List of Gene Editing/CRISPR Companies in China - 33

On August 20, 2024, Base Therapeutics announced that its AccuBase® base editor has officially received authorization for two patents from the United States Patent and Trademark Office (USPTO). This marks that both of Base Therapeutics' AccuBase® technologies have secured patent protection in China and overseas markets. Base Therapeutics' strategic positioning in the gene editing field is at the forefront internationally, making it one of the biopharmaceutical companies with the most authorizations domestically and abroad.

Shanghai BDgene Therapeutics Co., Ltd. was established in 2018. The founder team includes top scientists in the field of gene therapy, industrial experts with a background in international pharmaceutical companies, toxicology and safety pharmacology experts with preclinical drug evaluation experience.

BeiCell Therapeutics is a revolutionary cell therapy company stemming from Prof. Hongkui Deng's lab at Peking University, aiming to provide completely off-the-shelf cell therapy for cancer, autoimmune diseases treatment and regenerative medicine.

RNAi

Established in 2012, BravoVax is located at BravoVax Building, 858 Gaoxin Avenue in the state-level biopharma development zone, Donghu hi-tech park of Wuhan, China. Relying on the experienced management team and international channels from parent company BravoBio and strong support from Humanwell Healthcare Group, one of the top 20 Chinese pharmaceuticals, we have kept the momentum in its development, maintaining fast growth. We are committed to the research and development of human vaccines and other biological products. The new pilot facility and R&D center are built at Wuhan biopharaceutical industrial park as per cGMP. All staffs hold a college and above degree, and more than 30% of them have master and above degrees as well as senior professional titles.

At BRL Medicine Inc., we aim to become the world's leading Cellular & Gene pharmaceutical company in the era of new commercial civilization, with the mission of "Through innovation led by gene editing, develop breakthrough human therapeutics and benefit the whole world". With its independent R&D center and the "Shanghai Research Center for Gene Editing and Cell Therapy", jointly established by BRL Medicine and university, BRL Medicine has obtained more than 100 patent achievements, and initiated 5 Investigator-Initiated clinical Trials (IIT) in 8 leading hospitals in China, with many pipeline projects have entered into IND application stage. Among them, gene editing projects for β-thalassemia, PD1 knockout non-viral targeted integration CAR-T and UCART have achieved excellent clinical results and have published several academic papers in famous academic journals such as Nature Medicine and Nature biotechnology. BRL Medicine has built five technology platforms with independent intellectual property rights, including The Gene Editing Technology Innovation Platform, Hematopoietic Stem Cell Platform, The Quikin CAR-T Platform, Universal Cell Platform and Enhanced T Cell Platforms, and has a 7000 square meter GMP pilot plant and an operating team of more than 200 people, which strongly guarantees the rapid transformation and application of innovative research. BRL Medicine continuously promotes rapid update and iteration of R&D products through patient needs and clinical feedback. Driven by the era of new commercial civilization, BRL Medicine holds an open, shared, and win-win attitude, and works with global innovative biomedical ecological chain companies to accelerate the transformation and implementation of innovative drugs, so as to benefit global patients with genetic diseases and malignant tumors!

CARsgen is a biopharmaceutical company with operations in China and the U.S. focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors. The company has built an integrated cell therapy platform with in-house capabilities that span target discovery, antibody development, clinical trials, and commercial-scale manufacturing. CARsgen has internally developed novel technologies and a product pipeline with global rights to address major challenges of CAR T-cell therapies, such as improving the safety profile, enhancing the efficacy in treating solid tumors, and reducing treatment costs. Our vision is to become a global biopharmaceutical leader that brings innovative and differentiated cell therapies to cancer patients worldwide and makes cancer curable.

CorrectSequence Therapeutics (Correctseq) is a clinical-stage biotech company employing its proprietary transformer base editing (tBE) technology to pioneer next-generation gene editing therapies. Our leading pipeline candidate, CS-101, utilizing innovative base editor targeting HBG, curing β-hemoglobinopathies, has obtained IND approval from the China NMPA. Clinical data demonstrates its superior performance, indicating significant potential for CS-101 to become a best-in-class gene editing therapy for curing patients with β-hemoglobinopathies. Proof-of-concept (POC) data in mice for in vivo pipelines using tBE-editing therapies via lipid nanoparticle (LNP) delivery are available, including targets for metabolic dysfunction and associated diseases. Ex vivo multiplex editing of T cells on multiple targets simultaneously preserved T cell growth and function in vivo compared to non-edited cells, establishing tBE as the ideal gene editing tool for the next-generation cell therapy development. tBE offers significant advantages in controlling off-target effects and enhancing in vivo editing efficiency, making it ideal for both multiplex editing and precise single-target editing. It is compatible with various delivery system, including ex vivo editing in hematopoietic stem cells (HSCs) and T cells, as well as in vivo editing via LNPs or adeno-associated viruses (AAVs). We are developing multiple pipeline programs targeting genetic diseases, metabolic disorders, and cardiovascular diseases. Our focus on biotechnology innovation, research and development underscores our commitment to providing efficient, reliable and safe solution for unmet medical needs.

Cure Genetics is an innovative gene therapy company focused on the application and development of viral delivery systems and gene editing systems. In response to the clinical needs of complex genetic diseases and refractory tumors, it has conducted extensive international cooperation and established a breakthrough product pipeline, striving to become a benchmark in the global gene therapy field. The company was founded in 2016 and is located in Suzhou Industrial Park. It brings together core talents from different fields at home and abroad to build a complete differentiated platform around the AAV virus delivery system and CRISPR gene editing system. Possess core independent intellectual property rights and create core competitive barriers. At the same time, the company has established a scientific committee composed of internationally renowned scientists and executives from multinational pharmaceutical companies to lay a solid foundation for technology commercialization.

EdiGene is a clinical-stage company focused on translating gene-editing technologies into transformative genetic medicines for patients with significant unmet medical needs. Anchored by its proprietary technologies of gene editing, bioinformatics, and high throughput genome-editing screening, EdiGene advances the pipeline on therapeutic platforms of LEAPER-based in vivo RNA base editing, ex vivo gene-editing hematopoietic stem cells, and ex vivo gene-editing T cells for allogeneic CAR-T.

Elpiscience is a clinical-stage biopharmaceutical company dedicated to developing life-changing immuno-oncology therapies for cancer patients worldwide. The company’s innovative approach is focused on removing immunosuppressive factors in the tumor microenvironment, by targeting the adenosine pathway and myeloid checkpoints. A pipeline of novel molecules has been developed using its proprietary platforms including a powerful Bispecific Macrophage Engager (BiME®) technology that connects and activates macrophages for solid tumor killing without causing cytokine storms.

Epigenic Therapeutics is a frontier biotechnology company dedicated to developing next generation gene editing therapy utilizing regulation of epigenetic genome for a variety of diseases. Founded in 2021 by leading scientists focused on discovering gene editing technologies and developing gene editing therapies, the company has multiple product candidates in the pipeline, including treatment for metabolic, cardiovascular, viral hepatitis, ocular and rare diseases.

Hopstem Bioengineering Co., Ltd. was founded on January 2017 in Hangzhou, by neuroscientists and stem cell biologists from Johns Hopkins University. Hopstem has established world-leading iPSCs platform of neural differentiation and cell engineering. Hopstem has developed several critical patented technologies，as well as CMC platform for iPSC-derived cell therapy products. Hopstem has iPSC reprogramming patent, GMP iPSC line with global licensing rights, iPSC-derived cell product manufacturing and quality system, and a variety of cell therapy products and pipelines, targeting CNS diseases, optical diseases and tumor. Hopstem’s first clinical product - neural progenitor cell hNPC01 targets neural injury diseases such as stroke and traumatic brain injury, has received China's IND approval and entered clinical trials. Hopstem is committed to translating the most cutting-edge technologies to provide global patients with safe, efficient and affordable cell therapy products.

HuidaGene Biotechnology Co., Ltd (辉大基因) is a global biotechnology company focusing on the 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 patient's needs globally with various preclinical therapeutic programs covering ophthalmology, otology, myology, and neurology. Company's CRISPR-based therapeutics offer the potential to cure patients with life-threatening conditions by repairing the cause of their disease. We are committed to transform the future of genome-editing medicine.

ICE Bioscience was founded in 2010 as an Innovative CRO+ Explorer company. We specialize in early drug discovery services, spanning from target validation to the identification of pre-clinical candidates. Currently, ICE Bioscience offers a broad selection of off-the-shelf assays, including 100+ ion channels, 800+ Kinases/enzymes, 100+ GPCRs, and 40+ nuclear receptor assays, all accompanied by validation data and screening information. This comprehensive range of assays provides us with a competitive edge, as it covers a vast majority of druggable targets. Our company has established multiple platforms, encompassing Protein Engineering, Cell Line Development, Target Based Assays, Safety Pharmacology, Translational Biology, DMPK, In Vivo Pharmacology and Biophysical Assays, enabling us to smoothly execute the DMTA cycle (Design-Make-Test-Analysis cycle) for innovative drug discovery in collaboration with our clients.

Jacobio Pharma (1167.HK) is committed to developing and providing new and innovative products and solutions to improve people's health. Our pipeline revolves around novel molecular targets on six major signalling pathways: KRAS, immune checkpoints, tumor metabolism, P53, RB and MYC. We aim for our key projects to be among the top three in the world. Our vision is to become a global leader recognized for our impact in drug R&D together with our partners. Jacobio has R&D centers in Beijing, Shanghai and Boston with our Induced Allosteric Drug Discovery Platform (IADDP) and our iADC Platform.

Likang Life Sciences is an innovative enterprise dedicated to development of immune cell therapy, with tumor-specific neoantigen as the target. It aims to provide personalized diagnosis and treatment services for cancer patients with the most cutting-edge precise detection and treatment methods. By now, the company has established close cooperative relationship with Chinese PLA General Hospital (301 Hospital), Beijing Cancer Hospital, and Cancer Hospital Chinese Academy of Medical Sciences, and is preparing several clinical trials. The company's goal is to establish a top cancer treatment center with advanced concepts, leading technologies and standardized services, and lead cancer treatment into a new era of personalized tumor immunotherapy.

Neukio Biotherapeutics focuses on development of next generation allogenic cell therapies via the iPSC-CAR-NK axis, through internal discovery and external collaboration, with emphasis on innovative immuno-oncology products.

NOVOPROTEIN SCIENTIFIC , the mRNA reagent materials and recombinant proteins supplier from China. Novoprotein provides a one-stop solution of mRNA raw materials and services to support mRNA vaccines research and development. Our products include restriction enzymes, T7 RNA Polymerase, RNase inhibitor, Pyrophosphatase, DNaseI, Nucleotides, Vaccinia Capping Enzyme, mRNA 2´-O-Methyltransferase, Poly(A) Polymerase, RNase R, etc. The manufacturing processes are strictly controlled to ensure the products are animal-free and ampicillin-free. We guarantee the manufacturing and quality control comply with GMP regulation for tracking each step of the manufacturing process, including raw material sourcing. Novoprotein has developed more than ten thousand of recombinant proteins, like cytokines in cell therapy and organoid culture. We have researched and developed 7 technology platforms and 23 core technologies, including protein design and modification, protein production and quality control, protein application and evaluation. Novoprotein provides biomedical Industry customers with services from product development to technological innovation based on a comprehensive technology system and in-house innovative raw materials. James Pan - International Business Development email address: panyanzhi@novoprotein.com.cn

Shenzhen Pregin Biopharmaceutical Co., Ltd. is a national high-tech enterprise, Guangdong Provincial Cell and Gene Therapy Innovative Drug Engineering Technology Research Center, and Shenzhen Special New Enterprise. Investor shareholders include the National Small and Medium Enterprises Development Fund and the National Science and Technology Commission of the Chinese Academy of Sciences. Jiahe Fund, Haier Capital, Shenzhen Capital Group, Winbond Health ( 002004), etc. It has been deeply involved in the field of cell and gene therapy drugs for many years.

Qihan Biotech is a biotechnology company applying genome-editing technology to develop novel cell therapies and organs for transplantation. The company’s mission is to use high-throughput, multiplexable genome editing in combination with expertise in transplantation immunology to create immunologically privileged allogeneic cells and xenogeneic organs for use as therapies to treat cancer, organ failure, and other important medical conditions. With a vision to create a world in which cell and organ therapies are universally available to patients, Qihan Biotech has raised three rounds of financing and is advancing its cell therapy programs into IND-enabling investigations.

Recorna Bio is a researcher and developer of RNA-edited drugs, focusing on drug discovery and development by targeting RNA editing cutting-edge technology. The company has completed millions of USD in Angel round funding and collaborates with other RNA companies to discover novel therapies for lung disease.

SIMR is an innovation-oriented biopharmaceutical company dedicated to the discovery and development of new drugs for pain, inflammation and central nervous system related diseases. Since its establishment in 2014, the company has established a research and development platform of more than 3,000 square meters, with comprehensive drug development capabilities such as compound screening, CMC research, preclinical evaluation, and clinical development.

TargetRx is a clinical stage, high-tech pharmaceutical R&D company focused on developing next-generation targeted therapies for cancer patients, particularly those who are resistant or refractory to current treatments. TargetRx has developed a series of cutting-edge and efficient small molecule drug discovery platforms, which combine innovative research and development capabilities for drug design, screening, evaluation and optimization. Since its establishment, TargetRx has been granted over 170 patents from authorities in China, the US, Europe, Japan and other regions. Several potential best-in-class compounds are at different stages of multi-national clinical trials. BD Contact: shujun.mai@tjrbiosciences.com.

TransGen Biotech Co., Ltd is a researcher and manufacturer of molecular and cellular biology products.

VectorBuilder offer gene delivery technologies. VectorBuilder offers a full spectrum of gene delivery solutions covering virtually all research and clinical needs from bench to bedside

Vitalgen is a biopharmaceutical company that focuses on the research and development of gene therapeutics.

Westlake Genetech (Hangzhou) Co., Ltd. is an innovative biotech company, a start-up from scientific research achievements of Westlake University. We focus on the rapid development of first-in-class drugs by integrating the 3H (high throughput, high precision, high dimension) biotechnological data production and deep learning modeling. By building technology-driven gene therapy technology platforms and developing differentiated self-developed gene therapy products, we are committed to mapping the full-process implementation of AI-powered gene therapy. Westlake Genetech adheres to the development strategy of "platform + pipeline" dual-wheel drive, actively invests in R&D innovation that combines technological leadership and industrial feasibility, and promotes the technological and commercial landing of AI technology in the vertical field of biomedicine. Our self-developed AI-powered full-chain gene therapy technology platform has evolved to generate a large number of non-liver-targeted AAV capsids, developed gene editing tools with independent intellectual property rights, explored new targets, and developed safe, efficient, and accessible gene therapy innovative therapies for rare diseases, tumors, and other diseases. With the platform's empowerment, it will effectively expand the scope of gene therapy indications, significantly shorten the pipeline development time, improve the pipeline R&D efficiency, and achieve the optimization of cost reduction and efficiency improvement and technology tool selection. We are rapidly advancing our technological achievements to the field of disease treatment, laying out ex vivo and in vivo gene therapy, developing differentiated gene therapy products, and solving more unmet clinical needs.

As a global company with operations across Asia, Europe, and North America, WuXi AppTec provides a broad portfolio of R&D and manufacturing services that enable the global pharmaceutical and life sciences industry to advance discoveries and deliver groundbreaking treatments to patients. Through its unique business models, WuXi AppTec’s integrated, end-to-end services include chemistry drug CRDMO (Contract Research, Development and Manufacturing Organization), biology discovery, preclinical testing and clinical research services, advanced therapies CTDMO (Contract Testing, Development and Manufacturing Organization), helping customers improve the productivity of advancing healthcare products through cost-effective and efficient solutions. WuXi AppTec received an AA ESG rating from MSCI for the third consecutive year in 2023 and its open-access platform is enabling more than 6,000 customers from over 30 countries to improve the health of those in need – and to realize the vision that "every drug can be made and every disease can be treated."

YolTech Therapeutics is a biotech focuses on combining mRNA and gene editing technology to develop next-generation mRNA drugs and in vivo gene editing drugs.Our company is a pre-clinical stage gene editing biotech, has built leading genome editing platform and mRNA-LNP library. It possesses strong capability of novel Cas and base editors discovery and exceptional production capacity for LNP drug manufactory, with strong IP protection globally. It has created a pipeline with 10 genetic medicines focusing on cardiovascular diseases, infectious diseases and rare diseases.

Innovent Biologics Co is a biopharmaceutical company that focuses on developing innovative biotechnology solutions in the healthcare and pharmaceuticals industry.