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therapeutics","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"University of Oxford","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"University of Oxford \/ Evox Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"University of Oxford \/ Evox Therapeutics"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2021","type":"Collaboration","leadProduct":"Antisense oligonucleotide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Ultragenyx Pharmaceutical \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Ultragenyx Pharmaceutical \/ n-Lorem Foundation"},{"orgOrder":0,"company":"InveniAI","sponsor":"Bristol Myers Squibb","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"InveniAI","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Dosage Strength\/Form","sponsorNew":"InveniAI \/ Bristol Myers Squibb","highestDevelopmentStatusID":"2","companyTruncated":"InveniAI \/ Bristol Myers Squibb"},{"orgOrder":0,"company":"Cytiva","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Partnership","leadProduct":"Antisense Oligonucleotide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Cytiva","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Cytiva \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Cytiva \/ n-Lorem Foundation"},{"orgOrder":0,"company":"Parexel Biotech","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Partnership","leadProduct":"Antisense Oligonucleotide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Parexel Biotech","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Parexel Biotech \/ n-Lorem","highestDevelopmentStatusID":"2","companyTruncated":"Parexel Biotech \/ n-Lorem"},{"orgOrder":0,"company":"Creyon Bio","sponsor":"DCVC Bio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Series A Financing","leadProduct":"Oligonucleotide-based Medicine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Creyon Bio","amount2":0.040000000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0.040000000000000001,"dosageForm":"","sponsorNew":"Creyon Bio \/ DCVC Bio","highestDevelopmentStatusID":"2","companyTruncated":"Creyon Bio \/ DCVC Bio"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"The Conrad Prebys Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Funding","leadProduct":"Antisense Oligonucleotide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"n-Lorem Foundation","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","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":"","productType":"Small molecule","year":"2022","type":"Series B Financing","leadProduct":"Chaperon Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Octantbio","amount2":0.080000000000000002,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"Octantbio \/ Catalio Capital Management","highestDevelopmentStatusID":"2","companyTruncated":"Octantbio \/ Catalio Capital Management"},{"orgOrder":0,"company":"KBio","sponsor":"ZabBio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Partnership","leadProduct":"Monoclonal Antibody-based Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"KBio","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"KBio \/ ZabBio","highestDevelopmentStatusID":"2","companyTruncated":"KBio \/ ZabBio"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Acquisition","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","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":"","productType":"Cell and Gene therapy","year":"2022","type":"Acquisition","leadProduct":"AAV-based Gene Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"LogicBio Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"LogicBio Therapeutics \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"LogicBio Therapeutics \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Genomic Biopharma","sponsor":"POIC e dintorni","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Partnership","leadProduct":"RNA-based Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Genomic Biopharma","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Genomic Biopharma \/ POIC e dintorni","highestDevelopmentStatusID":"2","companyTruncated":"Genomic Biopharma \/ POIC e dintorni"},{"orgOrder":0,"company":"Ethris","sponsor":"Cipla","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Collaboration","leadProduct":"ETH45","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Ethris","amount2":0.02,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Inhalation","sponsorNew":"Ethris \/ Cipla","highestDevelopmentStatusID":"2","companyTruncated":"Ethris \/ Cipla"},{"orgOrder":0,"company":"GenKOre","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"GenKOre","amount2":0.29999999999999999,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":0.29999999999999999,"dosageForm":"","sponsorNew":"GenKOre \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"GenKOre \/ Undisclosed"},{"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":"Precision Genetic Medicine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Beam Therapeutics","amount2":1.3500000000000001,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","amount2New":1.3500000000000001,"dosageForm":"","sponsorNew":"Beam Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"2","companyTruncated":"Beam Therapeutics \/ Pfizer Inc"},{"orgOrder":0,"company":"Scenic Biotech","sponsor":"NICHD","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Agreement","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Scenic 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Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

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

                          Brand Name : RKD001

                          Molecule Type : 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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                          02

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : The agreement aims to proceed with research for an adeno-associated virus (AAV) vector mediated gene therapy for the treatment of Alexander disease, a fatal, ultra-rare disease.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          June 20, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : UMass Chan Medical School

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          03

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : The collaboration will seek to advance a novel TCR T-cell therapy, TCX-201, targeting recurrent respiratory papillomatosis (RRP), a rare orphan disease.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          June 04, 2024

                          Lead Product(s) : TCR T-cell Therapy

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : National Cancer Institute

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          04

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          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.

                          Brand Name : AMX-201

                          Molecule Type : 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

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          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.

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          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

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          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.

                          Brand Name : Undisclosed

                          Molecule 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

                          St. Jude Children’s Research Hospital

                          Country arrow
                          Medlab Asia & Asia Health
                          Not Confirmed

                          St. Jude Children’s Research Hospital

                          Country arrow
                          Medlab Asia & Asia Health
                          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.

                          Brand Name : Undisclosed

                          Molecule Type : Large molecule

                          Upfront Cash : Undisclosed

                          October 04, 2023

                          Lead Product(s) : Antisense Oligonucleotide

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : n-Lorem Foundation

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          08

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : 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 D...

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          July 10, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : Fulcrum Therapeutics

                          Deal Size : $70.0 million

                          Deal Type : Licensing Agreement

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                          09

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          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...

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          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

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : 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.

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          May 15, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery

                          Sponsor : British Patient Capital

                          Deal Size : $107.0 million

                          Deal Type : Series B Financing

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