The FDA has cleared an IND application for GC-1130A, a treatment for mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) being jointly developed by GC Biopharma Corp. and Novel Pharma Inc.
CSPC Pharmaceutical Group Ltd.’s anti-βKlotho monoclonal antibody drug JMT-202 has received clearance from China’s National Medical Products Administration (NMPA) to enter clinical trials to lower triglyceride levels in patients with hypertriglyceridemia.
CSPC Pharmaceutical Group Ltd.’s anti-βKlotho monoclonal antibody drug JMT-202 has received clearance from China’s National Medical Products Administration (NMPA) to enter clinical trials to lower triglyceride levels in patients with hypertriglyceridemia.
As a meeting looms of the U.S. FDA’s Endocrinologic and Metabolic Drugs Advisory Committee to evaluate a similar product from Novo Nordisk A/S, Eli Lilly and Co. made public positive top-line phase III data with its once-weekly insulin, efsitora alfa, in adults with type 2 diabetes using insulin for the first time and in those who require multiple daily injections.
The success of a vaccine, a gene editing design for an untreated disease, or achieving cell engraftment after several attempts, comes from years of accumulated basic science studies, thousands of experiments, and clinical trials. Innumerable steps precede hits in gene and cell therapies before a first-time revelation, and most of them are failures at the time. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Baltimore last week, several groups of scientists presented achievements that years ago looked impossible.
Centogene NV and Evotec SE have announced the discovery of a new small molecule with potential to treat patients with type 2 and type 3 Gaucher disease, or neuronopathic Gaucher disease.
Homerun success of Novo Nordisk A/S’ semaglutide, which recently became the U.S.’s biggest blockbuster drug, is serving as an “inflection point” for obesity therapeutics and fueling the drive for new and improved therapies, speakers said at Bio Korea 2024 on May 8.
Innovent Biologics Inc.’s glucagon-like peptide-1 receptor (GLP-1R) and glucagon receptor dual agonist, mazdutide, met the primary endpoint of superiority in a head-to-head phase III trial in Chinese patients with type 2 diabetes compared to Eli Lilly and Co.’s GLP-1, dulaglutide, for glycemic control.
From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.
Rhythm Pharmaceuticals Inc. has patented new Kir6.2/SUR1 activators reported to be useful for the treatment of disorders of sexual function, hyperinsulinemia, cancer, genitourinary, neurological and cardiovascular disorders.
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