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    [{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Chugai Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Chugai Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Ribomic","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Umedaptanib Pegol","moa":"FGFR-2\/FGFR-3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ribomic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ribomic \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Ribomic \/ Inapplicable"},{"orgOrder":0,"company":"Ribomic","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Umedaptanib Pegol","moa":"FGFR-2\/FGFR-3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ribomic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ribomic \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Ribomic \/ Inapplicable"},{"orgOrder":0,"company":"Ribomic","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Umedaptanib Pegol","moa":"FGFR-2\/FGFR-3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ribomic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ribomic \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Ribomic \/ Inapplicable"},{"orgOrder":0,"company":"Ribomic","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Umedaptanib Pegol","moa":"FGFR-2\/FGFR-3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ribomic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ribomic \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Ribomic \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Mirum Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Maralixibat","moa":"IBAT","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Takeda Pharmaceutical \/ Mirum Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Takeda Pharmaceutical \/ Mirum Pharmaceuticals"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Arrowhead Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Takeda Pharmaceutical \/ Arrowhead Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Takeda Pharmaceutical \/ Arrowhead Pharmaceuticals"},{"orgOrder":0,"company":"Shionogi","sponsor":"Jordan\u2019s Guardian Angels","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2025","type":"Collaboration","leadProduct":"Zatolmilast","moa":"PDE4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Shionogi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Shionogi \/ Jordan\u2019s Guardian Angels","highestDevelopmentStatusID":"8","companyTruncated":"Shionogi \/ Jordan\u2019s Guardian Angels"},{"orgOrder":0,"company":"Rege Nephro","sponsor":"DCI Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2024","type":"Series B Financing","leadProduct":"Tamibarotene","moa":"RAR-alpha","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Rege Nephro","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rege Nephro \/ DCI Partners","highestDevelopmentStatusID":"8","companyTruncated":"Rege Nephro \/ DCI Partners"},{"orgOrder":0,"company":"Rege Nephro","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2024","type":"Inapplicable","leadProduct":"Tamibarotene","moa":"RAR-alpha","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Rege Nephro","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rege Nephro \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Rege Nephro \/ Inapplicable"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Sanford Burnham Prebys","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"N-Acetyl-D-Leucine","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Daiichi Sankyo \/ Sanford Burnham Prebys","highestDevelopmentStatusID":"8","companyTruncated":"Daiichi Sankyo \/ Sanford Burnham Prebys"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Shionogi

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                            Shionogi

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                            Lead Product(s) : Zatolmilast

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Jordan’s Guardian Angels

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Shionogi, Jordan’s Guardian Angels Begin Jordan’s Syndrome Drug Study

                            Details : The collaboration aims to advance the clinical development of BPN14770 (zatolmilast), which is being evaluated in the mid-stage clinical trial studies for the treatment of Jordan's Syndrome.

                            Product Name : BPN14770

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            February 04, 2025

                            Lead Product(s) : Zatolmilast

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Jordan’s Guardian Angels

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            02

                            Rege Nephro

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                            Lead Product(s) : Tamibarotene

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : DCI Partners

                            Deal Size : $16.7 million

                            Deal Type : Series B Financing

                            DEALS_DEV arrow-down

                            Rege Nephro Completes Series B Funding For Kidney Disease Therapy Trials

                            Details : The funds will be used to advance the clinical trials of RN-014 (tamibarotene), company's retinoic acid receptor (RAR) agonist treatment, in Phase 2 for Autosomal Dominant Polycystic Kidney Disease.

                            Product Name : RN-014

                            Product Type : Vitamins/Minerals/Inorganic Salts

                            Upfront Cash : Undisclosed

                            July 10, 2024

                            Lead Product(s) : Tamibarotene

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : DCI Partners

                            Deal Size : $16.7 million

                            Deal Type : Series B Financing

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                            03

                            Rege Nephro

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                            Lead Product(s) : Tamibarotene

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Rege Nephro Enrolls Patients For Phase II Tamibarotene ADPKD Trial

                            Details : RN-014 (tamibarotene), under development for ADPKD, is a retinoic acid receptor (RAR) agonist expected to potently inhibit cyst formation and improve renal function.

                            Product Name : RN-014

                            Product Type : Vitamins/Minerals/Inorganic Salts

                            Upfront Cash : Inapplicable

                            May 28, 2024

                            Lead Product(s) : Tamibarotene

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Ribomic

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                            Ribomic

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                            Lead Product(s) : Umedaptanib Pegol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            RIBOMIC Shows Positive Results in Achondroplasia with Umedaptanib Pegol

                            Details : RBM-007 (umedaptanib pegol) is a novel oligonucleotide-based aptamer, with potent anti-FGF2 activity and is expected to treat Achondroplasia in pediatrics.

                            Product Name : RBM-007

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            May 11, 2024

                            Lead Product(s) : Umedaptanib Pegol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Ribomic

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                            Ribomic

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                            Lead Product(s) : Umedaptanib Pegol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            RIBOMIC Completes Enrollment in Early Phase 2 Achondroplasia Study

                            Details : RBM-007 (umedaptanib pegol) is a novel oligonucleotide-based aptamer, with potent anti-FGF2 activity and is expected to treat Achondroplasia.

                            Product Name : RBM-007

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            March 08, 2024

                            Lead Product(s) : Umedaptanib Pegol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Chugai Pharmaceutical

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                            Chugai Pharmaceutical

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                            Lead Product(s) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Chugai Files for Evrysdi Additional Indication for Pre-Symptomatic SMA in Infants-2 Months

                            Details : Evrysdi (risdiplam) is a survival of motor neuron 2 (SMN2) splicing modifier, which is being evaluated for the treatment of infants with pre-symptomatic spinal muscular atrophy.

                            Product Name : Evrysdi

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            February 15, 2024

                            Lead Product(s) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Takeda Pharmaceutical

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                            Takeda Pharmaceutical

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                            Lead Product(s) : Fazirsiran

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Arrowhead Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Arrowhead and Takeda Announce Topline Results from SEQUOIA Phase 2 Study of Fazirsiran in Patients with Alpha-...

                            Details : TAK-999 (fazirsiran) is a potential first-in-class investigational RNA interference (RNAi) therapy designed to reduce the production of mutant alpha-1 antitrypsin protein (Z-AAT) as a potential treatment for the rare genetic liver disease associated with...

                            Product Name : TAK-999

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            September 01, 2023

                            Lead Product(s) : Fazirsiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Arrowhead Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Daiichi Sankyo

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                            Daiichi Sankyo

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                            Lead Product(s) : N-Acetyl-D-Leucine

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Sanford Burnham Prebys

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Sanford Burnham Prebys Announces Start Of Phase 2 Clinical Trial Of DS-1211 In Individuals With PseudoXanthoma...

                            Details : DS-1211 is a potent and specific small-molecule inhibitor of TNAP. In vitro studies characterized the mode of inhibition and inhibitory effects of DS-1211 on three human ALP isozymes-TNAP, human intestinal ALP, human placental ALP.

                            Product Name : DS-1211

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 12, 2022

                            Lead Product(s) : N-Acetyl-D-Leucine

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Sanford Burnham Prebys

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Takeda Pharmaceutical

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                            Takeda Pharmaceutical

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                            Lead Product(s) : Maralixibat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Mirum Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Mirum Pharmaceuticals and Takeda Enter into Exclusive Licensing Agreement to Develop and Commercialize Maralix...

                            Details : Under the terms of the agreement, Takeda will be responsible for regulatory approval and commercialization of maralixibat in Japan. Takeda will also be responsible for development, including conducting clinical studies in cholestatic indications.

                            Product Name : LUM001

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            September 21, 2021

                            Lead Product(s) : Maralixibat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Mirum Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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