Exegenesis Bio To Present Five Posters at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, Los Angeles, CA, May 16-20, 2023

2023-04-05

基因疗法临床1期

PHILADELPHIA--(BUSINESS WIRE)-- Exegenesis Bio, a rapidly growing global gene therapy company is pleased to announce the presentation of five posters at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Los Angeles, California, May 16 to 20, 2023. Exegenesis Bio will present nonhuman primate data from its novel muscle-directed capsid development program, comparing the muscle transduction of its proprietary AAV capsid variants with the most potent muscle-directed capsids known to date. The company will also present further safety and efficacy data from its novel, clinical stage wet Age-Related Macular Degeneration (wAMD) gene therapy program, EXG102-031. Exegenesis Bio will present the following five posters at the 2023 ASGCT meeting: AAV9 Variants Engineered for More Efficient Muscle Transduction Using AAVartaTM (a Proprietary AI-Aided AAV Capsid Evolution Discovery Technology) Demonstrate Improved Muscle Transduction Versus AAV9 and MyoAAV4A Ongoing Safety and Efficacy of EXG102-031, a Novel Clinical Stage AAV-based Gene Therapy for Neovascular AMD Expressing a Therapeutic Fusion Protein that Binds/Neutralizes All Known Subtypes of VEGF and ANG2 Intravenous Administration of a Very Low Dose AAV-GLA Gene Therapy Corrects Substrate Levels and Demonstrates Strong Efficacy in the Mouse Model of Fabry Disease Safe and Efficient Intra-CSF Delivery of AAV Vector in Nonhuman Primates Leads to Improved Transgene Expression in the Central Nervous System and Demonstrates a Robust Safety Pro > A Next-Generation AAV Gene Therapy for Spinal Muscular Atrophy Can Avoid Systemic High-Dose AAV Administration and Improve Safety and Efficacy “I congratulate our team for advancing our proprietary AI-aided capsid discovery programs into nonhuman primates and generating industry-leading data, while also moving our clinical programs into IND-enabling and early-stage clinical studies. This is strong validation of the world-class capsid discovery platform that our company has built and the novel gene therapy constructs that our team has designed. We are now at an exciting stage of our journey where we are ready to partner with other companies to advance our highly promising muscle capsids into clinical development and bring our innovative gene therapies to patients worldwide," stated Zhenhua Wu, CEO of Exegenesis Bio. About AAVartaTM AI-Aided AAV Capsid Evolution Discovery Technology Platform The proprietary AAVarta platform utilizes cutting-edge AI technologies to design novel AAV capsids by developing a highly efficient sampling strategy and AI-powered scoring system. This approach has led to the rapid identification of a larger yield of highly potent capsid candidates from a larger library space. The platform has produced a variety of AAV9 variants that can significantly increase transduction in muscle cells versus the most potent AAV9 variants known to date, including MyoAAV2A and MyoAAV4A. Exegenesis Bio will share specific performance of its proprietary muscle-specific AAV9 capsid variants in poster sessions at the ASGCT meeting in May 2023. About EXG102-031 EXG102-031 is a clinical stage rAAV-based gene therapy for wet Age-Related Macular Degeneration (wAMD) that expresses a therapeutic fusion protein that is able to bind/neutralize all known subtypes of Vascular Endothelial Growth Factor (VEGF) and Angiopoietin-2 (ANG2), which are known to stimulate abnormal blood vessel formation and vascular leakage in the retina. The EXG102-031 Phase 1 clinical trial will evaluate safety and tolerability, as well as visual acuity and central retinal thickness in patients with wAMD. About Exegenesis Bio Exegenesis Bio is a clinical stage global gene therapy company with operations in Philadelphia, Boston and China. The company’s innovative gene therapy pipeline is based on proprietary capsids, promoters and unique protein engineering designs. Two programs have advanced to the clinical stage: (1) Type I Spinal Muscular Atrophy (SMA) AAV gene therapy in China and, (2) wAMD in USA. The company has built state-of-the-art cGMP manufacturing facilities that include 500L and 2,000L disposable bioreactors for viral vectors and 30L disposable fermenters for plasmids. Exegenesis Bio has raised over $150 M since inception in 2019 and currently employs over 200 scientific and operations staff worldwide. Collaboration and Technology Licensing Opportunities Please contact us to discuss licensing opportunities for our novel Muscle and CNS directed capsids and co-development opportunities for our wAMD, Fabry and SMA gene therapy programs.