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