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    [{"orgOrder":0,"company":"Opus Genetics","sponsor":"RD Fund","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","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":"","productType":"Cell and Gene therapy","year":"2021","type":"Agreement","leadProduct":"OPGx-003","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Opus 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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":"Technology","graph2":"Phase I\/II","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Opus Genetics \/ Ocuphire Pharma","highestDevelopmentStatusID":"14","companyTruncated":"Opus Genetics \/ Ocuphire Pharma"}]

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                            01

                            Opus Genetics

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                            CPhI India 2024
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                            Opus Genetics

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                            Lead Product(s) : APX3330

                            Therapeutic Area : Ophthalmology

                            Study Phase : Phase II

                            Sponsor : Ocuphire Pharma

                            Deal Size : Undisclosed

                            Deal Type : Merger

                            DEALS_DEV arrow-down

                            Ocuphire Pharma Announces Acquisition of Opus Genetics

                            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

                            Opus Genetics

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                            Lead Product(s) : OPGx-LCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Opus Genetics Receives FDA Rare Pediatric Disease Designation for OPGx-LCA5

                            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

                            Opus Genetics

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                            Lead Product(s) : OPGx-RHO

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Foundation Fighting Blindness

                            Deal Size : $1.7 million

                            Deal Type : Funding

                            DEALS_DEV arrow-down

                            Opus Genetics Announces $1.7 Million in Funding for Preclinical Programs

                            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

                            Opus Genetics

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                            Opus Genetics

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                            Lead Product(s) : OPGx-LCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Opus Genetics Completes Dosing in Phase 1/2 Trial of Gene Therapy OPGx-LCA5

                            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

                            Opus Genetics

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                            CPhI India 2024
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                            Opus Genetics

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                            Lead Product(s) : OPGx-LCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Opus Genetics Announces First Patient Dosed in Phase 1/2 Trial of Gene Therapy OPGx-LCA5 in Patients with Rare...

                            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

                            Opus Genetics

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                            Lead Product(s) : AAv-based Gene Therapy

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Recipient : IVERIC bio

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

                            DEALS_DEV arrow-down

                            Opus Genetics Announces Acquisition of the Rights to Two Gene Therapy Product Candidates for Inherited Retinal...

                            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

                            Opus Genetics

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                            CPhI India 2024
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                            Lead Product(s) : AAV8.hLCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Opus Genetics Receives FDA Clearance of IND Application for OPGx-001, a Gene Therapy Candidate Intended for th...

                            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

                            Opus Genetics

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                            Lead Product(s) : OPGx-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Opus Genetics Announces Promising New Data Highlighting Potential of AAV-based Gene Therapies for the Treatmen...

                            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

                            Opus Genetics

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                            CPhI India 2024
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                            Opus Genetics

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                            Lead Product(s) : OPGx-002

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            New Data on Potential of Opus AAV-based Gene Therapies for Rare Inherited Retinal Diseases to be Presented at ...

                            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

                            Opus Genetics

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                            Lead Product(s) : OPGx-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : National Resilience

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Opus Enters Strategic Collaboration with Resilience for AAV-based Gene Therapy Development and Manufacturing f...

                            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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