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[{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"The University Of Colorado Anschutz Medical Campus","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"BridgeBio Pharma and The University Of Colorado Anschutz Medical Campus Collaborate To Advance Medicines For Genetically Driven Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Undisclosed","productStatus":"Undisclosed","date":"October 2020","url1":"","url2":"","graph1":"Undisclosed","graph2":"BridgeBio Pharma"},{"orgOrder":0,"company":"Vipergen","sponsor":"Casma Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Casma Therapeutics and Vipergen Enter Into Drug Discovery Agreement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery Platform","country":"DENMARK","productType":"Small molecule","productStatus":"Undisclosed","date":"December 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Advance Ex Vivo Engineered Cell Therapy Programs","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Arbor Pharmaceuticals"},{"orgOrder":0,"company":"Integral Molecular","sponsor":"Optimeos Life Sciences","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Integral Molecular and Optimeos Life Sciences Enter Partnership to Develop mRNA and DNA-Based Gene Therapies Using Molecular Targeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Integral Molecular"},{"orgOrder":0,"company":"Ring Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ring Therapeutics Announces Issuance of U.S. Patent for its Anellovector\u2122 Compositions","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Large molecule","productStatus":"Undisclosed","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Ring Therapeutics"},{"orgOrder":0,"company":"Vyant Bio","sponsor":"The Loulou Foundation","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vyant Bio Announces Participation at the 2022 CDKL5 Forum Hosted by the Loulou Foundation on November 7-8, 2022","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Vyant Bio"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"GSK","pharmaFlowCategory":"D","amount":"$3,335.0 million","upfrontCash":"$170.0 million","newsHeadline":"GSK and Wave Life Sciences Announce Collaboration to Drive Discovery and Development of Oligonucleotide Therapeutics Focusing on Novel Genetic Targets","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Wave Life Sciences"}]

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            Details:

            The collaboration includes 2 components, first is a discovery collaboration which enables GSK to advance upto 8 programmes and Wave upto 3 programmes, using Wave’s PRISM platform in genetics and genomics. GSK will in-license Wave’s preclinical programme WVE-006 for AATD.

            Lead Product(s): RNA-based Therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Large molecule

            Partner/Sponsor/Collaborator: GSK

            Deal Size: $3,335.0 million Upfront Cash: $170.0 million

            Deal Type: Collaboration December 13, 2022

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            High-throughput functional screening to develop novel therapies for CDKL5-deficiency disorder using human iPSC-derived cortical organoids.CDKL5 stands for cyclin-dependent kinase-like 5, a protein whose gene is located on the X chromosome.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: The Loulou Foundation

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 27, 2022

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            Ring’s conception of Anellovectors™ as a broad bioplatform that could be applicable across diseases holds tremendous promise for unlocking the field of genetic medicines.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 20, 2022

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            The partnership combines Integral Molecular’s experience in antibody discovery and mRNA immunization with Optimeos’ technology for nanoparticle-based drug delivery systems having nanoparticle-based DNA Therapy.

            Lead Product(s): Nanoparticle-based DNA Therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Optimeos Life Sciences

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership June 08, 2022

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            Partnering with leading engineered cell therapy developers is a key component of Arbor’s strategy to broaden the potential of CRISPR discovery engine beyond the wholly-owned in vivo genetic medicine approaches for the benefit of even more patients.

            Lead Product(s): Ex-vivo Engineered Cell Therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: EdiGene

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement February 08, 2022

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            Proceeds from the financing will be used to advance new classes of disease-modifying drugs into the clinic and to further expand the Company’s unique and proprietary discovery platform.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Small molecule

            Partner/Sponsor/Collaborator: Lightspeed Venture Partners

            Deal Size: $51.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing November 17, 2021

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            The collaboration will combine Metagenomi’s next-generation CRISPR-based and other novel gene editing systems with Moderna’s mRNA and LNP technologies to accelerate the development of in vivo gene editing therapeutics.

            Lead Product(s): In-vivo Gene Editing Therapeutics

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Moderna Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration November 02, 2021

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            PeptiDream will select, optimize, and synthesize peptides for each receptor. Alnylam will then generate peptide-siRNA conjugates and perform in vitro and in vivo studies to support final peptide selection.

            Lead Product(s): Peptide-siRNA Conjugates

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Peptide

            Partner/Sponsor/Collaborator: Alnylam Pharmaceuticals

            Deal Size: $2,200.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration July 29, 2021

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            Deep Genomics uses AI and machine learning to program and prioritize transformational RNA therapeutics for genetic diseases. Company expands AI discovery platform for ‘Programmable’ RNA therapeutics and supports advancement of its portfolio into the clinic.

            Lead Product(s): RNA Therapeutics

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Large molecule

            Partner/Sponsor/Collaborator: SoftBank Vision Fund

            Deal Size: $180.0 million Upfront Cash: Undisclosed

            Deal Type: Series C Financing July 28, 2021

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            Enveda’s platform is the world’s most advanced drug discovery search engine for dark chemical space, building on years of cutting-edge advancements at the intersection of metabolomics and machine learning.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Small molecule

            Partner/Sponsor/Collaborator: Lux Capital

            Deal Size: $51.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing June 22, 2021

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