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June 25, 2025 GenAssist Ltd (GenAssist) announced that the United States (U.S.) Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to GEN6050X, the global first in vivo base editing drug for the treatment of Duchenne Muscular Dystrophy (DMD). Duchenne muscular dystrophy (DMD) is a serious or life-threatening disease that affects approximately […]

At the recent ASGCT 2025 Annual Meeting, GenAssist first showcased its cutting-edge HOPE (Human Originated Peptide Evolution) AAV evolution platform. This platform displays extremely enormous human genomic sequences （3B nt）on AAV via GenAssist’s proprietary primer library. HOPE technology enables the generation of about 10 billion random peptide libraries and more than 1 million AAV sequences, which facilitates the possibility of any tissue-specific

June 9, 2025 GenAssist Ltd (GenAssist) announced that the United States (U.S.) Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to GEN6050X, the company’s first in vivo base editing drug for the treatment of Duchenne Muscular Dystrophy (DMD). “DMD is a devasting progressive neuromuscular disorder that affects approximately 450,000 to 600,000 patients

May 17, 2025 – GenAssist Ltd (GenAssist) today announced updated efficacy results—highlighting new biomarker findings—from its ongoing investigator-initiated trial (IIT) of GEN6050X. The latest data were presented orally in the Gene Therapy for Muscle diseases session at the 28th Annual Meeting of the American Society of Gene & Cell Therapy. GEN6050X is an investigational base

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

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 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