Thursday, August 30, 2018
Reflection Biotechnologies Limited ("ReflectionBio"), a gene therapy company committed to the research and development of life-changing treatments for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ReflectionBio's RBIO-101 program (AAV.CYP4V2), an AAV-based gene therapy product for treating Bietti's Crystalline Dystrophy (BCD).
"Receiving orphan drug designation from the FDA is a milestone. It validates our R&D progress and provides us with various incentives in further developing BCD gene therapy. This brings hope to BCD patients and their families because BCD is a devastating blinding disease for which currently there is no approved treatment. BCD is estimated to affect more than 100,000 patients worldwide. As the next step, we plan to advance BCD gene therapy into human clinical trial," said Richard R. Yang, Founder and CEO of ReflectionBio.
“As a rare disease patient, I am proud to be driving R&D efforts for BCD gene therapy together with our collaborator, Dr. Stephen H. Tsang of Columbia University. The RBIO-101 preclinical data from Dr. Tsang’s lab support the clinical translation for patients with BCD. I am grateful for Dr. Tsang, his team and Columbia University for partnering with us, the patients, in fighting rare diseases,” Yang added.
“One out of every 10 people lives with at least one of more than 7,000 rare diseases. Many rare diseases cause premature death or life-long disabilities. Sadly, more than 90% of rare diseases do not have any approved treatment available. Rare disease patients shoulder the inevitable odds of genetic mutations for mankind, but are often ignored by society and under-supported by public resources. At ReflectionBio, we apply a ‘By Patients, For Patients’ approach for us, the patients, to join forces and to play a more active role in driving scientific and medical R&D. We partner with researchers, clinicians, CROs, CMOs and other stakeholders in accelerating and advancing orphan drug development. With faith, relentless efforts and the right partners, we, the patients, can make a difference in driving R&D to help ourselves and others,” Yang commented.
