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[{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Carmine Therapeutics","pharmaFlowCategory":"D","amount":"$900.0 million","upfrontCash":"Undisclosed","newsHeadline":"Takeda Partners up with Carmine Therapeutics to Develop and Market Rare Disease Candidates","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Takeda Pharmaceutical"},{"orgOrder":0,"company":"University of Oxford","sponsor":"Evox Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Evox Therapeutics Announces Launch of Strategic Collaboration with the University of Oxford","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"UNITED KINGDOM","productType":"Undisclosed","productStatus":"Undisclosed","date":"November 2020","url1":"","url2":"","graph1":"Undisclosed","graph2":"University of Oxford"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"N-Lorem Foundation Partners with Ultragenyx to Bolster Development of Personalized Medicines for Patients with Ultra-Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"Undisclosed","date":"April 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"InveniAI","sponsor":"Bristol Myers Squibb","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Relief Therapeutics and InveniAI Sign a Strategic Collaboration Agreement to Identify New Product Development Opportunities using Artificial Intelligence","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Undisclosed","productStatus":"Undisclosed","date":"November 2021","url1":"","url2":"","graph1":"Undisclosed","graph2":"InveniAI"},{"orgOrder":0,"company":"Cytiva","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"n-Lorem Foundation Partners with Cytiva to Bring Individualized ASO Medicines to Patients with Nano-rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Cytiva"},{"orgOrder":0,"company":"Parexel Biotech","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Parexel and n-Lorem Foundation Partner to Support Development of New Investigational Therapies for Nano-rare Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Parexel Biotech"},{"orgOrder":0,"company":"Creyon Bio","sponsor":"DCVC Bio","pharmaFlowCategory":"D","amount":"$40.0 million","upfrontCash":"Undisclosed","newsHeadline":"Creyon Bio Launches to Engineer Safe, Effective Precision Medicines on Demand for All Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Creyon Bio"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"The Conrad Prebys Foundation","pharmaFlowCategory":"D","amount":"$0.3 million","upfrontCash":"Undisclosed","newsHeadline":"The Conrad Prebys Foundation to Support the Discovery and Development of Personalized ASO Medicines for Nano-rare Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"n-Lorem Foundation"},{"orgOrder":0,"company":"Octantbio","sponsor":"Catalio Capital Management","pharmaFlowCategory":"D","amount":"$80.0 million","upfrontCash":"Undisclosed","newsHeadline":"Octant Raises $80 Million Series B Round to Support Expansion of Next-Generation Drug Discovery Platform, Announces Collaboration with Bristol Myers Squibb and Key Appointments","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Octantbio"},{"orgOrder":0,"company":"KBio","sponsor":"ZabBio","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"KBio Announces Strategic Partnership with ZabBio to Discover and Develop Antibody Therapeutics","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"KBio"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Alexion, AstraZeneca Rare Disease to Acquire LogicBio\u00ae Therapeutics to Accelerate Growth in Genomic Medicine","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"LogicBio Therapeutics"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Alexion, AstraZeneca Rare Disease, Completes Acquisition of LogicBio\u00ae Therapeutics","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"LogicBio Therapeutics"},{"orgOrder":0,"company":"Genomic Biopharma","sponsor":"POIC e dintorni","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Genomic Biopharma and Patient Advocacy Organization POIC e Dintorni APS Partner To Offer Whole Genome Sequencing To Bring Better Treatments And Cures To Those Living With Gastrointestinal Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"ITALY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Genomic Biopharma"},{"orgOrder":0,"company":"Ethris","sponsor":"Cipla","pharmaFlowCategory":"D","amount":"$16.0 million","upfrontCash":"Undisclosed","newsHeadline":"Cipla EU to Invest EUR 15 Million In Ethris, Initiating A Strategic Collaboration","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"GERMANY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Ethris"},{"orgOrder":0,"company":"GenKOre","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$300.0 million","upfrontCash":"Undisclosed","newsHeadline":"GenKOre Announces Collaboration with a US-based Company on In vivo Gene-editing Therapy","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"SOUTH KOREA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"GenKOre"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","amount":"$1,350.0 million","upfrontCash":"$300.0 million","newsHeadline":"Pfizer and Beam Enter Exclusive Multi-Target Research Collaboration to Advance Novel In Vivo Base Editing Programs for a Range of Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Beam Therapeutics"},{"orgOrder":0,"company":"Scenic Biotech","sponsor":"NICHD","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Scenic Biotech Enters Cooperative Research & Development Agreement with the National Institutes of Health for Genetic Modifier-Based Treatment Approach for Niemann-Pick Type C Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Scenic Biotech"},{"orgOrder":0,"company":"Evozyne","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","amount":"$400.0 million","upfrontCash":"Undisclosed","newsHeadline":"Evozyne Announces Multi-Target Research Collaboration and License Agreement with Takeda to Develop Next-Generation Gene Therapies","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Evozyne"},{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"British Patient Capital","pharmaFlowCategory":"D","amount":"$107.0 million","upfrontCash":"Undisclosed","newsHeadline":"OMass Therapeutics Announces Appointment of New Board Chair and Series B Extension","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"OMass Therapeutics"},{"orgOrder":0,"company":"Life Edit Therapeutics","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","amount":"$1,920.0 million","upfrontCash":"Undisclosed","newsHeadline":"Novo Nordisk and Life Edit Therapeutics Establish Multi-Target Collaboration to Discover and Develop Gene Editing Therapies for Rare and Cardiometabolic Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Life Edit Therapeutics"},{"orgOrder":0,"company":"CAMP4 Therapeutics","sponsor":"Fulcrum Therapeutics","pharmaFlowCategory":"D","amount":"$70.0 million","upfrontCash":"Undisclosed","newsHeadline":"Fulcrum Therapeutics Signs Exclusive Global License Agreement in Rare Hematology","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"CAMP4 Therapeutics"},{"orgOrder":0,"company":"St. Jude Children\u2019s Research Hospital","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"n-Lorem Foundation Collaborates to Support Children With Extremely Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"St. Jude Children\u2019s Research Hospital"}]

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            St. Jude Children’s Research Hospital

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            Details:

            The collaboration aims to accelerate the development of optimized experimental antisense oligonucleotide (ASO) medicines for pediatric patients with extremely rare genetic neurological disorders.

            Lead Product(s): Antisense Oligonucleotide

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Large molecule

            Partner/Sponsor/Collaborator: n-Lorem Foundation

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration October 04, 2023

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            Fulcrum gains worldwide license to intellectual property arising from CAMP4’s DBA program, including the rights for discovery, development, and commercialization of novel therapeutic agents against an undisclosed target for the potential treatment of Diamond-Blackfan Anemia.

            Lead Product(s): Undisclosed

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Small molecule

            Partner/Sponsor/Collaborator: Fulcrum Therapeutics

            Deal Size: $70.0 million Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement July 10, 2023

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            Under the collaboration, Novo Nordisk will leverage Life Edit’s suite of gene editing technologies to precisely edit the genome with the aim of developing therapies for rare genetic disorders and cardiometabolic diseases allowing for the development of up to seven programmes.

            Lead Product(s): Undisclosed

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Novo Nordisk

            Deal Size: $1,920.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration May 24, 2023

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            The financing will be used to to support OMass as it continues to progress its pipeline of small molecule therapeutics for rare diseases and immunological conditions targeting solute carriers, complex-bound proteins and GPCRs.

            Lead Product(s): Undisclosed

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Small molecule

            Partner/Sponsor/Collaborator: British Patient Capital

            Deal Size: $107.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing May 15, 2023

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            CRADA combines Scenic Biotech’s genetic modifier platform with NIH’s patient registry to unlock genetic modifiers as a path to novel therapies for NPC disease. The goal of the collaboration is to obtain clinical insights into genes that can reduce the severity of NPC.

            Lead Product(s): Undisclosed

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Small molecule

            Partner/Sponsor/Collaborator: NICHD

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement February 01, 2023

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            The collaboration will utilize GenKOre's proprietary CRISPR-Cas platform, TaRGET. The TaRGET platform is distinct from the most popular genome editing technology, CRISPR-Cas9, in that the whole editing module can be delivered with a single AAV vector.

            Lead Product(s): Undisclosed

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Undisclosed

            Deal Size: $300.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration January 26, 2023

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            ETH45 is a first-in-class inhalable form of Ethris’ proprietary mRNA for treatment of pulmonary alveolar proteinosis (PAP). ETH45 activates granulocyte-macrophage colony-stimulating factor (GM-CSF) signaling in the presence of anti-GM-CF antibodies.

            Lead Product(s): ETH45

            Therapeutic Area: Rare Diseases and Disorders Product Name: ETH45

            Highest Development Status: Discovery Product Type: Large molecule

            Partner/Sponsor/Collaborator: Cipla

            Deal Size: $16.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration December 29, 2022

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            Through the collaboration, all members of POIC e dintorni and their parents will have access to whole genome sequencing. Genomic Biopharma’s GBI AI, uses predictive algorithms and genomic data mining to support the identification of novel RNA-based therapeutics.

            Lead Product(s): RNA-based Therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Large molecule

            Partner/Sponsor/Collaborator: POIC e dintorni

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership December 12, 2022

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            The acquisition creates an opportunity to accelerate Alexion’s growth in genomic medicines through unique technology, an experienced rare disease R&D team, and expertise in pre-clinical development.

            Lead Product(s): AAV-based Gene Therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Alexion Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Acquisition November 16, 2022

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            LogicBio's gene delivery platforms, coupled with Alexion’s advancements with AstraZeneca, will drive future scientific possibilities and next generation medicines to treat rare genetic diseases.

            Lead Product(s): AAV-based Gene Therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Alexion Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Acquisition October 03, 2022

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