BioSpace News Archive

Another patient has died from acute liver failure after receiving Sarepta’s gene therapy for DMD ; After a quiet start to the year, M&A is back with one deal for a gene editing biotech reinvigorating that sector; and RFK Jr. installs a suite of new vaccine board members who share his skeptical views on vaccines.

The new version of the bill will still need to go through the entire House and Senate.

Speaking at BIO2025, Makary bemoaned what he called “unnecessary steps” and “avoidable delays” in the U.S. regulatory landscape.

The U.K.-based biotech is set to enter mid-stage studies for its depression drug this year, while two other GABA_A modulators are poised for clinical trials in 2026.

Sanofi paid a more than 300% premium on its acquisition of Vigil Neuroscience, suggesting a fierce battle to seal the deal. Across biopharma, companies are sometimes willing to put it all on the line for the right buyout. Novartis’ recent acquisition of Regulus for $800 million upfront provides a case study.

BioNTech said in 2022 that it faced “threats of a groundless patent infringement suit” from a company that was “unable to bring to market any product to help in the fight against COVID-19.” Now, the mRNA biotech is buying that very company.

Andembry is the first monthly Factor XIIa inhibitor for the prevention of HAE attacks and will be available to patients “before the end of June,” CSL announced.

Findings that U.S. companies can sue foreign rivals despite limited business operations in the country could dissuade drug developers from targeting the U.S. market, potentially benefiting domestic producers of biosimilars.

On the sidelines of BIO2025, Julie Gilmore, head of Lilly Gateway Labs, shares her thoughts on the $1.3 billion Verve Therapeutics buy, where Lilly’s therapeutic puck is potentially going and how the company is leveraging its unprecedented success in obesity to support young biotechs.

In combination with Eli Lilly’s tirzepatide, marketed as Zepbound for obesity, Scholar Rock’s monolonal antibody helped patients lose the same amount of weight as patients on tirzepatide alone while preserving more muscle mass.