Drug Development

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The prevalence of serious inflammatory safety issues such as cytokine release syndrome and immune effector cell–associated neurotoxicity syndrome limits the reach of these transformative cancer therapies.
After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told BioSpace, with both rare and chronic diseases headlining investor and R&D interest as JPM26 kicks off.
Recent breakthroughs and three decades of progress in treating Huntington’s disease
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RSV, influenza, COVID-19 and monkeypox have dominated vaccine discussions this year, but vaccines are also being developed for a host of other infectious diseases.
Neoleukin Therapeutics plans to slash its workforce by about 40% and drop NL-201, a de novo IL-2/IL-15 agonist currently in Phase I, according to its third-quarter report.
Beam Therapeutics has enrolled the first patient in a Phase I/II trial studying a gene therapy leveraging base editing in adults with severe sickle cell disease.
Surrozen announced it paused its Phase I study in Crohn’s disease and ulcerative colitis as several healthy participants showed increased levels of liver enzymes.
Stoke Therapeutics is gearing up for a pivotal trial of its lead program for a rare genetic epilepsy disease and announced positive results from its Phase I/IIa study in children with Dravet syndrome.
Topline results from the Phase III SUMMIT trial showed Satsuma Pharmaceuticals’ migraine candidate STS101 did not meet its primary efficacy endpoint.
MBX Biosciences secured $115 million in Series B financing to advance its pipeline of Precision Endocrine Peptides for endocrine disorders.
Roche announced its investigational antibody gantenerumab failed to meet the primary endpoints in GRADUATE I and II - two Phase III studies in Alzheimer’s disease.
Early data from a combination of Cue Biopharma’s CUE-101 and Keytruda shows the treatment is effective in patients with recurrent/metastatic HPV16+ head and neck cancer.
PACT Pharma shared results of a first-of-its-kind trial using CRISPR technology to swap a gene in a patient’s immune cell to treat solid tumors.