The deal would hand Lundbeck a treatment for a series of rare brain disorders that could, by the company's estimates, become a blockbuster product.

The agency agreed in court to allow compounding companies to continue producing copycat versions of Lilly’s fast-selling medicines while it reevaluates its recent decision to declare the drugs back in ...

The company has set high expectations for depemokimab, which it is also advancing in three other inflammatory diseases, including asthma.

The approval of Hympavzi Friday was a dose of good news for Pfizer, which is dealing with research setbacks and a challenge from an activist investor.

The French pharmaceutical giant said it’s in negotiations to sell a controlling stake of Opella to the private equity firm CD&R, in a deal that reportedly could be worth more than $16 billion.

To enhance patient outcomes and increased trial success, Convert Pharma is leveraging companion AI- and genetic biomarkers, a ...

The study, of Intellia’s treatment for hereditary angioedema, is the second Phase 3 trial the biotech has begun testing its “in vivo” gene editing medicines.

The Cambridge, Massachusetts-based biotech is using what it calls ligand-siRNA conjugates to reach drug targets in the kidney, which historically have been difficult to reach with RNA therapies.

Biotechnology company Purespring Therapeutics has raised about $105 million to fund its development of gene therapies for diseases of the kidney.

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Cumulatively, data showed the shot to be 63% effective across the three-year period. However, efficacy appeared to wane and GSK said it expects revaccination will eventually be needed.

Seven young boys given Bluebird's Skysona later developed blood cancers, findings that could shape how doctors balance the gene therapy’s risks against its benefit.