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Applicable","highestDevelopmentStatusID":"10","companyTruncated":"KalVista Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Sebetralstat","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"KalVista Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"KalVista Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"KalVista Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Spinogenix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"SPG601","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spinogenix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Spinogenix \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Spinogenix \/ Not Applicable"},{"orgOrder":0,"company":"Rege Nephro","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Tamibarotene","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Rege Nephro","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Rege Nephro \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Rege Nephro \/ Not Applicable"},{"orgOrder":0,"company":"Avenacy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Tranexamic Acid","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Avenacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Avenacy \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Avenacy \/ Not Applicable"},{"orgOrder":0,"company":"ReviR Therapeutics","sponsor":"CMT Research Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Partnership","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"ReviR Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"ReviR Therapeutics \/ CMT Research Foundation","highestDevelopmentStatusID":"3","companyTruncated":"ReviR Therapeutics \/ CMT Research Foundation"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"R-Bridge","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Financing","leadProduct":"Vamorolone","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Santhera Pharmaceuticals","amount2":0.040000000000000001,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"","sponsorNew":"Santhera Pharmaceuticals \/ R-Bridge","highestDevelopmentStatusID":"12","companyTruncated":"Santhera Pharmaceuticals \/ R-Bridge"},{"orgOrder":0,"company":"Sperogenix Therapeutics","sponsor":"Santhera Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Vamorolone","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Sperogenix Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Sperogenix Therapeutics \/ Santhera Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"Sperogenix Therapeutics \/ Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Sirolimus","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Biodexa Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"ReviR Therapeutics","sponsor":"Asieris Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Oncology","country":"","productType":"Small molecule","year":"2024","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"","graph1":"Genetic Disease","graph2":"Discovery Platform","graph3":"ReviR Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery Platform","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"ReviR Therapeutics \/ Asieris Pharmaceuticals","highestDevelopmentStatusID":"3","companyTruncated":"ReviR Therapeutics \/ Asieris Pharmaceuticals"}]

Find Clinical Drug Pipelines for Genetic Disease

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Penicillamine

                          Therapeutic Area : Genetic Disease

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Generic version of penicillamine is a copper chelating agent, small molecule drug candidate, which approved by USFDA for the treatment of Wilson's disease.

                          Brand Name : Cuprimine-Generic

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 03, 2020

                          Lead Product(s) : Penicillamine

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Granules India

                          02

                          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.

                          Brand Name : ABBV-2222

                          Molecule Type : Small molecule

                          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

                          03

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Altuviiio (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels, extending bleed protection in a once-weekly dose for people with haemophilia A.

                          Brand Name : Altuviiio

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          September 25, 2023

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          04

                          Details : Ztalmy (ganaxolone) is a positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS. It is used for the treatment of epileptic seizures associated with CDKL5 deficiency disorder.

                          Brand Name : Ztalmy

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          July 31, 2023

                          Lead Product(s) : Ganaxolone

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Marinus Pharmaceuticals

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Fermion Orion Company Banner

                          05

                          Lead Product(s) : Givosiran

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

                          Brand Name : SAR-439774

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          April 04, 2023

                          Lead Product(s) : Givosiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          06

                          Lead Product(s) : Eliglustat

                          Therapeutic Area : Genetic Disease

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Cerdelga (eliglustat tartrate) is a prescription medicine used for the long-term treatment of Gaucher disease type 1 (GD1) in adults who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs).

                          Brand Name : Cerdelga

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 01, 2023

                          Lead Product(s) : Eliglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          07

                          Details : Vertex will have the rights to conduct research using ImmunoGen's ADC technology to discover novel targeted conditioning agents for use with gene editing. CTX001 (exagamglogene autotemcel) is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edite...

                          Brand Name : CTX001

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : $15.0 million

                          March 01, 2023

                          Lead Product(s) : Exagamglogene Autotemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Vertex Pharmaceuticals

                          Deal Size : $352.0 million

                          Deal Type : Licensing Agreement

                          Immunogen

                          08

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Nexviazyme (avalglucosidase alfa) is an enzyme replacement therapy (ERT) designed to target the mannose-6-phosphate (M6P) receptor, the key pathway for uptake and transport of ERT. It is being developed for treatment of people living with pompe disease.

                          Brand Name : Nexviazyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          February 24, 2023

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          09

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Altuviiio (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophi...

                          Brand Name : Altuviiio

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          February 23, 2023

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          10

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophili...

                          Brand Name : BIVV001

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          January 25, 2023

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner