Huidagene Therapeutics Announces 19 Presentations Highlighting Advances in its Gene Therapy Technology Platforms at the 2023 ASGCT Annual Meeting

Ophthalmology

Gene replacement

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   replacement

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 SOD1^{G93A 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-editor

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-editor

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-editor

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 Cas12i

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-editor

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-editor

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 (http://annualmeeting.asgct.org/program).

“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 - 辉大基因

HuidaGene Therapeutics (辉大基因) 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