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[{"orgOrder":0,"company":"SparingVision","sponsor":"4BIO Capital","pharmaFlowCategory":"D","amount":"$51.9 million","upfrontCash":"Undisclosed","newsHeadline":"SparingVision Raises \u20ac44.5 Million and Appoints St\u00e9phane Boissel\n as Chief Executive Officer","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"SparingVision"},{"orgOrder":0,"company":"Lysogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Lysogene Receives MHRA and Research Ethics Committee Approvals to Initiate the Gene Therapy Clinical Trial in the UK with LYS-GM101","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Lysogene"},{"orgOrder":0,"company":"SparingVision","sponsor":"Jeito Capital","pharmaFlowCategory":"D","amount":"$75.0 million","upfrontCash":"Undisclosed","newsHeadline":"SparingVision Raises \u20ac75 Million Series B to Continue Building World-Leading Portfolio of Genomic Medicines for Ocular Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"SparingVision"},{"orgOrder":0,"company":"SparingVision","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"SparingVision's Lead Asset SPVN06 Clears IND Application in the US for the Treatment of Retinitis Pigmentosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"SparingVision"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IND for ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I\/R9 (LGMD2I\/R9), Cleared to Proceed By FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Atamyo Therapeutics"},{"orgOrder":0,"company":"Enyo Pharma","sponsor":"OrbiMed","pharmaFlowCategory":"D","amount":"$42.5 million","upfrontCash":"Undisclosed","newsHeadline":"ENYO Pharma Announces a \u20ac39 Million Series C Financing and FDA Clearance to Advance Vonafexor in a Phase 2 Clinical Trial for Patients With Alport Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Enyo Pharma"}]

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            Details:

            The financing will support the Phase 2 Alpestria-1 study and further profiling of EYP001 (vonafexor), a highly selective NR1H4 agonist, in other kidney diseases, such as Autosomal Dominant Polycystic Kidney Disease (ADPKD).

            Lead Product(s): Vonafexor

            Therapeutic Area: Genetic Disease Product Name: EYP001

            Highest Development Status: IND Enabling Product Type: Small molecule

            Partner/Sponsor/Collaborator: OrbiMed

            Deal Size: $42.5 million Upfront Cash: Undisclosed

            Deal Type: Series C Financing January 03, 2024

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            ATA-100 (GNT0006), a single-administration gene therapy candidate for LGMD2I/R9, delivers a normal copy of the gene for production of FKRP proteins, which is investigated for the treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9).

            Lead Product(s): GNT0006

            Therapeutic Area: Genetic Disease Product Name: ATA-100

            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 06, 2023

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            SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor and one enzyme reducing oxidative stress which, acting synergistically, aim at slowing or stopping the degeneration of cone photoreceptors.

            Lead Product(s): SPVN06

            Therapeutic Area: Genetic Disease Product Name: SPVN06

            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 December 01, 2022

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            Proceeds from the financing will be used to fund the first-in-human trials of the Company’s two lead gene-independent assets, SPVN06 and SPVN20, as well as the development of genome editing assets through its collaboration with Intellia Therapeutics.

            Lead Product(s): SPVN06

            Therapeutic Area: Genetic Disease Product Name: SPVN06

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

            Partner/Sponsor/Collaborator: Jeito Capital

            Deal Size: $75.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing September 14, 2022

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            First regulatory authorization to initiate this global, multi-center, single-arm, two-stage, adaptive-design clinical trial of LYS-GM101 in patients with a diagnosis of early or late infantile GM1 gangliosidosis.

            Lead Product(s): LYS-GM101

            Therapeutic Area: Genetic Disease Product Name: LYS-GM101

            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 January 11, 2021

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            Details:

            Proceeds from the financing will be primarily used to advance the development of SparingVision’s breakthrough treatment SPVN06 for the mutation-agnostic treatment of retinitis pigmentosa.

            Lead Product(s): SPVN06

            Therapeutic Area: Genetic Disease Product Name: SPVN06

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

            Partner/Sponsor/Collaborator: 4BIO Capital

            Deal Size: $51.9 million Upfront Cash: Undisclosed

            Deal Type: Financing October 21, 2020

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