Drugs in Dev. Genetic Disease
Phase I
Netherlands 
Lead Product(s) : ARGX-119
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Undisclosed
Product Name : Undisclosed
Product Type : Antibody
Upfront Cash : Inapplicable
May 31, 2024
Lead Product(s) : ARGX-119
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Nizubaglustat
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
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 : Other Small Molecule
Upfront Cash : Inapplicable
February 14, 2022
Lead Product(s) : Nizubaglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Nizubaglustat
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Azafaros Receives FDA Orphan Drug Designation for AZ-3102 in GM2 Gangliosidosis
Details : The ODD for AZ-3102, selectively inhibit two enzymes involved in glycolipid metabolism GCS and GbA2 for the treatment ofGM2 gangliosidosis has been granted based on efficacy demonstrated in a Sandhoff mouse model, including a clear effect on animal survi...
Product Name : Undisclosed
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
January 02, 2022
Lead Product(s) : Nizubaglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Nizubaglustat
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Phase 1 clinical study investigates safety and tolerability, pharmacokinetics and pharmacodynamic effects of first-in-class azasugar AZ-3102 in healthy subjects. AZ-3102 is being initially developed for the treatment of patients suffering from GM1 and GM...
Product Name : Undisclosed
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
June 04, 2021
Lead Product(s) : Nizubaglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : AMT-130
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
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 and Gene therapy
Upfront Cash : Inapplicable
February 27, 2020
Lead Product(s) : AMT-130
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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 : Undisclosed
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
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 : Undisclosed
Product Name : Undisclosed
Product Type : Other Small Molecule
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
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