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    [{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Bocidelpar","moa":"NR1C3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Astellas Pharma \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Astellas Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Shionogi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Zatolmilast","moa":"Phosphodiesterase 4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Shionogi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Shionogi \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Shionogi \/ 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":"Ileal bile acid transporter","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Takeda Pharmaceutical \/ Mirum Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Takeda Pharmaceutical \/ Mirum 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":"Phosphodiesterase 4D","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Shionogi","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Shionogi \/ Jordan\u2019s Guardian Angels","highestDevelopmentStatusID":"8","companyTruncated":"Shionogi \/ Jordan\u2019s Guardian Angels"},{"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":"Alkaline phosphatase, tissue-nonspecific isozyme","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Daiichi Sankyo \/ Sanford Burnham Prebys","highestDevelopmentStatusID":"8","companyTruncated":"Daiichi Sankyo \/ Sanford Burnham Prebys"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"PXE International","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"DS-1211b","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Daiichi Sankyo \/ PXE International","highestDevelopmentStatusID":"8","companyTruncated":"Daiichi Sankyo \/ PXE International"},{"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":"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":"Injection","sponsorNew":"Ribomic \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Ribomic \/ Inapplicable"},{"orgOrder":0,"company":"Daiichi Sankyo","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"DS-5141b","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Daiichi Sankyo","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Daiichi Sankyo \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Daiichi Sankyo \/ 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":"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":"Injection","sponsorNew":"Ribomic \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Ribomic \/ Inapplicable"},{"orgOrder":0,"company":"Nippon Shinyaku","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"NS-089","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nippon Shinyaku","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Nippon Shinyaku \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Nippon Shinyaku \/ Inapplicable"},{"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":"Retinoic acid receptor alpha | Retinoic acid receptor beta","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Rege Nephro","amount2":0.02,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"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":"Retinoic acid receptor alpha | Retinoic acid receptor beta","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"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            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 : Undisclosed

                            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

                            Shionogi

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                            Lead Product(s) : Zatolmilast

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of Zatolmilast (BPN14770) in Participants With PPP2R5D Neurodevelopmental Disorder (Jordan's Syndrome [J...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            December 05, 2024

                            Lead Product(s) : Zatolmilast

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            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 : Undisclosed

                            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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                            04

                            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 : Undisclosed

                            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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                            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 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 : Undisclosed

                            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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                            06

                            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 : Undisclosed

                            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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                            07

                            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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                            08

                            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 : Undisclosed

                            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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                            09

                            Daiichi Sankyo

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                            Daiichi Sankyo

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                            Lead Product(s) : DS-1211b

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : PXE International

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study of DS-1211b in Individuals With PseudoXanthoma Elasticum

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            October 06, 2022

                            Lead Product(s) : DS-1211b

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : PXE International

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            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 : Undisclosed

                            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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