Peking Union Medical College Hospital

SUZHOU, China I March 6, 2025 I 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: Topline efficacy data from patient 001 6 months after treatment 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 year 1 . 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 www.genassisttx.com , Contact: xinya@genassisttx.com . SOURCE: GenAssist Therapeutic

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

German drugmaker Boehringer Ingelheim didn’t share any data behind the top-line result, instead promising that the efficacy and safety findings would be unveiled in the second quarter.\n Boehringer Ingelheim has cemented the potential of its idiopathic pulmonary fibrosis (IPF) drug with a second phase 3 win in a related lung disease leading the drugmaker to expand the indications it will seek FDA approval for.The FIBRONEER-ILD trial saw 1,178 patients with progressive pulmonary fibrosis (PPF) receive either a 9 mg or 18 mg dose of the PDE4B inhibitor nerandomilast or placebo twice a day for 52 weeks. The study hit its primary endpoint of demonstrating an absolute change from baseline in forced vital capacity—a measure of the volume of air that a patient can breathe out—Boehringer said in a Feb. 10 release.The German drugmaker didn’t share any data behind the top-line result, instead promising that the efficacy and safety findings would be unveiled in the second quarter.“Initial data readouts of the FIBRONEER trials support a generally consistent safety and tolerability profile when compared to the phase 2 IPF study, with overall adverse events comparable to those seen in the placebo group,” the company said.Armed with the latest results, Boehringer said it will request that FDA and global regulators approve nerandomilast as a treatment for PPF, a type of interstitial lung disease. The company has already said it will seek approval for nerandomilast in IPF after proclaiming a win in a phase 3 trial for that lung disease in September 2024. Again, the company opted to hold back any concrete data from that trial for later in this year.When it comes to managing IPF, physicians already have Boehringer’s Ofev and Roche’s Esbriet. However, those drugs only slow the progression of fibrosis, while attempts to produce molecules that further slow, or ideally stop, progression have failed to prove their worth.By targeting phosphodiesterase 4B (PDE4B), nerandomilast is meant to have a mechanistic advantage over the incumbent products. Targeting PDE4 drives anti-inflammatory and antifibrotic effects, but attempts to develop oral inhibitors of the enzyme in respiratory diseases have been hindered by class-related systemic adverse events such as diarrhea and headaches.“The positive FIBRONEER-ILD topline result shows the potential of nerandomilast in progressive pulmonary fibrosis,” Shashank Deshpande, Head of Human Pharma at Boehringer, said in the release. “The hope is that the safety and tolerability profile we are initially seeing could potentially help to reduce treatment challenges.”