By Patients, For Patients

Alt-text for the human brain image in the banner: An image of a human brain signifying patients’ will and brain power in driving scientific and medical research and development.
ReflectionBio – By Patients, For Patients ®
Embracing the role of patients in driving scientific
and medical research and development.

ReflectionBio's By PATIENTS, FOR PATIENTS TM Approach and Belief

By Patients, For Patients TM
     – With faith, relentless efforts and the right partners, We, the PATIENTS, can make a difference in driving R&D to help ourselves and others.
ReflectionBio – By Patients, For Patients ®

Why Patient-driven R&D?

Urgent Unmet Medical Needs

There are 8,000 rare diseases.

Many cause premature death or life-long disabilities.

10% of the public suffer from at least one rare disease.

An image of a balance signifying the unbalance between the urgent unmet medical needs of rare disease patients and the sad reality.

Sad Reality

Rare disease = Less commercial and research interest.

Incentive policies have significantly increased the number of orphan drug approvals.

However, more than 90% of rare diseases still do not have any approved treatment available.

We, the rare disease patients, cannot afford to wait.


We, the patients, can make a difference in driving rare disease and orphan drug research and development in many ways, that is, to initiate, incubate, innovate and/or translate.


Many rare diseases lack basic research.
By initiating treatment oriented research, we, the patients, can make the critical 0 to 1 step.


Rare disease research is grossly under-supported by public funding sources.
We, the patients, can combine resources to incubate orphan drug R&D.


There are around 8,000 rare diseases. Information on rare diseases is scarce and scattered.  Therefore, rare disease experts are also rare.
Some rare disease patients became an expert of their own disease by reading and studying literature, talking to doctors and researchers, and combining their own experience as a patient.
From gene discovery to drug research and development, rare disease patients can proactively drive R&D and even contribute to the development of orphan drugs.


Latest scientific breakthroughs such as gene therapy have made it possible to develop treatments for many “untreatable” rare diseases.
We, the patients, can play an active role in driving R&D to translate the latest scientific breakthroughs to clinical applications.

Our Progress

Using Bietti’s Crystalline Dystrophy (BCD), a rare blinding disease as an example, ReflectionBio®, as a patient-driven biotech company, initiated and incubated the BCD gene therapy preclinical research, made innovations in BCD disease model and gene therapy drug candidates, obtained Orphan Drug Designation from the U.S. FDA and is in the process of advancing BCD gene therapy into human clinical trial.

To learn more about what ReflectionBio® has achieved in rare disease, orphan drug and Gene Therapy research and development with our By Patients, For Patients TM approach, please see Research + Development.

An image of the DNA

Our Vision

Going beyond BCD.

ReflectionBio’s progress in the research and development of gene therapy for treating Bietti’s Crystalline Dystrophy (BCD) was driven by our founder & CEO, Richard Yang. As a BCD patient, Richard initiated and drove the BCD gene therapy preclinical study in the process of going blind. With more patients and families join our efforts, we can do more beyond BCD and develop potential treatments for other rare retinal diseases and challenging disorders.