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

Nanoscope Therapeutics Announces First Patient Dosed in Phase 2b Clinical Trial of Optogenetic Gene Monotherapy to Restore Vision in Patients with Retinitis Pigmentosa

BEDFORD, Texas, July 19, 2021 / Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degenerative diseases, today announced that the first patient had been dosed in its Phase 2b clinical trial of MCO-010, an ambient-light activatable optogenetic monotherapy to restore vision in patients with retinitis pigmentosa (RP).

“I want to congratulate the Nanoscope team, our clinical partners, and patients participating in the trial. The results from our Phase 2b trial may provide the basis for consideration by the FDA for accelerated approval of MCO-010 for the treatment of RP. We look forward to working with the FDA and other regulatory agencies so that we can bring MCO-010 to RP patients,” said Sulagna Bhattacharya, Chief Executive Officer of Nanoscope.

Nanoscope’s optogenetics therapy uses an intravitreally delivered proprietary AAV2 vector to deliver Multi-Characteristic Opsin (MCO) genes into retinal cells, where they express polychromatic opsins enabling vision in different color environments. The therapy can be administered in a medical office setting without the need for goggles or other interventions. Based on preliminary evidence from the Company’s Phase 1/2a study, MCO-010 is potentially applicable not only for RP but other degenerative diseases of the eye irrespective of gene mutations. MCO-010 has received orphan drug designations for RP and Stargardt disease from the FDA.

The Phase 2b trial (NCT04945772) will include 27 participants in a randomized, double-blinded, sham-controlled, multi-center trial in the USA enrolling patients with advanced RP. The treatment will involve a single intravitreal injection of MCO-010 or sham to confirm safety, tolerability, and efficacy in improving patients’ vision and visual function. The study is expected to complete enrollment by the end of 2021 with 12-month results available by the end of 2022.

“This is an important milestone for both Nanoscope and the field of ophthalmic research. We remain steadfast in our commitment to bringing a safe and differentiated optogenetic monotherapy to the ophthalmology community,” said Dr. Mohamed Genead, Chief Development and Chief Medical Officer of Nanoscope. “Accordingly, we are enthusiastic about our MCO-010 development program, which we believe holds significant potential for the treatment of a broad range of retinal diseases.”

A completed Phase 1/2a open-label study of MCO-010 in 11 patients with RP demonstrated that the ambient light activatable optogenetic monotherapy was well-tolerated with improved quality of life consistent with significant functional vision and visual function improvement in advanced RP patients.

“We are excited by the huge interest received from the RP patients, caregivers, and clinical community. Our team is working hard to further expand the MCO-platform for other retinal degeneration including Stargardt disease, dry age-related macular degeneration (dAMD), and others,” said Samarendra Mohanty, Ph.D., Nanoscope’s President and Chief Scientific Officer.

About RP 
In normal eyes, opsins are expressed by photoreceptor cells and intrinsically photosensitive Retinal Ganglion Cells in the retina. When activated by light, these opsins trigger the physiological process of vision. RP, linked to over 60 different gene mutations, encompasses a group of rare genetic disorders where photoreceptors degrade gradually, leading to impaired vision and eventual blindness.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing optogenetic therapies for giving sight to the millions of blind individuals suffering from retinal degenerative diseases, for which no cure exists. The company’s pipeline includes optogenetics-based retinal regeneration therapies for patients with RP, Stargardt disease, and dAMD.

Contact:
Ananta Ayyagari
pr@nanostherapeutics.com
817-857-1186

Related Links

https://nanostherapeutics.com

Nanoscope Therapeutics Announces FDA Approval of IND for Phase 2b clinical trial of Optogenetic Gene Monotherapy to Restore Vision in Patients with Retinitis Pigmentosa

PRESS RELEASE – BEDFORD, TX (June 23, 2021) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degenerative diseases, today announced that its Investigational New Drug application (IND) is now open for Phase 2b clinical trial of MCO-010, an ambient-light activatable optogenetic monotherapy to restore vision in patients with advanced retinitis pigmentosa (RP).

“Congratulations to the Nanoscope team and I am excited to be part of it. We hope our therapy will restore vision in ambient light without any external device, eliminating risk of photo-toxicity” said Sulagna Bhattacharya, CEO of Nanoscope.

The impressive clinical data presented at the American Academy of Ophthalmology 2020 annual meeting has persisted till the end of Phase1/2a study. “These are major milestones for Nanoscope with potential benefit for patients suffering from retinal degeneration,” said Board Chairman Al Guillem, PhD.

The Phase 2b trial which starts in June, will be a randomized, double-blind, sham-controlled, multi-center trial in the USA enrolling patients with advanced RP. The treatment will involve a single intravitreal injection of MCO-010 to confirm its ability to improve the patients’ vision. MCO has received orphan drug designations for RP and Stargardt disease from the FDA.

Nanoscope’s gene therapy uses an AAV2 vector to deliver the MCO genes into bipolar retinal cells, where they express polychromatic opsins enabling vision in different color environments. The therapy can be administered in an office setting. Additionally, the therapy is applicable for RP regardless of underlying gene mutations.

A Phase 1/2a open label study of MCO-010 demonstrated the optogenetic monotherapy was safe with improved quality of life consistent with functional vision and visual function in advanced RP patients.

“Our gene therapies reprogram retinal cells and make them photosensitive to restore vision. MCO delivery with proprietary viral vector has allowed MCO-transduction in patients’ retina, confirmed by sustained fluorescence reporter expression,” explained Samarendra Mohanty, PhD, Nanoscope’s President and Chief Scientific Officer.

“We are excited by the guidance received from FDA regarding improvising the characterization of our product, the primary endpoint and potency assays that will hopefully accelerate our clinical program to make the restorative drug available to RP patients,” Dr. Mohanty added.

####

About RP
In normal eyes, opsins are expressed by photoreceptor cells and intrinsically photosensitive Retinal Ganglion Cells in the retina, and when activated by light they trigger the physiological process of vision. RP, linked to over 60 different gene mutations, encompasses a group of rare genetic disorders where photoreceptors degrade gradually, leading to impaired vision and eventual blindness. 

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing optogenetic therapies for giving sight to the millions of blind individuals suffering from retinal degenerative diseases, for which no cure exists. The company’s pipeline includes optogenetics based retinal regeneration therapy for patients with RP, Stargardt disease, and dry age-related macular degeneration.
Visit our website: nanostherapeutics.com

Contact:
Charles Craig
Opus Biotech Communications
charles.s.craig@gmail.com
404-245-0591

Nanoscope’s Optogenetic Gene Therapy Restores Clinically Meaningful Vision in 11 Patients Blinded by Retinitis Pigmentosa

Press Release – Bedford TX  – June 3, 2021

• First reported clinically meaningful functional improvement by optogenetic therapy
• Significant dose-dependent improvement of visual acuity at 16 weeks, previously reported at the American Academy of Ophthalmology 2020 annual meeting, continues through 52 weeks in severe retinitis pigmentosa patients
• Vision restoration in the ambient light environment using optogenetic gene monotherapy without the need of stimulating retinal implants or explants (goggles)
• One therapy for many–The first proof of restoring vision in patients blinded by various single as well as multiple genetic defect(s) in a mutation-independent manner
• Significant improvement in multiple mobility tasks and quality of life measures
• First gene therapy demonstrating gene transduction in patients by fluorescence reporter
• The Company expects to advance the therapy by launching a late-stage Phase 2b trial this summer with gene therapy that delivers Multi-Characteristic Opsin (MCO) to retinal cells

Nanoscope Therapeutics Inc., a clinical-stage biotechnology company that is developing gene therapies for the treatment of retinal diseases, today announced that vision improvements for all evaluated advanced retinitis pigmentosa (RP) patients persisted through one year following a single intravitreal injection in a Phase 1/2a clinical study with MCO.

“We expect to begin the first randomized, placebo-controlled, double-masked Phase 2b multi-center optogenetic trial in the US this summer to further validate our gene therapy’s ability to improve clinically meaningful vision in RP patients. If successful, it will be the first-ever restorative drug for millions of RP patients worldwide,” said Nanoscope CEO Sulagna Bhattacharya.

Three patients received low dose (1.75 × 10¹¹ VG per eye) and eight received a high dose of 3.5 × 10¹¹ VG per eye. Florescence imaging of retina revealed successful gene transduction. At completion, 6 out of 7 (86%) high dose MCO-therapy subjects gained >0.3 logMAR (15 letters). Due to COVID-19, one of the high dose subjects was not evaluated after 31 weeks of treatment.

“The safety and efficacy results from the clinical study have demonstrated that the benefit-risk balance is strongly in favor of MCO for the treatment of vision loss due to RP. The patients have improved irrespective of underlying gene mutation(s) that caused the disease,” said Nanoscope CMO and Ophthalmologist Sai Chavala, MD.

All subjects had objective and subjective improvement in functional vision. The shape discrimination accuracy improved to >90% in all subjects compared to baseline. Further, the performance in two different mobility tests improved by 50% reduction in time to touch lighted panel. These test outcomes were highly correlated with improved patient reported outcomes.

“After MCO-treatment, the patients reported long-lasting improvements in outdoor light sensitivity and daily activities. We were pleasantly surprised that after eight weeks of treatment, some subjects could attend their follow-up visits during the study without the assistance of a chaperone. Some of the patients even gained the ability to read letters on a wall or even the large text in a newspaper, use a cell phone, watch television, and could even thread a needle.” said the Principal Investigator Dr. Santosh Mahapatra, Ophthalmologist and Eye Surgeon.

Nanoscope’s RP gene therapy, which has received orphan drug designation from the US Food and Drug Administration, uses a proprietary AAV2 vector to deliver the MCO genes into the retina. This mutation-independent gene therapy involves a single injection through the eye administered in a doctor’s office.

Samarendra Mohanty, Ph.D., Nanoscope’s President, Chief Scientific Officer and inventor of the technology, said, “Optogenetics is a powerful research tool, but had limited scope of clinical benefit because the opsins had a narrow band of activation, unlike natural light environment. MCO is sensitive to broadband light and activatable by ambient light, thus eliminating the risk of photo-toxicity from long-term continuous use of external intense light stimulation devices.”

About RP 
In normal eyes, opsins are expressed by photoreceptor cells and intrinsically photosensitive Retinal Ganglion Cells (ipRGCs) in the retina, and when activated by light they trigger the physiological process of vision. RP encompasses a group of rare genetic disorders in which the retina’s photoreceptor cells degrade over time, leading to impaired vision and eventual blindness. These disorders are believed to be linked to over 60 different gene mutations.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics of Bedford, TX, is developing optogenetic gene therapies using light-sensitive molecules for giving sight to the millions of blind individuals suffering from retinal degenerative diseases, for which no cure exists. Nanoscope utilizes MCO molecules to re-sensitize the degenerated retina to restore vision in an ambient light environment. The company’s pipeline includes vision restoration in patients with retinitis pigmentosa, Stargardt disease, and age-related macular degeneration.

More information about Nanoscope Therapies 

Contact:
Charles Craig
Opus Biotech Communications
charles.s.craig@gmail.com 
404-245-0591

Nanoscope Therapeutics Announces New Clinical Advisory Board Appointments

BEDFORD, Texas, April 19, 2021 /PRNewswire/ — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of inherited retinal diseases, today announced an expansion of its Clinical Advisory Board with the appointment of four new members.

Nanoscope’s lead product is an optogenetic gene therapy, vMCO-010, that delivers light-sensitive Multi-Characteristics Opsin (MCO) into retinal cells to restore vision in patients with retinitis pigmentosa (RP) and Stargardt disease. Both applications have received orphan drug designation from the FDA. If successful, the optogenetic therapy would be the first treatment aimed at correcting these conditions.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics, of Bedford, TX, is advancing gene therapy using light-sensitive molecules for giving sight to the millions of blind individuals suffering from retinal degenerative disease, for which no cure exists. Nanoscope utilizes an ambient light-sensitive MCO molecule to re-sensitize the retina toward ambient light level. The company’s pipeline includes optogenetics based retinal regeneration therapy for vision restoration in patients with RP, Stargardt disease, and dry/end-stage wet age-related macular degeneration (AMD). For more information visit: https://nanostherapeutics.com/

Charles Craig
Opus Biotech Communications
charles.s.craig@gmail.com
404-245-0591