Nanoscope Therapeutics co-Founder and President to Present at World Orphan Drug Congress Europe 2021, Nov. 17, in Barcelona, Spain
Will discuss the current state of gene therapy clinical trials along with Nanoscope's unique mutation-agnostic gene therapy for restoring vision in people blinded by retinal degenerative diseases
BEDFORD, Texas, Nov. 15, 2021 /PRNewswire/ -- Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing optogenetic gene therapies for treatment of retinal degenerative diseases, today announced that Co-founder and President Samarendra Mohanty, Ph.D., will make a presentation on the Clinical Trial Landscape for Gene Therapies at the World Orphan Drug Congress Europe 2021, on Wednesday, Nov. 17 at 12:10 pm CEST, in Barcelona, Spain.
Dr. Mohanty will examine recent progress in clinical development of gene therapies, as well as challenges that remain before they can become a standard of care. In addition, Dr. Mohanty will explore the widening field of gene therapy that includes advances such as gene editing technology along with the emergence of non-viral gene delivery platforms as an alternative to viral vector-based gene therapy.
The talk will include Nanoscope's AAV-based optogenetic gene therapy platform for restoring vision in patients blinded by retinal degenerative diseases regardless of genetic mutations. It's lead product, MCO-010, is in a Phase 2b multicenter, randomized, double-masked, sham controlled clinical trial in the U.S. for treatment of retinitis pigmentosa (RP) (NCT04945772).
In a Phase 1/2a open label trial of MCO-010, all 11 RP patients experienced clinically meaningful restoration of vision. MCO also is in development for Stargardt disease and age-related macular degeneration. The gene therapy has received orphan drug designation from the FDA for RP and Stargardt disease.
Other areas that Dr. Mohanty will discuss includes Challenges in novel clinical endpoints and potency assays, as well as off-target effects as it pertains to long-term safety. He will also examine potential combination therapies involving gene delivery coupled with external activation. The conference, the world's largest meeting of stakeholders involved in rare disease drug development, is being held at the Melia Sitges Hotel Nov. 15 – 18.
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 other IRDs as well as macular degenerations. For more information visit: https://nanostherapeutics.com/
Contact:
Dan Eramian
Opus Biotech Communications
[email protected]
425-306-8716
SOURCE Nanoscope Therapeutics
Related Links
http://www.nanostherapeutics.com
WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM?
Newsrooms &
Influencers
Digital Media
Outlets
Journalists
Opted In
Share this article