DALLAS, Dec. 5, 2024 — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and geographic atrophy (GA), today announced that Ray Kaczmarek has joined the company as Chief Technology and Manufacturing Officer. Mr. Kaczmarek is a recognized expert in gene therapy manufacturing with decades of experience leading operations, technology, and commercial supply strategies at pharmaceutical and biotechnology companies.

“Ray brings a wealth of expertise in building and sustaining gene therapy manufacturing programs. His operational leadership will be crucial to developing the commercial supply of our future therapies as we advance towards potential commercialization,” said Sulagna Bhattacharya, Co-founder and Chief Executive Officer of Nanoscope.

Mr. Kaczmarek has an extensive background of executive leadership and operational expertise in the gene therapy manufacturing industry. Most recently, he served as Chief Executive Officer of Genezen, a contract development and manufacturing organization (CDMO) specializing in viral vector process development, GMP manufacturing, and analytical services. Prior to Genezen, Mr. Kaczmarek served as Vice President of GMP Operations at TriLink Biotechnologies, part of Maravai Lifesciences, a CDMO specializing in the synthesis of nucleic acids, GMP capping reagents, plasmids, and oligonucleotides. Mr. Kaczmarek also served as Vice President of Manufacturing and Supply Chain Operations at Pacira Biosciences, a leading provider of non-opioid pain management and regenerative health solutions, and, prior to that, he was President of Nitto Avecia, a CDMO specializing in end-to-end solutions for oligonucleotide programs. Mr. Kaczmarek is a graduate of Virginia Tech and a proud veteran of the United States Army.

“Nanoscope has reached a pivotal juncture in its mission to bring transformative gene therapy products to market,” said Mr. Kaczmarek. “I am excited to contribute my expertise to Nanoscope’s programs, and I look forward to leading the build-out of commercial product manufacturing to ensure Nanoscope is ready to bring these much-needed treatments to patients.”

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. Following positive end-of-study results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), the company announced its plan to initiate a BLA submission for MCO-010 to treat RP in Q1 2025. The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and plans to initiate a Phase 3 registrational trial in Q1 2025. MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
[email protected]

SOURCE Nanoscope Therapeutics

DALLAS, Dec. 2, 2024  — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases (IRDs) and geographic atrophy (GA) secondary to macular degeneration, today announced that Jordi Monés will provide a presentation of Nanoscope clinical data at the annual FLORetina conference taking place on December 5^th-8^th at the Fortezza da Basso in Florence Italy. Details for the presentation are as follows:

Title: Longitudinal Efficacy and Safety of MCO-010 Optogenetic Therapy for Vision Restoration in Patients With Severe Vision Loss Due to Retinitis Pigmentosa: 126-Week Results From a Long-Term Follow-Up Study (REMAIN)

Session Title: Retina Futura, Session 1
Session Date: December 6^th, 2024
Session Time: 12:18 pm CEST
Location: San Giovanni room, Fortezza da Basso, Florence
Presenter: Jordi Monés, MD, PhD, Director of Institut de la Màcula, and Barcelona Macula Foundation (Spain)

In addition to observed statistically significant improvements in best corrected visual acuity (BCVA) in Multi-Characteristic Opsin (MCO-010) treated retinitis pigmentosa (RP) patients of RESTORE multicenter, randomized, double-masked, sham-controlled clinical trial, Dr. Monés will present the durable efficacy and safety up to 126-week, observed in the REMAIN long term follow-up 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. Following positive end-of-study results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), the company announced its plan to initiate a BLA submission for MCO-010 to treat RP in Q1 2025. The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and plans to initiate a Phase 3 registrational trial in Q1 2025. MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for GA.

Investor Contact:
Argot Partners
212-600-1902
[email protected]

DALLAS, Nov. 11, 2024 — Nanoscope Therapeutics, Inc., a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degeneration (AMD), today announced presentations at the Advanced Therapies USA 2024 congress, being held November 12-13, 2024, in Philadelphia, PA. Details for the presentations are as follows:

Title: Democratizing gene modified cell therapy for neurodegenerative diseases
Session: Track 4: Gene Modified Cell Therapy
Session Date: Tuesday, November 12, 2024
Session Time: 1:30 PM to 1:50 PM EST 
Location: 120B, Pennsylvania Convention Center
Presenter: Samarendra Mohanty, PhD, President and Chief Scientific Officer

Panel Title: Demonstrating comparability to support regulatory approval
Session Title: Track 7: Viral Vector Manufacture
Session Date: Tuesday, November 12, 2024
Session Time: 3:30 PM to 4:10 PM EST
Location: 121B, Pennsylvania Convention Center
Panelist: Khandan Baradaran, PhD, SVP, Regulatory and Quality

Panel Title: Post Market Surveillance and Long-term follow up: How do we make sure the needs for patients are met?
Session Title: Track 9: Regulatory Affairs & Policy
Session Date: Wednesday, November 13, 2024
Session Time: 11:50 AM to 12:30 PM EST
Location: 120A, Pennsylvania Convention Center
Panelist: Samarendra Mohanty, PhD, President and Chief Scientific Officer

Panel Title: Safety in gene editing
Session Title: Track 5: Gene Therapy
Session Date: Wednesday, November 13, 2024
Session Time: 11:50 AM to 12:30 PM EST
Location: 120C, Pennsylvania Convention Center
Panelist: Khandan Baradaran, PhD, SVP, Regulatory and Quality

Session Title: Track 9: Regulatory Affairs & Policy
Session Date: Wednesday, November 13, 2024
Session Time: 3:35 PM EST
Location: 120A, Pennsylvania Convention Center
Chair: Khandan Baradaran, PhD, SVP, Regulatory and Quality

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. Following positive end-of-study results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), the company announced its plan to initiate a BLA submission for MCO-010 to treat RP in Q1 2025. The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and plans to initiate a Phase 3 registrational trial in Q1 2025. MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
[email protected]

DALLAS, Nov. 7, 2024  — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today announced that Sunil Gupta, MD, has joined the Company’s Board of Directors. Dr. Gupta is a nationally recognized retinal surgeon and a successful entrepreneur who brings deep experience in all phases of drug development and medical devices, including commercialization and manufacturing.

“Dr. Gupta possesses an extraordinary combination of medical and entrepreneurial expertise around the treatment of inherited retinal diseases and dry age-related macular degeneration. He is a renowned, patient-focused physician, as well as an effective leader in executing clinical trials and operations. Dr. Gupta has truly advanced care for retina patients across the world. He will be an invaluable member of Nanoscope’s board as we advance towards commercialization of our mutation-agnostic therapies to restore vision in millions of people blinded by retinal diseases and dry-AMD,” said Sulagna Bhattacharya, Co-Founder and Chief Executive Officer of Nanoscope.

“Nanoscope continues to advance work on its groundbreaking gene therapies for inherited retinal diseases such as retinitis pigmentosa (RP) and Stargardt, offering hope to patients for whom no cure currently exists,” said Dr. Gupta. “The company’s recent announcements highlighting the efficacy and safety results from its randomized Phase 2b trial in RP with subsequent BLA submission are revolutionary. With a Phase 3 registrational trial for Stargardt and a BLA filing in RP on the horizon, Nanoscope is closer to helping address serious unmet needs. Nanoscope has assembled a world-class team of scientists, physicians and business leaders, and I am excited to contribute.”

“I want to extend a warm welcome to Dr. Gupta and look forward to working with him. He joins us at a pivotal moment in Nanoscope’s journey,” said Glenn Sblendorio, Chairman of the Board. “With an enhanced regulatory pathway, we look forward to utilizing his strategic expertise as well as his network with the retinal community.”

Dr. Gupta is founder and clinical principal investigator at Retina Specialty Institute (RSI), one of the largest retinal research organizations in the United States serving patients in clinics across the south. He was also the founder and chief medical officer of USRetina—one of the largest associations of retina physicians in the United States specializing in drug distribution, GPO, and clinical operational processes and software development prior to its recent exit to McKesson. He founded and serves as chairman and chief medical officer of Intelligent Retinal Imaging Systems (IRIS), an FDA-cleared, cloud based, telemedicine platform for assessment of retinal eye disease in the primary care setting. Dr. Gupta’s IRIS team is working to end preventable blindness due to diabetic retinopathy and other conditions around the world. Dr. Gupta was the past chairman of the Andrews Institute Sports Medicine Center and served on the board of the Andrews Research & Education Institute. In these efforts and beyond, he bridges industry and clinical care delivery organizations to optimize patient experience and outcomes. Dr. Gupta earned his medical degree from the University of Cincinnati College of Medicine and completed his residency in Ophthalmology at the Cullen Eye Institute at Baylor College of Medicine in Houston, Texas. He completed a Fellowship in Vitreoretinal Diseases & Surgery at the University of Iowa.

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, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and recently announced plans to initiate a Phase 3 registrational trial in Q1 2025. MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both retinitis pigmentosa and Stargardt. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
(212) 600-1902
[email protected]

SOURCE Nanoscope Therapeutics

DALLAS, Oct. 31, 2024 / — Nanoscope Therapeutics, Inc., a BLA filing-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that positive 2-year results from the Phase 2b RESTORE randomized, controlled clinical trial (RCT) of its lead program, MCO-010, a mutation-agnostic gene therapy for patients with permanent and severe vision loss from advanced retinitis pigmentosa (RP) were presented by Allen C. Ho, MD, FACS, FASRS, Director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital, and Chief Medical Advisor of Nanoscope, on October 20, 2024 at the recently concluded AAO 2024 Annual Meeting.

During his presentation titled “Efficacy and Safety of MCO-010 Optogenetic Therapy for Vision Restoration in Patients With Severe Vision Loss Due to RP: Two-Year TOPLINE Results From a Phase 2b/3 Randomized, Sham-Controlled Clinical Trial (RESTORE),” Dr. Ho highlighted the measured benefit of ~0.3 LogMAR (3-line, 15-letter equivalent) mean best-corrected visual acuity (BCVA) improvement that was maintained at later time points over the course of the sham-controlled trial. Additional findings included:

• High-dose MCO-010 group had mean improvements of 0.337 ± 0.0829 and 0.539 ± 0.1032 LogMAR (vs. sham-control, p = 0.0209 and 0.0014) at weeks 52 (primary) and 76 (key secondary), respectively, and low-dose MCO-010 group had mean improvements of 0.382 ± 0.1244 and 0.374 ± 0.1332 LogMAR (vs. sham-control, p = 0.0290 and 0.0652) at the same timepoints (previously announced).
• At week 52, 7 of 18 (39%) MCO-010 dosed individuals had BCVA improvement ≥0.3 LogMAR; 10 of 18 (56%) reached this threshold at week 76.
• Longitudinal analysis shows mean BCVA improvement with statistical significance compared to sham-control at weeks 36-88 visits.
• BCVA area under the curve profiles up to weeks 52, 76, and 100 were 5 times greater and statistically significant for both MCO-010 dose groups in comparison to sham-control.
• MCO-010 was well tolerated with no serious adverse events reported in MCO-010 groups. Anterior chamber cell (44% MCO-010, 22% sham-control) and ocular hypertension (39% MCO-010, 11% sham-control) were the most commonly reported adverse events. These adverse events resolved or were controlled with topical medication.

“The durable statistically significant vision improvement achieved at multiple time points during the 2-year study is highly noteworthy. This degree of improvement has never been observed in a randomized, controlled trial of a highly heterogeneous severe vision-loss patient population,” said Dr. Ho. “These findings deliver hope to patients and ophthalmologists that a solution is close to being found. We are finally on the brink of an impactful in-office, mutation-agnostic gene therapy for people with severe vision loss.”

RESTORE is the first-ever RCT to show statistically significant visual acuity improvement in the RP patient population and a key part of the upcoming BLA submission planned for Nanoscope’s RP program. The RESTORE study design has been previously announced.

“Nanoscope is proud to be a leader in the optogenetic space, advancing a mutation and disease agnostic therapy designed for optimal performance with rapid kinetics and sensitivity across the visible light spectrum and at ambient light levels,” said Sulagna Bhattacharya, co-founder, and Chief Executive Officer of Nanoscope. “We recognize the high unmet need, and we are working diligently to bring MCO-010 to these patients with permanent and progressive vision loss from retinal degeneration. They are our North Star.”

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, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and recently announced plans to initiate a Phase 3 registrational trial in Q1 2025. MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
[email protected]

DALLAS, Oct. 21, 2024  — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for retinal degenerative diseases today announced a breakthrough discovery in Translational Vision Science & Technology (TVST, an ARVO journal) demonstrating that MCO-010 optogenetic treatment of the inner retina arrests further retinal degeneration in an animal model of retinitis pigmentosa (RP), as assessed by optical coherence tomography and immunohistochemical analysis.

Following intravitreal MCO-010 treatment (sonpiretigene isteparvovec), approximately 80% of bipolar cells were transduced in the retina, and no alterations in retinal thickness were observed, unlike the control group. The results described in the publication provide evidence that the expression of MCO-010 in the retinas of mice experiencing photoreceptor degeneration halts further loss and prevents further disorganization of the retinal cell layers.

“MCO-010 is the only broadband, fast, and highly sensitive opsin currently in clinical trials and has demonstrated significant improvement in vision in advanced RP patients. In addition to restoring vision by MCO-010 intravitreal injection, measured by behavioral and electrophysiological assays, this disease-modifying aspect of MCO-010 provides a key therapeutic advantage in optogenetic therapies by targeting bipolar cells in the inner retina,” said Dr. Vinit Mahajan, Professor and Vice Chair for Research, Department of Ophthalmology at Stanford University.

“This disease-modifying aspect of MCO-010 has also been observed in an animal model of Stargardt disease as well as the nonhuman primate model for geographic atrophy,” said Samarendra Mohanty, Ph.D., President and Chief Scientific Officer of Nanoscope. “We have preliminary evidence of stabilization of retina structure in the RP clinical trial, and we plan to conduct a long-term evaluation of this anatomical biomarker.”

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. Following positive end-of-study results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), the company announced its plan to initiate BLA submission for MCO-010 to treat RP in Q1 2025. MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and recently announced plans to initiate a Phase 3 registrational trial in Q1 2025. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
(212) 600-1902
[email protected]

SOURCE Nanoscope Therapeutics

DALLAS, Oct. 15, 2024 — Nanoscope Therapeutics, Inc., a BLA filing-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Nanoscope will present at the AAO 2024 Annual Meeting taking place October 18-21, 2024 in Chicago, Illinois, and the associated Eyecelerator @ AAO 2024 and Innovate Retina meetings both taking place on October 17^th, 2024. Details for the presentations are as follows:

Title: Efficacy and Safety of MCO-010 Optogenetic Therapy for Vision Restoration in Patients with Severe Vision Loss Due to RP: Two-Year TOPLINE Results from a Phase 2b/3 Randomized, Sham-Controlled Clinical Trial (RESTORE)
Session: Session PA048 (Retina, Vitreous Original Papers)
Session Date: Sunday, October 20^th, 2024
Session Time: 2:00PM – 3:15 PM CDT (In Person, On Demand)
Location: McCormick Place Convention Center, Room S405
Presenter: Allen Ho, MD, FACS, FASRS, Director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital, and Chief Medical Advisor of Nanoscope Therapeutics, Inc.

During his presentation, Dr. Ho will discuss end-of-study topline data from Nanoscope’s RESTORE, a randomized-controlled trial (RCT) of MCO-010 therapy in patients with severe vision loss due to retinitis pigmentosa (RP). RESTORE is the first-ever RCT to show statistically significant visual acuity improvement in this patient population and a key part of the upcoming BLA submission planned for Nanoscope’s RP program.

Session Title: Retina Showcase (Eyecelerator @ AAO 2024)
Session Date: Thursday, October 17^th, 2024
Session Time: 1:15 – 3:00 PM CDT
Location: McCormick Place Convention Center, room E450a
Presenter: Samuel Barone, MD, Chief Medical Officer

During his presentation, Dr. Barone will showcase Nanoscope’s next-generation Multi-Characteristic Opsin platform program for patients with geographic atrophy (GA) due to advanced AMD. The program utilizes MCO-020, a next-generation MCO gene therapy, and features Nanoscope’s proprietary spatially targeted, non-viral, optical gene delivery approach. Nanoscope’s GA program is currently in the clinical run-up to a Phase 1/2 trial in GA patients.

Session Title: The Future of Retinal Therapeutics (Innovate Retina)
Session Date: October 17^th, 2024
Session Time: 6:24 PM CDT
Location: InterContinental Chicago Magnificent Mile, Grand Ballroom
Panelist: Samarendra Mohanty, PhD, President and Chief Scientific Officer

Dr. Mohanty will participate in a panel covering the landscape of cutting-edge therapies in development to address retinal diseases.

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, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and recently announced plans to initiate a Phase 3 registrational trial in Q1 2025. MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
[email protected]

DALLAS, TX — Oct. 10, 2024  — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for retinal degenerative diseases today announced a productive FDA meeting for its clinical program evaluating MCO-010 for the treatment of retinitis pigmentosa (RP). Based on the regulatory feedback provided in the meeting, Nanoscope will commence with submission of a Biologics License Application (BLA) in Q1 2025.

In connection with the meeting, the FDA acknowledged Nanoscope’s proposed next steps to facilitate a BLA submission for MCO-010 for the treatment of severe vision loss due to RP. Nanoscope outlined its plans for a rolling submission of the MCO-010 BLA for the treatment of severe vision loss due to RP based on its Fast Track Designation.

Severe vision loss is often the outcome for individuals diagnosed with RP, ultimately leading to blindness. The current treatment paradigm focuses on vision rehabilitation, but patients still endure a lifetime of progressive vision impairment, thereby dramatically impacting quality of life.

“Preservation of baseline visual acuity over several years represents an important treatment effect that deviates from the expected natural history of RP,” said Allen C. Ho, MD, FACS, FASRS, Director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital, and Chief Medical Advisor of Nanoscope. “Feedback from the FDA has informed Nanoscope’s BLA submission plan, thereby presenting the potential for a viable, restorative option for patients whose vision has been lost due to the array of progressive retinal degenerations that comprise RP.”

“We are pleased with the positive interactions we have had with FDA as a result of the exceptional expertise and tireless commitment of the Nanoscope team,” said Sulagna Bhattacharya, Co-founder and Chief Executive Officer of Nanoscope. “Our shared goal is to change lives, and together, we have advanced MCO-010 to the point of BLA submission. With every step forward, we are focused on the patients who are waiting for meaningful sight restoration. Our team looks forward to continuing the important work we have begun, along with our partners, to bring this therapy to patients who have significant unmet need.”

“This productive meeting with the FDA also follows our recent End of Phase 2 meeting for our Stargardt macular degeneration program, which is advancing to a Phase 3 registrational trial,” said Samarendra Mohanty, Ph.D., President and Chief Scientific Officer of Nanoscope. “The evidence of improvements in visual acuity over 3 years across the RESTORE and REMAIN studies reinforces the strength of our commitment to bring this transformative therapy to patients.”

About Retinitis Pigmentosa
Retinitis pigmentosa, is a group of rare eye diseases that affect the retina, that presents symptoms in childhood and is a leading cause of blindness in working-age adults. It is an inherited disease, meaning it is passed on to children via faulty genes from their parents. There are more than several hundred gene mutations identified that can cause RP. In patients with RP, the light-sensing photoreceptors cells degrade leading to loss of vision. Currently, there are no approved treatments for severe vision loss due to RP. Slowing or preventing further deterioration of vision for patients with RP is a treatment goal.

About MCO-010
MCO-010 (sonpiretigene isteparvovec, suspension for intravitreal injection) is the only broadband, fast, and most sensitive opsin currently in clinical trials. Current gene therapies are aimed at treating patients with specific genetic mutations in the outer retinal cells, while ambient-light activatable MCO optogenetic monotherapy targeting abundant inner retinal neurons has the potential to restore vision loss due to advanced RP, with degenerated outer retinal cells. With bipolar cell targeting via mGluR6 promoter-enhancer, the MCO-010 expression cassette is designed for restoring high-quality vision in real-world environments. The proprietary AAV2 vector allows robust transduction of MCO-010 in bipolar cells upon intravitreal injection. The Phase 1/2 trial of MCO-010 in advanced RP patients demonstrated improvement in vision-guided mobility, shape discrimination, and visual acuity. A significant proportion of patients treated with MCO-010 in the randomized double-masked multicenter RESTORE trial exhibited improvements in best-corrected visual acuity as well as functional vision assessed by vision-guided mobility, and shape discrimination, along with a favorable safety profile.

Other Clinical Updates
The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and recently announced plans to initiate a Phase 3 registrational trial in Q1 2025.

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, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). 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
[email protected]

DALLAS, Sept. 30, 2024 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases (IRDs) and geographic atrophy secondary to macular degeneration, today announced that the company has acquired a license for the CatCh technology from Max Planck Innovation, the technology transfer organization of the Max Planck Society. Based on research findings from the Max Planck Institute for Biophysics, and the pioneering work of Prof. Ernst Bamberg in the field of optogenetics, this patented technology enhances the light sensitivity of a multi-characteristic opsin (MCO-010, a tri-protein fusion complex including CatCh) developed by Nanoscope as a gene therapy to restore vision in patients suffering from genetically caused visual impairments. Nanoscope’s groundbreaking MCO platform is the first optogenetic approach to combine multiple light-sensitive components, the sum of which produces a fusion protein that is sensitive across the visible spectrum at ambient light levels with fast kinetics. Nanoscope has already successfully completed several clinical studies on MCO-010 for the two leading inherited retinal degenerative diseases: retinitis pigmentosa (RP) and Stargardt disease. The company now plans to seek approval for the therapy and expand to broad therapeutic indications.

“We are excited to see the enhanced benefits of the MCO platform, which incorporates the CatCh technology from the Max Planck Institute for Biophysics, and offers new hope for restoring vision in those suffering from severe degenerative retinal conditions,” said Dr. Samarendra Mohanty, Co-Founder & President of Nanoscope Therapeutics. “Our agreement with Max Planck Innovation allows us to transform groundbreaking scientific discoveries into effective therapeutic solutions. With efficient viral delivery of the most complex bioengineered non-mammalian MCO-010 fusion protein to treat severe degenerative retinal conditions caused by many different genetic mutations, we are on the cusp of treating patients with high unmet needs and sustainably improving their quality of life.”

Dr. Mareike Göritz, Patent and Licensing manager at Max Planck Innovation, added: “The advanced properties of CatCh in combination with Nanoscope’s proprietary technology make this a very promising approach for gene therapy treatment of retinitis-related visual impairments. We are excited to follow the further development and would be delighted if patients were ultimately to benefit from this innovative approach.”

Blindness and visual impairments caused by genetic eye diseases such as retinitis pigmentosa, Stargardt disease, and age-related macular degeneration pose a significant medical challenge worldwide. These diseases often lead to a progressive loss of vision and have a substantial impact on the quality of life of those affected. As current treatment options are limited, innovative approaches like the MCO platform now offer new hope. By its ability to partially restore vision through gene therapy, Nanoscope’s technology platform could represent a significant breakthrough in the treatment of these serious eye diseases.

Therapeutic Application and Clinical Testing

The now-licensed CatCh technology has been integrated by Nanoscope Therapeutics as one of three subunits of the MCO-010 fusion protein, each of which contributes complementary light-activated properties, that together result in a treatment with the potential to restore vision in everyday settings. Nanoscope’s novel MCO-010 therapeutic approach has been successfully tested in multiple clinical studies for RP and Stargardt disease. Additionally, the MCO platform is already being tested and shown to be effective in non-human primates with geographic atrophies (GA) secondary to advanced age-related macular degeneration (AMD).

The further development and approval pathway for MCO-010 is currently being intensively reviewed. Nanoscope Therapeutics aims to obtain approval and is in discussions with the US Food and Drug Administration (FDA) to explore possible accelerated pathways to market. These efforts could help make the innovative therapy available to patients more quickly, who urgently need new treatment options.

The CatCh (calcium-transporting channelrhodopsin) technology was developed by Prof. Ernst Bamberg, one of the pioneers in the field of optogenetics, and his colleagues at the Max Planck Institute for Biophysics. CatCh, a mutant of Channelrhodopsin-2 with improved properties, offers significant advantages such as faster kinetics and increased blue light sensitivity.

About Nanoscope Therapeutics Inc.

Nanoscope Therapeutics is a clinical-stage biopharmaceutical company developing mutation-agnostic gene therapies to treat retinal degenerative diseases that cause vision impairment and blindness, for which no cure exists to date. The company recently announced the 100-week data from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also recently completed the Phase 2 STARLIGHT trial of MCO-010 therapy in patients with Stargardt disease (NCT05417126). MCO-010 has received FDA fast-track designations and FDA orphan drug designations for both RP and Stargardt disease. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for GA secondary to AMD.

Investor Contact:
Argot Partners
(212) 600-1902
[email protected]

SOURCE Nanoscope Therapeutics

DALLAS, Sept. 19, 2024 /PRNewswire/ — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases (IRDs) and geographic atrophy secondary to macular degeneration, today announced presentations at the 24^th Euretina congress taking place on September 19^th-22^nd in Barcelona, Spain.

Details for the presentations are as follows:

Title: Longitudinal AUC analysis of BCVA in patients treated with low- or high-dose MCO-010 mutation agnostic optogenetic therapy for retinitis pigmentosa: 100-week TOPLINE results from a Phase 2b/3 randomized, sham-controlled clinical trial (RESTORE)
Session Date: September 21, 2024
Session Time: 9:36 am CEST
Location:115, Barcelona International Convention Center
Presenter: Jordi Monés, MD, PhD, Director of Institut de la Màcula, and Barcelona Macula Foundation (Spain)

In addition to typical measures of efficacy at single time points (primary and key secondary endpoints), Dr. Monés will present the efficacy analysis for best corrected visual acuity (BCVA) in Multi-Characteristic Opsin (MCO-010) treated retinitis pigmentosa patients using the area under the curve (AUC) method that captures treatment benefit over the entire 2-year observation period in RESTORE. Prof. Jordi Monés (Spain) will also chair a satellite symposium focused on optogenetics therapeutics for IRDs and geographic atrophy. Details of the symposium are as follows.

Symposium Title: Optogenetic Therapies For Retinal Degenerative Disease – Clinical Developments And Updates
Date: September 20, 2024
Time: 9:45 am-10:45 am CEST
09:45 am CEST: Opening Remarks, Jordi Monés, MD, PhD (Spain)
09:48 am CEST: Degenerative Retinal Diseases, Arshad Khanani, MA, MD, FASRS (USA)
09:58 am CEST: Optogenetic Therapies for IRDs, Paulo Stanga, MD (UK)
10:08 am CEST: Optogenetic Therapies for Macular Degeneration, Jordi Monés, MD, PhD (Spain)
10:18 am CEST: Imaging and Efficacy Evaluations in Assessing Low-Vision Patients Treated with Optogenetics, Usha Chakravarthy, MD (UK)
10:28 CEST: Q&A, All Speakers, Moderated by Prof. Francesco Bandello, MD (Italy)
Location:118/119, Barcelona International Convention Center

The management team will be in attendance at the conference and accessible for meetings.

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, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and recently announced plans to initiate Phase 3 registrational trial. 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
[email protected]

SOURCE Nanoscope Therapeutics

DALLAS, Sept. 16, 2024  — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases, and geographic atrophy today announced presentations at the Euretina Innovation Spotlight and at Ophthalmology Futures Forum taking place on 18^th September in Barcelona, Spain. Details for the presentations are as follows:

Title: Breakthrough in Vision Restoration: Mutation-agnostic Optogenetics for Inherited Retinal Diseases and Geographic Atrophies
Session Title: Nonneovascular AMD and IRDs
Session Date: September 18, 2024
Session Time: 14:35 p.m. CEST
Location: Barcelona International Convention Center
Presenter: Samuel Barone, MD, Chief Medical Officer

In his presentation followed by a panel discussion, Dr. Barone will highlight the Multi-Characteristic Opsin (MCO) therapy pipelines of Nanoscope for the treatment of severe vision loss due to retinitis pigmentosa, Stargardt macular degeneration, and geographic atrophy.

Title: Democratizing Gene Therapy for Retinal Degenerative Diseases
Session Title: Company Presentations 3
Session Date: September 18, 2024
Session Time: 17:55 p.m. CEST
Location: Esferic Barcelona
Presenter: Samarendra Mohanty, PhD, President & Chief Scientific Officer

During his presentation, Dr. Mohanty will highlight the mutation-agnostic optogenetic gene therapy for vision restoration in inherited retinal dystrophies, and geographic atrophy. He will also participate in a panel discussion, chaired by Prof. Dominik Fischer, Professor at the University of Oxford.

Panel Title: Gene & Cell Therapy Outlook for Back of the Eye Diseases: Is this Really a Business?
Panel Date: September 18, 2024
Panel Time: 18:40 – 19:10 p.m. CEST
Location: Esferic Barcelona
Panelist: Samarendra Mohanty, PhD, President & Chief Scientific Officer

Both these events include networking opportunities with industry leaders, clinicians, innovators, regulators, and investors. The management team will be in attendance at these events and accessible for meetings.

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, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also completed the Phase 2 STARLIGHT 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
[email protected]

SOURCE Nanoscope Therapeutics

DALLAS, Sept. 12, 2024  — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for retinal degenerative diseases and age-related macular degeneration (AMD), today announced a productive End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) for its clinical program evaluating MCO-010 for the treatment of severe vision loss due to Stargardt Macular Degeneration (SMD), supporting advancement of MCO-010 to a Phase 3 registrational trial.

Key outcomes of the EOP2 meeting discussion included:

• The FDA provided clear input on the proposed Phase 3 registrational trial in patients with SMD intended to support safety and effectiveness of MCO-010. The proposed study design will consist of a single MCO-010 intravitreal dose group, randomized 1:1 to a control cohort receiving a sham injection.
• The FDA confirmed that a change in best corrected visual acuity (BCVA) that employs testing on the ETDRS Chart is appropriate for use in a primary efficacy endpoint assessing change from baseline to Week 52 in the proposed Phase 3 trial.
• The FDA provided valuable input on statistical methods for the proposed primary and secondary endpoints that will inform further engagement with FDA to align on statistical analyses.
• The FDA offered continued discussion and assistance to plan further evaluation of the multi-luminance shape discrimination test to support use as a key secondary endpoint.
• Alignment was achieved on the enrollment of legally blind patients with vision worse than 20/200, and as young as 12 years old, for the upcoming study, extending the population evaluated from that studied in Phase 2.
• The FDA agreed on the sufficiency of the current nonclinical package to support a future BLA submission.

“Patients suffering from Stargardt Macular Degeneration face a substantial disease burden with no restorative treatments presently available,” said Sulagna Bhattacharya, Co-founder and Chief Executive Officer of Nanoscope. “Following the positive results from the Phase 2 STARLIGHT clinical trial, the outcome of this meeting strengthens our conviction to advance clinical development of MCO-010 in this indication. We appreciate the FDA’s guidance and look forward to finalizing preparation for the Phase 3 trial.”

“The current paradigm for managing Stargardt disease is one of diagnosis, followed by vision rehabilitation and a lifetime sentence of declining vision to severe blindness. The advancement of Nanoscope’s groundbreaking therapy for Stargardt to a Phase 3 registrational trial moves us much closer to having a viable restorative option for patients whose vision has been lost due to this terrible disease,” said Samuel Barone, M.D., Chief Medical Officer.

The planned Phase 3 trial will be the first randomized, controlled gene therapy trial for Stargardt disease. Nanoscope’s mutation-agnostic therapeutic approach has the potential to improve vision for many others suffering from severe vision loss, including macular degeneration patients.

About Stargardt Disease
Stargardt disease, often called as juvenile macular dystrophy, is an eye disease that presents in childhood and a major cause of vision loss in children and young adults. It is an inherited disease, meaning it is passed on to children via faulty genes from their parents. In patients with Stargardt disease, the light-sensing photoreceptors cells in the macula degrade leading to loss of central vision. Currently, there are no approved treatments.

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, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for Retinitis Pigmentosa (RP) (NCT04945772). The company has also completed the STARLIGHT Phase 2 trial of MCO-010 in Stargardt disease patients (NCT05417126). MCO-010 has received FDA fast track designation and FDA orphan drug designation for both RP and Stargardt disease. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
(212) 600-1902
[email protected]

DALLAS, Sept. 9, 2024  — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases, today announced presentations at the Retina Society 57^th Annual Scientific Meeting and at Advanced Therapies Europe taking place during the 2^nd week of September in Lisbon, Portugal. Details for the presentations are as follows:

Title: BCVA Analysis of Patients Treated with Low- or High-Dose MCO-010 Mutation Agnostic Optogenetic Therapy for Retinitis Pigmentosa: 100-week TOPLINE Results from a Phase 2b/3 Randomized, Sham-Controlled Clinical Trial (RESTORE)
Session Title: Genetic Diseases, Dystrophies & Degenerations
Session Date: September 11, 2024
Session Time: 4:25 p.m. WEST
Location: Pedro Leitao Ballroom, Four Seasons Hotel Ritz, Lisbon
Presenter: Anat Loewenstein, MD, MHA, Professor and Director, Division of Ophthalmol., Tel Aviv University

In her presentation, Dr. Loewenstein will highlight the end-of-study 100 week RESTORE data from mutation-agnostic Multi-Characteristic Opsin (MCO) therapy of severe vision loss due to retinitis pigmentosa.

Title: Democratizing Gene Therapy for Neurodegenerative Diseases
Session Title:  Gene Therapy: Spotlight on Developments and Breakthroughs
Session Date: September 11, 2024
Session Time: 4:15 p.m. WEST
Location: Estoril Convention Center, Lisbon
Presenter: Samarendra Mohanty, PhD, President & Chief Scientific Officer

During his presentation, Dr. Mohanty will highlight the recent developments and breakthroughs on mutation-agnostic optogenetic gene therapy for vision restoration in inherited retinal dystrophies, and image-guided spatially targeted laser gene delivery platform for treating geographic atrophy. The presentation will be followed by a panel discussion at 4:45 pm. Dr. Mohanty will also chair a session titled “Flexibility in Action: Harnessing Adaptive Vector Platforms for Gene Therapy Manufacturing” on September 12, 2024, from 1:45 – 3: 00 p.m. WEST.

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, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also completed the Phase 2 STARLIGHT 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
[email protected]

DALLAS, TX — Aug. 8, 2024  — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today announced that Sulagna Bhattacharya, Chief Executive Officer and Samarendra Mohanty, President of Nanoscope Therapeutics, will present and participate in a panel discussion at the 2024 H.C. Wainwright 4^th Annual Ophthalmology Virtual Conference, taking place on August 15, 2024.

Details for the presentation are as follows:

Session Type: Presentation
Session Date: Thursday, August 15th
Session Time: 7:00 a.m. EST
Presenters: Sulagna Bhattacharya, Chief Executive Officer, and Samarendra Mohanty, PhD, President

Ms. Bhattacharya and Dr. Mohanty will provide a corporate update on Nanoscope’s upcoming activities following positive end-of-study top-line data from the Company’s randomized clinical trial of MCO-010 in patients with retinitis pigmentosa (RP) and Phase 2 trial in Stargardt disease. The presentation will also introduce the next-generation MCO-020 laser-delivery platform for non-viral gene therapy of geographic atrophies (GA) secondary to AMD. This will be followed by a panel discussion on advanced clinical programs targeting retinal indications moderated by Yi Chen, PhD, Managing Director, Equity Research at H.C. Wainright. The details for the panel are as follows:

Session Title: Derisked late-stage candidates for retinal disorders
Session Type: Panel Discussion
Session Date: Thursday, August 15th 
Session Time: 11:00 a.m. EST,
Panelist: Samarendra Mohanty, PhD, President

The panelists will discuss the paths they have forged for their respective programs toward advanced clinical status. This will include insights on the navigation of manufacturing, regulatory, and health economics on the path towards approval and commercial launch.

To register for the live webcast, please contact your H.C. Wainwright representative. A replay of the webcast will be available through H.C. Wainwright for a limited time following the event.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal diseases, for which no cure exists. The company’s lead asset, MCO-010, recently announced the 100-week data from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also recently completed the Phase 2 STARLIGHT trial of MCO-010 therapy in patients with Stargardt disease (NCT05417126). MCO-010 has received FDA fast-track designations and FDA orphan drug designations for both retinitis pigmentosa and Stargardt disease. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for GA secondary to AMD.

Investor Contact:
Argot Partners
212-600-1902
[email protected]

DALLAS, July 10, 2024  — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today announced that its Multi-Characteristic Opsin (MCO) gene therapy platform will be featured in a presentation by Dr. Michael Singer at the 2024 ASRS Annual Scientific Meeting taking place from July 17-July 20, 2024 at the Stockholmsmässan Convention Center in Stockholm, Sweden.

Details for the presentation are as follows:

Abstract Title: BCVA analysis of low- or high-dose MCO-010 mutation agnostic optogenetic therapy for retinitis pigmentosa: FIRST TIME 100-week TOPLINE Results from a Phase 2b/3 Randomized Sham-Controlled Clinical Trial (RESTORE)
Session Type: Late-Breaking Symposium Presentation
Session Title: Symposium 3: Hereditary Retinal Disease & Genetics
Session Date: Friday, July 19th
Session Time: 9:17am CEST
Presenter: Dr. Michael Singer

Dr. Singer will present end-of-study data from RESTORE, a randomized controlled trial of Nanoscope’s MCO optogenetic gene therapy in patients with severe vision loss related to Retinitis Pigmentosa (RP). MCO-treated subjects from RESTORE experienced clinically meaningful improvements in best-corrected visual acuity (BCVA) in a statistically significant manner. Dr. Singer will discuss the importance and magnitude of the vision restoration observed in MCO-treated patients from RESTORE in the context of the natural history of declining vision in this patient population.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal diseases, for which no cure exists. The company’s lead asset, MCO-010, recently announced the 100-week data from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also recently completed the Phase 2 STARLIGHT trial of MCO-010 therapy in patients with Stargardt disease (NCT05417126 ). MCO-010 has received FDA fast-track designations and FDA orphan drug designations for both retinitis pigmentosa and Stargardt disease. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophies secondary to AMD.

Investor Contact:
Argot Partners
212-600-1902
[email protected]

DALLAS, June 18, 2024  — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today announced that management will participate in one-on-one meetings with investors at the 2024 Leerink Partners Biopharma Private Company Connect, being held virtually on June 25-26, 2024.

About Nanoscope Therapeutics Inc.

Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal diseases, for which no cure exists. The company’s lead asset, MCO-010, recently announced the 100-week data from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also recently completed the Phase 2 STARLIGHT trial of MCO-010 therapy in patients with Stargardt disease (NCT05417126 ). MCO-010 has received FDA fast-track designations and FDA orphan drug designations for both retinitis pigmentosa and Stargardt disease. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophies secondary to AMD.

Investor Contact:
Argot Partners
212-600-1902
[email protected]

SOURCE Nanoscope Therapeutics

DALLAS, May 29, 2024 —  Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today announced that management will present at the BIO International Convention, taking place June 3-6, 2024, in San Diego, California.

Details for the presentations are as follows:

Session Date: Wednesday, June 5, 2024
Session Time: 4:30 – 4:45 p.m. PT
Location: San Diego Convention Center, Company Presentation Theater 1
Presenter: Sulagna Bhattacharya, Co-Founder and Chief Executive Officer

Ms. Bhattacharya presently serves on the board of directors of BIO. In her presentation, she will highlight Nanoscope’s Multi-Characteristic Opsin platform and give an overview of the Company’s mutation-agnostic approach. She will also detail the Company’s ongoing corporate activities. Nanoscope leadership will be available for meetings during the conference.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal diseases, for which no cure exists. The company’s lead asset, MCO-010, recently announced the 100-week data from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for RP (NCT04945772). The company has also recently completed the Phase 2 STARLIGHT trial of MCO-010 therapy in patients with Stargardt disease (NCT05417126 ). MCO-010 has received FDA fast-track designations and FDA orphan drug designations for both RP and Stargardt disease. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophies secondary to AMD.

Investor Contact:
Argot Partners
212-600-1902
[email protected]

SOURCE Nanoscope Therapeutics

DALLAS, TX (May 14, 2024) — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today announced the appointment of Khandan Baradaran, PhD, as Senior Vice President of Regulatory and Quality. Dr. Baradaran brings nearly two decades of extensive experience in manufacturing and regulatory review of gene therapy products, including regulatory Chemistry, Manufacturing, and Controls (CMC) experience.

“Khandan’s experience leading quality and compliance for rare genetic disease biopharmaceutical companies will be valuable as we continue to advance our versatile MCO platform, including MCO-010, which is driving our expanding pipeline of programs. This appointment supports the continued advancement of our mutation-agnostic therapies to restore vision in millions of people blinded by retinal diseases and highlights our continued growth as a company,” said Sulagna Bhattacharya, Co-Founder and Chief Executive Officer of Nanoscope.

“I am honored to join the highly motivated team at Nanoscope in the development of its novel approach to treating retinal degenerative diseases,” said Dr. Baradaran. “Following the impressive durable efficacy and safety results from the randomized control trial in retinitis pigmentosa (RP), I eagerly anticipate contributing to Nanoscope’s advancement as we navigate forthcoming regulatory milestones and prepare for commercial readiness.”

Dr. Baradaran was most recently Vice President of Regulatory CMC at Ultragenyx Pharmaceutical Inc., a biopharmaceutical company involved in the research and development of novel products for the treatment of rare and ultra-rare genetic diseases. Previously, Dr. Baradaran was Vice President and Head of Quality at Dimension Therapeutics, a gene therapy company focused on developing novel treatments for rare diseases. Prior to this, she held CMC and Regulatory positions at Biogen, Novartis, and Dyax. She holds a PhD in Virology from Harvard University and a BA in Molecular Biology from Wellesley College.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal diseases, for which no cure exists. The company’s lead asset, MCO-010, recently announced the 100-week data from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for RP (NCT04945772). The company has also recently completed the Phase 2 STARLIGHT trial of MCO-010 therapy in patients with Stargardt disease (NCT05417126 ). MCO-010 has received FDA fast-track designations and FDA orphan drug designations for both RP and Stargardt disease. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophies secondary to AMD.

Investor Contact:
Argot Partners
212-600-1902
[email protected]

SOURCE Nanoscope Therapeutics

DALLAS, May 7, 2024 /PRNewswire/ — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for retinal degenerative diseases, today announced that its viral and laser-delivered Multi-Characteristic Opsin (MCO) gene therapy platforms will be featured in multiple presentations at the 2024 ASGCT Annual Meeting and the Retina World Congress, being held in Baltimore, Maryland and Fort Lauderdale, Florida, respectively, in May.

Details are as follows:

2024 American Society of Gene + Cell Therapy (ASGCT) Annual Meeting

Abstract Title: Longitudinal Analysis of BCVA and Near-Field Object Recognition in Low- or High-Dose MCO-010 Mutation Agnostic Optogenetic Therapy for Retinitis Pigmentosa: 12-Month Results from a Phase 2b/3 Randomized, Sham-Controlled, Patient- and Assessor-Masked Clinical Trial (RESTORE)
Abstract/Poster Number: 917
Session Type: Poster Session
Session Title: Wednesday Posters: Gene Therapy Trials – In Vivo Modification
Session Date: May 8, 2024
Session Time: 12:00 – 7:00 p.m. ET
Presenter: Samuel Barone, MD, Chief Medical Officer

Presentation Title: Non-Viral Targeted Laser Delivery of Multi-Characteristic Opsin Genes for Treatment of Geographic Atrophy
Presentation Number: 426
Session Type: Oral Abstract Session
Session Title: Nonviral Approaches for Therapeutic Applications
Session Date: May 11, 2024
Session Time: 11:15 – 11:30 a.m. ET
Presenter: Samarendra Mohanty, PhD, President & Chief Scientific Officer

In this talk, Dr. Mohanty will highlight the advancement of near-infrared laser-delivered MCO-020 platform for the treatment of geographic atrophies (GA) secondary to age-related macular degeneration (AMD).

Retina World Congress 2024

Session Title: Gene Therapy, Pharmacotherapy and Optogenetics for IRD
Session Date: May 9, 2024
Session Time: 9:05 – 9:35 a.m. ET
Location: Fort Lauderdale Marriott Harbor Beach Hotel
Presenter: Samarendra Mohanty, PhD, Chief Scientific Officer

Dr. Mohanty will provide a corporate update on Nanoscope’s mutation-agnostic MCO-010 gene therapy for vision restoration in patients with inherited retinal degenerations (IRDs). This will be followed by a panel discussion moderated by Peter Kaiser, MD, Professor of Ophthalmology at Cole Eye Institute in Cleveland Clinic Lerner College of Medicine.

Presentation Title: Longitudinal BCVA and Safety Analysis of Mutation-Agnostic MCO-010 Optogenetic Therapy For Retinitis Pigmentosa: Patient Case From A Phase 2b Randomized, Sham-Controlled, Patient- And Assessor-Masked Clinical Trial (RESTORE)
Session Title: Gene and Cell Therapy
Session Date: May 10, 2024
Session Time: 2:21 – 2:26 p.m. ET
Location: Fort Lauderdale Marriott Harbor Beach Hotel
Presenter: Christine Kay, MD, Director of Electrophysiology, Retinal Genetics, and Clinical Trials at Vitreoretinal Associates in Gainesville, Florida, and Affiliate Assistant Professor at the University of South Florida.

Dr. Kay will detail the statistically significant and clinically meaningful improvements in visual acuity observed longitudinally for an individual with advanced retinitis pigmentosa (RP) treated with MCO-010 in the RESTORE trial.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal diseases, for which no cure exists. The company’s lead asset, MCO-010, recently announced the 100-week data from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for RP (NCT04945772). The company has also recently completed the Phase 2 STARLIGHT trial of MCO-010 therapy in patients with Stargardt disease (NCT05417126). MCO-010 has received FDA fast-track designations and FDA orphan drug designations for both RP and Stargardt disease. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for GA due to AMD.

Investor Contact:
Argot Partners
212-600-1902
[email protected]

SOURCE Nanoscope Therapeutics

DALLAS, May 2, 2024  — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for retinal degenerative diseases, today announced that its Multi-Characteristic Opsin (MCO) gene therapy platform will be featured in six presentations at the ARVO Annual Meeting, taking place May 5-9, 2024 in Seattle. The management team will be present at the conference and available to meet at the Nanoscope Therapeutics booth #4620.

Details for the presentations are as follows:

Presentation Title: Longitudinal BCVA analysis of low- or high-dose MCO-010 mutation agnostic optogenetic therapy for retinitis pigmentosa: 12-month results from a Phase 2b/3 randomized, sham-controlled, patient- and assessor-masked clinical trial (RESTORE)
Presentation Number: 2137
Presentation Type: Paper Session
Session Title: Retinitis Pigmentosa
Session Date: May 6, 2024
Session Time: 4:15 – 4:30 p.m. PT
Location: 612 – Seattle Convention Center – Arch Building
Presenter: Allen C. Ho, MD, Chief Medical Advisor

Abstract Title: Test-retest Agreement of the Freiburg Visual Acuity Test in Ultra-low Vision
Abstract/Poster Number: A0223
Session Type: Poster Session
Session Title: Low Vision Performance Assessment
Session Date: May 7, 2024
Session Time: 1:15 – 3:00 p.m. PT
Location: Exhibit Hall – Seattle Convention Center
Presenter: Samuel Barone, MD, Chief Medical Officer

Abstract Title: Longitudinal BCVA Analysis of Patients with Stargardt Disease and Macular Degeneration Treated With MCO-010, a Mutation-Agnostic Optogenetic Therapy: 48-Week Results From a Phase 2a Clinical Trial (STARLIGHT)
Abstract/Poster Number: B0094
Session Type: Poster Session
Session Title: Pediatric Ophthalmology and Inherited Eye Diseases
Session Date: May 8, 2024
Session Time: 2:15 – 4:00 p.m. PT
Location: Exhibit Hall – Seattle Convention Center
Presenter: Vinit B. Mahajan, MD, PhD, Professor of Ophthalmology at Stanford University

Abstract Title: Multi-characteristic opsin gene therapy attenuates retinal degeneration and restores vision in mouse models of retinitis pigmentosa
Abstract/Poster Number: B0293
Session Type: Poster Session
Session Title: Gene and Cell Therapies and Other Novel Therapeutics I
Session Date: May 8, 2024
Session Time: 2:15 – 4:00 p.m. PT
Location: Exhibit Hall – Seattle Convention Center
Presenter: Subrata Batabyal, PhD, Director, Non-Clinical Development

Abstract Title: Characterization of a Broadband Ambient Light Activatable Multi-Characteristic Opsin Supporting its Superior Performance in Restoring Vision
Abstract/Poster Number: B0924
Session Type: Poster Session
Session Title: Gene and Cell Therapies and Other Novel Therapeutics II
Session Date: May 9, 2024
Session Time: 8:00 – 9:45 a.m. PT
Location: Exhibit Hall – Seattle Convention Center
Presenter: Samarendra Mohanty, PhD, President and Chief Scientific Officer

Abstract Title: In Vivo Efficacy of a Novel Multi-Characteristic Opsin (MCO-010) in a Mouse Model of Leber Congenital Amaurosis
Abstract/Poster Number: B0942
Session Type: Poster Session
Session Title: Gene and Cell Therapies and Other Novel Therapeutics II
Session Date: May 9, 2024
Session Time: 8:00 – 9:45 a.m. PT
Location: Exhibit Hall – Seattle Convention Center
Presenter: Najam Sharif, PhD, DSc, Vice President of Global Research and Development

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal diseases, for which no cure exists. The company’s lead asset, MCO-010, recently announced the 100-week data from RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also recently completed the Phase 2 STARLIGHT trial of MCO-010 therapy in patients with Stargardt disease (NCT05417126). MCO-010 has received FDA fast-track designations and FDA orphan drug designations for both RP and Stargardt disease. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophies secondary to age-related macular degeneration.

Investor Contact:
Argot Partners
212-600-1902
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