Drug Development
Amid recent backlash stemming from market withdrawals and trial delays, the FDA seeks to further clarify its requirements for confirmatory trials for therapies seeking the shortened path to market.
FEATURED STORIES
Active immune therapies hold promise for preventing or slowing disease onset, but some experts warn of potential safety risks.
Ultracompact CRISPR systems, which are in some cases one-third the size of Cas9, are being designed to be more specific and enable in vivo gene editing in difficult to reach tissues.
With GLP-1 receptor agonists expected to dominate the weight loss market in the near term, several young companies are building on this mechanism, while others are taking a completely different approach.
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In the third episode of our mini series on artificial intelligence, Lori and guests continue their exploratory discussion on AI and the opportunity in patient lives.
The overall survival data from the late-stage trial will help Pfizer in its bid to expand the label for Talzenna and Xtandi, potentially covering all patients with metastatic castration-resistant prostate cancer regardless of biomarker status.
The pediatric patients, with a rare neurodegenerative disease, were treated with bluebird bio’s Skysona to slow the progression of neurologic dysfunction. Six patients developed myelodysplastic syndrome and one patient developed acute myeloid leukemia.
Cell and gene therapy investment rebounds; WuXi Biologics and other companies named in BIOSECURE Act mull options; Bayer, J&J and Pfizer’s recent layoffs; updates from the weight loss space.
Eli Lilly CEO David Ricks is confident his company and peer Novo Nordisk are years ahead in the weight loss space, as biotechs press on with compelling data.
On the heels of Keytruda’s success in a Phase III perioperative trial for a disease where it had previously failed to improve event-free survival, Merck touts an I&I deal with UK biotech Mestag.
Sage has decided to discontinue the development of dalzanemdor in Alzheimer’s disease. A study of the candidate in Huntington’s is ongoing, with early data expected later this year.
Tim Hunt, CEO of the Alliance for Regenerative Medicine, said Monday at the 2024 Cell & Gene Meeting on the Mesa that investments reached $10.9 billion in the first half of this year—outpacing 2019’s $9.8 billion total—but far below the pandemic peak.
With Monday’s data from SAPPHIRE, Scholar Rock is building toward regulatory submissions for apitegromab in spinal muscular atrophy in the first quarter of 2025.
J&J has recently pulled back from the infectious diseases space, including winding down R&D activity in this area in August 2023.