Suzhou Genassist Therapeutics Co Ltd.

SUZHOU, China, March 6, 2025 /PRNewswire/ -- 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 conduct clinical study globally for GEN6050X. "FDA clearance of our first IND is a significant milestone for our company. It validates the ability for our Targeted AID-mediated Mutagenesis (TAM) cytosine base editor technology to target diseases previously considered untreatable—broadening the full-body application of gene editing technology. As the second generation of CRISPR-Cas9, base editors offer immense potential with significantly lower off-target risks." said Dr. Chunyan He, CEO of GenAssist, "GenAssist is the first company to apply gene editing drug for DMD indication. By permanently repairing the mutated DMD gene, base editing may provide long-term benefit for DMD patients. We are excited about the potential of this program to bring new treatment for patients and demonstrate gene editing as a new therapeutic approach for DMD. In addition, GenAssist is advancing other DMD exon-skipping programs, which cover more than 30% DMD population." About GEN6050X Injection GEN6050X injection is an intravenous cytosine base editing drug 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 may permanently restore the expression of dystrophin through editing the mutated DMD gene. It provides an alternative solution for DMD patients. The IIT study is being conducted at Peking Union Medical College Hospital (NCT06392724) since August 2024. Until now, two patients have been dosed. The first 10-year-old patient has finished six-month follow-up. Safety Update of GEN6050X for the First two Participants Dosed: The drug was safe and tolerant with only transient and manageable SAEs. No SUSARs observed No hospitalizations reported All treatment-related AEs resolved with no sequelae No AEs of hepatic transaminitis observed, including no elevated gamma-glutamyl transferase levels Topline efficacy data from patient 001 6 months after treatment 1 point improvement in North Star Ambulatory Assessment (NSAA) 3 points increase in PUL2.0 scale, including 1 point increase in high level shoulder dimension and 2 points increase in mid-level elbow dimension items More than 100 meters increase in 6-Minute Walk Test Cardiac function keeps stable during follow up period More data will be presented at the 2025 MDA conference. 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 around age 12. Notably, patients reach a peak NSAA score of 26 at around 6.3 years old, after which the score declines by about 3 points per year1. As the disease advances, further complications, including heart and respiratory failure, occur, ultimately reducing the average life expectancy to about 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 GenAssist Ltd. (GenAssist) is a pioneering gene editing drug company with the operation entity located in China and subsidiaries in Boston (US) and Shanghai. The company is dedicated to developing base editing drugs against life-threatening diseases. Duchenne Muscular Disease (DMD) is the first therapeutic area. 1.F. Muntoni et al., PLoS ONE. 14, e0221097 (2019). For more information, please visit , Contact: [email protected]. SOURCE GenAssist Therapeutic Co.,Ltd WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

SUZHOU, China, May 6, 2023 /PRNewswire/ -- Suzhou GenAssist Therapeutic Co.,Ltd recently announced its pre-IND application of their first base editing product, GEN6050 and the acceptance by the FDA. GEN6050 is an in vivo base editing drug that targets exon 50 skipping in the Duchenne muscular dystrophy (DMD) gene. It is designed to restore the expression of dystrophin protein by base editing. DMD is a rare disease with a high incidence rate, with one DMD patient diagnosed per 4000 newborn boys. According to LEIDEN DMD data, exon 50 skipping can be used to treat 4% of DMD patients with large deletions [1]. Nowadays, DMD patients are often treated with marketed drugs such as glucocorticoids and ASOs, which target exon skipping at exon 45, 51, and 53. Until now, there is no approved drug targeting exon 50 skipping in the dystrophin gene, even in clinical stage. In addition, no AAV-delivered in vivo base editing drugs for any other indication have entered clinical trials globally. As one of the pioneering companies developing in vivo base editing drugs, Suzhou GenAssist Therapeutic has already established a comprehensive evaluation system for base editing drugs. Moreover, the Company is developing base editing products for more DMD mutations. The Pre-IND meeting usually refers to a voluntary meeting where the sponsor submits and communicates with the regulatory authority before conducting clinical trials of investigational new drugs (IND). The main purpose of the meeting is to review pharmacological study and non-clinical study, and to discuss whether existing research results are sufficient to support clinical research. About Suzhou GenAssist Therapeutic Suzhou GenAssist Therapeutic is a globally leading innovative company founded in July 2020. The headquarter of Suzhou GenAssist Therapeutic locates in Suzhou, China, with subsidiaries in Shanghai, China and Boston, USA. Suzhou GenAssist owns the global rights of TAM base editor, which is licensed from Chinese Academy of Nutrition and Health and Westlake University. Suzhou GenAssist Therapeutic is devoted to transform cutting-edging science into products that bring significant benefits to patients. For more information, please visit . [1] Data Source: Leiden Duchenne Muscular Dystrophy pages (version March 11, 2008). SOURCE Suzhou GenAssist Therapeutic Co.,Ltd

SUZHOU, China, May 6, 2023 /PRNewswire/ -- Suzhou GenAssist Therapeutic Co.,Ltd recently announced its pre-IND application of their first base editing product, GEN6050 and the acceptance by the FDA. GEN6050 is an in vivo base editing drug that targets exon 50 skipping in the Duchenne muscular dystrophy (DMD) gene. It is designed to restore the expression of dystrophin protein by base editing. DMD is a rare disease with a high incidence rate, with one DMD patient diagnosed per 4000 newborn boys. According to LEIDEN DMD data, exon 50 skipping can be used to treat 4% of DMD patients with large deletions [1]. Nowadays, DMD patients are often treated with marketed drugs such as glucocorticoids and ASOs, which target exon skipping at exon 45, 51, and 53. Until now, there is no approved drug targeting exon 50 skipping in the dystrophin gene, even in clinical stage. In addition, no AAV-delivered in vivo base editing drugs for any other indication have entered clinical trials globally. As one of the pioneering companies developing in vivo base editing drugs, Suzhou GenAssist Therapeutic has already established a comprehensive evaluation system for base editing drugs. Moreover, the Company is developing base editing products for more DMD mutations. The Pre-IND meeting usually refers to a voluntary meeting where the sponsor submits and communicates with the regulatory authority before conducting clinical trials of investigational new drugs (IND). The main purpose of the meeting is to review pharmacological study and non-clinical study, and to discuss whether existing research results are sufficient to support clinical research. About Suzhou GenAssist Therapeutic Suzhou GenAssist Therapeutic is a globally leading innovative company founded in July 2020. The headquarter of Suzhou GenAssist Therapeutic locates in Suzhou, China, with subsidiaries in Shanghai, China and Boston, USA. Suzhou GenAssist owns the global rights of TAM base editor, which is licensed from Chinese Academy of Nutrition and Health and Westlake University. Suzhou GenAssist Therapeutic is devoted to transform cutting-edging science into products that bring significant benefits to patients. For more information, please visit . [1] Data Source: Leiden Duchenne Muscular Dystrophy pages (version March 11, 2008). SOURCE Suzhou GenAssist Therapeutic Co.,Ltd