DALLAS, Feb. 7, 2022  — Nanoscope Therapeutics, a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced full enrollment of its Phase 2b clinical trial of MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic monotherapy to restore vision in patients with retinitis pigmentosa (RP). Topline results are expected in Q1 of next year.

“Completing enrollment for this trial is another important milestone for Nanoscope and millions suffering from retinal diseases for which there are no effective treatments,” said Sulagna Bhattacharya, CEO of Nanoscope. “Positive results from this trial will bring us closer to meeting this significant unmet medical need.”

A Completed Phase 1/2a study of MCO-010 in 11 patients with RP demonstrated that the optogenetic therapy was well-tolerated, with improved quality of life consistent with significant functional vision improvement in advanced RP patients.

The Phase 2b trial (NCT04945772) enrolled 27 participants with severe vision loss due to advanced RP in a randomized, double-masked, sham-controlled, multi-center trial. In this study, patients receive either an injection of MCO-010 or a sham injection to evaluate the efficacy and safety of MCO-010 therapy.

“RP is a progressive inherited condition caused by a spectrum of different gene mutations. This optogenetic therapy has the potential to impact patients with various genetic types of RP,” said Byron Lam, MD, Neuro-Ophthalmology Professor at Bascom Palmer Eye Institute and leading expert on RP.

Nanoscope’s optogenetic therapy uses a proprietary AAV2 vector to deliver MCO genes into retinal cells enabling vision in natural environments. The therapy is administered as a single eye injection for in-office delivery without the need for any other devices or interventions. Based on evidence from the Phase 1/2a study, MCO-010 is potentially applicable for vision restoration in retinal degenerative diseases irrespective of gene mutations. MCO-010 has received orphan drug designations for RP and Stargardt disease from the FDA. A Phase-2 study in Stargardt macular degeneration is also being initiated.

About RP 
RP, linked to over 100 different gene mutations, encompasses a group of rare disorders where photoreceptors degrade, leading to blindness in approximately 2 million individuals.

About Nanoscope Therapeutics
Nanoscope Therapeutics is developing optogenetic therapies to restore sight in millions of blind individuals suffering from retinal diseases, for which no cure exists. The pipeline includes optogenetic therapies for RP, Stargardt, and dry-AMD.

Contact:
Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com
425-306-8716

BEDFORD, Texas, Feb. 1, 2022  — Nanoscope Therapeutics Inc, a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced an expansion of its Clinical and Scientific Advisory Board with the appointment of five new members.

“We are honored to have such distinguished experts in retinal disorders and gene therapy join us as advisers. The fact that top key opinion leaders are joining us speaks to how innovative our therapies are and how impactful they will be on patients’ lives,” said Nanoscope President and CSO, Samarendra Mohanty, Ph.D. “Their guidance will be invaluable as we advance our clinical development of gene therapies to restore vision in patients blinded by retinal diseases.”

New members include:

• David S. Boyer, M.D., Senior Partner, Retina-Vitreous Associates Medical Group in Southern California.
• Jeff Heier, M.D., Co-President and Medical Director, Ophthalmic Consultants of Boston and Assistant Professor, Ophthalmology, Tufts University School of Medicine
• Peter Kaiser, M.D., Director of the Center of Ocular Research and Evaluation, Cleveland Clinic’s Cole Eye Institute and Professor of Ophthalmology
• Stephen Kaminsky, Ph.D., Professor of Research in Genetic Medicine and Associate Director, Gene Therapy Core, Weill Cornell Medical College.
• Steven Gray, Ph.D., Associate Professor, University of Texas Southwestern Medical Center and Director of the Viral Vector Facility.

They join an existing group of distinguished advisory board members (SriniVas R. Sadda, M.D., Michael Singer, M.D., Stephen H. Tsang, M.D, Ph.D.; Vittorio Porciatti, D.Sc.; Paul Yang, M.D., Ph.D.; Thomas Yorio, Ph.D.; and Sai Chavala, M.D.), who are advising Nanoscope on inherited retinal disorders, macular degenerations, scientific affairs, and cell-gene therapies.

Nanoscope has received orphan drug designations from the U.S. Food and Drug Administration for Retinitis Pigmentosa (RP) and Stargardt disease. The company is conducting double-masked, sham-controlled, multisite Phase 2b clinical trials in the U.S. for RP and is expanding into macular degeneration with another multisite Phase 2 trial on Stargardt disease.

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 inherited retinal disorders as well as macular degenerations. Nanoscope is also advancing a proprietary non-viral laser gene delivery platform for targeted gene therapy of geographic atrophies. For more information visit: https://nanostherapeutics.com/

Contact:

Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com
425-306-8716

BEDFORD, TX (January 25, 2022) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced it received IND clearance from the FDA to begin a Phase 2 trial of its Multi-Characteristic Opsin (MCO-010) ambient-light activatable optogenetic monotherapy to restore vision in Stargardt patients.

“This is another important milestone for Nanoscope, giving us the opportunity of conducting a second clinical trial in the U.S. of our proprietary MCO gene therapy platform, designed to restore vision for patients suffering from retinal degenerative diseases,” said Sulagna Bhattacharya, CEO of Nanoscope. “Currently there are no approved treatments for these conditions.”

This Phase 2 clinical trial for Stargardt is expected to begin in H1-2022. Nanoscope is currently conducting a Phase 2b multicenter, randomized, sham-controlled and double-masked study of MCO-010 for RP patients. MCO-010 has received orphan drug designations from the FDA for RP and Stargardt. Stargardt is a macular degeneration (MD) affecting children and adults. Progress on Nanoscope’s Stargardt program trial has implications for MCO as a platform and its impact beyond rare diseases.

“Presently all existing trials attempt to slow down the progression of vision loss in patients with Stargardt disease, Optogenetic approach is to restore vision. This can be a groundbreaking attempt to evaluate optogenetic gene therapy to improve vision in Stargardt patients. I’m excited by the potential MCO-010 has to restore vision for many patients with sight loss caused by outer retinal dystrophies including dry age-related macular degeneration”, said David S. Boyer, M.D., Retina-Vitreous Associates Medical Group and adjunct clinical professor of ophthalmology with Keck School of Medicine at the University of Southern California.

Stargardt is an inherited retinal disease involving dysfunction/degeneration of photoreceptors. Nanoscope’s MCO-010 gene therapy reprograms healthy retinal cells to make them photosensitive. It uses proprietary AAV2 vectors to deliver the MCO genes into the cells where they express polychromatic opsins enabling vision in different color environments. The therapy, involving a single intravitreal injection given in a medical office setting, is applicable for RP and Stargardt regardless of underlying gene mutations.

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, and dry age-related MD.

Contact:

Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com
425-306-8716

BEDFORD, Texas, Jan. 19, 2022 /– Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing optogenetic gene therapies for the treatment of retinal degenerative diseases, will be featured in several presentations at BiOS, the world’s largest biomedical optics and biophotonics conference, as a part of SPIE Photonics West 2022, in San Francisco, January 22–27, 2022 at the Moscone Center.

“We are proud to be featured this year and look forward to highlighting our efforts to address retinal degenerative diseases with the goal of bringing relief to the millions of patients worldwide suffering from vision loss,” said Sulagna Bhattacharya, CEO of Nanoscope.

Nanoscope’s President and Chief Scientific Officer Samarendra Mohanty, Ph.D., is co-chair of Conference on Optogenetics and Optical Manipulation. The program will feature three presentations on Nanoscope’s technologies, including one by Dr. Mohanty titled Optogenetic Vision Restoration with Multi-Characteristic Opsins in Bipolar Cells, Jan. 23, 9:30 am, Rm 105 (Level 1 South Lobby).

In addition, Dr. Mohanty will present: Ultrafast Laser Based Transient Perforation of Cellular Membranes for In Vivo Neuromodulation, highlighting Nanoscope’s patented non-viral laser gene delivery technology. The presentation, Jan. 23, 2 pm, Rm 153 (Upper Mezzanine South), is part of the program on Frontiers in Ultrafast Optics: Biomedical, Scientific, and Industrial Applications XXII.

Nanoscope is also presenting:

• Spatially Targeted Non-Viral Optical Delivery of Opsin-Encoding Genes into Primate Retina, Jan. 22, 2 pm, Rm 105(Level 1 South Lobby), by Sanghoon Kim, Ph.D.
• Multi-characteristic opsin restores vision in Stargardt mice model, Jan. 22, 3:30 pm, Rm 105 (Level 1 South), by Subrata Batabyal, Ph.D.

Nanoscope’s lead product, MCO-010, is in multicenter, randomized, double-masked Phase 2b clinical trials in the U.S. for treatment of RP (NCT04945772). In a Phase 1/2a open label trial of MCO-010, RP patients experienced restoration of vision to a level sufficient to resume activities of daily living.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing optogenetic therapies to give sight to millions of patients suffering from retinal degenerative diseases, for which no cure exists. The company’s pipeline includes optogenetic monotherapy for patients with retinitis pigmentosa, Stargardt disease, and other IRDs as well as macular degenerations. Nanoscope is also advancing a proprietary non-viral laser gene delivery platform for targeted MCO therapy of geographic atrophies. For more information visit: https://nanostherapeutics.com/

Contact:
Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com
425-306-8716

Bedford, TX, January 12, 2022 –  Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing optogenetic gene therapies for treatment of retinal degenerative diseases, today announced that it has been named winner in the “Biotech Innovation” category at the D CEO and Dallas Innovates third annual 2022 Innovation Awards, which honors disruptors and trailblazers contributing to the North Texas Hi-Tech corridor.

Nanoscope Therapeutics team accepting the 2022 Biotech Innovation Award. From left: Anthony Togba, CFO; Sulagna Bhattacharya, Co-Founder and CEO; and Samarendra Mohanty, Co-Founder and President.

In accepting the award, Nanoscope Co-Founder and CEO Sulagna Bhattacharya stated, “A big thanks to the hard-working and talented team at Nanoscope. I am also thankful to Dallas Innovates and D CEO magazine for their support of the Biotech industry and for recognizing companies like ours, focused on finding cures for previously-untreatable diseases. This award highlights the potential of our gene therapy to address unmet medical needs for millions of patients with retinal diseases around the world”.

Nanoscope’s proprietary Multi-Characteristic Opsin (MCO) gene therapy platform is in late-stage clinical development for restoration of vision in patients blinded by retinal degenerative diseases, such as retinitis pigmentosa (RP), regardless of genetic mutations and type of retinal degenerative disease.

“Biotech Innovation requires passion and perseverance. When we started 12 years back, gene therapy held promise and hope. Now, in this exciting era of gene therapy, we are on the cusp of bringing restorative therapy for blindness caused by retinal degenerative diseases,” said Samarendra Mohanty, Co-Founder and President of Nanoscope.

The company’s lead product, MCO-010, is in a Phase 2b multicenter, randomized, double-masked, sham controlled clinical trial in the US for treatment of RP (NCT04945772), and is expanding into macular degeneration with another Phase 2 trial on Stargardt disease. In a Phase 1/2a trial of MCO-010, all 11 RP patients experienced restoration of vision to a level sufficient to resume activities of daily living. Nanoscope has received orphan drug designation from the U.S. FDA for RP and Stargardt disease.

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 retinitis pigmentosa, Stargardt disease, and other IRDs as well as macular degenerations. Nanoscope is also advancing a proprietary non-viral laser gene delivery platform for targeted MCO therapy of geographic atrophies. For more information visit: https://nanostherapeutics.com/

Contact:
Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com
425-306-8716

BEDFORD, Texas, Jan 10, 2022 — Nanoscope Therapeutics Inc, a clinical-stage biotechnology company developing optogenetic gene therapies for retinal degenerative diseases, today announced that Nanoscope has been awarded U.S. patent 11,180,537 covering its Multi-Characteristic Opsin (MCO) optogenetic gene therapy platform for restoring vision. This award bolsters Nanoscope’s existing intellectual property position on composition and methods for the MCO therapeutics in Australia (AU2017372351) and Japan (JP6779546).

“This foundational patent with protection through 2037 is another milestone for Nanoscope and our efforts to bring the hope of light to people suffering from retinal diseases by enabling them to see again and regain their quality of life,” said Sulagna Battacharya, CEO of Nanoscope.

The patent, titled “Optogenetic modulation by Multi-Characteristic Opsins for vision restoration and other applications,” from the USPTO provides broad protections for MCO, and their therapeutic applications.

Nanoscope aims to treat retinal diseases such as retinitis pigmentosa (RP) and macular degenerations with MCO gene therapy, regardless of genetic mutations. MCO therapy is used to reprogram surviving retinal cells to sense light again in real-world conditions. MCO’s unique mechanism of action restores fast, broadband, and ambient-light sensitivity, thus allowing vision restoration in different natural and recreational situations without use of external light stimulation devices such as goggles.

“This patent represents one of over half a dozen patents awarded for our innovations on optogenetic molecules, and methods of delivery/treatment. Our mission is to develop therapies that restores light sensitivity under everyday circumstances. Our proprietary technology provides a competitive advantage as we bring first-in-class therapies to market towards that mission,” said Samarendra Mohanty, MCO-platform Inventor and President of Nanoscope.

The company is conducting double-masked, sham-controlled, multisite Phase 2b clinical trials in the U.S. for RP and is expanding into macular degeneration with another Phase 2 trial on Stargardt disease. It has received orphan drug designation from the U.S. Food and Drug Administration for RP and Stargardt disease.

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 retinal disorders. Nanoscope is also advancing a proprietary non-viral laser gene delivery platform for targeted MCO therapy of geographic atrophies. For more information visit: https://nanostherapeutics.com/

Contact:
Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com
425-306-8716

Bedford, TX,  December 22, 2021 –  Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing optogenetic gene therapies for treatment of retinal degenerative diseases, has been named a finalist for the D CEO and Dallas Innovates third annual Innovation Awards, which recognize companies that contribute to making Dallas-Fort Worth an innovation hub.

“We are proud to be part of the Dallas-Fort Worth biotech community and we greatly appreciate that D CEO and Dallas Innovates recognize our team’s efforts to develop a new treatment – a first-of-its kind gene therapy – to restore vision in patients blinded by retinal diseases,” said Nanoscope Co-Founder and CEO Sulagna Battacharya.

The 2022 Innovation Awards honor companies as well as business executives, entrepreneurs, and other leaders. Nanoscope was named a finalist in the category of Innovation in Biotech. All finalists will be recognized in the January/February issue of D CEO magazine and online at DallasInnovates.com. Winners will be revealed at an exclusive awards event in January.

Nanoscope’s proprietary Multi-Characteristic Opsin (MCO) gene therapy platform is being developed for restoration of vision in patients blinded by retinal degenerative diseases, such as retinitis pigmentosa (RP), regardless of genetic mutations.

The company’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 RP (NCT04945772). Results are expected in Q4 2022. In a Phase 1/2a open label trial of MCO-010, all 11 RP patients experienced restoration of vision to a level sufficient to resume activities of daily living.

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 retinitis pigmentosa, Stargardt disease, and other IRDs as well as macular degenerations. Nanoscope is also advancing a proprietary non-viral laser gene delivery platform for targeted MCO therapy of geographic atrophies. For more information visit: https://nanostherapeutics.com/

Contact:
Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com
425-306-8716

BEDFORD, Texas, Dec. 8, 2021 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing optogenetic gene therapies for treatment of retinal degenerative diseases, announced today the company’s Multi-Characteristic Opsin (MCO) gene therapy platform will be featured in a presentation by Michael Singer, M.D., a member of Nanoscope’s Clinical Advisory Board, at Stanford University’s Innovate Retina summit on December 11, 2021

Dr. Singer is Clinical Professor of Ophthalmology at the University of Texas Health Science Center in San Antonio, Texas, and Director of Clinical Research at Medical Center Ophthalmology Associates in San Antonio. His presentation is part of a program titled “Global Innovation in Retina” organized by Stanford University School of Medicine’s Byers Eye Institute. Online registration is free.

Nanoscope’s MCO optogenetic gene therapy is in clinical development for vision restoration in people blinded by retinitis pigmentosa (RP) regardless of genetic mutations. Nanoscope is conducting a multicenter Phase 2b trial in the U.S. (NCT04945772). Results are expected in Q4 2022. In a Phase 1/2a trial, the RP patients experienced restoration of vision to a level sufficient to resume activities of daily living.

“I am very thrilled about the potential for this new therapy for patients with retinitis pigmentosa,” Dr. Singer said. “The prospect of visual restoration in these nearly blind patients is incredibly exciting.”

Dr. Singer’s presentation, titled “Nanoscope Therapeutics: Introduction and Perspectives,” begins at 8:42 am Pacific Standard Time followed by his participation in a panel discussion.

“We are pleased Dr. Singer is presenting our unique MCO platform aimed at helping millions of people worldwide who suffer from retinal diseases,” said Sulagna Battacharya, CEO of Nanoscope. “Dr. Singer has been instrumental in advising the company as its advances its clinical development programs not only in RP, but also for Stargardt disease and age-related macular degeneration.”

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 retinitis pigmentosa, Stargardt disease, and other inherited retinal disorders as well as macular degenerations. Nanoscope is also advancing a proprietary non-viral laser gene delivery platform for targeted MCO therapy of geographic atrophies. For more information visit: https://nanostherapeutics.com/

Contact:
Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com
425-306-8716

BEDFORD, Texas, Dec. 3, 2021 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing optogenetic gene therapies for treatment of retinal degenerative diseases, was awarded as the Top Retina Company at the Eyecelerator conference at the 2021 meeting of the American Academy of Ophthalmology (AAO) in New Orleans, LA.

Nanoscope won top company honors in the retina innovation category for exciting development of proprietary Multi-Characteristic Opsin (MCO) gene therapy platform for restoring vision in patients blinded by retinal degenerative diseases, such as retinitis pigmentosa (RP), regardless of genetic mutations. The technology was featured in a presentation by Samarendra Mohanty, Ph.D., Nanoscope Co-Founder and President.

“Nanoscope was recognized as an up-and-coming company with an innovative optogenetic gene therapy that may offer a different approach to treating retinal diseases,” said Allen C. Ho, MD, Attending Surgeon and Director of Retina Research Wills Eye Hospital and Eyecelerator program Co-chair.

The Eyecelerator conference is organized by AAO and the American Society of Cataract and Refractive Surgery to showcase emerging technology, groundbreaking research and inventive concepts in ophthalmology.

“We thank the organizers for recognizing our progress in development of a unique gene therapy to restore meaningful vision for the millions of people worldwide who are blinded by retinal degenerative diseases,” said Sulagna Bhattacharya, CEO of Nanoscope.

Nanoscope’s lead asset, MCO-010, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for treatment of RP (NCT04945772). In Phase 1/2a trial of MCO-010, the RP patients experienced restoration of vision to a level sufficient to independently resume everyday activities.

The company’s MCO gene therapy has a unique mechanism of action and characteristics of being fast, broadband, and ambient-light sensitive allowing vision restoration in everyday settings. MCO-010 has received orphan drug designations from the FDA for RP and Stargardt disease.

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 inherited retinal disorders as well as macular degenerations. Nanoscope is also advancing a proprietary non-viral laser gene delivery platform for targeted MCO therapy of geographic atrophies. For more information visit: https://nanostherapeutics.com/

Contact:
Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com
425-306-8716

BEDFORD, Texas (Nov. 15, 2021) — 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
pr@nanostherapeutics.com
425-306-8716

BEDFORD, Texas, Nov. 9, 2021 — 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 at the Eyecelerator on November 11, 2021, as part of the annual meeting of the American Academy of Ophthalmology (AAO) in New Orleans, LA. AAO is the world’s largest association of eye physicians and surgeons.

Dr. Mohanty will discuss Nanoscope’s proprietary Multi-Characteristic Opsin (MCO) gene therapy platform for restoring vision in patients blinded by retinal degenerative diseases, such as retinitis pigmentosa (RP), regardless of genetic mutations. MCO has a unique mechanism of action and characteristics of being fast, broadband, and ambient-light sensitive allowing vision restoration in different natural or recreational light surroundings.

“We think the key to addressing retinal degenerative diseases is to bring forth therapies that restore meaningful vision in everyday conditions”, said Sulagna Bhattacharya, CEO of Nanoscope.

Nanoscope’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 RP (NCT04945772). Results are expected in Q4 2022. In a Phase 1/2a open label trial of MCO-010, all 11 RP patients experienced restoration of vision to a level sufficient to resume activities of daily living.

Nanoscope is also developing MCO for Stargardt disease and geographic atrophy. MCO gene therapy has received orphan drug designations from the FDA for RP and Stargardt disease.

Dr. Mohanty’s presentation will be part of the Eyecelerator’s Retina Spotlight session on “Geographic Atrophy and Optogenetic Treatment Advances on the Horizon” from 11:15 am to 12:00 pm in the Ernest N. Morial Convention Center.

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 retinitis pigmentosa, Stargardt disease, and other IRDs as well as macular degenerations. Nanoscope is also advancing a proprietary non-viral laser gene delivery platform for targeted MCO therapy of geographic atrophies. For more information visit: https://nanostherapeutics.com/

Contact:
Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com
425-306-8716

Positive Data from Nanoscope’s Phase 1/2a Trial of Gene Therapy to Restore Vision in Patients Blinded by Retinitis Pigmentosa to be Featured at 2021 American Society of Retina Specialists Meeting

Two presentations will highlight positive data from the 52-week follow-up of patients who received a single intravitreal injection of Multi-Characteristic Opsin Gene Therapy

BEDFORD, Texas, (October 8, 2021)  — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degenerative diseases, today announced findings from a Phase 1/2a clinical trial of its Multi-Characteristic Opsin (MCO) gene therapy for people blinded by retinitis pigmentosa (RP) will be highlighted in two presentations during the 2021 Annual Meeting of the American Society of Retina Specialists (ASRS) October 8 – 12 at JWMarriott San Antonio, TX.

The presentations will be made by Dr. Sai Chavala, MD, a professor at TCU-UNTHSC Medical School. Dr. Chavala’s presentation titled, “Intravitreal AAV2 Optogenetic Vision Restoration in Retinal Degenerative Patients with ABCA4 Mutation,” will be on Sunday, October 10, 2:46 pm -2:50 pm, during an In the Pipeline session exploring new therapies.

Dr. Chavala will detail findings from four RP patients with ABCA4 mutation from the Phase1/2a trial who showed overall improvement in vision after 52 weeks of receiving a single intravitreal injection of MCO-010.

Dr. Chavala will discuss data from all 11 RP patients in the Phase1/2a trial in a Video on Demand, titled “Safety and Efficacy of Intravitreal Optogenetic Gene Therapy for Advanced Retinitis Pigmentosa,” as part of the ASRS Virtual Annual Meeting Program. Results showed the therapy was well-tolerated and all patients experienced improvement in vision following MCO-010 injection.

MCO-010 uses a proprietary AAV2 vector to deliver MCO genes into retinal cells where they express polychromatic opsins sensitive to ambient light for vision restoration in different color environments. The therapy focuses on disease phenotype, enabling treatment of retinal degenerative diseases regardless of genetic mutations.

Results of a randomized, double-masked Phase 2b multi-center trial (NCT04945772) of MCO-010s are expected in Q4 2022. MCO-010 has orphan drug designations from the FDA for RP and Stargardt disease, which are known to be associated with ABCA4 mutation.

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 gene therapies for patients with RP, Stargardt disease, and dry age-related macular degeneration.

Contact:
Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com
425-306-8716

Nanoscope President to Present at Company Showcase of the Ophthalmology Innovation Summit (OIS) Retina October 7, 2021, in San Antonio, TX

Nanoscope has demonstrated functional vision restoration in blind patients using MCO optogenetic gene monotherapy and is advancing proprietary non-viral laser gene delivery platform for geographic atrophies

Positive Data of Optogenetic Gene Therapy for Patients Blinded by Retinitis Pigmentosa to be presented at Retina Society’s Annual Conference

52 weeks follow-up from MCO-010 Vision Restoration in Retinal Degeneration patients to be presented

Results of the Nanoscope’s on-going Phase 2b US Trial (RESTORE) of its optogenetic gene therapy to restore vision for retinitis pigmentosa patients expected in Q4 2022

BEDFORD, Texas, Sept. 29, 2021  — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degeneration diseases, today announced findings from its clinical study of its optogenetic gene therapy for people blinded by ABCA4 mutation will be presented at The Retina Society’s 54^th Annual Scientific Meeting in Chicago September 29 – October 2, 2021.

The presentation by Dr. Sai Chavala, M.D. titled “Optogenetic Vision Restoration in ABCA4 Mutation Retinal Degeneration Patients,” is scheduled for Friday, October 1, 2021, at 11:03 am in the Grand Ballroom of the Ritz Carlton Hotel in Chicago. The Retina Society was founded in 1968 for educational and scientific purposes concerning the diagnosis, care and treatment of diseases and injuries to the retina.

Dr. Chavala, a professor at TCU-UNTHSC Medical School in Fort Worth, TX, will present findings from retinitis pigmentosa (RP) patients dosed intravitreally with Nanoscope’s Multi-Characteristic Opsin (MCO) gene therapy. The RP patients with ABCA4 gene mutation received a single intravitreal injection of the gene therapy, were followed for 52 weeks, and all patients experienced improved visual acuity and function with no serious adverse events.

Nanoscope’s optogenetics therapy uses a proprietary AAV2 vector to deliver MCO genes into retinal cells where they express polychromatic opsins sensitive to ambient light for vision restoration in different color environments. The therapy, which can be administered in a medical office, focuses on disease phenotype, enabling treatment of retinal degenerative diseases, such as RP, regardless of genetic mutations.

MCO-010 currently is in a randomized, double-masked Phase 2b multi-center trial (NCT04945772) in the US. Results are expected in Q4 2022. MCO-010 has orphan drug designations from the US Food and Drug Administration for RP and Stargardt disease, which are known to be associated with ABCA4 mutation.

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 gene therapies for patients with RP, Stargardt disease, and dry age-related macular degeneration.

Contact:
Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com
425-306-8716

Nanoscope Awarded $1.5M Phase2B SBIR Grant to Advance Ambient Light Activatable Opsin Gene Therapy to Restore Vision for AMD Patients

BEDFORD, TX (September 14, 2021)—Nanoscope researchers were awarded a $1.5 million Phase2b Small Business Research Innovation (SBIR) grant by the National Eye Institute (NEI)of the National Institutes of Health (NIH) to advance the company’s ambient light activatable optogenetic gene therapy for age-related macular degeneration(AMD).

“Winning the NIH grant via a highly competitive review process is a testament to the innovativeness and clinical significance of our first-in-class optogenetic therapy based on our Multi-Characteristic Opsin (MCO),which is a patent-protected ambient light activatable protein for restoration of vision in people with AMD,”said Sulagna Bhattacharya, CEO of Nanoscope.“Our goal is to apply our therapy to relieve the suffering of millions of people worldwide with AMD.”’

Nanoscope’s lead MCO gene therapy, MCO-010, is in a late-stage Phase 2b trial for retinitis pigmentosa (RP) in the US. MCO-010 has orphan drug designations for RP and Stargardt disease from the US Food and Drug Administration. A Phase 1/2a trial of MCO-010 showed patients blinded by RP experienced clinically meaningful vision restoration.

Nanoscope’s optogenetic gene therapy uses a proprietary AAV2 vector to deliver MCO genes into bipolar retinal cells where they express MCOs engineered to be fast and polychromatic, enabling vision in different color environments. The MCOs reprogram bipolar cells to act like photoreceptor cells damaged not only by AMD, but also the full range of inherited retinal disorders (IRDs) regardless of underlying disease-causing gene mutations. The therapy involves a single intravitreal injection and can be administered in a medical office setting.

“Our preliminary data show MCO therapy can be applied in a mutation-independent manner for specific IRDs and can serve as a retinal disease-agnostic platform therapy,” said Samarendra Mohanty, PhD, Principal Investigator of the recently awarded NIHgrant and Nanoscope’s President and Chief Scientific Officer.“ With this grant we can accelerate advancement of our MCO therapy for geographic atrophies of the macula for juveniles as well as adults.”

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 age-related macular degeneration.

Contact:
Charles Craig
Opus Biotech Communications
pr@nanostherapeutics.com
404-245-0591

Nanoscope Therapeutics Announces Presentation of Positive Results from Optogenetic Gene Therapy for Retinal Degenerative Diseases at the EURETINA 2021 Virtual Conference

Company’s lead product, an optogenetic gene therapy to restore vision for patients blinded by retinitis pigmentosa, is currently in late-stage Phase 2b trials in US

BEDFORD, TX (September 8, 2021) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degeneration diseases, today announced Dr. Santosh Mahapatra, principal investigator of the company’s clinical study on optogenetic gene therapy for vision restoration in people blinded by different gene mutations including ABCA4, will present findings from the study at the EURETINA 2021 Virtual Conference September 9 – 12. The conference is conducted by the European Society of Retina Specialists.

Dr. Mahapatra’s presentation, “52 Weeks Safety and Efficacy of Multi-Characteristic Opsin Enabled Vision Restoration in Patients with ABCA4 Mutation”, is Sunday, September 12, in Prize Papers 13 session on New Drug Treatments & Technologies.

ABCA4 mutations are associated with retinal degeneration diseases, including retinitis pigmentosa and Stargardt disease. Patients in the study received a single intravitreal injection of Nanoscope’s Multi-Characteristic Opsin (MCO) gene therapy and were followed for 52 weeks. All patients experienced improvement in visual acuity and function with no serious adverse events.

Nanoscope’s optogenetics therapy delivers MCO genes in proprietary AAV2 vectors into retinal cells leading to expression of ambient light-sensitive polychromatic opsins for vision restoration in different color environments. The therapy, which can be administered in a medical office, focuses on disease phenotype, enabling treatment of retinal diseases regardless of genetic mutations.

Nanoscope researchers also developed a Low-Vision Multi-Parameter Test (LVMPT) for assessing different attributes of functional vision. Michael Carlson’s presentation of the “Low-Vision Multi-Parameter Test for Monitoring Visual Function of Patients with Advanced Retinal Diseases” is Thursday, September 9, in the New Drug Treatment and Technology session.

The MCO-010 gene therapy and LVMPT device are being evaluated in a Phase 2b trial (NCT04945772) in the US. MCO-010 has received orphan drug designations from the US Food and Drug Administration for RP and Stargardt disease. 

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 ocular gene therapies for patients with RP, Stargardt disease, and geographic atrophies.

Contact:

Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com

425-306-8716