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

The European Commission has designated Orphan Drug status to Avrobio Inc's 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.