On April 24, 2025 — GenAssist Ltd (GenAssist) announced meaningful functional improvements in the second patient in the ongoing IIT study of its groundbreaking DMD base editing drug GEN6050X after the reported positive response from the first patient. Up to date, two patients have finished 6 months follow-up. Dosing of a third patient is underway. […]
On Mar 19,2025 GenAssist Ltd (GenAssist) disclosed the interim results of its base editing DMD drug, GEN6050X, from its investigator-initiated trial (IIT) (NCT06392724) at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. The early findings indicated the encouraging therapeutic potential for this in vivo base editing drug. As of the reporting date, the
On March 06, 2025, GenAssist Ltd (GenAssist), a pioneering gene-editing biotechnology company specializing in genome medicines, is thrilled to announce that it has received clearance from the U.S Food and Drug Administration(FDA) for its Investigational New Drug (IND) application for GEN6050X, a first-in-class base editing drug for Duchenne Muscular Dystrophy (DMD). GenAssist is planning to
On January 20, 2025, GenAssist Ltd (GenAssist), a pioneering gene-editing biotechnology company specializing in genome medicines, is excited to announce that one late-breaking oral presentation and six posters presentation will be released at the 2025 Muscular Dystrophy Association (MDA) Conference. The conference will take place from March 16-19th, 2025 at Hilton Anatole, Dallas, TX. Oral
GenAssist Ltd Announces Oral Presentations at the 2025 MDA Conference Read More »
On November 7, 2024, GenAssist and the JIREN Charity Foundation held the launch ceremony for the “YOUXING Fund” at AstraZeneca’s booth during the 7th China International Import Expo (CIIE). The event was witnessed and supported by AstraZeneca China. During the ceremony, the representative from GenAssist highlighted the global challenges faced in the treatment of rare
GenAssist & JIREN Charity Foundation’s “YOUXING Fund” Launch Ceremony Held at CIIE Read More »
GenAssist Ltd (GenAssist), a pioneering gene-editing biotechnology company for genome medicines, announce that two of our posters for presentation will be released at American Society of Gene and Cell Therapy’s (ASGCT) Breakthroughs in Muscular Dystrophy, which will take place November 19-20, 2024, at The Westin Michigan Avenue Chicago, IL. Several latest advancements in first DMD
GenAssist Ltd (GenAssist), a pioneering gene-editing biotechnology company for genome medicines, released six posters for presentation at the ESGCT 31st Annual Congress, which is held from October 22-25, 2024, in Rome, Italy. Several latest advancements in base editing, DMD, and AAV vector design were featured by poster presentations from GenAssist at ESGCT, which covered a
On September 06, 2024, GenAssist Ltd (GenAssist), announced that the first DMD patient has been successfully dosed with its base editing drug, GEN6050X injection, in an investigator-initiated trial (IIT). “To date, no gene editing drug has entered clinical trials for DMD indication. This first-in-human dosing marks that DMD treatment has entered the gene editing era”,
On July 29, 2024, Suzhou GenAssist Therapeutical Co., Ltd, the operation entity of GenAssist Ltd (GenAssist), announced that Pre-IND (PIND) meeting request for its first base editing DMD drug, GEN6050X, has be submitted to NMPA. Previously, the PIND meeting to FDA was held in April 2023. The PIND filing to FDA and NMPA indicated the
On May 30, 2024, Suzhou GenAssist Therapeutical Co., Ltd, the operation entity of GenAssist Ltd (GenAssist), announced that the IIT(NCT06392724) kickoff meeting for its first base editing DMD drug, GEN6050X, has be successfully held in Peking Union Medical College Hospital (PUMCH). This program is sponsored by Suzhou GenAssist Therapeutics and led by Professor Yi Dai,
Senior pharmacology and toxicology advisor
Pharmacology, Shang University of Traditional Chinese Medicine.
Senior Vice General Manager at Shanghai InnoStar Biotechnology Co., Ltd.
More than 20 years in preclinical pharmacology and toxicology evaluation of new drugs.
Senior Scientific Advisor
Ph.D., Molecular Cell Biology and Bioengineering, Max Planck Research School.
Senior Director of genome engineering department, AstraZeneca, Gothenburg, Sweden.
More than 10 years’ experience in gene editing tool and drug development.
Senior Clinical Physician Advisor
Ph.D. , Peking Union Medical College, Tsinghua University.
Associate Chief physician. Neurology Department. Peking Union Medical College Hospital, Beijing.
Member of the Neurology Branch of the Chinese Medical Association.
Member of the DMD/BMD Study Group of the Chinese Rare Disease Alliance.
Specializes in complex and rare neurological disorders, gene diagnosis of monogenic.
Senior strategic Advisor
Over 30 years of experience in drug discovery, clinical translation, clinical practice, and operational management Former President of Chongqing Medical and Pharmaceutical College.
Ex-Vice President of the Technical Center at Fosun Pharma.
Senior Biostatistics Advisor
Ph.D. Biostatistics, University of Michigan, USA.
Bettyann Asche Murray Distinguished Professor, Deputy Chair.
Department of Biostatistics, University of Texas, MD Anderson Cancer Center.
Inventor of Bayesian Optimal lnterval PhaseⅠ/ⅡTrial Design (BOlN12); Participated in clinical trial design of many anti-cancer treatment.
Co-funder, Senior scientific Advisor
Ph.D. , Pathology, Ohio State University
Associate Professor at West Lake University, Young Scientist in the 973 Program
Thousand Youth Talents Program, National Outstanding Young Scientist
Inventor of Targeted AID-mediated Mutagenesis (TAM) base editor
CEO, Co-Founder
Ph.D. , Immunology, Shanghai Jiao Tong University.
MBA, Manchester Business School.
Over 20 years of experience in pharmaceutical industry, covering R&D, BD, Investment and Incubation.
Quality management advisor
Former Director of the Quality Assurance Department and Laboratory at the Shanghai Institute of Biological Products
Research and establishment of quality standards for various viral bioproducts as well as registration applications
Committee Member of the Chinese Pharmacopoeia Commission,
Expert Member of the Shanghai Science and Technology Commission
Committee Member of the Quality Management Committee of the Shanghai Medical Industry Association
Member of the New Technology/Skills Professional Committee.