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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":"Evozyne","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Evozyne and Takeda Announce Strategic Collaboration and License Agreement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Large 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Platform","country":"U.S.A","productType":"Small molecule","productStatus":"Undisclosed","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Enveda Bioscience"},{"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 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"}]

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Vyant Bio

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Vyant Bio Announces Participation at the 2022 CDKL5 Forum Hosted by the Loulou Foundation on November 7-8, 2022

Details:

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 Therapeutics

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Ring Therapeutics Announces Issuance of U.S. Patent for its Anellovector™ Compositions

Details:

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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Integral Molecular

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Integral Molecular and Optimeos Life Sciences Enter Partnership to Develop mRNA and DNA-Based Gene Therapies Using Molecular Targeting

Details:

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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Arbor Pharmaceuticals

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EdiGene and Arbor Biotechnologies Announce Worldwide Non-exclusive License Agreement to Advance Ex Vivo Engineered Cell Therapy Programs

Details:

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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Protego Biopharma

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Protego Biopharma Raises $51 Million Series A Financing to Advance the Treatment of Protein Misfolding Diseases

Details:

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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Metagenomi

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Moderna and Metagenomi Establish Collaboration to Develop Next-Generation In Vivo Gene Editing Therapeutics

Details:

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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Enveda Bioscience

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Enveda Biosciences Closes $51M Series A Financing Led by Lux Capital

Details:

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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Obsidian Therapeutics

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Vertex and Obsidian Therapeutics Establish Collaboration to Discover Novel Therapies That Regulate Gene Editing

Details:

This multi-year strategic research collaboration leverages Obsidian’s proprietary cytoDRiVE® platform to discover controllable genetic therapies to treat serious diseases.

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: Vertex

Deal Size: $1,300.0 million Upfront Cash: $75.0 million

Deal Type: Collaboration April 22, 2021

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Evozyne

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Evozyne and Takeda Announce Strategic Collaboration and License Agreement

Details:

In partnership with Takeda, Evozyne will create novel protein sequences for advancement as gene therapies. At completion Takeda has the option to obtain an exclusive license to develop and commercialize the novel protein sequences as part of its gene therapy program.

Lead Product(s): Synthetic proteins

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Discovery Platform Product Type: Large molecule

Partner/Sponsor/Collaborator: Takeda Pharmaceutical

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Collaboration January 25, 2021

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BridgeBio Pharma

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BridgeBio Pharma and The University Of Colorado Anschutz Medical Campus Collaborate To Advance Medicines For Genetically Driven Diseases

Details:

Under the new, expanded collaboration, BridgeBio will support early-discovery research already underway in CU Anschutz labs and will accelerate promising therapies into the clinic in order to develop and ultimately commercialize therapies for patients.

Lead Product(s): Undisclosed

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Discovery Platform Product Type: Undisclosed

Partner/Sponsor/Collaborator: The University Of Colorado Anschutz Medical Campus

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Collaboration October 21, 2020

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