Oncologists' experience with CGT has increased, with more patients treated and earlier lines of therapy being utilized.

Shortly after taking reins at BioMarin, CEO Alexander Hardy laid out three paths the company could take for its struggling ...

"Eli Lilly buys Adverum in eye disease gene therapy punt" was originally created and published by Pharmaceutical Technology, ...

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...

The novel treatment, partly developed by CHOP, eliminates the need for regular infusions while preventing the ...

BioMarin Pharmaceutical BMRN reported third-quarter 2025 adjusted earnings per share (EPS) of 12 cents against the Zacks Consensus Estimate of a loss of 15 cents. Quarterly earnings plummeted nearly ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

With Eli Lilly's recent acquisition of Verve Therapeutics and its PCSK9 candidate, delve into what the target represents.

An increasing demand for gene therapies is focusing the industry on enhancing production capabilities, transitioning from ...

BioMarin no longer expects to hit its $4 billion revenue target by 2027, citing various market factors such as impending ...

A patient dosed in the MAGNITUDE study has suffered a Grade 4 liver transaminases and increased total bilirubin.