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Inapplicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Deutivacaftor","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Eplontersen Sodium","moa":"TTR mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Medis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Other Small Molecule","year":"2025","type":"Agreement","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Italfarmaco S.p.A","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"Italfarmaco S.p.A \/ Medis","highestDevelopmentStatusID":"15","companyTruncated":"Italfarmaco S.p.A \/ Medis"},{"orgOrder":0,"company":"Nexus Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Tranexamic Acid","moa":"Plasminogen","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Nexus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Nexus Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Nexus Pharmaceuticals \/ Inapplicable"}]

Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                          01

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Tranexamic Acid in Sodium Chloride Injection is an antifibrinolytic indicated in patients with hemophilia for shortterm use to reduce or prevent hemorrhage.

                          Product Name : Tranexamic Acid Sodium Chloride Injection

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 19, 2025

                          Lead Product(s) : Tranexamic Acid

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Under the agreement, Medis will have exclusive distribution rights of Duvyzat (Givinostat HCl). It is being indicated for the treatment of DMD.

                          Product Name : Duvyzat

                          Product Type : Other Small Molecule

                          Upfront Cash : Undisclosed

                          March 18, 2025

                          Lead Product(s) : Givinostat HCl

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Medis

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          03

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Waiznua (eplontersen) is a transthyretin-directed antisense oligonucleotide. It is indicated for adults with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis.

                          Product Name : Wainzua

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          March 10, 2025

                          Lead Product(s) : Eplontersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Alyftrek (deutivacaftor/tezacaftor/vanzacaftor), a once-daily next-in-class CFTR modulator, which is indicated for the treatment of cystic fibrosis.

                          Product Name : Alyftrek

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 07, 2025

                          Lead Product(s) : Deutivacaftor,Tezacaftor,Vanzacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Daybue (trofinetide) is a inhibitor of CYP3A4, indicated for Rett syndrome, a rare neurodevelopmental disorder in 2 year and older.

                          Product Name : Daybue

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          March 05, 2025

                          Lead Product(s) : Trofinetide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Sucraid (sacrosidase) oral solution is indicated for treatment of sucrase deficiency, which is part of congenital sucrase-isomaltase deficiency, in adult & pediatric patients 5 months of age & older.

                          Product Name : Sucraid

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          March 01, 2025

                          Lead Product(s) : Sacrosidase

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Vyjuvek (beremagene geperpavec) is a non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds.

                          Product Name : Vyjuvek

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 28, 2025

                          Lead Product(s) : Beremagene Geperpavec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Takhzyro (lanadelumab) is an antibody that specifically binds and decreases plasma kallikrein activity and is indicated for prophylaxis to prevent attacks of HAE in patients aged 12 years and older.

                          Product Name : Takhzyro

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          February 24, 2025

                          Lead Product(s) : Lanadelumab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Andembry (garadacimab) is a novel Factor XIIa-inhibitory monoclonal antibody. It is under development as a once-monthly subcutaneous prophylactic treatment for attacks related to HAE.

                          Product Name : Andembry

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          February 20, 2025

                          Lead Product(s) : Garadacimab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Under the agreement, Taiba will be responsible for the distribution and commercialization of Xolremdi (mavorixafor), an oral, once-daily treatment for WHIM syndrome in MENA region.

                          Product Name : Xolremdi

                          Product Type : Other Small Molecule

                          Upfront Cash : Undisclosed

                          February 19, 2025

                          Lead Product(s) : Mavorixafor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Taiba Healthcare

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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