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    [{"orgOrder":0,"company":"Vyant Bio","sponsor":"The Loulou Foundation","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 Platform","graph3":"Vyant Bio","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vyant Bio \/ The Loulou Foundation","highestDevelopmentStatusID":"3","companyTruncated":"Vyant Bio \/ The Loulou Foundation"},{"orgOrder":0,"company":"Andelyn Biosciences","sponsor":"UMass Chan Medical School","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 Platform","graph3":"Andelyn Biosciences","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Andelyn Biosciences \/ UMass Chan Medical School","highestDevelopmentStatusID":"3","companyTruncated":"Andelyn Biosciences \/ UMass Chan Medical School"},{"orgOrder":0,"company":"Capsida","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 Platform","graph3":"Capsida","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Capsida \/ Inapplicable","highestDevelopmentStatusID":"3","companyTruncated":"Capsida \/ Inapplicable"},{"orgOrder":0,"company":"Hongene Biotech","sponsor":"n-Lorem Foundation","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 Platform","graph3":"Hongene Biotech","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Hongene Biotech \/ n-Lorem Foundation","highestDevelopmentStatusID":"3","companyTruncated":"Hongene Biotech \/ n-Lorem Foundation"},{"orgOrder":0,"company":"AbCellera","sponsor":"Rallybio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"AbCellera","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"AbCellera \/ Rallybio","highestDevelopmentStatusID":"3","companyTruncated":"AbCellera \/ Rallybio"},{"orgOrder":0,"company":"Adrestia Therapeutics","sponsor":"GSK","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 Platform","graph3":"Adrestia Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Adrestia Therapeutics \/ GSK","highestDevelopmentStatusID":"3","companyTruncated":"Adrestia Therapeutics \/ GSK"},{"orgOrder":0,"company":"Healx","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2024","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Healx","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Healx \/ Sanofi","highestDevelopmentStatusID":"3","companyTruncated":"Healx \/ Sanofi"},{"orgOrder":0,"company":"EDIRNA","sponsor":"Sirnaomics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Large Molecule","year":"2023","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"EDIRNA","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"EDIRNA \/ Sirnaomics","highestDevelopmentStatusID":"3","companyTruncated":"EDIRNA \/ Sirnaomics"},{"orgOrder":0,"company":"Airna","sponsor":"Arch Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Large Molecule","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Airna","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Airna \/ Arch Ventures","highestDevelopmentStatusID":"3","companyTruncated":"Airna \/ Arch Ventures"},{"orgOrder":0,"company":"Generian Pharmaceuticals","sponsor":"Mitobridge","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 Platform","graph3":"Generian Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Generian Pharmaceuticals \/ Mitobridge","highestDevelopmentStatusID":"3","companyTruncated":"Generian Pharmaceuticals \/ Mitobridge"},{"orgOrder":0,"company":"Syneron Bio","sponsor":"AstraZeneca","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Peptide","year":"2025","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Syneron Bio","amount2":3.48,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":3.48,"dosageForm":"Undisclosed","sponsorNew":"Syneron Bio \/ AstraZeneca","highestDevelopmentStatusID":"3","companyTruncated":"Syneron Bio \/ AstraZeneca"},{"orgOrder":0,"company":"NanoVation Therapeutics","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Undisclosed","year":"2024","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"NanoVation Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NanoVation Therapeutics \/ Novo Nordisk","highestDevelopmentStatusID":"3","companyTruncated":"NanoVation Therapeutics \/ Novo Nordisk"},{"orgOrder":0,"company":"CAMP4 Therapeutics","sponsor":"BioMarin Pharmaceutical","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 Platform","graph3":"CAMP4 Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CAMP4 Therapeutics \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"3","companyTruncated":"CAMP4 Therapeutics \/ BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Mina Therapeutics","sponsor":"Nippon Shinyaku","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"Mina Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mina Therapeutics \/ Nippon Shinyaku","highestDevelopmentStatusID":"3","companyTruncated":"Mina Therapeutics \/ Nippon Shinyaku"},{"orgOrder":0,"company":"AbCellera","sponsor":"Kodiak Sciences","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Antibody","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"AbCellera","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"AbCellera \/ Kodiak Sciences","highestDevelopmentStatusID":"3","companyTruncated":"AbCellera \/ Kodiak Sciences"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Rett Syndrome Research Trust","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Discovery Platform","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ProQR Therapeutics \/ Rett Syndrome Research Trust","highestDevelopmentStatusID":"3","companyTruncated":"ProQR Therapeutics \/ Rett Syndrome Research Trust"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Syneron Bio

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : AstraZeneca

                            Deal Size : $3,475.0 million

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Syneron Bio Announces Strategic Collaboration with AstraZeneca

                            Details : Under this collaboration, AstraZeneca will gain access to Syneron's Synova platform to support the advancements of research programmes exploring possible future treatments of chronic disease.

                            Product Name : Undisclosed

                            Product Type : Peptide

                            Upfront Cash : $75.0 million

                            March 21, 2025

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Discovery Platform

                            Sponsor : AstraZeneca

                            Deal Size : $3,475.0 million

                            Deal Type : Collaboration

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                            02

                            Healx

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : Sanofi

                            Deal Size : Undisclosed

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Healx Partners with Sanofi To Explore Rare Diseases with AI Drug Discovery

                            Details : Under the agreement, Sanofi will provide data related to a late stage discontinued asset to identify new disease indications using Healnet, Healx’s AI-driven drug discovery platform.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            November 13, 2024

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Discovery Platform

                            Sponsor : Sanofi

                            Deal Size : Undisclosed

                            Deal Type : Agreement

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                            03

                            NanoVation Therapeutics

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : Novo Nordisk

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            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.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : $600.0 million

                            September 18, 2024

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Discovery Platform

                            Sponsor : Novo Nordisk

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            04

                            Andelyn Biosciences

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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

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                            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.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            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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                            05

                            ProQR Therapeutics

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                            ProQR Therapeutics

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : Rett Syndrome Research Trust

                            Deal Size : $1.0 million

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            ProQR and Rett Syndrome Research Trust Join Forces with Axiomer™ RNA Editing Collaboration

                            Details : The collaboration focuses on developing editing oligonucleotides (EONs) using ProQR’s Axiomer technology to target MECP2 and correct specific mutations.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            May 01, 2024

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Discovery Platform

                            Sponsor : Rett Syndrome Research Trust

                            Deal Size : $1.0 million

                            Deal Type : Collaboration

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                            06

                            Hongene Biotech

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : n-Lorem Foundation

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            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.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            April 24, 2024

                            Lead Product(s) : Undisclosed

                            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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                            07

                            Mina Therapeutics

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                            Mina Therapeutics

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                            Lead Product(s) : Undisclosed

                            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.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            April 04, 2024

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Discovery Platform

                            Sponsor : Nippon Shinyaku

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            08

                            CAMP4 Therapeutics

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                            CAMP4 Therapeutics

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                            Lead Product(s) : Undisclosed

                            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.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            January 10, 2024

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Discovery Platform

                            Sponsor : BioMarin Pharmaceutical

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            09

                            Airna

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                            Airna

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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Discovery Platform

                            Sponsor : Arch Ventures

                            Deal Size : $30.0 million

                            Deal Type : Financing

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                            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...

                            Product Name : Undisclosed

                            Product Type : Other Large Molecule

                            Upfront Cash : Undisclosed

                            September 19, 2023

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Discovery Platform

                            Sponsor : Arch Ventures

                            Deal Size : $30.0 million

                            Deal Type : Financing

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                            10

                            EDIRNA

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                            EDIRNA

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                            Lead Product(s) : Undisclosed

                            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.

                            Product Name : Undisclosed

                            Product Type : Other Large Molecule

                            Upfront Cash : Undisclosed

                            May 07, 2023

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Discovery Platform

                            Sponsor : Sirnaomics

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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