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Day One Biopharmaceuticals announced positive early data assessing tovorafenib as a once-a-week treatment in people aged six months to 25 years with relapsed or progressive pediatric low-grade glioma.
Researchers from the Dana-Farber Cancer Institute looked deeper into a non-coding portion of the human genome, uncovering mechanisms that might drive or suppress cancer development.
On Tuesday, Stealth Biotherapeutics announced that it plans to meet with the FDA to discuss a potential NDA for its therapeutic, elamipretide, which is intended to treat Barth syndrome.
The FDA has approved Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy in adults.
Evotec SE has inked a deal with Janssen Pharmaceutical hat showcases its end-to-end integrated drug discovery and development platform.
Soleno announced promising results of its diazoxide choline extended-release tablets in patients with Prader-Willi syndrome at ENDO 2022.
Eli Lilly’s Olumiant (baricitinib) received approval from the FDA for the treatment of severe alopecia areata, making it the first systemic treatment for adults living with the disease.
Treatments for overactive bladder are not one size fits all, which is why Urovant is advancing URO-902, an injectable gene therapy that is currently being investigated in Phase IIa clinical trials.
Shares of CRISPR Therapeutics fell more than 11% on Monday as investors react negatively to the endorsement of a rival beta-thalassemia gene therapy developed by bluebird bio.
OptiNose announced positive top-line results from its Phase III ReOpen2 clinical trial testing OptiNose’s XHANCE, a fluticasone propionate nasal spray for chronic sinusitis.
BioSpace looks at several companies developing new gene therapies in the wake of the FDA recommending two of bluebird bio’s lentiviral vector gene therapies.
Versanis Bio’s data reveals the results of a pooled analysis, showing that its lead asset, bimagrumab, improves body composition in both diabetic and non-diabetic patients.