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    [{"orgOrder":0,"company":"Adrestia Therapeutics","sponsor":"GSK","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Adrestia Therapeutics","amount2":1.1499999999999999,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":1.1499999999999999,"dosageForm":"","sponsorNew":"Adrestia Therapeutics \/ GlaxoSmithKline","highestDevelopmentStatusID":"3","companyTruncated":"Adrestia Therapeutics \/ GlaxoSmithKline"},{"orgOrder":0,"company":"Capsida","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AAV9-based Gene Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Capsida","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Capsida \/ Not Applicable","highestDevelopmentStatusID":"3","companyTruncated":"Capsida \/ Not Applicable"},{"orgOrder":0,"company":"Generian Pharmaceuticals","sponsor":"Mitobridge","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Generian Pharmaceuticals","amount2":0.17999999999999999,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0.17999999999999999,"dosageForm":"","sponsorNew":"Generian Pharmaceuticals \/ Mitobridge","highestDevelopmentStatusID":"3","companyTruncated":"Generian Pharmaceuticals \/ Mitobridge"},{"orgOrder":0,"company":"AbCellera","sponsor":"Rallybio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2022","type":"Collaboration","leadProduct":"Antibody-based Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"AbCellera","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"AbCellera \/ Rallybio","highestDevelopmentStatusID":"3","companyTruncated":"AbCellera \/ Rallybio"},{"orgOrder":0,"company":"EDIRNA","sponsor":"Sirnaomics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Partnership","leadProduct":"RNA-editing Therapeutic","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"EDIRNA","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"EDIRNA \/ Sirnaomics","highestDevelopmentStatusID":"3","companyTruncated":"EDIRNA \/ Sirnaomics"},{"orgOrder":0,"company":"Airna","sponsor":"ARCH Venture Partners","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Financing","leadProduct":"RNA Editing Therapeutic","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Airna","amount2":0.029999999999999999,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"Airna \/ ARCH Venture Partners","highestDevelopmentStatusID":"3","companyTruncated":"Airna \/ ARCH Venture Partners"},{"orgOrder":0,"company":"Mina Therapeutics","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Collaboration","leadProduct":"RNAa-based Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Mina Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","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","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2024","type":"Partnership","leadProduct":"ASO-based Therapy","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Hongene Biotech","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Hongene Biotech \/ n-Lorem Foundation","highestDevelopmentStatusID":"3","companyTruncated":"Hongene Biotech \/ n-Lorem Foundation"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            CAMP4 Therapeutics

                            U.S.A arrow
                            World Vaccine Congress
                            Not Confirmed

                            CAMP4 Therapeutics

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                            World Vaccine Congress
                            Not Confirmed
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                            • WHO-GMP
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                            Lead Product(s) : regRNA-based Therapy

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : BioMarin Pharmaceutical

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            CAMP4 Partners on Research to Develop RNA-Targeting Medicines for Rare Diseases

                            Details : The collaboration combines BioMarin’s expertise in drug development with CAMP4’s platform-based target discovery capabilities to advance novel regRNA-targeting medicines for rare genetic conditions.

                            Brand Name : Undisclosed

                            Molecule Type : Large molecule

                            Upfront Cash : Undisclosed

                            October 01, 2024

                            Lead Product(s) : regRNA-based Therapy

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Discovery Platform

                            Sponsor : BioMarin Pharmaceutical

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            02

                            NanoVation Therapeutics

                            Canada arrow
                            World Vaccine Congress
                            Not Confirmed

                            NanoVation Therapeutics

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                            World Vaccine Congress
                            Not Confirmed
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                            • EDQM
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                            Lead Product(s) : RNA-based Therapy

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : Novo Nordisk

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            Novo Nordisk Bets $600M on NanoVation for Genetic Medicines Beyond Liver

                            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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                            03

                            Andelyn Biosciences

                            U.S.A arrow
                            World Vaccine Congress
                            Not Confirmed

                            Andelyn Biosciences

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                            World Vaccine Congress
                            Not Confirmed
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : UMass Chan Medical School

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            Andelyn Biosciences, UMass Partner on Cockayne Syndrome

                            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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                            04

                            Hongene Biotech

                            U.S.A arrow
                            World Vaccine Congress
                            Not Confirmed

                            Hongene Biotech

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                            World Vaccine Congress
                            Not Confirmed
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                            • WHO-GMP
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                            Lead Product(s) : ASO-based Therapy

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : n-Lorem Foundation

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            n-Lorem Partners with Hongene for Personalized ASO Medicines

                            Details : The partnership will discover personalized antisense oligonucleotide medicines for nano-rare disease patients, with Hongene providing essential compounds to n-Lorem to offset development costs.

                            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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                            05

                            Mina Therapeutics

                            United Kingdom arrow
                            World Vaccine Congress
                            Not Confirmed

                            Mina Therapeutics

                            United Kingdom arrow
                            World Vaccine Congress
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : RNAa-based Therapy

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : Nippon Shinyaku

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            MiNA Therapeutics Collaborates with Nippon Shinyaku On RNAa Therapies

                            Details : MiNA Therapeutics will use its RNAa algorithm and platform to identify RNAa molecules targeting rare CNS genetic diseases under the collaboration.

                            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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                            06

                            Airna

                            U.S.A arrow
                            SupplySide West 2024
                            Not Confirmed

                            Airna

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                            SupplySide West 2024
                            Not Confirmed
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                            Lead Product(s) : RNA Editing Therapeutic

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : ARCH Venture Partners

                            Deal Size : $30.0 million

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            AIRNA Launches to Develop New Class of RNA Editing Therapeutics for Rare and Common Diseases

                            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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                            07

                            EDIRNA

                            U.S.A arrow
                            SupplySide West 2024
                            Not Confirmed

                            EDIRNA

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                            SupplySide West 2024
                            Not Confirmed
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                            • EDQM
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                            Lead Product(s) : RNA-editing Therapeutic

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : Sirnaomics

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            Sirnaomics Establishes a Strategic Partnership with EDIRNA Inc., an RNA-Editing Therapeutic Company

                            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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                            08

                            AbCellera

                            Canada arrow
                            SupplySide West 2024
                            Not Confirmed

                            AbCellera

                            Canada arrow
                            SupplySide West 2024
                            Not Confirmed
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                            • EDQM
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                            Lead Product(s) : Antibody-based Therapy

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : Rallybio

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            AbCellera and Rallybio Announce Strategic Alliance to Discover, Develop, and Commercialize Novel Antibody-Base...

                            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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                            09

                            Generian Pharmaceuticals

                            U.S.A arrow
                            SupplySide West 2024
                            Not Confirmed

                            Generian Pharmaceuticals

                            U.S.A arrow
                            SupplySide West 2024
                            Not Confirmed
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : Mitobridge

                            Deal Size : $180.0 million

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Generian and Astellas Enter into Collaboration and Exclusive License Agreement to Discover and Develop Novel S...

                            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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                            10

                            Capsida

                            U.S.A arrow
                            SupplySide West 2024
                            Not Confirmed

                            Capsida

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                            SupplySide West 2024
                            Not Confirmed
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                            • EDQM
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                            Lead Product(s) : AAV9-based Gene Therapy

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Capsida Biotherapeutics Presents Data Demonstrating Unique Industry-Leading Technology and Capabilities With P...

                            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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