Facing increasing competition to PI3Kα inhibitor Piqray (alpelisib) in breast cancer, Novartis AG looks to bolster its pipeline with next-generation programs, including phase I/II-stage candidate ...

In what the U.S. FDA has dubbed a milestone move toward fewer animal studies in drug development, the agency published a draft guidance to help sponsors validate new approach methodologies that can ...

Congruence Therapeutics Inc. has closed a $39.5 million financing to advance into its portfolio of small-molecule correctors for diseases of protein misfolding into the clinic.

Further weight loss details are yet to come this year, but Eli Lilly and Co. scored positive top-line results in Transcend-T2D-1, a phase III trial evaluating the efficacy and safety of retatrutide, ...

There is broad agreement that psychiatric diagnoses in their current form are not reflective of any underlying biology, and ...

With top-line pivotal data with gene therapy RGX-202 for Duchenne muscular dystrophy (DMD) due in the next quarter, Regenxbio ...

Otsuka Pharmaceutical Co. Ltd. reaped positive results from an open-label phase II study of repinatrabit (JNT-517) in adolescents with phenylketonuria (PKU), a rare disease marked by the inability to ...

Neurodegenerative disease and cognitive decline cannot be explained by a single process. Beta-amyloid plaques, hyperphosphorylated tau, alpha-synuclein, activated microglia and astrocytes, altered ...

Mestag Therapeutics Ltd. has raised a $40 million round from existing investors as it completes final preparations for its lead program MST-0312 to enter clinical development in mid-2026. MST-0312 is ...

Entering a cell and watching its entire inner machinery at work, how DNA is copied, how proteins are assembled, or how it splits in two, has been, for decades, an impossible dream. Now, scientists at ...