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[{"orgOrder":0,"company":"Adrestia Therapeutics","sponsor":"GSK","pharmaFlowCategory":"D","amount":"$1,150.0 million","upfrontCash":"Undisclosed","newsHeadline":"GSK and Ahren Announce Co-Led Series A Investment in Adrestia, and Multi-Project Discovery Collaboration for Five Projects","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"UNITED KINGDOM","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Undisclosed","graph2":"Adrestia Therapeutics"},{"orgOrder":0,"company":"Capsida","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Capsida Biotherapeutics Presents Data Demonstrating Unique Industry-Leading Technology and Capabilities With Potential to Treat Both Rare and Common Diseases at the Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Capsida"},{"orgOrder":0,"company":"Generian Pharmaceuticals","sponsor":"Mitobridge","pharmaFlowCategory":"D","amount":"$180.0 million","upfrontCash":"Undisclosed","newsHeadline":"Generian and Astellas Enter into Collaboration and Exclusive License Agreement to Discover and Develop Novel Small Molecules For Undruggable Targets","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Small molecule","productStatus":"Undisclosed","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Generian Pharmaceuticals"},{"orgOrder":0,"company":"AbCellera","sponsor":"Rallybio","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AbCellera and Rallybio Announce Strategic Alliance to Discover, Develop, and Commercialize Novel Antibody-Based Therapeutics for Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"CANADA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"AbCellera"},{"orgOrder":0,"company":"EDIRNA","sponsor":"Sirnaomics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Sirnaomics Establishes a Strategic Partnership with EDIRNA Inc., an RNA-Editing Therapeutic Company","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"EDIRNA"},{"orgOrder":0,"company":"Airna","sponsor":"ARCH Venture Partners","pharmaFlowCategory":"D","amount":"$30.0 million","upfrontCash":"Undisclosed","newsHeadline":"AIRNA Launches to Develop New Class of RNA Editing Therapeutics for Rare and Common Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Airna"},{"orgOrder":0,"company":"Mina Therapeutics","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"MiNA Therapeutics Enters Research Collaboration with Nippon Shinyaku to Develop RNAa Therapeutics Targeting Rare Neurodegenerative Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Mina Therapeutics"}]

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            Details:

            Under the collaboration, MiNA Therapeutics will utilize its proprietary RNAa algorithm and technology platform to identify and characterise RNAa molecules targeting rare genetic diseases of the central nervous system (CNS).

            Lead Product(s): RNAa-based Therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Large molecule

            Partner/Sponsor/Collaborator: Nippon Shinyaku

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration April 04, 2024

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            The financing will advance company's pipeline of RNA editing therapeutics driven by its flexible RNA editing platform, RESTORE+TM. AIRNA is advancing development of a best-in-class product candidate to treat the inherited genetic disease alpha-1 antitrypsin deficiency.

            Lead Product(s): RNA Editing Therapeutic

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Large molecule

            Partner/Sponsor/Collaborator: ARCH Venture Partners

            Deal Size: $30.0 million Upfront Cash: Undisclosed

            Deal Type: Financing September 19, 2023

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            The partnership accelerates EDIRNA's proprietary Edit-to-Cure Therapeutics™ platform for RNA-Editing therapeutics, targeting diseases with high unmet clinical needs.

            Lead Product(s): RNA-editing Therapeutic

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Large molecule

            Partner/Sponsor/Collaborator: Sirnaomics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership July 05, 2023

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            Under the terms of the agreement, AbCellera and Rallybio will co-develop up to five rare disease novel antibody-based therapeutic targets, which will be chosen together by both companies.

            Lead Product(s): Antibody-based Therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Large molecule

            Partner/Sponsor/Collaborator: Rallybio

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration December 01, 2022

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            Under the terms of the agreement, the companies will jointly conduct research and preclinical development activities to identify novel monovalent small molecules that modulate target proteins through activation, stabilization or degradation as potential development candidates.

            Lead Product(s): Undisclosed

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Small molecule

            Partner/Sponsor/Collaborator: Mitobridge

            Deal Size: $180.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration June 29, 2022

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            Adeno-Associated Virus Engineering Platform, Platform utilizes combinatorial engineering and automation to screen in high-throughput, generating capsids greatly improved over AAV9 in CNS efficiency and specificity.

            Lead Product(s): AAV9-based Gene Therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 18, 2022

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            The collaboration will combine Adrestia’s world leading, synthetic viability platform with GSK’s deep scientific expertise in human genetics, functional genomics, screening, and bioinformatics.

            Lead Product(s): Undisclosed

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Undisclosed

            Partner/Sponsor/Collaborator: GSK

            Deal Size: $1,150.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration December 18, 2020

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