CRISPR Medicine

HuiGene Therapeutics

Company Type: Therapeutics

Main focus: Development of therapeutics based on RNA-editing tools (CRISPR/Cas) and delivery via adeno-associated virus (AAV)

Company stage: Pre-clinical

Diseases: Ophthalmology, central nervous system, muscle dystrophies, otology (ear), hepatology

Genome-editing tool: CRISPR/Cas-based RNA-editing tools

Funding stage: Series C

Location: Pudong New Area, Shangai

Website: www.huidagene.com/en/

Pipeline: www.huidagene.com/en/goods/pipeline.html

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HuiGene Therapeutics is a pre-clinical stage company with multiple R&D pipelines for ophthalmology, neurology, otology, myology and hepatology. The company aims to treat genetic conditions – including rare diseases – via CRISPR-Cas-based RNA-editing tools and adeno-associated virus (AAV) delivery vectors. Their pre-clinical studies are supported by an in-house facility focused on generating CRISPR-edited animal models to showcase the efficacy of their therapeutics.

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