US FDA Grants Orphan Drug Status to EXG110 for Fabry Disease

Exegenesis Bio, a clinical-stage global gene therapy company, has announced that the US FDA has granted Orphan Drug Designation (ODD) to its novel gene therapy, EXG110, designed to treat Fabry disease. Fabry disease is a rare lysosomal disorder characterized by excessive lipid deposition in tissues, leading to significant health issues such as renal failure, cardiac disease, and strokes. EXG110 is intended as a one-time treatment, delivering a genetic payload directly to liver and heart cells, enhancing efficacy, safety, and dosing. The therapy is currently in clinical trials in China, with plans to initiate trials in the United States.

The FDA’s Office of Orphan Drug Products provides ODD to support the development of treatments for rare diseases affecting fewer than 200,000 people in the U.S. This designation offers several incentives, including tax credits for qualified clinical trials, exemptions from user fees, and the potential for seven years of market exclusivity post-approval.

Exegenesis Bio’s CEO, Dr. Zhenhua Wu, expressed his satisfaction with the FDA’s decision, highlighting the urgent need for improved treatments for Fabry disease. Dr. Wu emphasized that this recognition validates the company’s scientific rigor, innovation, and commitment to patients with rare diseases. The company has also shared progress on other clinical programs, such as EXG102/202 for wet Age-Related Macular Degeneration (wAMD) and EMC023, a muscle-specific capsid for gene therapies targeting muscle diseases.

EXG102 is an investigational therapy for wAMD that is administered through subretinal injection. The company is also developing EXG202, a next-generation therapy that uses a highly specific ocular capsid to deliver the treatment via intravitreal injection, a less invasive method. In non-human primate studies, EXG202 has shown a significant increase in retinal transduction efficiency compared to other ocular-specific capsids.

Dr. Wu elaborated on the company’s approach to treating wAMD, which involves targeting multiple pathways in neovascular disease, packaging the genetic material in a proprietary capsid for efficient delivery to photoreceptors and retinal pigment epithelium, and administering the treatment through a safe, non-surgical procedure. The company is conducting clinical trials for these therapies in the USA and China and anticipates sharing further data in the coming months.

EMC023 is another promising development from Exegenesis Bio. This muscle-specific capsid targets and transduces skeletal muscle tissues more effectively than current benchmarks, demonstrating a significant increase in vector genome copies and transgene mRNA levels in non-human primate studies. Dr. Wu expressed excitement about the potential of this novel delivery platform to treat various muscle diseases, including Duchenne Muscular Dystrophy.

Dr. Wu also commended the company’s global team for their progress across all pipeline programs and platform technologies. Exegenesis Bio boasts full in-house capabilities, including R&D, CMC, Quality, and Regulatory, and has advanced three clinical-stage programs in the USA and China: wAMD, Spinal Muscular Atrophy, and Fabry Disease. The company is committed to delivering these much-needed treatments to patients globally.

Looking ahead, Exegenesis Bio plans to provide further updates on its R&D efforts for Fabry Disease, wAMD, Spinal Muscular Atrophy, and Muscle Tropic Capsid programs during JP Morgan week in San Francisco in January 2025.

Fabry disease, a rare X-linked lysosomal disorder, results from mutations in the gene for alpha-Gal A. The deficiency of this enzyme causes lipid buildup in tissues, leading to severe health problems. Wet Age-Related Macular Degeneration (wAMD) is a severe eye disease caused by abnormal blood vessel growth and leakage in the retina, leading to vision loss. Duchenne Muscular Dystrophy (DMD) is a genetic disorder characterized by progressive muscle deterioration, affecting multiple body systems.

In summary, Exegenesis Bio is advancing innovative gene therapy solutions for a range of serious health conditions, leveraging proprietary technologies to improve treatment efficacy and patient outcomes.