Nanoscope Therapeutics CEO Sulagna Bhattacharya Joins BIO Health Section Governing Board

DALLAS, TX (February 13, 2023) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Sulagna Bhattacharya, Chief Executive Officer and Co-Founder of Nanoscope Therapeutics, has been named to the Health Section Governing Board of the Biotechnology Innovation Organization (BIO) Board of Directors.

Ms. Bhattacharya was named to the BIO Board of Directors in February 2022 and currently serves on the Emerging Companies Section Governing Board, on which she will continue to serve in addition to her new role on the Health Section Governing Board. She has also been selected as a member of BIO’s Nominating Committee.

The BIO Health Section Governing Board is comprised of thought leaders who bring their expertise to major issues affecting their specific area of the biotechnology industry.

“Accelerating development of innovative therapies is a true passion of mine,” said Ms. Bhattacharya. “I look forward to working with the other biopharma leaders on the BIO Health Section Governing Board to promote policy strategies that enable this acceleration across the Biotech sector.”

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Nanoscope Therapeutics to Present at the BIO CEO & Investor Conference

DALLAS, Feb. 2, 2023 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Sulagna Bhattacharya, Chief Executive Officer and Co-Founder of Nanoscope Therapeutics, will present at the BIO CEO & Investor Conference, taking place February 6-9, 2023, in New York.

Ms. Bhattacharya will provide a company update on Nanoscope’s clinical and corporate activities on Tuesday, February 7, from 2:00-2:15 p.m. Eastern Time in the Winter Garden Room (6^th floor) at the Marriott Marquis. Nanoscope leadership will be available for meetings during the conference from Monday, February 6 to Thursday, February 9. Those interested in scheduling a meeting may do so through the BIO One-on-One Partnering system: https://www.bio.org/events/bio-ceo-investor-digital-conference/partnering.

Ms. Bhattacharya presently serves on the board of directors of BIO. She is also a member of multiple BIO sub-committees for capital formation, strategy advocating for public policy beneficial to small-cap and private biotech companies.

Nanoscope Therapeutics is developing gene-agnostic, sight restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

SOURCE Nanoscope Therapeutics

Nanoscope Therapeutics Receives Fast Track Designation by the FDA for MCO-010 for the Treatment of Stargardt Disease

DALLAS, Jan. 30, 2023 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic monotherapy to restore vision in blind patients, for the treatment of Stargardt disease to improve visual function.

“We are pleased to receive the FDA’s decision to grant Fast Track Designation for the second indication of our vision restoring therapy, and we look forward to sharing six-month data from the Phase 2 STARLIGHT clinical trial of MCO-010 therapy in Stargardt patients this quarter,” said Sulagna Bhattacharya, CEO of Nanoscope. “Receiving Fast Track Designation reinforces the urgency of our mission to find an effective solution for Stargardt patients and is an acknowledgement of MCO-010’s potential to address this unmet medical need.”

In September 2022, enrollment was completed in the Phase 2 open-label STARLIGHT clinical trial (NCT05417126) of MCO-010 in six patients with advanced vision loss due to a clinical or genetic diagnosis of Stargardt disease. In this study, all subjects received the same single intravitreal dose of 1.2E11gc/eye of MCO-010. Six-month data from the STARLIGHT trial are expected in Q1 2023.

The FDA’s Fast Track designation is intended to facilitate the development and expedite the review of novel therapies to treat serious conditions for which there is unmet medical need. With the Fast Track designation, Nanoscope Therapeutics is eligible for more frequent regulatory meetings and communications with the FDA.

In February 2022, enrollment was completed in the Phase 2b multicenter, randomized, double-masked, sham-controlled RESTORE clinical trial (NCT04945772) of MCO-010. Top line data from the RESTORE trial are also expected in Q1 2023.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation and FDA orphan drug designations for both RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

SOURCE Nanoscope Therapeutics

Nanoscope Therapeutics Announces Presentations at the Optogenetics and Optical Manipulation Conference

DALLAS, Jan. 23, 2023  — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced an oral presentation and a poster presentation at the Optogenetics and Optical Manipulation Conference as part of the SPIE Photonics West BiOS Program, taking place January 28-February 2, 2023 in San Francisco.

Details for the oral presentation are as follows:

Abstract Title: Functionalizing inner retina with multi-characteristic opsin arrested further degeneration and functional deterioration
Abstract Number: 12366-10
Session Name: 3. Clinical Applications
Session Date: Sunday, January 29, 2023
Presentation Time: 11:40 a.m.-12:00 p.m. PT
Location: Moscone Center, Room 101 (Level 1 South Lobby)
Presenter: Dr. Samarendra Mohanty, President and Chief Scientific Officer of Nanoscope Therapeutics

In addition to highlighting the benefits of MCO gene therapy, Dr. Mohanty will discuss the results of intravitreally delivered AAV-carried MCO-010 in multiple mice models of retinal degeneration, including retinitis pigmentosa and Stargardt disease, that showed arrest of further retinal degeneration as evaluated by optical coherence tomography (OCT), electroretinogram (ERG) and immuno-histochemistry (IHC).

Details for the poster presentation are as follows:

Abstract Title: OCT-guided variable spot electrophysiology platform for correlated structural and functional assessment of optogenetic intervention
Abstract Number: 12366-24
Session Name: BiOS poster session
Session Date: Sunday, January 29, 2023
Presentation Time: 5:30-7:00 p.m. PT
Location: Moscone Center, Level 2 West

Dr. Mohanty will detail the development of image-guided spatially resolved functional assessment of retina using OCT-guided variable-spot stimulated electroretinogram (vsERG). The OCT-vsERG platform is currently used to monitor diseased areas in retina of patients with Stargardt macular degeneration and to measure functional changes after optogenetic therapy.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

SOURCE Nanoscope Therapeutics

Nanoscope Therapeutics to Present at the Next Generation Ophthalmic Drug Delivery Summit

DALLAS, Jan. 19, 2023  — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Dr. Samarendra Mohanty, President and Chief Scientific Officer of Nanoscope Therapeutics, will give a presentation titled “Pioneering Laser-Targeted Optical Delivery of Therapeutics to the Eye” and participate in a panel discussion at the Next Generation Ophthalmic Drug Delivery Summit, taking place January 24-26, 2023 in San Francisco.

Details for the presentation and panel are as follows:

Pioneering Laser-Targeted Optical Delivery of Therapeutics to the Eye
Date and Time: Thursday, January 26, 2023; 11:45 a.m. PT
Location: Hyatt Centric Fisherman’s Wharf

In his presentation, Dr. Mohanty will discuss the application of laser-targeted optical delivery as a therapeutic platform, and he will detail Nanoscope’s proof-of-concept to deliver genes and other molecules.

Panel Discussion: What is the Ideal System to Test Durable Ophthalmic Therapies? Discussing Desired
Attributes for a Benchmark System
Date and Time: Thursday, January 26, 2023; 9:15 a.m. PT
Location: Hyatt Centric Fisherman’s Wharf

Nanoscope Therapeutics is developing gene-agnostic, sight restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Nanoscope’s Top Executive Team to Participate in Advanced Therapies Week

DALLAS, TX (January 11, 2023) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Dr. Samarendra Mohanty, President and Chief Scientific Officer, will give a presentation titled “Pioneering a New Wave of Gene-Agnostic Gene Therapy” in the session “The Next Wave: Cell and Gene Therapy’s Commercial Shift” at Advanced Therapies Week, taking place January 17-20, 2023 in Miami. He will also participate in the closing panel discussion.

Details for the talk and panel are as follows:

Title: Pioneering a New Wave of Gene-Agnostic Gene Therapy
Session: The Next Wave: Cell and Gene Therapy’s Commercial Shift
Date and Time: Thursday, January 19, 2023; 3:35 p.m.- 4:05 p.m. ET (talk) and 4:35 p.m.-5:00 p.m. ET (panel)

Location: Miami Beach Convention Center

Advanced Therapies Week brings together industry leaders in healthcare for knowledge sharing and relationship building to advance the next pillar of medicine.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Nanoscope Therapeutics Appoints Massoud Motamed, PhD, as VP of Quality

DALLAS, TX (January 5, 2023) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced the appointment of Massoud Motamed, PhD, as Vice President of Quality. Dr. Motamed brings nearly a decade of CGMP quality and compliance experience, including extensive experience in manufacturing and regulatory review of gene therapy products.

“Massoud’s experience leading quality and compliance in CGMP manufacturing for gene therapies will be valuable as we progress in our Chemistry, Manufacturing and Controls (CMC) capabilities. This appointment supports the continued advancement of our optogenetic therapies to restore vision in millions of people blinded by retinal diseases and highlights our evolution as a company,” said Sulagna Bhattacharya, CEO of Nanoscope.

“I am thrilled to join the highly motivated team at Nanoscope in the development of its novel approach to treating retinal degenerative disease,” said Dr. Motamed. “I look forward to helping Nanoscope navigate advanced clinical development and upcoming regulatory submissions as the Company continues to progress in its clinical trials.”

Dr. Motamed was most recently a CMC reviewer for the Gene Therapy Branch at the Center for Biologics Evaluation and Research (CBER), the center within the US Food and Drug Administration (FDA) that regulates biological products for human use under applicable federal laws. Dr. Motamed has received many awards including FDA’s Outstanding Service award from the Office of Regulatory Affairs. Previously, he served as Head of Quality for the Viral Vector Core at UT Southwestern Medical Center. Prior to this, Dr. Motamed held appointments as Head of Auditing and Compliance at Cipla, a global pharmaceutical company, as well as an FDA inspector for pharmaceutical quality and compliance. He holds a PhD in Biochemistry from UT Southwestern Medical Center, an MS in Organic Chemistry from the University of California, Berkeley and a BS in Biochemistry from Southern Methodist University.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Nanoscope Therapeutics to Present at the 41st Annual J.P. Morgan Healthcare Conference

DALLAS, Jan. 3, 2023 /PRNewswire/ — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that management will present at the 41st Annual J.P. Morgan Healthcare Conference taking place January 9-12, 2023, in San Francisco.

Chief Executive Officer, Sulagna Bhattacharya, and Chief Medical Officer, Aaron Osborne will provide a company update on Nanoscope’s clinical and corporate activities on Wednesday, January 11, during 2:00-2:25 p.m. Pacific Time at Mission Bay (32nd Floor) in the Westin St. Francis. Nanoscope leadership will be available for in-person meetings during the conference from Tuesday, January 10 to Thursday, January 12.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Nanoscope Therapeutics to Present at the Piper Sandler 34th Annual Healthcare Conference

DALLAS, TX (November 22, 2022) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that management will present at the Piper Sandler 34th Annual Healthcare Conference on Thursday, December 1, 2022, in New York.

Chief Executive Officer, Sulagna Bhattacharya, and Chief Medical Officer, Aaron Osborne will provide a company update on Nanoscope’s clinical and corporate activities on Thursday, December 1, at 2:10 PM Eastern Time in the Harlem Track of the Lotte New York Palace. Nanoscope leadership will be available for in-person meetings at the conference on Thursday, December 1.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Nanoscope Therapeutics to Host Clinical Perspectives Panel to Discuss MCO-010 and the Treatment Landscape for Retinitis Pigmentosa and Stargardt Disease

Webcast to be held on Thursday, November 17^th from 4:30-5:30 pm ET

DALLAS, TX. – November 10, 2022 – Nanoscope Therapeutics, Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, will host a clinical perspectives event on Thursday, November 17, 2022 from 4:30 – 5:30 p.m. ET. The event will focus on Nanoscope’s lead candidate, MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy, and its potential role in treating retinitis pigmentosa (RP) and Stargardt disease, two inherited retinal diseases that are leading causes of blindness in the working-age population.

Sulagna Bhattacharya, Co-Founder and CEO of Nanoscope Therapeutics, will introduce the event and provide an introduction to MCO-010. This will be followed by presentations on the treatment landscape, unmet medical need, and a Q&A panel with renowned experts in RP and Stargardt disease, including:

Stephen Tsang, M.D., Ph.D.
László Bitó Professor, Ophthalmology, Pathology and Cell Biology, Institute of Human Nutrition, and Columbia Stem Cell Initiative, Columbia University and Co-director for Stem Cells, Genome Engineering and Regenerative Medicine Course

Victor H. Gonzalez, M.D.
Founder/Chief Executive, Valley Retina Institute, McAllen, TX. Dr. Gonzalez is also affiliated with Valley Baptist Medical Center-Harlingen, South Texas Health System, Harlingen Medical Center, Mission Regional Medical Center, Rio Grande Regional Hospital, Knapp Medical Center, and Doctor’s Hospital at Renaissance.

Samar Mohanty, Ph.D., Co-Founder & President, and Aaron Osborne, MBBS, Chief Medical Officer & Chief Development Officer of Nanoscope Therapeutics will also participate in the panel discussion.

Webcast Details
A live webcast of the event will be available on the Events page of the Nanoscope Therapeutics website and may be accessed through https://nanoscope-therapeutics-conference-call.open-exchange.net/.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Nanoscope Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Nanoscope Therapeutics Expands Clinical and CMC Leadership Team with Gene Therapy Pharma Experience

Dallas, TX (Nov 7, 2022) — Nanoscope Therapeutics, Inc., a clinical-stage biotechnology company developing gene therapies to treat retinal degenerative diseases, today announced that the Company has expanded its clinical and Chemistry, Manufacturing and Controls (CMC) team with the appointments of Jean Chang, as VP of Clinical Operations and Victor Adeniyi as Senior Director of CMC.

“We are excited to welcome Jean and Victor, both experienced biopharmaceutical leaders in gene therapy, to the Nanoscope team, and we look forward to the integral roles they will play in bolstering our clinical and CMC capabilities,” said Sulagna Bhattacharya, CEO of Nanoscope. “As we make steady progress with our clinical trials, their expertise will help support the continued advancement of our optogenetic gene therapies to restore vision in millions of people blinded by retinal diseases.”

Ms. Chang brings more than 20 years of experience in clinical operations and project management resulting in successful clinical trials in various therapeutic areas including CNS, cardiorenal, hematology, oncology, and ophthalmology. Previously, she served as Executive Director of Clinical Operations at Catalyst Biosciences, and Senior Director, Clinical Operations at Adverum Biotechnologies. Ms. Chang holds a B.S. in Medical Technology from China Medical University in Taiwan and an M.S. in Immunology from Wayne State University in Michigan.

Mr. Adeniyi has a decade of experience in cGMP manufacturing of biologics, including cell and gene therapy, leading successful manufacturing of clinical trial materials. Previously, he served as Head of Manufacturing and Tech Transfer at UT Southwestern Medical Center and manufacturing expert at Novartis Pharmaceuticals. Mr. Adeniyi also brings extensive experience in cGMP manufacturing while leading teams at Lonza, and Fujifilm Diosynth Biotechnologies. He earned his M.S. in Biotechnology and M.B.A. from the University of Houston.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Charles River and Nanoscope Therapeutics Announce Multifaceted Gene Therapy Manufacturing Partnership

Charles River will manufacture both plasmid DNA and viral vectors for late phase clinical trials targeting degenerative ocular diseases with no known cure

WILMINGTON, Mass. & DALLAS, Tx. – October 18, 2022 – Charles River Laboratories International, Inc. and Nanoscope Therapeutics, Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced a comprehensive manufacturing collaboration utilizing Charles River’s extensive contract development and manufacturing (CDMO) services in both plasmid DNA and viral vectors.

“We are excited to continue to support Nanoscope Therapeutics’ efforts in the production of gene therapies that are focused on restoring vision for people suffering from retinal degenerative diseases with no known cure. Their work is incredibly important to patients, and we look forward to continuing to serve as a partner,” said Kerstin Dolph, Corporate Senior Vice President of Biologics Solutions at Charles River.

“Nanoscope looks forward to working with Charles River to support our accelerated development program. We have high expectations for our program and are counting on Charles River leadership, resources and scale to deliver in this partnership,” said Anil Lalwani, Vice President of CMC at Nanoscope Therapeutics, Inc.

A Robust Manufacturing Collaboration
Through this partnership, Nanoscope will have access to established manufacturing platforms and multiple Charles River CDMO centers of excellence, leveraging a comprehensive range of services including but not limited to GMP cell banking, High Quality (HQ) and GMP-grade plasmid DNA manufacture, and GMP adeno-associated virus (AAV) production.

This gene therapy manufacturing partnership builds on Charles River’s acquisitions of Cognate BioServices, Cobra Biologics, and Vigene Biosciences in 2021 that expanded its comprehensive cell and gene therapy (C&GT) portfolio to span each of the major CDMO platforms – cell therapy, viral vector, and plasmid DNA production.

Treating Degenerative Retinal Diseases
Nanoscope Therapeutics is developing gene-agnostic, sight restoring Multi-Characteristic Opsin (MCO) optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. Nanoscope’s optogenetic therapy uses a proprietary AAV2 vector to deliver MCO genes into retinal cells to enable vision in different color environments. The therapy is administered as a single intravitreal injection for in-office delivery without the need for any other devices or interventions.

About Charles River
Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit www.criver.com.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top line data expected H1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Nanoscope Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Charles River Investor Contact:
Todd Spencer
Corporate Vice President,
Investor Relations
781.222.6455
todd.spencer@crl.com

Charles River Media Contact:
Amy Cianciaruso
Corporate Vice President,
Chief Communications Officer
781.222.6168
amy.cianciaruso@crl.com

Nanoscope Therapeutics Receives Fast Track Designation by the FDA for MCO-010 for the Treatment of Retinitis Pigmentosa

DALLAS, TX (October 10, 2022) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic monotherapy to restore vision in blind patients, for the treatment of retinitis pigmentosa (RP) via intravitreal injection.

“The FDA’s decision to grant FTD underscores the importance of MCO-010 to address a serious unmet need and validates its potential as an effective therapeutic for patients with RP,” said Sulagna Bhattacharya, CEO of Nanoscope. “We are proud to have the support of the FDA and look forward to collaboratively interacting with FDA to assess next steps in the clinical development and future regulatory review of MCO-010.”

In January 2022, 27 RP patients with advanced vision loss were enrolled in a multicenter, randomized, double-masked, sham-controlled RESTORE clinical trial (NCT04945772) of MCO-010. Top line data from the clinical trial are expected in H1 2023.

In September of this year, enrollment was completed in the Phase 2 open-label STARLIGHT clinical trial (NCT05417126) of MCO-010 in patients with advanced vision loss due to Stargardt disease. Six-month data from the clinical trial are expected in H1 2023.

The FDA’s Fast Track designation is intended to facilitate the development and expedite the review of novel therapies to treat serious conditions for which there is unmet medical need. With the Fast Track designation, Nanoscope Therapeutics is eligible for more frequent regulatory meetings and communications with the FDA.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top line data expected H1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

SOURCE Nanoscope Therapeutics

Nanoscope Therapeutics to Present at Chardan’s 6th Annual Genetic Medicines Conference

DALLAS, TX (September 30, 2022) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that management will present at Chardan’s 6th Annual Genetic Medicines Conference on Monday, October 3, 2022, in New York.

Chief Executive Officer, Sulagna Bhattacharya, and Chief Medical Officer, Aaron Osborne will present a company update on Nanoscope clinical and corporate activities on Monday, October 3, at 5:00 PM Eastern Time in the Embassy room. Nanoscope leadership will be available for in-person meetings at the event on both Monday, October 3, and Tuesday, October 4.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top line data expected H1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

SOURCE Nanoscope Therapeutics

Nanoscope Therapeutics to Present at Eyecelerator during the American Academy of Ophthalmology (AAO) 2022 Annual Meeting

DALLAS, Sept. 26, 2022 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Dr. Samarendra Mohanty, Co-Founder and President, will give a presentation on the clinical development of Nanoscope’s Multi-Characteristic Opsin (MCO) gene therapy platform at Eyecelerator during the American Academy of Ophthalmology (AAO) 2022 Annual Meeting, taking place Thursday, September 29, 2022, in Chicago, IL.

Pioneering Optogenetic Therapeutics and Optical Gene Delivery for Vision Restoration
Date and Time: Thursday, September 29, 2022; 2:51 p.m. CT
Location: McCormick Place Convention Center

In addition to highlighting the benefits of MCO gene therapy, Dr. Mohanty will profile Nanoscope’s Phase 2b RESTORE trial from its lead clinical program as well as introduce the Company’s groundbreaking non-viral laser-delivered MCO-020 gene therapy for geographic atrophy. RESTORE trial results, along with six-month data from the Phase 2 STARLIGHT trial for Stargardt disease, are expected in H1 2023.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top line data expected H1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

SOURCE Nanoscope Therapeutics

Nanoscope Therapeutics Announces Completion of Enrollment in STARLIGHT Phase 2 Clinical Trial of MCO-010 Optogenetic Gene Therapy for Stargardt Disease

Six-month data from Phase 2 STARLIGHT trial expected in H1 2023

DALLAS, Sept. 13, 2022  — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced full enrollment of its Phase 2 clinical trial of MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic monotherapy to restore vision in blind patients, for Stargardt disease. Six-month data from the Phase 2 STARLIGHT trial are expected in H1 2023.

“We are excited by the therapeutic potential of MCO-010, supported by robust data from previous preclinical and clinical studies,” said Sulagna Bhattacharya, CEO of Nanoscope. “Completing the quick two-month enrollment of this Phase 2 trial, which will evaluate the safety and effects of a single intravitreal injection of MCO-010, brings us another major step forward in developing this novel therapy with broad therapeutic application.”

The Phase 2 STARLIGHT open-label trial (NCT05417126) enrolled six subjects with advanced vision loss due to a clinical or genetic diagnosis of Stargardt disease.  In this study, all subjects received the same single intravitreal dose of 1.2E11gc/eye of MCO-010 as used in the Company’s Phase 2b retinitis pigmentosa (RP) study.

“Stargardt disease is a blinding condition affecting adults and children alike. It is the most common macular dystrophy and has no established treatment,” said Byron Lam, MD, Robert Z. & Nancy J. Greene Chair and Professor of Ophthalmology at the University of Miami and principal investigator in the Phase 2 STARLIGHT trial. “MCO-010 has the potential to improve visual function in RP and Stargardt patients, and we look forward to seeing the data from this study.”

Nanoscope’s optogenetic therapy uses a proprietary AAV2 vector to deliver MCO genes into retinal cells to enable vision in different color environments. The therapy is administered as a single intravitreal injection for in-office delivery without the need for any other devices or interventions. MCO-010 has received orphan drug designations for RP and Stargardt disease from the FDA and is concurrently being evaluated in Nanoscope’s Phase 2b RESTORE trial in patients with RP, with trial results also expected in H1 2023.

Nanoscope Therapeutics is developing gene-agnostic, sight restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top line data expected H1 2023. The company has also initiated a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

SOURCE Nanoscope Therapeutics

Nanoscope Therapeutics to Present at the 3rd Annual Gene Therapy for Ophthalmic Disorders Meeting

DALLAS, Sept. 8, 2022  — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Dr. Samarendra Mohanty, Co-Founder and President, will give a presentation on the clinical development of Nanoscope’s Multi-Characteristic Opsin (MCO) gene therapy platform and participate in a panel titled “Gene Therapy for Ophthalmic Disorders – A Year in Review” at the 3rd Annual Gene Therapy for Ophthalmic Disorders Meeting, taking place September 13-16, 2022 in Boston, MA.

Details for the presentation are as follows:

Exploring Ambient Light Activatable Optogenetics for Vision Restoration in Retinal Degenerative Diseases
Date and Time: Thursday, September 15, 2022; 9:00-9:30 a.m. ET
Location: DoubleTree by Hilton Boston North Shore

In addition to highlighting the benefits of MCO gene therapy, Dr. Mohanty will detail Nanoscope’s Phase 2b RESTORE trial from its lead clinical program. RESTORE trial results, along with six-month data from the Phase 2 STARLIGHT trial for Stargardt disease, are expected in H1 2023. He will also participate in the panel on Wednesday, September 14, 2022; 8:25-9:05 am ET.

Two other additional technical leaders of Nanoscope, Dr. Subrata Batabyal and Dr. Sanghoon Kim, will be featured in a pre-conference workshop highlighting non-viral spatially targeted, laser gene delivery to retina (details below). Nanoscope’s MCO-020 gene therapy program is based on targeted laser-delivery of optogenes to areas of geographic atrophy in advanced AMD patients, and currently undergoing IND-enabling studies.

Workshop A: Discussing the Use of Non-Viral Gene Delivery as a Therapeutic Modality to the Eye
Date and Time: Tuesday, September 13, 2022; 8:30-11:00 am ET
Location: DoubleTree by Hilton Boston North Shore

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Nanoscope Therapeutics to Present at Baird’s 2022 Global Healthcare Conference

DALLAS, Sept. 7, 2022  — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that management will present at Baird’s 2022 Global Healthcare Conference on Wednesday, September 14, 2022, in New York.

Chief Executive Officer, Sulagna Bhattacharya, and Chief Medical Officer, Aaron Osborne will present a company update on Nanoscope clinical and corporate activities on Wednesday, September 14, at 12:15 PM Eastern Time in the Morgan Suite (Mezzanine Level). Nanoscope leadership will be available for in-person meetings at the event on both Tuesday, September 13, and Wednesday, September 14.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Nanoscope Announces Presentations at the 22^nd Euretina Congress

Results of Phase 2b RESTORE trial for retinitis pigmentosa and six-month data for Phase 2 STARLIGHT trial for Stargardt disease expected in H1 2023

DALLAS, TX (August 29, 2022) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that clinical development of its Multi-Characteristic Opsin (MCO) gene therapy platform will be featured in two presentations at the 22^nd Euretina Congress, taking place September 1-4, 2022, in Hamburg, Germany and online.

Details for the presentations are as follows:

Development of Novel Endpoints for the Study of Multi-Characteristic Opsin Enabled Vision Restoration in Patients with Advanced Retinitis Pigmentosa; Double-masked, Randomized, sham-controlled, Multicenter Phase 2b study
Presenter: Victor Hugo Gonzalez, M.D., Valley Retina Institute, McAllen, TX.
Session: IRD I
Date and Time: Thursday, September 1, 2022; 3:05 p.m. CEST
Location: Hall X1, Congress Centre Hamburg

Dr. Gonzalez will discuss Nanoscope’s Phase 2b RESTORE trial, its most advanced clinical program. RESTORE trial results, along with six-month data from the Phase 2 STARLIGHT trial for Stargardt disease, are expected in H1 2023.

52 Week Safety and Efficacy of Optogenetic Therapy for Vision Restoration in Retinitis Pigmentosa Patients
Presenter: SriniVas R. Sadda, M.D., President and Chief Scientific Officer of the Doheny Eye Institute, the Stephen J. Ryan – Arnold and Mabel Beckman Endowed Chair, and Professor of Ophthalmology at the University of California – Los Angeles (UCLA), David Geffen School of Medicine.
Session: IRD II
Date and Time: Friday, September 2, 2022; 3:29 p.m. CEST
Location: Hall X1, Congress Centre Hamburg

Dr. Sadda will discuss the safety and efficacy of intravitreal MCO Optogenetic Therapy for vision restoration in advanced retinitis pigmentosa patients in a Phase 1/2a study.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top line data expected H1 2023. The company has also initiated a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Nanoscope Therapeutics to Present at the Retina Forum 2022

DALLAS, Aug. 26, 2022 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Aaron Osborne, Chief Medical Officer, will give a presentation on clinical development of Nanoscope’s Multi-Characteristic Opsin (MCO) gene therapy platform and participate in a panel titled “Gene Therapy Platforms for Macular Disease – Are They Worth the Money?” at the Retina Forum, taking place August 31, 2022, in Hamburg, Germany and online as part of the Ophthalmology Futures Forums.

Details for the presentation are as follows:

Pioneering a New Wave of Optogenetic Therapeutics for Vision Restoration
Presenter: Aaron Osborne, MBBS, Nanoscope’s Chief Medical Officer
Date and Time: Wednesday, August 31, 2022; 4:51 p.m. CEST
Location: The Fontenay Hotel

In addition to highlighting the benefits of MCO gene therapy, Dr. Osborne will detail Nanoscope’s Phase 2b RESTORE trial from its lead clinical program. RESTORE trial results, along with six-month data from the Phase 2 STARLIGHT trial for Stargardt disease, are expected in H1 2023.

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 exists. The company’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top line data expected H1 2023. The company has also initiated a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com