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Genetics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Dosage Strength\/Form","sponsorNew":"Opus Genetics \/ Massachusetts Eye and Ear and Harvard Medical School","highestDevelopmentStatusID":"4","companyTruncated":"Opus Genetics \/ Massachusetts Eye and Ear and Harvard Medical School"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"National Resilience","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","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":"","sponsorNew":"Opus Genetics \/ National Resilience","highestDevelopmentStatusID":"5","companyTruncated":"Opus Genetics \/ National Resilience"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","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":"","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":"","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":"","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":"","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 Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Opus Genetics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Not Applicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","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":"Injection","sponsorNew":"Opus Genetics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Opus Genetics \/ Not Applicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Foundation Fighting Blindness","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","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":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"OPGx-LCA5","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Opus Genetics \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"Opus Genetics \/ Not Applicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Ocuphire Pharma","pharmaFlowCategory":"D","therapeuticArea":"Ophthalmology","country":"","productType":"Small molecule","year":"2024","type":"Merger","leadProduct":"APX3330","moa":"","graph1":null,"graph2":null,"graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":null,"therapeuticAreaShortName":null,"amount2New":0,"dosageForm":"","sponsorNew":"Opus Genetics \/ Ocuphire Pharma","highestDevelopmentStatusID":null,"companyTruncated":"Opus Genetics \/ Ocuphire Pharma"}]

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

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          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.

                          Brand Name : APX3330

                          Molecule 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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                          02

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          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.

                          Brand Name : OPGx-LCA5

                          Molecule 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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                          03

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          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).

                          Brand Name : OPGx-RHO

                          Molecule 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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                          04

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          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.

                          Brand Name : OPGx-LCA5

                          Molecule 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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                          05

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          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).

                          Brand Name : OPGx-LCA5

                          Molecule 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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                          06

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : Opus Genetics' AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : $0.5 million

                          December 28, 2022

                          Lead Product(s) : AAv-based Gene Therapy

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Preclinical

                          Recipient : IVERIC bio

                          Deal Size : Undisclosed

                          Deal Type : Acquisition

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                          07

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : OPGx-001 is an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5gene to retinal photoreceptors. Preclinical studies in in vitro and in vivo models of LCA5 have provided support for the safety and efficacy of OPGx-001.

                          Brand Name : OPGx-001

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          December 01, 2022

                          Lead Product(s) : AAV8.hLCA5

                          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

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : Preclinical data, including faithful animal and human iPSC models, have demonstrated preservation of visual function when OPGx-001 was administered prior to peak disease severity, indicating the potential for a broad therapeutic window for patients with ...

                          Brand Name : OPGx-001

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          May 04, 2022

                          Lead Product(s) : OPGx-001

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : Data support continued development of OPGx-001, designed to address mutations in LCA5 gene and OPGx-002, restoring protein expression caused by mutations in RDH12 gene, to address mutations in genes causing forms of Leber congenital amaurosis.

                          Brand Name : OPGx-002

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          April 28, 2022

                          Lead Product(s) : OPGx-002

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Preclinical

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : Resilience will provide process and analytical development, quality control testing, and GMP manufacturing services for IND-enabling toxicology and first-in-human material for Opus’ adeno-associated viral (AAV) vector-based gene therapies including OPG...

                          Brand Name : OPGx-001

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          April 11, 2022

                          Lead Product(s) : OPGx-001

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Preclinical

                          Sponsor : National Resilience

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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