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Phacilitate’s annual event dawns as cell and gene therapies reach a new tipping point: the science has hit new heights just as regulatory and government policies spark momentum and frustration.
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With the biopharma industry performing better of late, analysts, executives and other industry watchers are “cautiously optimistic”—a term heard all over the streets of San Francisco at the J.P. Morgan Healthcare Conference earlier this month.
Bristol Myers Squibb, GSK and Merck are contributing drug ingredients as part of their deals with the White House but are keeping many of the terms of their agreements private.
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
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The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
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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.
Active immune therapies hold promise for preventing or slowing disease onset, but some experts warn of potential safety risks.
GSK, Moderna and Pfizer are all looking at potential respiratory syncytial virus vaccine sales slumps thanks to recently updated CDC guidelines regarding the use of RSV shots in seniors.
Approved under the regulator’s accelerated pathway, Tecelra is also the first new synovial sarcoma therapy in more than a decade, according to Adaptimmune Therapeutics.
In pursuit of Merck’s blockbuster Keytruda, GSK’s Jemperli scored its own broad FDA label expansion, allowing its use in first-line endometrial cancer regardless of biomarker status.
As part of a major reorganization, Vir Biotechnology has discontinued the bulk of its virology work and pivoted to cancer in an exclusive licensing deal with Sanofi.
Poor efficacy data for two early-stage candidates for the rare disease alpha-1 antitrypsin deficiency have convinced Vertex Pharmaceuticals to terminate their development.
Moderna’s stock price plunged more than 20% in Thursday morning trading after the company lowered its 2024 revenue guidance due to weak demand for its coronavirus vaccine in the second quarter.
With an upfront $800 million payment and $325 million in potential milestones, Otsuka Pharmaceutical is acquiring Jnana Therapeutics’ drug discovery platform and rare disease candidate, the companies announced Thursday.
Eli Lilly on Thursday released late-stage data showing a 38% reduction in the risk of heart failure outcomes, as it plays catch-up with Novo Nordisk’s semaglutide which won the FDA’s cardio nod in March.