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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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Platform","country":"JAPAN","productType":"Peptide","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Peptide","graph2":"PeptiDream"},{"orgOrder":0,"company":"Deep Genomics","sponsor":"SoftBank Vision Fund","pharmaFlowCategory":"D","amount":"$180.0 million","upfrontCash":"Undisclosed","newsHeadline":"Deep Genomics Raises $180M in Series C Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery Platform","country":"CANADA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Deep Genomics"},{"orgOrder":0,"company":"Metagenomi","sponsor":"Moderna Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Moderna and Metagenomi Establish Collaboration to Develop Next-Generation In Vivo Gene Editing Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"November 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Metagenomi"},{"orgOrder":0,"company":"Protego Biopharma","sponsor":"Lightspeed Venture Partners","pharmaFlowCategory":"D","amount":"$51.0 million","upfrontCash":"Undisclosed","newsHeadline":"Protego Biopharma Raises $51 Million Series A Financing to Advance the Treatment of Protein Misfolding Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Small molecule","productStatus":"Undisclosed","date":"November 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Protego Biopharma"},{"orgOrder":0,"company":"Arbor Pharmaceuticals","sponsor":"EdiGene","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"EdiGene and Arbor Biotechnologies Announce Worldwide Non-exclusive License Agreement to 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            Details:

            Under the collaboration, Bristol Myers Squibb gains access to Scenic’s Cell-Seq technology platform to unlock the underlying genetic interactions of cellular pathways for undisclosed targets.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Small molecule

            Partner/Sponsor/Collaborator: Bristol Myers Squibb

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration April 30, 2024

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            The collaboration combines Intellia’s leading CRISPR-based platform, including its DNA writing technology, with ReCode’s proprietary selective Organ Targeting lipid nanoparticle to precisely correct one or more cystic fibrosis disease-causing gene mutations.

            Lead Product(s): CRISPR-based Therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: ReCode Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration February 15, 2024

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            The collaboration will focus on the design and development of editing oligonucleotides (EONs) using ProQR’s Axiomer technology platform targeting the transcription factor Methyl CpG binding protein 2 (MECP2) and correcting mutations of interest.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Large molecule

            Partner/Sponsor/Collaborator: Rett Syndrome Research Trust

            Deal Size: $1.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration January 05, 2024

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            The collaboration aims to accelerate the development of new and more stable enzymes. Creating improved engineering processes for designer enzymes could lead to safer and more effective therapeutics, such as enzyme replacement therapy, for a broad range of genetic diseases.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Large molecule

            Partner/Sponsor/Collaborator: University of Edinburgh

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration December 14, 2023

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            Under the partnership, X-Chem will develop lead candidate, iBRD9, a BRD9 inhibitor, which is an epigenetic reader, as a novel therapeutic target for the treatment of Huntington’s Disease (HD).

            Lead Product(s): iBRD9

            Therapeutic Area: Genetic Disease Product Name: iBRD9

            Highest Development Status: Discovery Platform Product Type: Small molecule

            Partner/Sponsor/Collaborator: X-Chem

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership December 05, 2023

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            The proceeds from the round will fuel the rapid development of next generation of genetic nanomedicines using the polyNaut® platform, expand the scientific and management teams and establish industry-leading laboratory facilities.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Undisclosed

            Partner/Sponsor/Collaborator: SomaServe

            Deal Size: $25.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing November 13, 2023

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            The combined company will focus on the advancement of Korro Bio’s portfolio of RNA editing programs including, OPERA platform which harnesses the body’s natural base editing system, specifically ADAR, to make targeted edits to a single RNA base.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Large molecule

            Partner/Sponsor/Collaborator: Korro Bio

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Merger November 03, 2023

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            The financing will be used to develop novel DNA-based medicines using Rampart’s proprietary DNA-based medicines platform, called HALO, developed using cues from nature to overcome the key limitations and safety concerns of viral and early non-viral gene approaches.

            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Large molecule

            Partner/Sponsor/Collaborator: OrbiMed

            Deal Size: $85.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing October 24, 2023

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            Details:

            The financing will be used to advance the company's platform and first drug candidates towards the clinic for a first indication in Stop Codon Disease.

            Lead Product(s): tRNA Medicine

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Large molecule

            Partner/Sponsor/Collaborator: Flagship Pioneering

            Deal Size: $109.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing August 09, 2023

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            The funding will be used to advance a first-of-its-kind RNA editing platform which enables multi-kilobase edits, allowing a single genetic medicine to treat diseases with high allelic diversity.

            Lead Product(s): Genetic Medicine

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Playground Global

            Deal Size: $26.0 million Upfront Cash: Undisclosed

            Deal Type: Financing August 03, 2023

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