Sarepta has begun evaluating a regimen meant to lower the risk of liver damage in Elevidys recipients. Elsewhere, Inovio laid off staff and a Bayer kidney drug notched another trial victory.

Testing revealed weight loss effects some Wall Street analysts believe compare favorably to Lilly’s orforglipron, which regulators could approve within weeks.

Company shares spiked more than 60% after the therapy surpassed Wall Street benchmarks in Phase 1 testing against late-line colorectal tumors.

Ugur Sahin and Özlem Türeci, who directed the COVID vaccine maker’s rise to prominence, will helm an as-yet-unnamed company ...

The trial clearance for Shanghai-based Unixell is another step forward for the rapidly advancing Chinese biotech ecosystem, which has invested heavily in cell and gene therapy technology.

More early-stage data show the drug, called salanersen, appeared to have positive effects on neurodegeneration and motor function in patients previously treated with Novartis' Zolgensma.

Xenon shares skyrocketed nearly 50% on results that handily beat Wall Street expectations. Elsewhere, Capricor got a decision date for a spurned cell therapy and AbbVie and Regeneron showcased obesity ...

The proposed acquisition would hand Servier a couple experimental drugs in human testing as well as Ojemda, an approved ...

Combining the disparate databases used to detect potential issues with drugs, vaccines and other products into a single dashboard will enhance their utility and cut costs, the agency said.

Incubated by Atlas Venture, the biotech startup is advancing a daily pill it sees potentially helping people with dystonia or ...

The agency acted after reviewing mouse data submitted in 2024, making the timing of the new pause “confusing,” one analyst wrote.

Prasad contributed to an uncertain regulatory climate for gene therapy makers focused on rare conditions. Shares of many ...