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Undisclosed"},{"orgOrder":0,"company":"MyoPax","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"GERMANY","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Satori-01","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"MyoPax","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"MyoPax \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"MyoPax \/ Undisclosed"},{"orgOrder":0,"company":"Sixera Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWEDEN","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"SXR1096","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Sixera Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Cream","sponsorNew":"Sixera Pharma \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sixera Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"UX053","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"UX053","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Xenon Pharmaceuticals Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Undisclosed","year":"2010","type":"Inapplicable","leadProduct":"XPF-001","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Xenon Pharmaceuticals Inc","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Xenon Pharmaceuticals Inc \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Xenon Pharmaceuticals Inc \/ Undisclosed"},{"orgOrder":0,"company":"OBiO Technology","sponsor":"Chinagene","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell & Gene Therapy","year":"2023","type":"Collaboration","leadProduct":"ZVS101e","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"OBiO Technology","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"OBiO Technology \/ Chinagene","highestDevelopmentStatusID":"7","companyTruncated":"OBiO Technology \/ Chinagene"},{"orgOrder":0,"company":"Bioray Laboratories","sponsor":"923rd Hospital of Joint Logistics Support Force of PLA","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"\u0392-Globin Restored Autologous Hematopoietic Stem Cell","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Bioray Laboratories","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bioray Laboratories \/ 923rd Hospital of Joint Logistics Support Force of PLA","highestDevelopmentStatusID":"7","companyTruncated":"Bioray Laboratories \/ 923rd Hospital of Joint Logistics Support Force of PLA"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"QR-421a","moa":"Usherin messenger RNA (USH2A mRNA)","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"ProQR Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ProQR Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"EXG-001","moa":"||ZSCAN4","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Elixirgen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Elixirgen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"EXG-34217","moa":"ZSCAN4","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Elixirgen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Elixirgen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EXG-34217","moa":"ZSCAN4","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Elixirgen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Elixirgen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"EXG-34217","moa":"ZSCAN4","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Elixirgen Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Elixirgen Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Elixirgen Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Acquisition","leadProduct":"CLN-301","moa":"CLN3","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Alcyone Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alcyone Therapeutics \/ Biogen","highestDevelopmentStatusID":"7","companyTruncated":"Alcyone Therapeutics \/ Biogen"},{"orgOrder":0,"company":"MavriX Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"MVX-220","moa":"UBE3A gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"MavriX Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"MavriX Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"MavriX Bio \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ALN-CFB","moa":"CFB","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Pat...

                            Details : Olipudase Alfa is a Enzyme drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Niemann-Pick Disease, Type A.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            November 17, 2014

                            Lead Product(s) : Olipudase Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : SAR421869

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

                            Details : SAR421869 is a drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Usher Syndromes.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            January 06, 2012

                            Lead Product(s) : SAR421869

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Regeneron Pharmaceuticals

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            Regeneron Pharmaceuticals

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : ALN-CFB

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Fa...

                            Details : ALN-CFB is a Oligonucleotide drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Hemoglobinuria, Paroxysmal.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            September 23, 2025

                            Lead Product(s) : ALN-CFB

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Alcyone Therapeutics

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            Alcyone Therapeutics

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : CLN-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Biogen

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

                            DEALS_DEV arrow-down

                            Biogen to Acquire Alcyone Therapeutics, Expanding Drug Delivery Solution Portfolio

                            Details : With the acquisition, Biogen Alcyone’s remaining therapeutic assets, including CLN-301 will be divested into a new independent company, Neela Therapeutics.

                            Product Name : CLN-301

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            September 18, 2025

                            Lead Product(s) : CLN-301

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Biogen

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

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                            05

                            MavriX Bio

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            MavriX Bio

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : MVX-220

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome

                            Details : MVX-220 is a Cell & Gene Therapy drug candidate, which is currently being evaluated in Phase I/ Phase II clinical studies for the treatment of Angelman Syndrome.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            September 18, 2025

                            Lead Product(s) : MVX-220

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Taysha Gene Therapies

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            Taysha Gene Therapies

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : TSHA-102

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Jefferies

                            Deal Size : Undisclosed

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Taysha Gene Therapies Announces Offering of Common Stock and Pre-Funded Warrants

                            Details : The proceeds will be used to fund clinical, regulatory and manufacturing development for TSHA-102, a self-complementary intrathecally delivered AAV9 gene transfer therapy, in Rett syndrome.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            May 28, 2025

                            Lead Product(s) : TSHA-102

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Jefferies

                            Deal Size : Undisclosed

                            Deal Type : Public Offering

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                            07

                            Taysha Gene Therapies

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            Taysha Gene Therapies

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : TSHA-102

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Jefferies

                            Deal Size : $200.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Taysha Gene Therapies Announces Pricing of Stock and Pre-Funded Warrants

                            Details : The company will fund clinical, regulatory and manufacturing development for TSHA-102, a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy, in Rett syndrome.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            May 28, 2025

                            Lead Product(s) : TSHA-102

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Jefferies

                            Deal Size : $200.0 million

                            Deal Type : Public Offering

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                            08

                            Theranexus

                            France arrow
                            OTS 2025
                            Not Confirmed

                            Theranexus

                            France arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Beyond Batten Disease Foundation

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Theranexus & BBDF Reveal Real-World Data Supporting Batten-1 for Batten

                            Details : Batten-1 is a novel and exclusive proprietary drug containing the active ingredient miglustat. The MOA of this substance blocks the accumulation of glycosphingolipids and neuroinflammation.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 13, 2025

                            Lead Product(s) : Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Beyond Batten Disease Foundation

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Opus Genetics

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            Opus Genetics

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : AAV8.hLCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Opus gets RMAT Designation for OPGx-LCA5 Gene Therapy in Vision Restoration

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

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            May 06, 2025

                            Lead Product(s) : AAV8.hLCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Opus Genetics

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            Opus Genetics

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : AAV8.hLCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Opus Shares One-Month Data from Pediatric Gene Therapy OPGx-LCA5 Trial

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

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            April 08, 2025

                            Lead Product(s) : AAV8.hLCA5

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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