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[{"orgOrder":0,"company":"BrainVectis","sponsor":"AskBio","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AskBio Acquires BrainVectis to Expand its Clinical Pipeline for Neurodegenerative Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"BrainVectis"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vivet\u2019s Second Gene Therapy Product, VTX-803 for PFIC3, Receives US and European Orphan Drug Designation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 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Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"SATT Conectus"},{"orgOrder":0,"company":"Variant","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"VARIANT Receives European Orphan Drug Designation for its Gene Therapy Product Candidate, VAR002, for the Treatment of Both Leber Congenital Amaurosis and Cone-Rod Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Variant"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not 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Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Cellectis"},{"orgOrder":0,"company":"SparingVision","sponsor":"Tenpoint Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"SparingVision Sells Ex Vivo GIRK Technology to Tenpoint Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"SparingVision"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"French Government","pharmaFlowCategory":"D","amount":"$5.2 million","upfrontCash":"Undisclosed","newsHeadline":"Vivet Therapeutics Receives EUR 4.9 Million to Advance Development of a Gene Therapy for the Treatment of Cerebrotendinous Xanthomatosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Vivet Therapeutics"}]

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            Vivet will use the funds to further develop its gene therapy product VTX-806 as an effective treatment option to stop or reverse disease progression over the long-term, or potentially cure cerebrotendinous xanthomatosis in patients.

            Lead Product(s): VTX-806

            Therapeutic Area: Genetic Disease Product Name: VTX-806

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: French Government

            Deal Size: $5.2 million Upfront Cash: Undisclosed

            Deal Type: Funding February 01, 2024

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            Through the divestment, Tenpoint has acquired SparingVision's ex vivo GIRK technology. SparingVision will remain focused on in vivo genomic medicines, with SPVN20, a GIRK-based gene therapy candidate.

            Lead Product(s): SPVN20

            Therapeutic Area: Genetic Disease Product Name: SPVN20

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Tenpoint Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Divestment July 12, 2023

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            Primera is advancing pioneering mtDNA gene editing therapies developed in the Mayo Clinic lab to precisely fix a patient’s mutated mtDNA and potentially achieve a cure for the disease.

            Lead Product(s): mtDNA-based Therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Primera Therapeutics

            Deal Size: $750.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration December 29, 2022

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            The pre-clinical data presented at ESGCT further demonstrate our ability to leverage TALEN® gene editing technology to potentially address genetic diseases, namely, sickle cell disease and lysosomal storage diseases.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 11, 2022

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            The publication of a peer-reviewed article for FXS01 in EMBO Molecular Medicine showing that adeno-associated viral (AAV) vector-delivered diacylglycerol kinase (DGKk) achieves long-term rescue of fragile X syndrome (FXS) in a mouse model of disease.

            Lead Product(s): FXS01

            Therapeutic Area: Genetic Disease Product Name: FXS01

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 21, 2022

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            First approval of ATA-100, was received from UK MHRA its gene therapy for the treatment of the fukutin-related protein limb-girdle muscular dystrophy Type 2I/R9.

            Lead Product(s): ATA-100

            Therapeutic Area: Genetic Disease Product Name: ATA-100

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 06, 2021

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            VAR002 is a CRX-expressing AAV vector designed as a relevant therapeutic approach for gene supplementation and a promising drug candidate for mutation-agnostic gene therapy. VAR002 prevents photoreceptor degeneration without adverse effects linked with CRX overexpression.

            Lead Product(s): VAR002

            Therapeutic Area: Genetic Disease Product Name: VAR002

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 24, 2021

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            Details:

            Lysogene will be responsible for the preclinical and clinical development, manufacturing, regulatory activities, and commercialization of the drug candidate, globally. which includes FXS01, a gene therapy candidate for the treatment of Fragile X syndrome.

            Lead Product(s): FXS01

            Therapeutic Area: Genetic Disease Product Name: FXS01

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Lysogene

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement June 11, 2021

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            Vivet will continue to advance the preclinical studies for VTX-803 and VTX-802 for PFIC3 and PFIC2. Mirum has the exclusive option to license the programs after which Mirum would lead the clinical development and any future commercialization of the programs.

            Lead Product(s): VTX-803

            Therapeutic Area: Genetic Disease Product Name: VTX-803

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Mirum Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration April 12, 2021

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            The funding round supports company's program focusing on identifying new vectors. Using the company's VECTrans® platform, nanoparticles of mRNA, as well as ASOs and siRNAs, can be vectorized in order to treat both diseases with a genetic component and cancers.

            Lead Product(s): Nucleic acid based therapeutics

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Undisclosed

            Deal Size: $14.5 million Upfront Cash: Undisclosed

            Deal Type: Series D Financing January 20, 2021

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