ClinicaSpace

In the race to make the most tolerable obesity drug, there seems to be no clear winner—at least not according to analysts parsing the data presented at the American Diabetes Association annual meeting this week.

The Inflation Reduction Act includes an exemption for orphan drugs for a single indication, but experts say this is far from sufficient to maintain momentum in the rare disease space.

After the delayed approval of its next-generation COVID-19 vaccine, Novavax now awaits the first meeting of the recently overhauled CDC vaccine advisory committee next week. Throughout a tumultuous season, the Maryland-based company is relying on agility and a diverse pipeline to stay ahead of rapidly changing regulations.

The well-respected director of the FDA’s cell and gene therapy office was seen as a stabilizing and trustworthy voice inside the quickly reshaping FDA, especially since the late-March exit of CBER Director Peter Marks.

AstraZeneca, Pfizer and more are leveraging the computational power of AI to better design trials, predict the potential efficacy and safety profiles of their molecules and synthesize massive multi-omic information to gain a more complete understanding of challenging cancers.

While cancelled NIH grants and regulatory uncertainty are less hospitable to clinical research in the U.S., Europe must play its cards right to attract more studies.

As multiple companies vie to expand on Alnylam’s success in commercializing RNAi therapeutics, the pioneering company has set a goal of targeting small interfering RNA to any tissue by 2030.

Vaccine skepticism is at an all-time high in the U.S., and HHS Secretary Robert F. Kennedy Jr. is making some drastic moves in the name of reversing that trend. But misinformation and inconsistencies within the country’s healthcare agencies highlight problems with his approach.

Analysts reacted positively to the news that uniQure is in alignment with the FDA on an accelerated approval pathway and on target for a Q1 2026 submission for its one-time gene therapy for Huntington’s disease—but patients have been here before.

Here’s how companies can ensure they’re in compliance with new requirements that go into effect in August.