Nanoscope Therapeutics is at the forefront of advancing gene therapy through the use of light-sensitive molecules and light-assisted gene delivery. The company is committed to providing sight to millions of blind individuals afflicted with retinal degenerative diseases for which there is currently no cure. Nanoscope focuses on developing proprietary ambient light-activated optogenetic therapy to restore vision in people suffering from various inherited retinal degenerative diseases, including Retinitis Pigmentosa, Stargardt, and dry AMD. In genetic retinal degenerative diseases, vision loss occurs as cells of the retina gradually become dysfunctional. Nanoscope's gene therapies aim to address this condition by delivering light-sensitive molecules known as Multi-Characteristic Opsins (MCO) into retinal cells.

Tags

Eye Diseases

Congenital Disorders

Other Diseases

AAV based gene therapy

Disease domain score

A glimpse into the focused therapeutic areas

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Technology Platform

Most used technologies in drug development

Targets

Most frequently developed targets

Top 5 Drug Type	Count

AAV based gene therapy	3

Top 5 Target	Count

Opsin receptors	1
RP4(Rhodopsin)	1

Drugs associated with Nanoscope Therapeutics, Inc.

Sonpiretigene Isteparvovec

Target

Opsin receptors

Mechanism

Opsin modulators

Active Org.

Nanoscope Therapeutics, Inc.Startup

Originator Org.

Nanoscope Therapeutics, Inc.Startup

Active Indication

Stargardt Disease [+2]

Inactive Indication-

Drug Highest PhasePhase 2

First Approval Ctry. / Loc.-

First Approval Date-

MCO-020

Target

RP4

Mechanism

RHO modulators

Active Org.

Nanoscope Therapeutics, Inc.Startup

Originator Org.

Nanoscope Therapeutics, Inc.Startup

Active Indication

Geographic Atrophy [+1]

Inactive Indication-

Drug Highest PhasePreclinical

First Approval Ctry. / Loc.-

First Approval Date-

MCO-030

Target-

Mechanism

Gene transference

Active Org.

Nanoscope Therapeutics, Inc.Startup

Originator Org.

Nanoscope Therapeutics, Inc.Startup

Active Indication

Wet age-related macular degeneration

Inactive Indication-

Drug Highest PhasePreclinical

First Approval Ctry. / Loc.-

First Approval Date-

Clinical Trials associated with Nanoscope Therapeutics, Inc.

NCT06805474

/ Not yet recruitingNot Applicable

A Prospective Observational Study to Assess the Reliability and Validity of the Multi-Luminance Shape Discrimination Test (MLSDT) With 9 Objects

The goal of this observational study is to assess the reliability and validity of the 9-object MLSDT for evaluation of participants with moderate to severe vision impairment when tested without a wearable low-vision magnifying aid (eGlasses) and then with eGlasses. These results will be compared to ETDRS testing results for the same participants without eGlasses and then with eGlasses. Two cohorts will consist of participants who have vision loss due to STGD or geographic atrophy (GA) due to age-related macular degenerations (AMD). Normally sighted participants will provide a control group.

Start Date06 Feb 2025

Sponsor / Collaborator

Nanoscope Therapeutics, Inc.Startup

NCT06162585

/ Enrolling by invitationNot Applicable

Long Term Follow-up for Subjects Who Previously Participated in the NTXMCO-002 RESTORE Study

This study will be conducted following Good Clinical Practice (GCP) and International Conference on Harmonization (ICH) guidelines. Eligible subjects will be consented to return for scheduled study visits for this study following their completion in study NTXMCO-002 (RESTORE). They will not receive a second treatment with MCO-010 (or a repeated sham injection) in this study

Start Date08 Dec 2023

Sponsor / Collaborator

Nanoscope Therapeutics, Inc.Startup

NCT06048185

/ Enrolling by invitationNot Applicable

Long Term Follow-up for Participants Who Previously Received a Single Intravitreal Injection of MCO-010 Optogenetic Therapy in the STARLIGHT Study

The current study is a non-interventional long-term safety follow-up of the subjects who completed STARLIGHT, in accordance with FDA guidance on recipients of human gene therapy products.

Start Date19 Oct 2023

Sponsor / Collaborator

Nanoscope Therapeutics, Inc.Startup

100 Clinical Results associated with Nanoscope Therapeutics, Inc.

0 Patents (Medical) associated with Nanoscope Therapeutics, Inc.

Literatures (Medical) associated with Nanoscope Therapeutics, Inc.

01 Dec 2024·JOURNAL OF OCULAR PHARMACOLOGY AND THERAPEUTICS

Efficacy of Intravitreal Multi-Characteristic Opsin (MCO-010) Optogenetic Gene Therapy in a Mouse Model of Leber Congenital Amaurosis

Article

Author: Sharif, Najam A. ; Batabyal, Subrata ; Kim, Sanghoon ; Mohanty, Samarendra ; Carlson, Michael ; Dibas, Adnan

Purpose: Leber congenital amaurosis (LCA) is a sight-threatening inherited retinal disorder (IRD) caused by numerous genetic mutations. Multi-characteristic opsin (MCO)-based optogenetic therapy allows the recruitment of residual cells of the retina in LCA for alternative vision transduction while being mutation-agnostic. Using rd12 mice, we investigated the in vivo efficacy of an adeno-associated virus2 (AAV2)-transduced ambient light-activatable MCO (MCO-010) containing a metabotropic glutamate receptor-6 bipolar cell-specific promoter/enhancer. Methods: Mice requiring > 40 s to reach and board a dimly lit hidden platform in a water-maze were selected and randomly divided into 2 cohorts. These mice were intravitreally (IVT) injected with either 1.7E9 gene copies/eye of MCO-010 or control AAV2 and re-tested in the water-maze. Spectral-domain optical coherence tomography (SD-OCT), hematoxylin and eosin staining of retinas, and electroretinographic (ERG) studies were also conducted. Results: Safety of MCO-010 in rd12 mice was confirmed by the lack of significant detrimental changes in the mouse behavior, b-wave amplitudes and in retinal thickness. rd12 control mice performed relatively poorly in the water-maze test requiring ≥ 30-60 s to find and board the platform. MCO-010-treated rd12 mice reached the platform much faster than the AAV2-treated rd12 mice, with some mice only requiring < 5 s to achieve this goal (P < 0.01-0.0024). Conclusions: IVT MCO-010 treatment was well tolerated by rd12 mice, and it prevented the decrease in retinal thickness, and preserved ERG parameters. It also significantly improved the vision in rd12 mice relative to control AAV2-injected mice. MCO-010 therefore represents a novel and efficacious optogenetic therapeutic to treat LCA and other IRDs irrespective of the genetic defect(s).

16 Oct 2024·Translational Vision Science & Technology

Multi-Characteristic Opsin Therapy to Functionalize Retina, Attenuate Retinal Degeneration, and Restore Vision in Mouse Models of Retinitis Pigmentosa

Article

Author: Mohanty, Samarendra ; Sharif, Najam A. ; Dibas, Adnan ; Narcisse, Darryl ; Kim, Sanghoon ; Tchedre, Kissaou ; Batabyal, Subrata ; Carlson, Michael

Purpose:

Retinal degeneration 1 and 10 (rd1 and rd10) mice are useful animal models of retinitis pigmentosa (RP) with rapidly and slowly progressive pathologies, respectively. Our study aims were to determine the effect of adeno-associated viral vector 2 (AAV2)-delivered multi-characteristic opsin (MCO-010; under the control of a metabotropic glutamate receptor-6 promoter enhancer) on the morphological and functional characteristics of vision in both rd1 and rd10 mice.

Methods:

Various retinal measures of MCO-010 transduction and electrophysiological, behavioral, and other routine blood analyses were performed in the rd1 and/or rd10 mice after intravitreal injection of 1 µL of MCO-010 or AAV2 vehicle. Functional tests included electroretinogram, visually evoked potential, and behavior assay (optomotor and water maze). Retinal thickness, intraocular pressure, and plasma cytokine levels were also determined.

Results:

Following intravitreal MCO-010 injection, approximately 80% of bipolar cells were transduced in the retina, and no alterations in retinal thickness were observed at 4 months post-injection. However, retinal thickness significantly decreased in control mice. MCO-010 treatment increased head movements and induced faster navigation of mice to the platform in a water-maze test. The MCO-010 gene therapy helped preserve visually evoked electrical response in the retina and visual cortex. No ocular toxicity, immunotoxicity, or phototoxicity was observed in the MCO-010-treated mice, even under chronic intense light conditions.

Conclusions:

Intravitreal MCO-010 was well tolerated in rd1 and rd10 mice models of RP, and it appeared to attenuate retinal photoreceptor degeneration based on retinal structure and functional outcome measures.

Translational Relevance:

As reported here, optogenetic treatment of the inner retina attenuates further retinal degeneration in addition to photosensitizing higher order neurons, and this disease-modifying aspect should be evaluated in optogenetic clinical trials.

01 Aug 2023·Ophthalmology. Retina

Prevalence of Macular Atrophy in the MARINA Study of Ranibizumab versus Sham for Neovascular Age-Related Macular Degeneration.

Article

Author: Osborne, Aaron ; Gune, Shamika ; Corkery, Ellie ; Blotner, Steve ; Domalpally, Amitha ; Blodi, Barbara A ; Grzeschik, Susanna M

OBJECTIVE:

Determine prevalence, progression rates, and associations of newly detectable macular atrophy (MA) in patients with choroidal neovascularization (CNV) secondary to neovascular age-related macular degeneration (nAMD) with/without ranibizumab treatment.

DESIGN:

Post hoc analysis of MA in patients with occult/minimally classic nAMD who received monthly intravitreal ranibizumab (0.3 or 0.5 mg) or sham injections for 24 months (M) in MARINA, a phase III trial in treatment-naive patients (NCT00056836).

PARTICIPANTS:

Seven hundred six patients with nAMD: ranibizumab 0.3 mg, n = 236; 0.5 mg, n = 237; sham, n = 233.

METHODS:

Macular atrophy, assessed by color fundus photographs/fluorescein angiography, was classified as "within," "adjacent," or "nonadjacent" to the original CNV lesion. Factors associated with MA were assessed by multivariate logistic regression.

MAIN OUTCOME MEASURES:

Prevalence/incidence of newly detectable MA over time, association with CNV area, MA progression rate, association of MA with visual acuity (VA), changes in CNV/leakage area, and factors predictive of new MA at 24M.

RESULTS:

At 24M, new MA was detected in 36.8%, 40.4%, and 21.0% of eyes for ranibizumab 0.3 mg, 0.5 mg, and sham, respectively, most frequently within the area of the baseline CNV lesion (93.2%, 85.0%, and 69.0%). Rate of MA progression was similar across arms (∼ 0.3 to 0.4 mm/year). There was strong association between absence of fibrosis and detectable MA (odds ratio, 2.7; 95% confidence interval [CI], 1.29-5.56), whereas an association was not identified between detectable MA and baseline VA, baseline fellow eye atrophy, ranibizumab treatment, or change in leakage/CNV area at 24M. Ranibizumab-treated eyes gained VA with (0.3 mg: 5.3 letters [95% CI, -3.3, 13.8]; 0.5 mg: 9.8 [4.7-15.0]) or without new MA (0.3 mg: 6.4 [4.1-8.6]; 0.5 mg: 8.0 [5.3-10.6]), whereas VA in sham-treated eyes deteriorated with/without new MA (-14.7 [-23.6, -5.8] and -14.0 [-16.9, -11.1], respectively).

CONCLUSIONS:

New MA was more frequently detected in ranibizumab-treated than sham-treated eyes. Macular atrophy progression was similar across arms. Multivariate analysis showed that absence of fibrosis was the only variable associated with increased MA. Regardless of MA presence/location at baseline or throughout the study, ranibizumab-treated eyes showed clinically significant improvements in VA, whereas VA in sham-treated eyes worsened.

FINANCIAL DISCLOSURE(S):

Proprietary or commercial disclosure may be found after the references.

News (Medical) associated with Nanoscope Therapeutics, Inc.

06 Feb 2025

·www.prnewswire.com

Nanoscope Therapeutics to Present at the BIO CEO and Investor Conference

DALLAS, Feb. 6, 2025 /PRNewswire/ -- Nanoscope Therapeutics Inc., a biotechnology company committed to restoring vision in blind patients in real-world settings by developing and commercializing novel gene therapies for retinal degenerative diseases, today announced that Sulagna Bhattacharya, Chief Executive Officer and Co-Founder of Nanoscope Therapeutics, will present at the BIO CEO & Investor Conference, taking place February 10-11, 2025, in New York, NY. Details for the presentation are as follows: Ms. Bhattacharya will provide an update on Nanoscope's clinical and corporate activities on Monday, February 10, at 4:30 PM Eastern Time in the Plymouth Room. Nanoscope leadership will be available for meetings during the conference. Attendees interested in scheduling a meeting may do so through the BIO One-on-One Partnering system: . "With our upcoming BLA submission for MCO-010, Nanoscope is on the cusp of providing meaningful sight restoration for patients suffering from retinitis pigmentosa," said Ms. Bhattacharya. "I appreciate this opportunity to highlight for attendees the benefits and applications of MCO-010 therapy as well as discuss our other clinical programs in development for indications with significant unmet need. I'm looking forward to another productive event." 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 currently 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 programs include an IND-ready non-viral laser-delivered MCO-020 asset for GA, as well as an AAV asset for Leber Congenital Amaurosis, in IND-enabling studies. Investor Contact: Argot Partners 212-600-1902 [email protected] SOURCE Nanoscope Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Orphan DrugFast TrackGene Therapy

11 Nov 2024

·www.prnewswire.com

Nanoscope Announces Presentations at the Advanced Therapies USA Congress in Philadelphia

DALLAS, Nov. 11, 2024 /PRNewswire/ -- 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] SOURCE Nanoscope Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Fast TrackGene TherapyOrphan Drug

07 Nov 2024

·www.prnewswire.com

Renowned Retinal Surgeon and Medical Entrepreneur, Sunil Gupta, MD, Joins Nanoscope Board of Directors

DALLAS, Nov. 7, 2024 /PRNewswire/ -- 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 WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Fast TrackOrphan DrugExecutive ChangeClinical Result

100 Deals associated with Nanoscope Therapeutics, Inc.

100 Translational Medicine associated with Nanoscope Therapeutics, Inc.

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Drug(Targets)	Indications	Global Highest Phase

Sonpiretigene Isteparvovec ( Opsin receptors )	Retinitis Pigmentosa More	Phase 2
MCO-020 ( RP4 )	Retinitis Pigmentosa More	Preclinical
MCO-030	Wet age-related macular degeneration More	Preclinical
NOD-800	Dystrophy, Macular More	Discontinued

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