Benzinga | Mar 1, 2021 12:00PM EST

Avrobio's Cystinosis Gene Therapy Gets Orphan Drug Status In Europe

The European Commission has designated Orphan Drug status to Avrobio Inc's (NASDAQ: AVRO) AVR-RD-04, its gene therapy to treat cystinosis.

* AVR-RD-04 consists of the patient's hematopoietic stem cells, genetically modified to express cystinosin, the protein deficient in cystinosis patients.

* AVR-RD-04 is currently being evaluated in a Phase 1/2 trial sponsored by its academic collaborator at the University of California, San Diego.

* Orphan drug tag gives companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants, and ten years of market exclusivity following regulatory approval.

* AVR-RD-04 has also received orphan drug designation in the U.S.

* Cystinosis is a rare, progressive disease marked by cystine accumulation in cellular organelles known as lysosomes. This buildup can cause debilitating symptoms, including kidney failure, corneal damage, and thyroid dysfunction.

* Price Action: AVRO shares are up 4.09% at $11.45 in market trading hours on the last checked Monday.