Market Overview

Patient-Driven Gene Therapy Company ReflectionBio Receives Orphan Drug Designation From U.S. FDA For the Treatment of Bietti's Crystalline Dystrophy (BCD)


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.

About Bietti's Crystalline Dystrophy (BCD): BCD is a rare retinal
degeneration. BCD was first identified by Italian ophthalmologist Dr.
G.B. Bietti in 1937. In BCD, progressive atrophy and degeneration of the
retinal pigment epithelium (RPE) lead to symptoms similar to those of
other forms of retinal degeneration, including retinitis pigmentosa
(RP). Yellow-white crystals are seen in the retina of BCD patients,
which may disappear in advanced stages. Onset usually occurs between
early teenage years and the third decade of life, leading to legal
blindness by the 3rd to 6th decade of life. Since
it is inherited in an autosomal recessive pattern, some BCD patients may
have no family history. Based on the BCD incidence rate (1:67,000)1,
BCD is estimated to affect more than 100,000 patients globally. BCD has
been reported in patients from Asia, Europe, North and South America,
the Middle East, and Africa. It is more common in East Asia, especially
among Chinese and Japanese populations. Researchers suggested that BCD
may be underdiagnosed because its symptoms are similar to those of other
eye disorders that progressively damage the retina.

About U.S. Orphan Drug Designation: Orphan drug designation is
granted by the FDA to a drug or biological product ("drug") that
demonstrates promise for the treatment of rare diseases or conditions
affecting fewer than 200,000 patients in the U.S. The designation allows
the drug developer to be eligible for a seven-year period of U.S.
marketing exclusivity upon approval of the drug, as well as tax credits
for clinical research costs, and the waiver of Prescription Drug User
Fee Act (PDUFA) filing fees.

About ReflectionBio – By Patients, For Patients: Reflection
Biotechnologies Limited (ReflectionBio) is a patient-driven biotech
company founded by a rare disease patient. ReflectionBio focuses on
developing gene therapy and other novel treatments for diseases that are
considered "incurable" today. Our founders collectively have more than
25 years of experience in science, law, finance, and technology
collaboration and management. ReflectionBio created and applies a "By
Patients, For Patients
" approach for patients to combine efforts in
driving research and development related to rare diseases and other
challenging diseases. For example, through initiation, collaboration,
incubation and translation of, or even innovation in, R&D projects, we,
the patients, can make a difference in advancing and accelerating drug
discovery and development. Moreover, ReflectionBio provides patient
advisory, advocacy and information services related to rare diseases,
treatment options, and driving R&D and drug development as patients.

1 Source for BCD incidence rate:

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