AMSTERDAM, The Netherlands — Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today new data showing that a one-time administration of its lead product GlyberaTM results in significant long-term health benefits. Long-term follow-up data from two clinical trials show that one administration with...
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ROCKVILLE, Md. – GlycoMimetics, Inc. (Nasdaq: GLYC) announced today that clinicians at University of California (UC) Davis Comprehensive Cancer Center initiated dosing of the first patient in a clinical study of uproleselan combined with venetoclax and azacitidine for the treatment of older or unfit patients with treatment-naïve acute myeloid leukemia...
SAN CARLOS, Calif. – Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, today announced the publication in Molecular Genetics and Metabolism of a key finding from a natural history study of phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), a rare pediatric orphan disease. In this study, the levels...
A group of genetically modified monkeys with glow-in-the-dark feet could help advance research into neurological illnesses such as Parkinson’s and Huntington’s. Genetically modified monkeys in scientific research are not uncommon, however, these are the first family to pass a manufactured genetic trait onto a second generation.The monkeys, which were created...
TAIPEI, Taiwan — Golden Biotechnology Corp., an advanced biopharmaceutical drug development company, had unveiled the findings of its Phase II clinical trial investigating Antroquinonol (HOCENA) in combination with the standard of care (SOC): nab-paclitaxel + gemcitabine, as first-line treatment for metastatic pancreatic cancer. The trial showed a positive median overall...
SAN FRANCISCO, Calif. — Gordian Biotechnology, an in vivo drug discovery and development company, today announced its platform that enables patient predictive, in vivo screening of hundreds of gene targets for FDA-recognized diseases of aging at a scale never before possible. The company has raised $60 million to date from...
Governor M. Jodi today signed a bill that requires birthing hospitals to screen newborns for cystic fibrosis, an inherited disorder that clogs the lungs, causes infections and affects digestion. “Every baby deserves the best start in life and that is a promise society must never abandon,” Governor Rell said during...
Shanghai, China – This study is led by Prof. Wei-Li Zhao (Shanghai Institute of Hematology, State Key Laboratory of Medical Genomics, National Research Center for Translational Medicine at Shanghai, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine). G-protein coupled receptors (G-protein ) are critical targets for viral...
MENLO PARK, Calif. — Grace Science, LLC announced today that the first patient was dosed in an open-label Phase 1/2/3 clinical trial of GS-100, an AAV9 gene replacement therapy for the treatment of NGLY1 Deficiency. Part 1/2 of the study will investigate dose-escalation and the safety and efficacy of a single...
MENLO PARK, Calif. – Grace Science, LLC announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GS-100, an investigational AAV9 gene replacement therapy for the treatment of NGLY1 deficiency. The FDA grants orphan drug designation to investigational medicines and biologics intended for the...