Nanoscope Therapeutics Receives Orphan Drug Designation in Saudi Arabia

Got story updates? Submit your updates here. ›

Nanoscope Therapeutics, a biotechnology company based in Dallas, Texas, has received Orphan Drug Designation from the Saudi Food and Drug Authority for its MCO-010 gene therapy treatment. This designation provides regulatory and financial incentives to support the development of treatments for rare diseases and is an important milestone in Nanoscope's global expansion efforts.

Why it matters

Orphan Drug Designation is a significant regulatory achievement that can accelerate the development and approval process for rare disease treatments. This designation in Saudi Arabia builds on Nanoscope's existing Orphan Drug status in the United States and European Union, further solidifying the company's leadership in the global gene therapy market.

The details

MCO-010 is Nanoscope's lead gene therapy candidate, designed to treat a rare form of inherited retinal degeneration. The Orphan Drug Designation in Saudi Arabia provides Nanoscope with regulatory benefits, including protocol assistance, reduced regulatory fees, and potential market exclusivity upon approval.

• Nanoscope Therapeutics received the Orphan Drug Designation from the Saudi Food and Drug Authority on March 30, 2026.

What’s next

With this latest Orphan Drug Designation, Nanoscope Therapeutics will continue to work closely with regulatory authorities in Saudi Arabia, the United States, and the European Union to advance the clinical development and potential approval of MCO-010 for patients with rare retinal diseases.