Suzhou Xingming Youjian Biotech, a Fosun Pharma-Backed Gene Therapy Company, Wins 2024 ASGCT Outstanding Poster Award for Its First-in-Class Optogenetic Therapy UGX-201

Recently, Suzhou UgeneX Therapeutics Co., Ltd. (hereinafter referred to as "UgeneX") announced its participation in the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) held in Baltimore, USA, from May 7 to 11, 2024. The ASGCT Annual Meeting is the largest global event in the field of cell and gene therapy. At the conference, UgeneX presented two research posters.

Dr. Sun Sijie, Executive President of the company, shared on-site UgeneX's new drug research and development achievements in applying optogenetic gene therapy to restore vision for patients with advanced retinal degenerative diseases, including newly developedAnimal Efficacy Data of Novel High-Performance Ion Channel Optogenetic ProteinsAndPartial results of the IIT (Investigator-Initiated Trial) for the UGX-201 pipeline conducted at the First Affiliated Hospital of Soochow University. The peers present at the conference showed a strong interest in the latest developments of UgeneX's research.

In this annual meeting, the title is "Optogenetic Therapy UGX-201 for Patients with Advanced Retinitis Pigmentosa: Interim Results of an Investigator-Initiated Trial to Evaluate the Safety, Tolerability, and Preliminary Efficacy at the First Affiliated Hospital of Soochow University"UGX-201 Optogenetic Therapy for Advanced Retinitis PigmentosaPatient: The interim results of the safety, tolerability, and preliminary efficacy evaluation trial initiated by researchers at the First Affiliated Hospital of Soochow University" poster received the conference's "2024 Outstanding Poster Award." Globally, several companies have conducted clinical studies using optogenetic technology to treat various degenerative photoreceptor diseases. However, UgeneX's UGX-201 pipeline is the first to enter clinical research (investigator-initiated) using a GPCR-type photosensitive protein as the drug molecule, and the interim results are exciting. Therefore,Many researchers and pharmaceutical companies attending the conference were very interested in this pipeline. Winning the award further demonstrates that the clinical research results of the UGX-201 pipeline have garnered significant attention and recognition from the conference organizers and fellow attendees.。

UGX-201 isA broad-spectrum gene therapy product for retinal photoreceptor degenerative diseases (such as retinitis pigmentosa, dry age-related macular degeneration, etc.). The patient's retinal photoreceptor cells undergo degenerative changes due to the disease, and in the late stage, most or nearly all of the photoreceptor cells are lost, causing the retina to lose its photosensitive ability, resulting in a significant decline in vision or even blindness. UGX-201Using adeno-associated virus vectors to deliver the gene of recombinant opsin (an artificially modified light-sensitive protein of the GPCR type) into retinal ganglion cells, enabling them to possess the light-sensing function originally belonging to photoreceptor cells, thereby rescuing vision.UGX-201 is a global first-in-class product independently developed by UgeneX, with full intellectual property rights and global interests. It is also the first product of its kind to enter clinical research worldwide. In mid-2022, an investigator-initiated clinical study (IIT) was launched, and the interim results have already demonstrated excellent safety and efficacy.

About UgeneX

UgeneX was founded in July 2021, incubated by the new drug innovation fund of Fosun Pharma's Fosun Health Capital. It focuses on the development of in vivo gene therapy products for ophthalmic diseases. The company has a professional neurobiology and visual science R&D team with over 15 years of research experience.Targeting a vast market of 300 million patients globally with degenerative diseases of photoreceptor cells and optic nerves, UgeneX utilizes proprietary optogenetics and in-situ transdifferentiation technologies. Through intravitreal injection of innovative drugs, light-sensitive proteins or transcription factors are delivered, allowing the blind to see the world again.。

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