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II","graph3":"PepGen","amount2":0.12,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Leerink Partners"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Private Placement","leadProduct":"Tralesinidase Alfa","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0.050000000000000003,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Infusion","sponsorNew":"Spruce Biosciences \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Leerink Partners"},{"orgOrder":0,"company":"Ribomic","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Umedaptanib Pegol","moa":"FGF2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ribomic","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ribomic \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ribomic \/ Undisclosed"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"WVE-N531","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Wave Life Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Wave Life Sciences \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Wave Life Sciences \/ Undisclosed"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"MRM-3379","moa":"Type 4 cyclic nucleotide phosphodiesterase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Mirum Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Mirum Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"BAY 3401016","moa":"SEMA3A","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Macupatide","moa":"GIPR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eli Lilly \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Eli Lilly \/ Undisclosed"},{"orgOrder":0,"company":"The Emmes Company, LLC","sponsor":"Rachel G. Greenberg, MD, MB, MHS","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Guanfacine","moa":"Adrenergic receptor alpha-2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"The Emmes Company, LLC","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"The Emmes Company, LLC \/ Rachel G. Greenberg, MD, MB, MHS","highestDevelopmentStatusID":"8","companyTruncated":"The Emmes Company, LLC \/ Rachel G. Greenberg, MD, MB, MHS"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Scott R. Plotkin, MD, PhD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Brigatinib","moa":"ALK tyrosine kinase receptor | Epidermal growth factor receptor erbB1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Takeda Pharmaceutical \/ Scott R. Plotkin, MD, PhD","highestDevelopmentStatusID":"8","companyTruncated":"Takeda Pharmaceutical \/ Scott R. Plotkin, MD, PhD"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Region Skane","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Miscellaneous","year":"2018","type":"Inapplicable","leadProduct":"Trametinib","moa":"Dual specificity mitogen-activated protein kinase kinase; MEK1\/2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Region Skane","highestDevelopmentStatusID":"8","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Region Skane"},{"orgOrder":0,"company":"Healx","sponsor":"Craig Erickson","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Gaboxadol","moa":"GABA-A receptor; agonist GABA site","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Healx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Healx \/ Craig Erickson","highestDevelopmentStatusID":"8","companyTruncated":"Healx \/ Craig Erickson"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Jerry Vockley","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Acer Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Acer Therapeutics \/ Jerry Vockley","highestDevelopmentStatusID":"8","companyTruncated":"Acer Therapeutics \/ Jerry Vockley"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Jerry Vockley","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zevra Therapeutics \/ Jerry Vockley","highestDevelopmentStatusID":"8","companyTruncated":"Zevra Therapeutics \/ Jerry Vockley"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Michael Campos, MD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2012","type":"Inapplicable","leadProduct":"Alpha 1-Antitrypsin","moa":"Leukocyte elastase","graph1":"Genetic Disease","graph2":"Phase II","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Michael Campos, MD","highestDevelopmentStatusID":"8","companyTruncated":"CSL Behring \/ Michael Campos, MD"},{"orgOrder":0,"company":"Dompe Farmaceutici","sponsor":"Ospedale San Raffaele","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Protein","year":"2015","type":"Inapplicable","leadProduct":"Recombinant Human Nerve Growth Factor","moa":"Neurotrophic tyrosine kinase receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Dompe Farmaceutici","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Dompe Farmaceutici \/ Ospedale San Raffaele","highestDevelopmentStatusID":"8","companyTruncated":"Dompe Farmaceutici \/ Ospedale San Raffaele"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"John Mark Sloan","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Idelalisib","moa":"PI3-kinase p110-delta subunit","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gilead Sciences \/ John Mark Sloan","highestDevelopmentStatusID":"8","companyTruncated":"Gilead Sciences \/ John Mark Sloan"},{"orgOrder":0,"company":"Boehringer Ingelheim GmbH","sponsor":"Romain Lazor","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Nintedanib Esylate","moa":"Platelet-derived growth factor receptor","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Boehringer Ingelheim GmbH","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Boehringer Ingelheim GmbH \/ Romain Lazor","highestDevelopmentStatusID":"8","companyTruncated":"Boehringer Ingelheim GmbH \/ Romain Lazor"},{"orgOrder":0,"company":"Genentech","sponsor":"Jesse Roman","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Pirfenidone","moa":"TNF","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Genentech \/ Jesse Roman","highestDevelopmentStatusID":"8","companyTruncated":"Genentech \/ Jesse Roman"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Jerry Vockley","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Jerry Vockley","highestDevelopmentStatusID":"8","companyTruncated":"Ultragenyx Pharmaceutical \/ Jerry Vockley"},{"orgOrder":0,"company":"Incyte Corporation","sponsor":"Aaron Logan","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Ruxolitinib Phosphate","moa":"Tyrosine-protein kinase JAK1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Incyte Corporation","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Incyte Corporation \/ Aaron Logan","highestDevelopmentStatusID":"8","companyTruncated":"Incyte Corporation \/ Aaron Logan"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"David Williams","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Autologous CD34+ Stem Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bluebird Bio \/ David Williams","highestDevelopmentStatusID":"8","companyTruncated":"Bluebird Bio \/ David Williams"}]

Find Drugs for Genetic Disease in Phase II Clinical Development

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Olipudase Alfa is a Enzyme drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Niemann-Pick Diseases.

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          April 29, 2025

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Details : ABBV-CLS-628 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Polycystic Kidney, Autosomal Dominant.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          March 30, 2025

                          Lead Product(s) : ABBV-CLS-628

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Calico Life Sciences

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Abbvie Company Banner

                          03

                          Details : Under the terms of the agreement, Sionna will assume all development for ABBV-2222 (galicaftor), which is being evaluated in combination with navocaftor for the treatment of Cystic Fibrosis.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Undisclosed

                          July 16, 2024

                          Lead Product(s) : Galicaftor,Navocaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Sionna Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

                          Abbvie Company Banner

                          04

                          Lead Product(s) : SAR447537

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : SAR447537 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of alpha 1-Antitrypsin Deficiency.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          June 09, 2023

                          Lead Product(s) : SAR447537

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          05

                          Lead Product(s) : SAR447537

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : SAR447537 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of alpha 1-Antitrypsin Deficiency.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          May 12, 2023

                          Lead Product(s) : SAR447537

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          06

                          Details : Galicaftor is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Cystic Fibrosis.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          April 21, 2021

                          Lead Product(s) : Galicaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Abbvie Company Banner

                          07

                          Details : ABBV-3067 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Cystic Fibrosis.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          May 31, 2019

                          Lead Product(s) : Navocaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Abbvie Company Banner

                          08

                          Lead Product(s) : Tesevatinib

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Tesevatinib is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Polycystic Kidney, Autosomal Dominant.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          June 29, 2017

                          Lead Product(s) : Tesevatinib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          09

                          Lead Product(s) : Lademirsen

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Lademirsen is a Oligonucleotide drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Nephritis, Hereditary.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          August 04, 2016

                          Lead Product(s) : Lademirsen

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          10

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Venglustat is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Gaucher Disease.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          July 25, 2016

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner