RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), Regenerative Medicine Advanced Therapy (United States), Orphan Drug (Saudi Arabia), Regenerative Medicine Advanced Therapy (Japan), SAKIGAKE (Japan)

Structure/Sequence

Sequence Code 1098900043

Source: *****

Clinical Trials associated with Sonpiretigene Isteparvovec

NCT06162585

/ Active, not recruitingNot 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.

NCT06048185

/ Active, not recruitingNot 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.

NCT05921162

/ Enrolling by invitationNot Applicable

A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection

This study "A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection" is an observational study and will be conducted following Good Clinical Practice (GCP)- International Conference on Harmonization (ICH) guidelines. Eligible subjects satisfying all inclusion and none of the exclusion criteria will be enrolled. All subject who completed the parent clinical study (NSCT/CT/18/01) will undergo safety and efficacy assessments up to 5 years post study drug injection

Start Date14 Jul 2023

Sponsor / Collaborator

Nanoscope Therapeutics, Inc.

100 Clinical Results associated with Sonpiretigene Isteparvovec

100 Translational Medicine associated with Sonpiretigene Isteparvovec

100 Patents (Medical) associated with Sonpiretigene Isteparvovec

Literatures (Medical) associated with Sonpiretigene Isteparvovec

01 Sep 2025·EClinicalMedicine

Safety and efficacy of MCO-010 optogenetic therapy in patients with Stargardt disease in USA (STARLIGHT): an open-label multi-center Ph2 trial

Article

Author: Ayyagari, Ananta ; Chang, Jean ; Koester, John ; Mahajan, Vinit B ; Ho, Allen C ; Barone, Samuel B ; Sharif, Najam A ; Chavala, Sai H ; Lane, Hayley ; Gregori, Ninel Z ; Carlson, Michael ; Von Tress, Mark ; Lam, Byron L ; Gonzalez, Victor H ; Batabyal, Subrata ; Kim, Sanghoon ; Mohanty, Samarendra K ; Piltz-Seymour, Jody ; Choudry, Nozhat ; Tsang, Stephen H ; Boyer, David S ; Zak, Vitaliy

Background:

Stargardt disease (SD) is an inherited degenerative retinal disease affecting rod and cone photoreceptors and retinal pigment epithelial (RPE) cells, leading to severe and irreversible vision loss. Optogenetics is a promising approach to restoring vision by photosensitizing spared healthy retinal neurons.

Methods:

The STARLIGHT phase 2 open-label study (NCT05417126) was conducted over 48-weeks at two US sites to assess the safety and efficacy of MCO-010 administered via a single intravitreal injection in the worse-seeing eye of SD patients. The study began on July 5, 2022, and was completed on September 28, 2023. MCO-010 targets bipolar cells rather than retinal ganglion cells (RGCs) to utilize more abundant cells that respond to ambient light while preserving natural visual processing pathways after treatment. Six adults (mean age 50 years, range 32-71 years, four males and two females) received 1.2E11 genome copies (gc)/eye of MCO-010. The primary outcome was the incidence, nature, severity of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), intraocular inflammation, retinal thickness, best-corrected visual acuity (BCVA), and lesion size. Secondary endpoints included change in BCVA, vision-guided mobility, and shape determination accuracy. Visual field perimetry and Michigan Retinal Degeneration Questionnaire (MRDQ) were exploratory endpoints.

Findings:

All six participants had at least one ocular TEAE; non-ocular TEAEs occurred in three participants. The most common TEAEs were conjunctival hemorrhage, ocular hypertension, and vitreous cells (two subjects). There were no deaths, hospitalization, loss of eye, retinal detachment, endophthalmitis, or TEAEs leading to study discontinuation. The BCVA (mean ± SD) of the six treated eyes at baseline and 48-week follow-up was 22.8 ± 9.87 (range 9-35), and 28.3 ± 13.28 (range 4-42) ETDRS letters, respectively. The BCVA change from baseline was 7.2 ± 11.74, 4.2 ± 14.81, and 5.5 ± 12.29 at 12, 24, and 48 weeks, respectively. With a wearable magnifier (low-vision glasses), the BCVA change was 17.8 ± 13.35, 15.7 ± 17.37, 13.3 ± 21.37 at 12, 24, and 48 weeks, respectively. The improvement in mean defect in visual field perimetry was 1.02 ± 3.54, 2.47 ± 5.00, and 2.63 ± 5.26 dB at 12, 24, and 48 weeks, respectively. Specific improvements were noted in reading, and color, and contrast domains of the MRDQ.

Interpretation:

MCO-010 optogenetic phase 2 results support further investigation for treatment SD. To our knowledge, this is the first report of improving on(eye)-chart vision of SD participants utilizing optogenetics.

Funding:

Nanoscope Therapeutics Inc.

01 Aug 2025·Journal of Managed Care & Specialty Pharmacy

The effectiveness and value of sonpiretigene isteparvovec for the treatment of advanced retinitis pigmentosa

Article

Author: Rind, David M. ; McKenna, Avery ; Agboola, Foluso ; Phillips, Marie ; Richardson, Marina ; Makam, Anil N. ; Lee, Woojung ; Herce-Hagiwara, Belen

01 May 2025·MOLECULAR THERAPY

Perspectives and open questions in vision restoration with vMCO-010

Letter

Author: Lindner, Moritz

125

News (Medical) associated with Sonpiretigene Isteparvovec

10 Jul 2026

·www.prnewswire.com

Nanoscope Therapeutics to Present at 2026 American Society of Retina Specialists Annual Scientific Meeting

Jordi Monés, MD, PhD, to Introduce First Direct-to-Phase 2 Randomized, Controlled Trial of Optogenetic Therapy in Macular Degeneration Benjamin Bakall, MD, PhD, to Present Durable 3-Year REMAIN Follow-Up Data from the RESTORE Phase 2b/3 Trial of MCO-010 in Retinitis Pigmentosa MONTREAL, July 10, 2026 /PRNewswire/ -- Nanoscope Therapeutics, Inc., a biotechnology company committed to developing and commercializing novel, disease-agnostic therapies for patients with photoreceptor loss and vision impairment due to retinal degeneration, today announced that clinical data from its optogenetic therapy programs will be featured at the American Society of Retina Specialists (ASRS) 44th Annual Scientific Meeting, taking place July 15–18, 2026, at the Palais des Congrès de Montréal in Montreal, Canada. The presentations will highlight Nanoscope's entry into age-related macular degeneration (AMD) with a new clinical trial of optogenetic therapy in geographic atrophy (GA) evaluating vision improvement, and long-term durability data for MCO-010 in retinitis pigmentosa (RP). Presentation Details: Title: Vision Improvement with Sonpiretigene Isteparvovec Optogenetic Therapy in Macular Degeneration: First Direct-to-Phase 2 Randomized, Controlled Trial Presenter: Jordi Mones, MD, PhD Session: AMD – Non-neovascular Symposium 1: "Beyond Atrophy: Restoring Vision and Redefining Care in Geographic Atrophy" Date & Time: Thursday, July 16 | 1:46 PM Title: Durable Vision Improvement with MCO-010 Optogenetic Therapy: 3-Year REMAIN Follow-Up Data from the RESTORE Phase 2b/3 Trial for Retinitis Pigmentosa Presenter: Benjamin Bakall, MD, PhD Format: Paper-on-Demand Presentation About the MCO Platform Multi-characteristic opsin (MCO) is a one-time, in-office, intravitreal disease-agnostic therapy platform designed to restore vision in patients with photoreceptor degeneration, including Retinitis Pigmentosa (RP), Stargardt disease (SD), and geographic atrophy (GA). By activating highly dense bipolar retinal cells to become light sensitive, MCO utilizes the remaining visual circuitry following photoreceptor death. MCO treatment does not require genetic testing, invasive surgery, or repeat dosing, enabling broad patient applicability within existing retina office workflows. About Nanoscope Therapeutics Nanoscope Therapeutics is developing disease-agnostic, vision-restoring optogenetic therapy for millions of patients blinded by retinal degenerative diseases. Following positive results from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), a BLA has been submitted to the FDA. If approved, MCO-010 has the potential to be the standard of care for RP patients, administered as a one-time, in-office injection without the need for genetic testing. The company has also shown promising results in the STARLIGHT Phase 2 clinical trial of MCO-010 in SD (NCT05417126) and plans to initiate a Phase 3 registrational trial in 2026. MCO-010 has received FDA Fast Track and Orphan Drug designations for both RP and SD, along with RMAT designation for SD, and EMA Orphan designations to cover non-syndromic and syndromic rod- and cone-dominant dystrophies, as well as macular dystrophies. MCO-010 has received Sakigake and orphan designations for inherited retinal dystrophies (IRDs) in Japan and an orphan designation for IRDs in Saudi Arabia. A Phase 2 program for MCO in GA is expected to start in 2026. Other IND-ready programs include Leber congenital amaurosis (LCA). Contact: Nanoscope Therapeutics, Inc (817) 857-1186 [email protected] SOURCE Nanoscope Therapeutics 21% more press release views with Request a Demo

Phase 2Clinical ResultOrphan DrugFast Track

08 Jul 2026

·www.fiercepharma.com

Catalent sells UK facility to Codis, expands Nanoscope partnership

Catalent is shedding one of its U.K. manufacturing facilities as it inks a separate partnership deal to advance a new gene therapy, expanding in certain areas while scaling back in others. First up, Codis is set to acquire Catalent’s Nottingham facility in England for an undisclosed price, equipping the St. Louis-based CDMO with oral solid dose development and small-scale manufacturing capabilities, the company said in a July 6 press release. The transaction is expected to close by the end of the third quarter this year. Codis said the facility will help it move toward becoming Europe’s leading source for spray dry solutions, running the gamut from development through to final oral dose forms. The site also fits with Codis’ large-scale commercial spray-drying facility located in Haverhill, U.K. “This strategic acquisition will further establish Codis as a leading European provider of comprehensive spray dry solutions,” Nicolas Fortin, Codis chief executive, said in a statement. “By bringing formulation development and GMP manufacturing together, we provide the fastest, lowest-risk path to unlocking a molecule's full potential.” Codis also has manufacturing facilities in Missouri and Seymour, Indiana. Catalent, which was acquired by Novo Nordisk's parent company, Novo Holdings, for $16.5 billion in 2024, still operates four production plants in the U.K. and a clinical trial supply facility in Scotland. In a separate deal, Catalent expanded an ongoing partnership with Nanoscope Therapeutics to support late-phase clinical and commercial supply of the Dallas-based biotech’s lead optogenetic gene therapy, MCO-010. Optogenetic gene therapy integrates gene delivery with light to control cell activity. In order to target cells, a harmless virus inserts a light-sensitive protein gene into target cells that can be turned on or off with specific wavelengths of light, with the goal of restoring vision for patients with degenerative eye diseases such as retinitis pigmentosa and Stargardt disease. Under the partnership, Catalent is responsible for commercially compliant packaging and distribution for MCO-010, and the CDMO has already secured commercial packaging validation in support of Nanoscope's rolling submission to the FDA, the company said in a July 7 press release. Financial terms of the deal weren’t disclosed. “As we continue to manufacture MCO-010 commercial drug product, having a packaging partner who can scale with us to meet the needs of global commercialization is essential,” Sulagna Bhattacharya, co-Founder and CEO of Nanoscope, said in a statement. “Catalent’s expertise and reliability give us confidence as we prepare for the next stage of this journey.”

AcquisitionGene Therapy

08 Jul 2026

·www.pharmamanufacturing.com

Catalent, Nanoscope Therapeutics expand gene therapy agreement

Catalent, a global contract development and manufacturing organization (CDMO) headquartered in Tampa, Florida, has expanded its partnership with Nanoscope Therapeutics, a Dallas-based biotechnology company, to support late-phase clinical and commercial supply of MCO-010, an optogenetic gene therapy being developed to restore vision in patients with advanced retinal degenerative diseases including retinitis pigmentosa and Stargardt disease. Under the expanded agreement, Catalent will provide commercial-compliant packaging and distribution for MCO-010 and has secured a commercial packaging validation program to support Nanoscope’s Biologics License Application with the FDA, according to the announcement. A rolling BLA submission is currently underway, the company said. MCO-010 is designed to reprogram retinal cells to become light-sensitive, targeting patients with late-stage disease who currently have limited therapeutic options, according to the announcement. Sulagna Bhattacharya, CEO of Nanoscope Therapeutics, said in a statement that having a packaging partner able to scale to meet the needs of global commercialization is essential as the company continues to manufacture MCO-010 commercial drug product. The announcement follows two recent developments at Catalent. Earlier this week, the company agreed to sell its Nottingham, UK facility to Codis , a CDMO specializing in spray drying and particle engineering, as part of Codis’ expansion of its integrated oral solid dose and spray drying network in Europe. In June, Catalent launched its first enterprise AI tool to accelerate root cause analysis and corrective action development for quality management processes across its manufacturing network.

Gene TherapyLicense out/in

100 Deals associated with Sonpiretigene Isteparvovec

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Retinitis Pigmentosa	NDA/BLA	United States	Nanoscope Therapeutics, Inc.	14 Jul 2025
Stargardt Disease	Phase 2	United States	Nanoscope Therapeutics, Inc.	05 Jul 2022
Retinitis	Phase 2	United States	Nanoscope Therapeutics, Inc.	13 Jul 2021
Geographic Atrophy	Preclinical	United States	Nanoscope Therapeutics, Inc.	01 Apr 2026
Leber Congenital Amaurosis	Preclinical	United States	Nanoscope Therapeutics, Inc.	01 Apr 2025

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


RESTORE (EURETINA2025)	Phase 2/3	Retinitis Pigmentosa	27	MCO-010 low-dose	qthsolslom(ualyjjzyiz) = mptzddpcbu jjvwobadyt (ogyqepqryq, 0.14) View more	Positive	04 Sep 2025
				MCO-010 high-dose	qthsolslom(ualyjjzyiz) = vmdlbadyeg jjvwobadyt (ogyqepqryq, 0.112) View more
NCT04945772 (PRNewswire) Manual	Phase 2	Retinitis Pigmentosa	-	MCO-010 gene therapy	rldmccicef(jratchaikf) = tzywjlncpn lgzoursvdo (hqsmqiyfev ) View more	Positive	31 Oct 2024
				sham-control	ncfcoewdvp(zdfsrtlhuo) = keytlojtob hbhsqetobx (lqifmvhikp ) View more
NCT04945772 (RESTORE, Biospace) Manual	Phase 2	Retinitis Pigmentosa	27	MCO-010 high dose	ycpwfttyxo(hgrevwbjrv) = xvxcvvyhuq ufazkgjrmf (kukazkqwmk, 0.0829) View more	Positive	26 Mar 2024
				MCO-010 low dose	ycpwfttyxo(hgrevwbjrv) = bzfkyuhjiv ufazkgjrmf (kukazkqwmk, 0.1244) View more
NCT05417126 (STARLIGHT, ARVO2023) Manual	Phase 2	Stargardt Disease	6	MCO-010	fsrqcycayn(rhpzayhgjq) = uwdhetlhkd xqlraalgtm (swaudrpxxf ) View more	Positive	01 Jun 2023

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