SUZHOU, China, Sept. 9, 2024 /PRNewswire/ — On September 06, 2024, GenAssist Ltd (GenAssist), announced the first DMD patient dosed with its base editing drug, GEN6050X injection, in an investigator-initiated trial (IIT).
“This is the first-in-human trial for DMD gene editing therapy. It marks that DMD treatment has entered the gene editing era”, said Chelsie He, CEO of GenAssist, “As the second generation of CRISPR-Cas9, base editors own huge application potential with lower off-target risk. DMD is such a devastating disease and lack effective drugs. DMD families suffer a lot from this disease. Leveraging this groundbreaking technology, we are dedicated to developing better treatments for DMD population.”
About GEN6050X Injection
GEN6050X injection is an intravenous cytosine base editing drug using dual AAV9 vectors, designed for DMD patients amenable to exon 50 skipping. GEN6050X is based on GenAssist’s unique RNA editing-free Targeted AID-mediated Mutagenesis (TAM) cytosine base editor technology. Through one-shot systemic administration, GEN6050X can permanently restore the expression of dystrophin through editing the mutated DMD gene. It may provide a curative solution for DMD. The IIT study is being conducted in the Peking Union Medical College Hospital (NCT06392724). At the same time, the IND filing of GEN6050X (US and China) is expected in November 2024.
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a rare, X-linked recessive hereditary disease caused by mutations in the dystrophin gene, leading to a loss of dystrophin protein vital for muscle cell membrane stability. DMD primarily affects skeletal and heart muscles, with progressive muscle wasting symptoms leading to loss of ambulation by around age 12, followed by further complications including heart and respiratory failure, with an average life expectancy of 26 years.
DMD affects approximately 1 in 3,500 to 5,000 live male births. According to LEIDEN data, about 80% of DMD patients could potentially benefit from exon skipping, with 4% specifically eligible for exon 50 skipping.
About GenAssist Ltd.
Founded in July 2020, GenAssist Ltd. (GenAssist) is a pioneering gene editing drug company. The operation entity of GenAssist is located in Suzhou, China, with two subsidiaries in Shanghai (China) and Boston (US). GenAssist owns the global rights of Targeted AID-mediated Mutagenesis cytosine base editor technology. The company is dedicated to developing base editing drugs against life-threatening diseases.
Check out our AAV CDMO service to expedite your gene therapy research
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
Related News
World-First CAR-T Therapy Using Donor Cells Sends Autoimmune Diseases into Remission
In a groundbreaking advancement, three individuals suffering from severe autoimmune diseases have achieved remission after receiving a revolutionary therapy using donor-derived immune cells. This pioneering treatment, which utilized bioengineered immune cells known as...
Eli Lilly to Invest $4.5 Billion in New Drug Manufacturing and Research Facility
Eli Lilly is making a major investment to enhance its drug manufacturing capabilities, announcing plans to build a $4.5 billion facility in Lebanon, Indiana. The new center, named the Lilly Medicine Foundry, will serve a dual purpose: developing innovative...
Avidity Biosciences Announces FDA Removed Partial Clinical Hold on Delpacibart Etedesiran (del-desiran/AOC 1001)
SAN DIEGO, Oct. 3, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug...
Quell Therapeutics Taps eXmoor Pharma to Manufacture CAR-Tregs
U.K. developer Quell Therapeutics hired eXmoor Pharma to make supplies of its pipeline of novel immunosuppressive cell therapies—called CAR-Tregs—at its new cell and gene therapy facility. Quell recently teamed with fellow U.K.-based eXmoor in a “strategic...
Related Services
AAV Packaging Services
READ MORE
Off-the-Shelf AAV Products
READ MORE
