Gaucher Disease Articles & Analysis
6 news found
“Earlier in the year, we made the strategic decision to reprioritize our pipeline activities and streamline our organization, which not only strengthened our cash position, but also focused our efforts to further advance our cystinosis and Gaucher disease type 3 programs into late-stage trials. “Collectively, our pipeline targets more than 40,000 ...
Dosed a third patient in our Phase 1/2 GUARD1 clinical trial in Gaucher disease type 1 Deprioritized Fabry disease program in January 2022 to focus pipeline on Gaucher disease programs, cystinosis, Hunter syndrome and Pompe disease programs, extending cash runway into the first quarter of 2024 ...
(Nasdaq: GANX) (“Gain”), a biotechnology company transforming the drug discovery paradigm with structurally targeted allosteric regulators (STARs) identified with its proprietary computational discovery platform, today presented new pre-clinical data from its Gaucher Disease (GD) program. These results were highlighted in a late breaking abstract ...
Capitalizing on significant advances in computational biology and drug discovery, the Company has applied its proprietary computational SEE-Tx® platform to generate its lead programs in GBA1 Parkinson’s disease, Gaucher disease as well as four additional programs to advance the treatment of lysosomal storage disorders. In addition, the ...
Tiago Fleming Outeiro, PhD (University Medical Center Göttingen) will discuss protein misfolding in neurodegenerative diseases. Ricardo A. Feldman, PhD (University of Maryland School of Medicine) will present recent data generated in patient-derived iPSCs with Gain Therapeutics’ structurally targeted allosteric regulators for the treatment of GBA1-associated ...
Pioneering technology platform rapidly uncovers the genetic causes of unsolved diseases and redefines the understanding of rare and complex genetic diseases. ...