutics offer the potential to heal patients with life-threatening diseases by addressing the root cause of their disease. HuidaGene aims to change the future of genome-editing medicine.
For more information, see http://www.HuidaGene.com
or follow us on LinkedIn at http://www.linkedin.com/company/HuidaGene
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