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[{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nanoscope Therapeutics Receives Orphan Drug Designation for Gene Therapy of Blindness","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nanoscope President to Deliver Talk on Gene Therapy for Retinal Diseases at Association for Ocular Pharmacology and Therapeutics Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nanoscope's Optogenetic Gene Therapy Restores Clinically Meaningful Vision in 11 Patients Blinded by Retinitis Pigmentosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nanoscope's Patient Dosed in Phase 2b clinical trial of Optogenetic Gene therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"National Eye Institute","pharmaFlowCategory":"D","amount":"$1.5 million","upfrontCash":"Undisclosed","newsHeadline":"Nanoscope Awarded $1.5M Phase 2B SBIR Grant to Advance Ambient Light Activatable Opsin Gene Therapy to Restore Vision for AMD Patients","therapeuticArea":"Ophthalmology","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Ophthalmology","graph2":"IND Enabling"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nanoscope 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Entity","date":"September 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Charles River Laboratories, Inc","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Charles River and Nanoscope Therapeutics Announce Multifaceted Gene Therapy Manufacturing Partnership","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nanoscope Therapeutics Announces Positive Topline Results from Phase 2b RESTORE Trial of MCO-010 for the Treatment of Retinitis Pigmentosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"}]

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            Details:

            MCO-010 (sonpiretigene isteparvovec) is an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy for vision restoration in advanced retinitis pigmentosa (RP).

            Lead Product(s): Sonpiretigene Isteparvovec

            Therapeutic Area: Genetic Disease Product Name: MCO-010

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 30, 2023

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            Through this partnership, Nanoscope will have access to established manufacturing platforms and multiple Charles River CDMO centers of excellence. The vMCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin gene expression cassette.

            Lead Product(s): vMCO-010

            Therapeutic Area: Genetic Disease Product Name: vMCO-010

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Recipient: Charles River Laboratories, Inc

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership October 18, 2022

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            This multicenter, randomized, double-masked, sham-controlled, dose-ranging study will evaluate 2 dose levels of vMCO-010 in up to 18 subjects with retinitis pigmentosa (9 per dose).

            Lead Product(s): vMCO-010

            Therapeutic Area: Genetic Disease Product Name: vMCO-010

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 13, 2022

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            Nanoscope’s MCO-020 gene therapy program is based on targeted laser-delivery of optogenes to areas of geographic atrophy in advanced AMD patients, and currently undergoing IND-enabling studies.

            Lead Product(s): MCO-020

            Therapeutic Area: Ophthalmology Product Name: MCO-020

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 08, 2022

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            The purpose of the study is to evaluate the safety and efficacy of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010).

            Lead Product(s): vMCO-010

            Therapeutic Area: Genetic Disease Product Name: vMCO-010

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 29, 2022

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            Details:

            This multicenter, randomized, double-masked, sham-controlled, dose-ranging study will evaluate 2 dose levels of vMCO-010 in up to 18 subjects with retinitis pigmentosa (9 per dose). Nine subjects will receive sham injection.

            Lead Product(s): vMCO-010

            Therapeutic Area: Genetic Disease Product Name: vMCO-010

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 26, 2022

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            Details:

            vMCO-010, an investigational AAV gene therapy designed to restore functional vision to patients with severe vision loss due to retinal degeneration, demonstrated a favorable safety profile and several patients experienced restoration of vision in Phase 1/2a open-label trial.

            Lead Product(s): MCO-010

            Therapeutic Area: Genetic Disease Product Name: vMCO-010

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 26, 2022

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            Details:

            Nanoscope Therapeutics lead product MCO-010, is in a Phase 2b multicenter, randomized, double-masked, sham controlled clinical trial in the U.S. for treatment of retinitis pigmentosa.

            Lead Product(s): MCO-010

            Therapeutic Area: Ophthalmology Product Name: MCO-010

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 15, 2021

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            Details:

            Nanoscope’s lead MCO gene therapy, MCO-010, is in a late-stage Phase 2b trial for retinitis pigmentosa (RP) in the US. MCO-010 has orphan drug designations for RP and Stargardt disease from the US Food and Drug Administration.

            Lead Product(s): MCO-010

            Therapeutic Area: Ophthalmology Product Name: MCO-010

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: National Eye Institute

            Deal Size: $1.5 million Upfront Cash: Undisclosed

            Deal Type: Funding September 14, 2021

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            Details:

            The treatment will involve a single intravitreal injection of MCO-010 or sham to confirm safety, tolerability, and efficacy in improving patients’ vision and visual function.

            Lead Product(s): MCO-010

            Therapeutic Area: Genetic Disease Product Name: vMCO-010

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 19, 2021

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