Recently, gene-editing tool company HuiGene Therapeutics announced the completion of hundreds of millions of yuan of round C financing. HuiGene Therapeutics has completed four rounds of financing. This round of financing will be used for the company's technological innovation and pipeline R&D, product registration and clinical development, GMP pilot production base construction, and team expansion.

Founded in 2018, HuiGene Therapeutics is committed to the R&D and production of gene therapy drugs such as gene substitution and gene editing for rare single-gene genetic diseases and chronic diseases threatening human health. The technical platform covers the whole chain of preclinical drug development such as drug target screening, drug optimization, animal pharmacological and toxicological evaluation, and drug process development. The R&D pipeline includes the central nervous system, ophthalmology, muscle, hearing, and other fields, with integrated R&D capabilities from drug process development, production transformation, CMC and drug clinical and registration application. HuiGene Therapeutics now has R&D laboratories, drug process development and analysis laboratories, model animal test centers, and other R&D bases. The company's R&D team accounts for more than 70% of the company's total employees.

Gene therapy refers to the treatment of diseases by improving human genes, which is divided into in vivo gene therapy and in vitro gene therapy. Compared with other adenovirus-related diseases, adenovirus-associated gene therapy has higher safety due to its low gene expression in the body. HuiGene Therapeutics delivery vector platform adopts high-throughput screening, local transformation, and other methods to design and develop AAV vectors with high safety, low immunogenicity, and high tissue specificity for different types of diseases. HuiGene Therapeutics has developed a variety of AAV vectors and has independent property rights. At present, HuiGene Therapeutics has more than 10 patent applications, all of which are invention patents, mainly related to RNA targeting, genome, and other fields.