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


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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.

27% profits every 20 days?

This is what Nic Chahine averages with his options buys. Not selling covered calls or spreads... BUYING options. Most traders don't even have a winning percentage of 27% buying options. He has an 83% win rate. Here's how he does it.


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Posted In: BiotechNewsHealth CareSmall CapFDAGeneralEuropean Commission