[{"orgOrder":0,"company":"Genomic Biopharma","sponsor":"POIC e dintorni","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"ITALY","productType":"Undisclosed","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Genomic Biopharma","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genomic Biopharma \/ POIC e dintorni","highestDevelopmentStatusID":"2","companyTruncated":"Genomic Biopharma \/ POIC e dintorni"},{"orgOrder":0,"company":"Exicure","sponsor":"Ipsen","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"IRELAND","productType":"Oligonucleotide","year":"2022","type":"Termination","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Exicure","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Exicure \/ Ipsen","highestDevelopmentStatusID":"2","companyTruncated":"Exicure \/ Ipsen"},{"orgOrder":0,"company":"Scenic Biotech","sponsor":"Barth Syndrome Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Undisclosed","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Scenic Biotech","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Scenic Biotech \/ Barth Syndrome Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Scenic Biotech \/ Barth Syndrome Foundation"},{"orgOrder":0,"company":"ReviR Therapeutics","sponsor":"Kennedy's Disease Association","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"ReviR Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ReviR Therapeutics \/ Kennedy's Disease Association","highestDevelopmentStatusID":"2","companyTruncated":"ReviR Therapeutics \/ Kennedy's Disease Association"},{"orgOrder":0,"company":"Replay Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Replay Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Replay Bio \/ Inapplicable","highestDevelopmentStatusID":"2","companyTruncated":"Replay Bio \/ Inapplicable"},{"orgOrder":0,"company":"Codexis","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Codexis","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Codexis \/ Inapplicable","highestDevelopmentStatusID":"2","companyTruncated":"Codexis \/ Inapplicable"},{"orgOrder":0,"company":"LUCA Science","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"LUCA Science","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"LUCA Science \/ Kyowa Kirin","highestDevelopmentStatusID":"2","companyTruncated":"LUCA Science \/ Kyowa Kirin"},{"orgOrder":0,"company":"Cytiva","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Large Molecule","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Cytiva","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cytiva \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Cytiva \/ n-Lorem Foundation"},{"orgOrder":0,"company":"Scenic Biotech","sponsor":"NICHD","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Undisclosed","year":"2023","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Scenic Biotech","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Scenic Biotech \/ NICHD","highestDevelopmentStatusID":"2","companyTruncated":"Scenic Biotech \/ NICHD"},{"orgOrder":0,"company":"Ethris","sponsor":"Cipla","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"GERMANY","productType":"Oligonucleotide","year":"2022","type":"Collaboration","leadProduct":"ETH45","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Ethris","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Ethris \/ Cipla","highestDevelopmentStatusID":"2","companyTruncated":"Ethris \/ Cipla"},{"orgOrder":0,"company":"Creyon Bio","sponsor":"DCVC Bio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Creyon Bio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Creyon Bio \/ DCVC Bio","highestDevelopmentStatusID":"2","companyTruncated":"Creyon Bio \/ DCVC Bio"},{"orgOrder":0,"company":"Invitae","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Invitae","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Invitae \/ BridgeBio Pharma","highestDevelopmentStatusID":"2","companyTruncated":"Invitae \/ BridgeBio Pharma"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"The Conrad Prebys Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Large Molecule","year":"2022","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"n-Lorem Foundation \/ The Conrad Prebys Foundation","highestDevelopmentStatusID":"2","companyTruncated":"n-Lorem Foundation \/ The Conrad Prebys Foundation"},{"orgOrder":0,"company":"Octantbio","sponsor":"Catalio Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Octantbio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Octantbio \/ Catalio Capital Management","highestDevelopmentStatusID":"2","companyTruncated":"Octantbio \/ Catalio Capital Management"},{"orgOrder":0,"company":"InveniAI","sponsor":"Bristol Myers Squibb","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"InveniAI","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"InveniAI \/ Bristol Myers Squibb","highestDevelopmentStatusID":"2","companyTruncated":"InveniAI \/ Bristol Myers Squibb"},{"orgOrder":0,"company":"Evozyne","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Evozyne","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Evozyne \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"2","companyTruncated":"Evozyne \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Carmine Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Takeda Pharmaceutical \/ Carmine Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Takeda Pharmaceutical \/ Carmine Therapeutics"},{"orgOrder":0,"company":"GenKOre","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SOUTH KOREA","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"GenKOre","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"GenKOre \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"GenKOre \/ Undisclosed"},{"orgOrder":0,"company":"University of Oxford","sponsor":"Evox Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"University of Oxford","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"University of Oxford \/ Evox Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"University of Oxford \/ Evox Therapeutics"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Beam Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Beam Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"2","companyTruncated":"Beam Therapeutics \/ Pfizer Inc"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Acquisition","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"LogicBio Therapeutics \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"LogicBio Therapeutics \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Acquisition","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"LogicBio Therapeutics \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"LogicBio Therapeutics \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Parexel Biotech","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Large Molecule","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Parexel Biotech","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Parexel Biotech \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Parexel Biotech \/ n-Lorem Foundation"},{"orgOrder":0,"company":"Life Edit Therapeutics","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Life Edit Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Life Edit Therapeutics \/ Novo Nordisk","highestDevelopmentStatusID":"2","companyTruncated":"Life Edit Therapeutics \/ Novo Nordisk"},{"orgOrder":0,"company":"RevolKa","sponsor":"DEEPCORE","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Other Large Molecule","year":"2024","type":"Series A Financing","leadProduct":"RKD001","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"RevolKa","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"RevolKa \/ DEEPCORE","highestDevelopmentStatusID":"2","companyTruncated":"RevolKa \/ DEEPCORE"},{"orgOrder":0,"company":"Massachusetts General Hospital","sponsor":"DIMERx","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Licensing 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Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Skyhawk Therapeutics \/ Ipsen","highestDevelopmentStatusID":"2","companyTruncated":"Skyhawk Therapeutics \/ Ipsen"},{"orgOrder":0,"company":"VivaVision","sponsor":"Everads Therapy","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"VivaVision","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"VivaVision \/ Everads Therapy","highestDevelopmentStatusID":"2","companyTruncated":"VivaVision \/ Everads Therapy"},{"orgOrder":0,"company":"SpliceBio","sponsor":"Spark Therapeutics, Inc","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SPAIN","productType":"Undisclosed","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"SpliceBio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"SpliceBio \/ Spark Therapeutics, Inc","highestDevelopmentStatusID":"2","companyTruncated":"SpliceBio \/ Spark Therapeutics, Inc"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"RSRT","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2024","type":"Collaboration","leadProduct":"AX-2402","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ProQR Therapeutics \/ RSRT","highestDevelopmentStatusID":"2","companyTruncated":"ProQR Therapeutics \/ RSRT"}]

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                          01

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : The collaboration will focus on designing and developing AX-2402, a Methyl CpG Binding Protein 2 (MECP2) therapy, for the treatment of Rett syndrome.

                          Product Name : AX-2402

                          Product Type : Oligonucleotide

                          Upfront Cash : Undisclosed

                          December 11, 2024

                          Lead Product(s) : AX-2402

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : RSRT

                          Deal Size : $9.1 million

                          Deal Type : Collaboration

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                          02

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : The parternship aims to identify a small molecule that targets mutant AR mRNA splicing and causes selective destruction of the disease. ReviR aims to develop a potential therapeutic for SBMA.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          November 25, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : Kennedy's Disease Association

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          03

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : The proceeds will accelerate RevolKa’s drug discovery and development activities, including RKD001, a protein in discovery for rare diseases.

                          Product Name : RKD001

                          Product Type : Other Large Molecule

                          Upfront Cash : Undisclosed

                          August 29, 2024

                          Lead Product(s) : RKD001

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : DEEPCORE

                          Deal Size : $1.7 million

                          Deal Type : Series A Financing

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                          04

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : Under the term of license agreement, DIMERx aims to advance the development of AMX-201, currently being evaluated in early-stage trials for treating challenging paradigms across various rare diseases.

                          Product Name : AMX-201

                          Product Type : Other Small Molecule

                          Upfront Cash : Undisclosed

                          May 22, 2024

                          Lead Product(s) : AMX-201

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : DIMERx

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

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                          05

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : The collaboration aims to develop novel small molecules that modulate RNA for rare neurological diseases, including an option for Ipsen to acquire license for successful candidates.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          April 22, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : Ipsen

                          Deal Size : $1,800.0 million

                          Deal Type : Collaboration

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                          06

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : The goal of the collaboration is to generate new insights focused on genetic modifiers and the discovery of novel therapeutic targets for rare diseases and other unmet medical needs.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          January 30, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : BridgeBio Pharma

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          07

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : Under the agreement, Spark secures exclusive worldwide rights to SpliceBio's proprietary Protein Splicing platform to develop, manufacture, and commercialize a gene therapy targeting an undisclosed inherited retinal disease.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          October 17, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : Spark Therapeutics, Inc

                          Deal Size : $216.0 million

                          Deal Type : Licensing Agreement

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                          08

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : Under the terms of collaboration agreement, VivaVision and Everads will jointly conduct pre-clinical research activities, in order to identify lead candidates for future clinical development and commercialization.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          June 01, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : Everads Therapy

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          09

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : 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...

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Undisclosed

                          May 24, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : Novo Nordisk

                          Deal Size : $1,920.0 million

                          Deal Type : Collaboration

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                          10

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

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

                          Product Name : Undisclosed

                          Product Type : Other Large Molecule

                          Upfront Cash : Undisclosed

                          April 10, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : n-Lorem Foundation

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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