Nanoscope plans to apply for FDA approval for its optogenetic therapy for retinitis pigmentosa

Key Takeaways

  • MCO-010, a gene therapy that works regardless of the underlying mutation, demonstrated vision improvement in patients with permanent and severe vision loss from advanced retinitis pigmentosa (RP).
  • Nanoscope plans to submit a biologics license application to the FDA for MCO-010 later this year. 

Nanoscope Therapeutics has announced that it met the primary and secondary endpoints for its Phase 2b RESTORE clinical trial of MCO-010, an optogenetic therapy for restoring some vision to people with advanced vision loss from retinitis pigmentosa, a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina. As a result, the company plans to submit a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) in the second half of 2024. Approval of the BLA would enable the company to market MCO-010 to patients in the US.

MCO-010 is designed for people who have lost most or all of their photoreceptors, the light-sensing cells in the retina that make vision possible. The treatment, a small drop of liquid delivered by intravitreal injection, uses a human-engineered virus to deliver copies of a Multi-Characteristic Opsin (MCO) gene to bipolar cells — cells that don’t normally sense light but often survive after photoreceptors are lost to advanced retinal disease. The company says that MCO-010 is enabling the recipient’s bipolar cells to sense ambient light, thereby working as a backup system for lost photoreceptors. The approach doesn’t use goggles or eyewear to enhance the visual information coming into the retinas. It’s also designed to work regardless of the mutated gene causing the RP.

“MCO-010 would, for the first time, provide ophthalmologists a treatment option that actually gives vision back to the patients suffering permanent vision loss from advanced retinitis pigmentosa (RP), no matter which of the 100-plus mutations caused the retinal degeneration,” Samuel Barone, MD, chief medical officer of Nanoscope, told Healio. “Since MCO-010 is delivered via simple in-office intravitreal injection, we anticipate rapid adoption globally, once approved.”


“Nanoscope Plans to Apply for FDA Approval for its Optogenetic Therapy.” Eye On the Cure Research News, Foundation Fighting Blindness, March 27, 2024; see source article Anthony DeFino, “Gene therapy improves vision in patients with retinitis pigmentosa in phase2b trial.” Healio Ocular Surgery News, March 27, 2024; see source article