[{"orgOrder":0,"company":"Opus Genetics","sponsor":"RD Fund","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Financing","leadProduct":"OPGx-001","moa":"LCA5 gene mutation","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Opus Genetics \/ RD Fund","highestDevelopmentStatusID":"4","companyTruncated":"Opus Genetics \/ RD Fund"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Massachusetts Eye and Ear and Harvard Medical School","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Agreement","leadProduct":"OPGx-003","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Opus 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Resilience"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"OPGx-002","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Opus Genetics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Opus Genetics \/ Not Applicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"OPGx-001","moa":"LCA5 gene","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Opus Genetics \/ Not Applicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AAV8.hLCA5","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Opus Genetics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Not Applicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"IVERIC bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Acquisition","leadProduct":"AAv-based Gene Therapy","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Opus Genetics \/ Opus Genetics","highestDevelopmentStatusID":"4","companyTruncated":"Opus Genetics \/ Opus Genetics"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"OPGx-LCA5","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare 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Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Funding","leadProduct":"OPGx-RHO","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Opus Genetics \/ Foundation Fighting Blindness","highestDevelopmentStatusID":"4","companyTruncated":"Opus Genetics \/ Foundation Fighting Blindness"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"OPGx-LCA5","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Opus Genetics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Not Applicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Ocuphire Pharma","pharmaFlowCategory":"D","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Merger","leadProduct":"APX3330","moa":"","graph1":"Ophthalmology","graph2":"Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"","sponsorNew":"Opus Genetics \/ Ocuphire Pharma","highestDevelopmentStatusID":"8","companyTruncated":"Opus Genetics \/ Ocuphire Pharma"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"APX3330","moa":"","graph1":"Ophthalmology","graph2":"Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"","sponsorNew":"Opus Genetics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Opus Genetics \/ Not Applicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"AAV8.hLCA5","moa":"LCA5 gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Inapplicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Phentolamine Mesylate","moa":"Adrenergic receptor alpha-1\/2","graph1":"Ophthalmology","graph2":"Phase III","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Ophthalmic Solution","sponsorNew":"Opus Genetics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Opus Genetics \/ Inapplicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Perceptive Advisors","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Public Offering","leadProduct":"OPGx-LCA5","moa":"LCA5 gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Opus Genetics","amount2":0.02,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Perceptive Advisors","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Perceptive Advisors"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Phentolamine Mesylate","moa":"Adrenergic receptor-alpha 1\/2","graph1":"Ophthalmology","graph2":"Phase III","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Ophthalmic Solution","sponsorNew":"Opus Genetics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Opus Genetics \/ Inapplicable"}]

Find Clinical Drug Pipeline Developments & Deals by Opus Genetics

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : Opus intends to use the net proceeds to fund clinical development of its lead gene therapy programs, OPGx-LCA5 for the treatment of leber congenital amaurosis.

                          Product Name : OPGx-LCA5

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          March 21, 2025

                          Lead Product(s) : OPGx-LCA5

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Perceptive Advisors

                          Deal Size : $21.4 million

                          Deal Type : Public Offering

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                          02

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : Nyxol (phentolamine ophthalmic solution 0.75%) is being evaluated for the treatment in patients with dim light disturbances and decreased mesopic vision.

                          Product Name : Nyxol

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 21, 2025

                          Lead Product(s) : Phentolamine Mesylate

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : Nyxol (phentolamine mesylate) an alpha1/2 adrenergic receptor inhibitor, is being investigated for decreased visual acuity under low light conditions following keratorefractive surgery.

                          Product Name : Nyxol

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 26, 2025

                          Lead Product(s) : Phentolamine Mesylate

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : OPGx-LCA5 is an investigational gene therapy, which is currently being evaluated for the treatment of patients with leber congenital amaurosis.

                          Product Name : OPGx-LCA5

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 18, 2025

                          Lead Product(s) : AAV8.hLCA5

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : APX3330 is an oral small-molecule inhibitor of Ref-1, which is currently being evaluated for the treatment of patients with non-proliferative diabetic retinopathy.

                          Product Name : Undisclosed

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 19, 2024

                          Lead Product(s) : APX3330

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : The acquisition creates a leading, clinical-stage company focused on developing gene therapies, including APX3330, an oral small-molecule inhibitor of Ref-1 for non-proliferative diabetic retinopathy.

                          Product Name : APX3330

                          Product Type : Small molecule

                          Upfront Cash : Undisclosed

                          October 22, 2024

                          Lead Product(s) : APX3330

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : Phase II

                          Sponsor : Ocuphire Pharma

                          Deal Size : Undisclosed

                          Deal Type : Merger

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                          07

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : OPGx-LCA5 is an AAV8 vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis resulting from biallelic mutations in the LCA5 gene.

                          Product Name : OPGx-LCA5

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          August 20, 2024

                          Lead Product(s) : OPGx-LCA5

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : The proceeds will be used to conduct a preclinical safety study for OPGx-RHO, a gene therapy that targets autosomal dominant retinitis pigmentosa caused by RHO mutations (RHO-adRP).

                          Product Name : OPGx-RHO

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          June 13, 2024

                          Lead Product(s) : OPGx-RHO

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Preclinical

                          Sponsor : Foundation Fighting Blindness

                          Deal Size : $1.7 million

                          Deal Type : Funding

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                          09

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : OPGx-LCA5 uses an AAV8 vector to deliver a functional LCA5 gene to the retina for patients with Leber congenital amaurosis from LCA5 mutations.

                          Product Name : OPGx-LCA5

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          March 26, 2024

                          Lead Product(s) : OPGx-LCA5

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5).

                          Product Name : OPGx-LCA5

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          September 07, 2023

                          Lead Product(s) : OPGx-LCA5

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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