
Gene Therapy Drug Developer

A Warm Spring Day Opens a New Chapter, the Pharmaceutical Industry Invites All Talents to the Grand Event.March 1, 2025 to March 2, 2025,IBI EXPO 2025 (3rd) Biopharmaceutical Innovation Industry ConferenceWill be inSuzhou International Expo CenterGrandly held.
Conference FocusCell therapy, gene therapy, novel antibody drugs, stem cells and regenerative medicine, gene editing, nucleic acid drugs, exosomes, novel deliveryetc.In the frontier field of biopharmaceutical technology, fromNew Drug R&D, Non-clinical Evaluation, CMC Development, Drug Registration, Clinical Trials, Commercial Transformationetc.The entire lifecycle of biopharmaceuticals will be covered, and leading enterprises from various细分 fields in China, emerging companies, as well as representatives of leading companies that have made significant progress in the past year will be invited to present an unprecedented annual event in the field of biopharmaceutical innovation.

Since AAV vectors were first used in human patients, AAV gene therapy has evolved over nearly 30 years and has become one of the popular tracks in the innovative drug field. According to publicly available information,As of June 21, 2024, eight AAV gene therapies have been approved for marketing worldwide, with over 900 pipelines concurrently under research and development.These therapies inRare diseases, tumor-related diseases, hematological disorders, retinal diseases, hereditary deafness, etc.Significant breakthroughs have been made in the field, bringing new treatment hope to many patients.Global AAV Gene Therapy Sales Reached $1.5 Billion Last Year, Expected to Reach $22.3 Billion by 2029. It can be seen that the future of AAV gene therapy is promising.
China’s AAV gene therapy started later compared to foreign countries, but it is developing very rapidly. This year, China's AAV gene therapy has achieved many significant breakthroughs.
On July 24, Belief BioMed announced that the marketing application for its AAV gene therapy BBM-H901 was accepted by the NMPA for the treatment of adult patients with hemophilia B. The therapy uses a liver-targeting AAV843 capsid-based vector, which indicates that China-produced AAV gene therapies are about to enter the commercialization stage. Belief BioMed has already established AAV commercial production facilities.
In October, Lingyi Biotech announced that a "prospective, single-center, open, single-arm clinical study evaluating the safety and efficacy of a single intravenous infusion of LY-M001 injection in adolescent patients with Type I Gaucher disease" was successfully conducted and achieved therapeutic effects at the National Children's Medical Center, Shanghai Children's Medical Center affiliated with Shanghai Jiao Tong University School of Medicine. LY-M001 is China’s first independently developed AAV gene therapy drug targeting Type I and Type III Gaucher disease, which can express the glucocerebrosidase required by patients after a single injection.
In September, LY-M003 Injection, another product by Lingyi Biotech, completed the first dosing in a gene therapy patient with Wilson's disease.
In September, the rAAV gene therapy FT-002 injection developed by Fangtuo Biotech received Phase II IND approval from the U.S. FDA. This is the first rAAV gene therapy produced in China to be granted FDA approval for directly conducting Phase II clinical trials in the United States. Previously, clinical research data published by Fangtuo Biotech on FT-002, with the longest follow-up of one year among 18 patients, showed that all subjects demonstrated good safety and tolerability, and certain indicators such as retinal sensitivity and visual function significantly improved in specific dose groups.
In September, the IND application for RM-101 from Ruifeng Bio was officially approved by the U.S. FDA for the treatment of Usher syndrome. The press release noted that RM-101 is the world’s first AAV-based gene-editing candidate drug for this indication. RM-101 is a candidate AAV gene therapy targeting USH2A gene-related retinitis pigmentosa. Administered via subretinal injection, RM-101 specifically infects photoreceptor cells, inducing exon 13 skipping in USH2A to produce a truncated but biologically functional Usherin protein, thereby maintaining the normal structure and function of photoreceptor cells.
In the same month, the IND for Jiyin Biotech's EXG110 injection was accepted by the CDE. The EXG110 injection uses a recombinant AAV vector to deliver the therapeutic gene GLA to liver and muscle cells, aiming to treat Fabry disease.
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Moreover, the rapid development of China's AAV gene therapy field is not only reflected in clinical progress but also in technological innovation and international cooperation. Multiple companies are actively exploring new technical pathways, aiming toImprove gene delivery efficiency, enhance treatment safety, and broaden the range of indications. For example, by optimizing AAV capsid design, developing new gene editing tools, and utilizing artificial intelligence-assisted drug discovery, the advancement of AAV gene therapy is continuously propelled.More precise, more efficient, more secureThe direction of development.
In addition, with the development of AAV gene therapy, it can also be seenFrom raw material supply, vector production, plasmid preparation to clinical trial services, a series of professional service providers have emerged, offering strong support for the research, development, and commercialization of AAV gene therapies. This has not only reduced R&D costs but also improved overall R&D efficiency, accelerating the clinical translation of innovative achievements.
As the industry is about to enter the commercialization stage, in order to boost the development of the industry,IBI EXPO 2025 (3rd) Biopharmaceutical Innovation Industry ConferenceSpecialThe 5th Gene Editing and Gene Therapy Technology Seminar, itsAAV Gene Therapy Sub-venueSpecially invitedLingyi Biotech Founder and CEO Lin QingServed as a co-planner, exploring with participants from multiple perspectivesThe Development Path of AAV Gene Therapy, seize opportunities at critical moments, build a more complete industrial ecosystem, and boost the track's takeoff.
Introduction to the Planner

Dr. Lin Qing
Founder and CEO of Lingyi Biotech
Former Head of WuXi AppTec Rare Disease Center
Lin Qing, Ph.D. in Biology from Zhejiang University, worked as a lecturer at the Institute of Genetics at Fudan University after graduation. In 1999, she went to Vanderbilt University in the United States to engage in functional genomics research for nearly 10 years, advancing from postdoctoral researcher to assistant professor.Nature, Nature Biotech, PNASPublished more than 20 papers in important journals. Also has over a decade of experience in the pharmaceutical industry, holding key leadership positions at companies such as Sundia, Pfizer, and WuXi AppTec. Has extensive successful R&D cases in the fields of hereditary rare diseases, tumor immunology, and autoimmune diseases.

AAV Gene Therapy Sub-venue
Key Content and Hot Topics
New Capsid Modification and Application
New DNA Delivery Technology (LNP-DNAVDNA Electroporation)
Roundtable Discussion:Market Trends of AAV Gene Therapy in China
This forum is for enterprises and scientific research institutions guestsOpen Report Seats, to showcase the latest achievements to the industry and create more cooperation opportunities. Welcome to register!

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