Orphan Drug Designation Has Been Granted to a Gene Therapy for Bietti’s Crystalline Dystrophy

Reflection Biotechnologies has announced that the US Food and Drug Administration has awarded Orphan Drug Designation to their RBIO-101 program, which is focused on a gene therapy designed to treat Bietti’s crystalline dystrophy. Reflection Biotechnologies is a patient-driven gene therapy company that was founded by a patient with a rare disease. The company is dedicated to researching potential treatments for rare diseases. For more detailed information, you can read the source press release here, at Business Wire.

About Bietti’s Crystalline Dystrophy

According to the NIH, Bietti cystralline dystrophy (BCD) is a disorder that is characterised by the accumulation of fatty compounds in the back of the eye (the retina). This causes damage and may lead to loss of vision. Typically, people affected by BCD first begin to notice changes to their vision in their teens of twenties, which may include night blindness (difficulty seeing when its dark), and less sharp vision. People with BCD may also lose areas of their vision, usually the sides, and colour vision may worsen. However, each eye may show different symptoms.

BCD can be caused by alterations to the CYP4V2 gene. This gene is involved in the production of a specific enzyme that is thought to be involved in breaking down lipids to make energy (fatty acid oxidation), although the enzyme’s role remains poorly understood.

About the RBIO-101 Program

Reflection Biotechnologies are researching an investigational AAV-based gene therapy for the potential treatment of BCD. The next step of the development program is to test the BCD gene therapy in a human clinical trial.

About Orphan Drug Designation

The US FDA has awarded Orphan Drug Designation the RBIO-101 program. This designation is reserved for drugs that are intended to treat rare diseases – defined as conditions that affect fewer than 200,000 Americans. It is designed to support the development of drugs and products for rare diseases, and provides several benefits to the developers. These can include tax credits for clinical testing, a longer period of marketing exclusivity, and, in some cases, a waived drug user fee. For more information about Orphan Drug status, you can click here.


Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

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