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Announces $200 Million Private Placement Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Tyra Biosciences"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Neurocrine Biosciences","pharmaFlowCategory":"D","amount":"$1,865.0 million","upfrontCash":"$165.0 million","newsHeadline":"Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich's Ataxia in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Voyager Therapeutics, Inc"}]

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            Details:

            Novasep will be in charge of developing and manufacturing the two AAV vectors designed for the Sensorion OTOF-GT project and will supply Drug Product batches to support preclinical and clinical studies.

            Lead Product(s): OTOF-GT,Aav Vectors

            Therapeutic Area: Genetic Disease Product Name: OTOF-GT

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

            Partner/Sponsor/Collaborator: Sensorion

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement October 27, 2020

            Axplora CB

            TMF Summit 2024

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            envelop Contact Supplier

            Details:

            Through collabraotion, Neurocrine will to focus on the development and commercialization of VY-FXN01, which helps to restore FXN protein levels. It is being evaluated in the treatment of Friedreich’s ataxia.

            Lead Product(s): VY-FXN01

            Therapeutic Area: Genetic Disease Product Name: VY-FXN01

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

            Partner/Sponsor/Collaborator: Neurocrine Biosciences

            Deal Size: $1,865.0 million Upfront Cash: $165.0 million

            Deal Type: Collaboration February 26, 2024

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            The net proceeds will be used for continued research and development of next-generation gene editing technology to develop company's therapeutic portfolio, including metabolic disease, hemophilia A, cardiovascular disease, central nervous system diseases and cystic fibrosis.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: J.P. Morgan

            Deal Size: $93.7 million Upfront Cash: Undisclosed

            Deal Type: Public Offering February 13, 2024

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            The net proceeds will be used for continued research and development of next-generation gene editing technology to develop company's therapeutic portfolio, including metabolic disease, hemophilia A, cardiovascular disease, central nervous system diseases and cystic fibrosis.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: J.P. Morgan

            Deal Size: $93.7 million Upfront Cash: Undisclosed

            Deal Type: Public Offering February 08, 2024

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            The net proceeds will be used for continued research and development of next-generation gene editing technology to develop company's therapeutic portfolio, including metabolic disease, hemophilia A, cardiovascular disease, central nervous system diseases and cystic fibrosis.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: J.P. Morgan

            Deal Size: $86.9 million Upfront Cash: Undisclosed

            Deal Type: Public Offering February 05, 2024

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            The net proceeds will be used for the development of TYRA-300 an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of achondroplasia.

            Lead Product(s): TYRA-300

            Therapeutic Area: Genetic Disease Product Name: TYRA-300

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: RA Capital Management

            Deal Size: $200.0 million Upfront Cash: Undisclosed

            Deal Type: Private Placement February 02, 2024

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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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            Under the agreement, BlueRock exercised its option to exclusively license OpCT-001, an induced pluripotent stem cell (iPSC) derived cell therapy candidate for the treatment of primary photoreceptor diseases.

            Lead Product(s): OpCT-001

            Therapeutic Area: Genetic Disease Product Name: OpCT-001

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

            Partner/Sponsor/Collaborator: BlueRock Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement January 23, 2024

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            KRRO-110 is based on Korro’s proprietary RNA editing platform, OPERA™. It is being evaluated in preclinical studies for the treatment of Alpha-1 Antitrypsin Deficiency.

            Lead Product(s): KRRO-110

            Therapeutic Area: Genetic Disease Product Name: KRRO-110

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 18, 2024

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            The company will use the net proceeds to develop its first-in-class therapeutic pipeline which targets mitochondria dysfunction linked to the progression of rare diseases such as the neuromuscular disorder Duchenne muscular dystrophy, Huntington’s disease.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Wren Capital

            Deal Size: $5.0 million Upfront Cash: Undisclosed

            Deal Type: Financing December 13, 2023

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