DALLAS, Jan. 20, 2025 /PRNewswire/ -- Nanoscope Therapeutics Inc., a biotechnology company committed to restoring vision in blind patients in real-world settings by developing and commercializing novel gene therapies for retinal degenerative diseases, today announced several presentations by company leadership at the 21^st  Annual Advanced Therapies Week Conference, taking place from January 20-23, 2025 at the Kay Bailey Hutchinson Convention Center in Dallas, Texas. Details for the presentations are as follows:

Title: Navigating the Challenges of Gene Therapies for Rare Diseases Session Title: Gene Therapy for Rare Disease
Session Date: January 21, 2025
Session Time: 12:15 p.m. C.T.
Presenter: Samarendra Mohanty, PhD, President & Chief Scientific Officer

Dr. Mohanty will discuss key challenges to modern gene therapy programs for rare disease indications. These will include the general lack of suitable animal models, established clinical endpoints, and validated analytics and manufacturing processes, which in most cases need to be implemented by the gene therapy developer.

Title: Partnering for Commercialization, a CMC Prospective

Session Title: Partnering for Gene Therapy Success
Session Date: January 21, 2025
Session Time: 4:30 p.m. C.T.
Presenter: Raymond Kaczmarek, Chief Technical and Manufacturing Officer

During his presentation, Mr. Kaczmarek will highlight several key internal and external considerations paramount to ensuring a successful CDMO relationship and overall manufacturing program for gene therapy programs.

Session Title: International Convergence and Regulatory Alignment for CGT
Session Date: January 22, 2025
Session Time: 11:00 a.m. C.T.
Chair: Khandan Baradaran, PhD, SVP Regulatory and Quality

Dr. Baradaran will host a fireside chat with Dr. Nicole Verdun, Super Office Director of the Office of Therapeutic Products, CBER, discussing how gene and cell therapy developers can forge productive interactions with regulators to ensure an efficient path toward approval.

Title: Optical Approaches to Non-Viral Redosable Gene Therapies Session Title: Are Redosable Gene Therapies on the Horizon?

Session Date: January 22, 2025
Session Time: 11:00 a.m. C.T.
Presenter: Subrata Batabyal, PhD, Director, Non-Clinical Development

During his presentation, Dr. Batabyal will discuss the dosing limitations of AAV gene therapies, and how Nanoscope's non-viral, optical dosing technologies can overcome this limitation by eliminating concerns related to immunogenicity.

Title: First-Line Gene Therapies for Inherited Blindness Session Title: Innovation Series Company Presentations
Session Date: January 22, 2025
Session Time: 4:00 p.m. C.T.
Presenter: Sulagna Bhattacharya, CEO

During her talk, Ms. Bhattacharya will provide a corporate update on Nanoscope's recent and upcoming progress across pipeline programs.  She will specifically cover Nanoscope's lead asset MCO-010, which is slated for BLA submission, as well as the advancement of Nanoscope's Stargardt program to a registrational Phase 3 trial. 

Title: Ensuring Flexibility and Scalability in AAV Gene Therapy Manufacturing Session Title: Bioprocess Workflows & Analytics for Flexibility and Scalability
Session Date: January 22, 2025
Session Time: 5:00 p.m. C.T.
Presenter: Victor Adeniyi, Senior Director, CMC

During his presentation, Adeniyi will provide an overview of the landscape of analytical and scalability challenges in gene therapy, and discuss the value of designing and incorporating flexibility to address these challenges over time.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure currently exists. Following positive end-of-study results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), the company announced its plan to initiate a BLA submission for MCO-010 to treat RP in Q1 2025. The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and plans to initiate a Phase 3 registrational trial in Q1 2025. MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical programs include an IND-ready non-viral laser-delivered MCO-020 asset for GA, as well as an AAV asset for Leber Congenital Amaurosis, in IND-enabling studies.

Investor Contact:
Argot Partners
212-600-1902
PR@nanostherapeutics.com

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SOURCE Nanoscope Therapeutics