[{"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":"","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"December 2020","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Adrestia Therapeutics","amount2":1.1499999999999999,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery Platform","highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":1.1499999999999999,"dosageForm":"","sponsorNew":"Adrestia Therapeutics \/ GlaxoSmithKline","highestDevelopmentStatusID":"3","companyTruncated":"Adrestia Therapeutics \/ GlaxoSmithKline"},{"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":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","year":"2022","type":"Not Applicable","leadProduct":"AAV9-based Gene Therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Capsida","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery Platform","highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Capsida \/ Not Applicable","highestDevelopmentStatusID":"3","companyTruncated":"Capsida \/ Not Applicable"},{"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":"","productType":"Small molecule","productStatus":"Undisclosed","date":"June 2022","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Generian Pharmaceuticals","amount2":0.17999999999999999,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery Platform","highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0.17999999999999999,"dosageForm":"","sponsorNew":"Generian Pharmaceuticals \/ Mitobridge","highestDevelopmentStatusID":"3","companyTruncated":"Generian Pharmaceuticals \/ Mitobridge"},{"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":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 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Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2024","year":"2024","type":"Collaboration","leadProduct":"RNAa-based Therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Mina Therapeutics","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery Platform","highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Mina Therapeutics \/ Nippon Shinyaku","highestDevelopmentStatusID":"3","companyTruncated":"Mina Therapeutics \/ Nippon Shinyaku"},{"orgOrder":0,"company":"Hongene Biotech","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"n-Lorem Foundation Partners with Hongene Biotech Corporation to Support the Discovery of Personalized ASO Medicines for Nano-rare Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2024","year":"2024","type":"Partnership","leadProduct":"ASO-based Therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Hongene Biotech","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery Platform","highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Hongene Biotech \/ n-Lorem Foundation","highestDevelopmentStatusID":"3","companyTruncated":"Hongene Biotech \/ n-Lorem Foundation"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          Discovery on Target
                          Not Confirmed
                          Discovery on Target
                          Not Confirmed

                          Details : The partnership combines NanoVation’s proprietary lcLNP™ technology for RNA delivery with Novo Nordisk’s expertise in cardiometabolic and rare disease R&D and clinical translation.

                          Brand Name : Undisclosed

                          Molecule Type : Large molecule

                          Upfront Cash : $600.0 million

                          September 18, 2024

                          Lead Product(s) : RNA-based Therapy

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery Platform

                          Sponsor : Novo Nordisk

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          02

                          Discovery on Target
                          Not Confirmed
                          Discovery on Target
                          Not Confirmed

                          Details : Through the partnership, Andelyn Biosciences will leverage suspension AAV Curator Platform to manufacture clinical grade AAV9-CSA vector to treat Cockayne syndrome.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          July 18, 2024

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery Platform

                          Sponsor : UMass Chan Medical School

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          03

                          Discovery on Target
                          Not Confirmed
                          Discovery on Target
                          Not Confirmed

                          Details : The partnership aims to discover and provide personalized experimental antisense oligonucleotide medicines to nano-rare disease patients. Hongene will provide amidites, compounds essential to developing ASO medicines, to n-Lorem to offset the costs of de...

                          Brand Name : Undiclosed

                          Molecule Type : Large molecule

                          Upfront Cash : Undisclosed

                          April 24, 2024

                          Lead Product(s) : ASO-based Therapy

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery Platform

                          Sponsor : n-Lorem Foundation

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          04

                          Discovery on Target
                          Not Confirmed
                          Discovery on Target
                          Not Confirmed

                          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).

                          Brand Name : Undisclosed

                          Molecule Type : Large molecule

                          Upfront Cash : Undisclosed

                          April 04, 2024

                          Lead Product(s) : RNAa-based Therapy

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery Platform

                          Sponsor : Nippon Shinyaku

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          05

                          Discovery on Target
                          Not Confirmed
                          Discovery on Target
                          Not Confirmed

                          Details : 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 antitryp...

                          Brand Name : Undisclosed

                          Molecule Type : Large molecule

                          Upfront Cash : Undisclosed

                          September 19, 2023

                          Lead Product(s) : RNA Editing Therapeutic

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery Platform

                          Sponsor : ARCH Venture Partners

                          Deal Size : $30.0 million

                          Deal Type : Financing

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                          06

                          Discovery on Target
                          Not Confirmed
                          Discovery on Target
                          Not Confirmed

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

                          Brand Name : Undisclosed

                          Molecule Type : Large molecule

                          Upfront Cash : Undisclosed

                          July 05, 2023

                          Lead Product(s) : RNA-editing Therapeutic

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery Platform

                          Sponsor : Sirnaomics

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          07

                          Discovery on Target
                          Not Confirmed
                          Discovery on Target
                          Not Confirmed

                          Details : 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.

                          Brand Name : Undisclosed

                          Molecule Type : Large molecule

                          Upfront Cash : Undisclosed

                          December 01, 2022

                          Lead Product(s) : Antibody-based Therapy

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery Platform

                          Sponsor : Rallybio

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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                          08

                          Discovery on Target
                          Not Confirmed
                          Discovery on Target
                          Not Confirmed

                          Details : 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 ...

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          June 29, 2022

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery Platform

                          Sponsor : Mitobridge

                          Deal Size : $180.0 million

                          Deal Type : Collaboration

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                          09

                          Discovery on Target
                          Not Confirmed
                          Discovery on Target
                          Not Confirmed

                          Details : 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.

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          May 18, 2022

                          Lead Product(s) : AAV9-based Gene Therapy

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery Platform

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Discovery on Target
                          Not Confirmed
                          Discovery on Target
                          Not Confirmed

                          Details : 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.

                          Brand Name : Undisclosed

                          Molecule Type : Undisclosed

                          Upfront Cash : Undisclosed

                          December 18, 2020

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Discovery Platform

                          Sponsor : GSK

                          Deal Size : $1,150.0 million

                          Deal Type : Collaboration

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