List of Adeno-associated viral vector (AAV) Companies in China - 21

At Anlong Bio, patients are our priority. Since launching, our goal has been to deliver transformative gene therapy medicines to patients faster and at low cost, in China and globally. We aim to provide unmatched access to treatments for patients through our extensive resources, our experienced leadership and advisory teams, and our trusted network of partners.

Founded in 2018, Belief BioMed is aiming to become a globally leading gene therapy company by being committed to providing innovative therapies with improved efficacy for monogenic disorder diseases, age-related degenerative diseases, and certain malignant diseases through its AAV vector technology from early discovery to commercialization. The R&D and production strengths of Belief BioMed have been recognized by top investment institutions and enterprises. Belief BioMed has offices, research centers and manufacturing facilities in Shanghai, Hong Kong, Beijing and Suzhou China and North Carolina US.

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!

CANbridge Pharmaceuticals is a pharmaceutical company that focuses on developing and commercializing innovative medicines to address unmet medical needs.

CanSino Biologics Inc. (CanSinoBIO, SHSE: 688185, HKEX:06185) is an innovative biopharmaceutical company dedicated to exploring best solutions to the prevention of diseases through cutting-edge research & development, advanced manufacturing and commercialization of innovative vaccine products for human use worldwide. Since its establishment in Tianjin, China in 2009, CanSinoBIO has experienced tremendous growth with 17 vaccines preventing 12 diseases, including approved vaccines for Ebola virus disease (Ad5-EBOV), COVID-19 (Ad5-nCoV, trade name: Convidecia), and meningitis (MCV2, trade name: Menphecia). CanSinoBIO has been listed on the Main Board of Hong Kong Exchange and Clearing Limited (HKEx) in March 2019. A year later, CanSinoBIO has successfully listed on the Sci-Tech Innovation Board (STAR Market) of the Shanghai Stock Exchange, making it the first "A+H" dual listing vaccine company. CanSinoBIO is focusing on continually expanding manufacturing capacity for its current vaccine candidates and further enhancing the competitiveness and the scope of its portfolio by promoting the R&D of new vaccine candidates. Leveraging broad experiences of our accomplished team of scientists and business leaders who had previously held technical and senior management positions at many of the leading pharmaceutical companies in the world, including Sanofi Pasteur, Astra Zeneca, Wyeth (now Pfizer) and CNBG (China), CanSinoBIO has developed five key platform technologies, including viral vector-based technology, synthetic vaccine technology, protein structure design and recombinant technology, mRNA technology as well as formulation and delivery technology.

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.

Exegenesis Bio is a clinical stage global gene therapy company with operations in Philadelphia, Boston and China. The company’s innovative gene therapy pipeline is based on proprietary capsids, promoters and unique protein engineering designs. Two programs have advanced to the clinical stage: (1) Type I Spinal Muscular Atrophy (SMA) AAV gene therapy in China (2) neurovascular AMD (wet AMD) rAAV gene therapy in USA The company has built state-of-the-art cGMP manufacturing facilities that include 500L and 2,000L disposable bioreactors for viral vectors and 30L disposable fermenters for plasmids. Exegenesis Bio has raised over $150 M since inception in 2019 and currently employs over 200 scientific and operations staff worldwide. Website: https://www.exegenesisbio.com/ Contacts: Company Information: Contact@ExegenesisBio.com Business Development: BD@ExegenesisBio.com Careers: Careers@ExegenesisBio.com

Frontera is a leading clinical-stage biotechnology company that seeks to develop the best gene therapy medicines to improve the lives of patients across multiple disease areas. Frontera's APEX Technology & Manufacturing Platform is an innovative adeno-associated virus (AAV) gene expression system. It includes novel and clinically validated AAV vectors, efficient and fully-integrated CMC processes, and access to early clinical data, making it possible to develop novel gene therapy products with quicker speed-to-market and lower cost.

Gritgen Therapeutics (here in after designated as "Gritgen") was founded in China and is a biotechnology company focused on gene therapy. They have launched a commercial GMP facility in China and have opened a good manufacturing practice (GMP) facility in Suzhou Industrial Park. Gritgen provides an important vector foundation for the company to realize gene therapy for hemophilia A. The company has also launched a Phase 1 clinical trial to assess the safety of gene therapy for hemophilia A.

Hengyu Biotechnology is a CXO platform founded by a scientific research team that provides biosafety testing and related process validation. It is a comprehensive provider founded by a group of top scientists, dedicated to providing innovative solutions in the biotech and biopharma space.

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.

At Neurophth, we walk from obstacle to obstacle without loss of enthusiasm. 在纽福斯公司，我们是从一个障碍走向另一个障碍而不丧失热情。 We are China’s first gene therapy company for ophthalmic diseases. Our mission is building a brighter future for patients by innovative gene therapies. Headquartered in Wuhan with subsidiaries in Shanghai, Suzhou, and USA, we are striving to discover and develop gene therapies for patients suffering from blindness and other eye diseases globally. We leverage our AAV and CMC platforms to regain vision in patients suffering from Leber’s hereditary optic neuropathy (LHON), autosomal dominant optic atrophy, vascular retinopathy (wAMD, DME, etc), optic neuropathy (glaucoma, etc) and other genetic diseases. Our most advanced investigational candidate, NR082 (rAAV2-ND4), in development for the treatment of LHON associated with ND4 mutation using a single administration through less invasive intravitreal injection, has received orphan disease designations in the U.S. We are always seeking highly motivated and talented individuals with diverse experiences, abilities and interests who are seeking to inspire the world and help patients to restore vision to join us on our journey. To learn more about Neurophth and our open positions, visit www.neurophth.com.

A global biotech company commited to developing new and effective therapies for psychiatric and neurological disorders affecting millions of patients in need. We design novel CNS-tropic AAV capsids and innovative gene expression cassettes to achieve efficient, effective, and safe gene therapies for severe CNS disorders.

Shanghai Sunway Biotech is a biopharmaceutical company that develops, manufactures, and commercializes cancer biotherapeutics based on its oncolytic virus platform. The company has been involved in clinical trials and has received approval for oncolytic virus therapy for cancer treatment in China. Shanghai Sunway Biotech aims to provide innovative and effective treatments for cancer patients.

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.