Exegenesis Bio, a clinical stage global gene therapy company, announced that US FDA has granted Orphan Drug Designation (“ODD”) to EXG110, a novel gene therapy for Fabry disease, a rare lysosomal disorder that results in excessive deposition of lipids in tissues, eventually leading to renal failure, cardiac disease and strokes. EXG110 is a one-time treatment that delivers the genetic payload directly to liver and heart cells with improved efficacy, safety and dosing. The first patient has been dosed in a China clinical trial and the company plans to initiate a US clinical trial.
The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation qualifies a company for incentives, including tax credits for qualified clinical trials, exemptions from user fees, and the potential for seven years of market exclusivity after approval.
“The FDA’s decision to grant orphan drug designation to EXG110 highlights the need for better approaches to treat Fabry disease, a debilitating condition that affects thousands of people worldwide. We believe this is validation of our rigorous science, our innovation culture and our strong commitment to rare disease patients,” stated Dr. Zhenhua Wu, CEO of Exegenesis Bio. “This is another milestone in our journey to become a truly Global Gene Therapy company. We recently shared updates on our other clinical programs and technology platforms at the European Society for Gene and Cell Therapy meeting, including updates on EXG102/202, a clinical stage gene therapy for wet Age-Related Macular Degeneration (wAMD) and EMC023, a muscle-specific capsid that enables targeted delivery of gene therapies for muscle diseases.”
EXG102 is an investigational gene therapy for wAMD that is administered by subretinal injection. Exegenesis Bio is developing a next generation version, EXG202, that incorporates the same genetic cargo, but is delivered to deep retinal cells by a highly-specific ocular capsid that enables administration by intravitreal injection, a less invasive, non-surgical approach. EXG202 has demonstrated a 3 to 5-fold increase in retinal transduction efficiency over currently known ocular-specific capsids in a non-human primate study.
“Our unique approach to wAMD is built on three pillars: (1) targeting multiple pathways involved in neovascular disease, including VEGF subtypes A, B, C, D and Angiopoietin 2; (2) packaging our cargo in a proprietary capsid that targets photoreceptors and retinal pigment epithelium more efficiently, and (3) administering the treatment by intravitreal injection, a safe, non-surgical procedure,” emphasizes Dr. Wu. “We believe this will result in a "Best-In-Class" treatment for wAMD and other ocular diseases. We are currently conducting clinical trials in USA and China and hope to share further data in the coming months.”
EMC023 is Exegenesis Bio’s proprietary muscle-specific capsid that targets and transduces skeletal muscle tissues more effectively than current benchmark AAV muscle-specific capsids. EMC023 has demonstrated increased skeletal muscle transduction in a non-human primate study – up to 3-fold increase in vector genome copies and up 13-fold increase in transgene mRNA levels.
“We believe that we have discovered one of the most specific skeletal muscle targeting capsids known to date,” states Dr. Wu. We are excited to explore the potential of this novel delivery platform in a variety of muscle diseases, including Duchenne Muscular Dystrophy.”
“I would like to congratulate our global team for accelerating progress on all of our pipeline programs and platform technologies. We have built a company with full in-house R&D, CMC, Quality and Regulatory capabilities, and we now have three clinical stage programs in USA and/or China: (i) wet Age Related Macular Degeneration, (ii) Spinal Muscular Atrophy, and (iii) Fabry Disease. Our team is operating with a sense of urgency to bring these much-needed treatments to patients worldwide,” emphasizes Dr. Wu.
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