Drugs in Dev.
Genetic Disease
Phase I
Netherlands 
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Lead Product(s) : ARGX-119
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : ARGX-119 is a Antibody drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Myasthenic Syndromes, Congenital.
Product Name : Undisclosed
Product Type : Antibody, Unconjugated
Upfront Cash : Inapplicable
May 31, 2024
Lead Product(s) : ARGX-119
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Nizubaglustat
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Positive safety, tolerability and pharmacodynamics data from multiple dose levels in healthy volunteers support evaluation of AZ-3102, a small molecule designed to be a potent and selective inhibitor of two target enzymes involved in glycolipid metabolis...
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
February 14, 2022
Lead Product(s) : Nizubaglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Antimony pentoxide
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Up to two years of follow-up in large transgenic huntington’s disease model demonstrates stable mHTT protein lowering.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
February 27, 2020
Lead Product(s) : Antimony pentoxide
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : QR-010
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : European Commission
Deal Size : Inapplicable
Deal Type : Inapplicable
Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation
Details : QR-010 is a Oligonucleotide drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Cystic Fibrosis.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
September 30, 2015
Lead Product(s) : QR-010
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : European Commission
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : KH176
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : University Ghent
Deal Size : Inapplicable
Deal Type : Inapplicable
A Dose-escalating Clinical Trial With KH176
Details : KH176 is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of MELAS Syndrome.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
September 09, 2015
Lead Product(s) : KH176
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : University Ghent
Deal Size : Inapplicable
Deal Type : Inapplicable
