PHILADELPHIA--(BUSINESS WIRE)--Exegenesis Bio, a clinical stage global gene therapy company is pleased to announce 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.
Exegenesis Bio plans to provide further R&D updates on Fabry Disease, wAMD, Spinal Muscular Atrophy and Muscle Tropic Capsid programs during JP Morgan week in San Francisco, Jan 13-16, 2025.
About Fabry Disease
Fabry disease is a rare X-linked lysosomal disorder that results in excessive deposition of lipids in the tissues. Researchers have identified hundreds of mutations causing Fabry disease in the gene for alpha-Gal A, located on the X chromosome. Deficiency of alpha-Gal A is the main cause for the lysosomal buildup of glycosphingolipids. Progressive glycolipid accumulation can lead to renal failure, cardiac disease and strokes.
About wet Age Related Macular Degeneration (wAMD)
Nearly 20 million people in the US are living with Age-Related Macular Degeneration (AMD), a progressive eye disease caused by damage to the macula/retina that can lead to blurred vision and blindness. Almost 2 million people have a more severe form of the disease, “wet” AMD (wAMD), which is caused by abnormal blood vessel growth and blood or fluid leakage into the macula, leading to scarring and rapid loss of central vision. wAMD accounts for approximately 90 percent of all AMD-related blindness worldwide.
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy is a genetic disorder characterized by the progressive loss of muscle. It is a multi-systemic condition, affecting many parts of the body, resulting in deterioration of the skeletal, heart, and lung muscles. Over a million people are living with DMD and other muscle dystrophies worldwide.
About EXG110 Gene Therapy for Fabry Disease
EXG110 is a one-time treatment for Fabry disease in children and adults, engineered with a proprietary capsid to increase delivery to target heart and liver cells, potentially improving efficacy and safety, and enabling lower dosing. The first patient has been dosed in China and a US clinical trial is planned in 2025.
About EXG102-031 Subretinal Injection for wet Age Related Macular Degeneration
EXG102-031 is a rAAV-based gene therapy expressing a therapeutic fusion protein that is able to bind/neutralize all known subtypes of Vascular Endothelial Growth Factor (VEGF A, B, C, D) and Angiopoietin-2, which are known to stimulate abnormal blood vessel formation and vascular leakage in the retina. The US and China based clinical trials will evaluate safety and tolerability, as well as visual acuity and central retinal thickness in patients with wAMD.
About EXG202 Intravitreal Injection for wet Age Related Macular Degeneration
EXG202 is a next generation gene therapy that incorporates the same genetic cargo as EXG102-031 – and 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
About Exegenesis Bio
Exegenesis Bio is a clinical stage global gene therapy company with operations in Philadelphia, Boston, Singapore and China. The company’s innovative gene therapy pipeline is based on proprietary capsids, promoters and unique protein engineering designs. Three programs have advanced to the clinical stage: (1) wAMD in USA and China, (2) Spinal Muscular Atrophy (SMA) in China and (3) Fabry disease in China. Exegenesis Bio 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. The company employs over 200 scientific and operations staff worldwide.
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Company Website: www.ExegenesisBio.com
