Nanoscope Therapeutics Announces FDA Approval of IND for Phase 2b clinical trial of Optogenetic Gene Monotherapy to Restore Vision in Patients with Retinitis Pigmentosa
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- Published on Thursday, 24 June 2021 11:05
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BEDFORD, TX, USA I June 23, 2021 I Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degenerative diseases, today announced that its Investigational New Drug application (IND) is now open for Phase 2b clinical trial of MCO-010, an ambient-light activatable optogenetic monotherapy to restore vision in patients with advanced retinitis pigmentosa (RP).
"Congratulations to the Nanoscope team and I am excited to be part of it. We hope our therapy will restore vision in ambient light without any external device, eliminating risk of photo-toxicity," said Sulagna Bhattacharya, CEO of Nanoscope.
The impressive clinical data presented at the American Academy of Ophthalmology 2020 annual meeting has persisted till the end of Phase1/2a study. "These are major milestones for Nanoscope with potential benefit for patients suffering from retinal degeneration," said Board Chairman Al Guillem, PhD.
The Phase 2b trial which starts in June, will be a randomized, double-blind, sham-controlled, multi-center trial in the USA enrolling patients with advanced RP. The treatment will involve a single intravitreal injection of MCO-010 to confirm its ability to improve the patients' vision. MCO has received orphan drug designations for RP and Stargardt disease from the FDA.
Nanoscope's gene therapy uses an AAV2 vector to deliver the MCO genes into bipolar retinal cells, where they express polychromatic opsins enabling vision in different color environments. The therapy can be administered in an office setting. Additionally, the therapy is applicable for RP regardless of underlying gene mutations.
A Phase 1/2a open label study of MCO-010 demonstrated the optogenetic monotherapy was safe with improved quality of life consistent with functional vision and visual function in advanced RP patients.
"Our gene therapies reprogram retinal cells and make them photosensitive to restore vision. MCO delivery with proprietary viral vector has allowed MCO-transduction in patients' retina, confirmed by sustained fluorescence reporter expression," explained Samarendra Mohanty, PhD, Nanoscope's President and Chief Scientific Officer.
"We are excited by the guidance received from FDA regarding improvising the characterization of our product, the primary endpoint and potency assays that will hopefully accelerate our clinical program to make the restorative drug available to RP patients," Dr. Mohanty added.
In normal eyes, opsins are expressed by photoreceptor cells and intrinsically photosensitive Retinal Ganglion Cells in the retina, and when activated by light they trigger the physiological process of vision. RP, linked to over 60 different gene mutations, encompasses a group of rare genetic disorders where photoreceptors degrade gradually, leading to impaired vision and eventual blindness.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing optogenetic therapies for giving sight to the millions of blind individuals suffering from retinal degenerative diseases, for which no cure exists. The company's pipeline includes optogenetics based retinal regeneration therapy for patients with RP, Stargardt disease, and dry age-related macular degeneration.
SOURCE: Nanoscope Therapeutics