Drug Development
Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the floor. At a roundtable last week, FDA leaders promised faster approvals and broad support to the industry.
FEATURED STORIES
Beam Therapeutics and Verve Therapeutics have each built their lead candidates on a technique billed as a safer alternative to conventional CRISPR. Clinical results have so far been promising.
As tariffs, HHS workforce cuts and the ouster of CBER Director Peter Marks threaten the “lifeblood” of the cell and gene therapy space, experts express wariness over the unknowns and optimism that Marks’ legacy will carry on.
Presentations at this year’s American Association for Cancer Research meeting could have a broad impact on the treatment landscape for head and neck and lung cancer, and implications for specific drug modalities like TIGIT and VEGF.
Subscribe to ClinicaSpace
Clinical trial results, research news, the latest in cancer and cell and gene therapy, in your inbox every Monday
THE LATEST
The canceled contract comes as Moderna nears a critical FDA decision for its next-generation COVID-19 vaccine.
InflaRx’s vilobelimab met the bar for futility in a Phase III trial for the rare skin disease pyoderma gangrenosum.
R&D spending across the global pharmaceutical sector climbed 1.5% in 2024, according to unreleased data from Evaluate Pharma.
In addition to a $140 million series D, GRIN Therapeutics has signed a global licensing deal for the epilepsy disorder drug radiprodil worth $50 million upfront.
Acute systemic infection caused the patient to develop fatal capillary leak syndrome, highlighting the unpredictability of gene therapies and potentially challenging investment in the space, analysts say.
With $90 million to start, Syndeio has a lead asset in Phase II clinical trials for major depressive disorder, with plans to soon launch a biomarker trial in Alzheimer’s disease.
Prothena has already discontinued the development of birtamimab, and anticipates further spending cuts, including workforce reduction.
As the World Health Organization initiates a new agreement for coordinating global responses to future pandemics, the future of vaccine development in the U.S. faces growing challenges, including waning funding and regulatory changes, that threaten next-gen COVID-19 vaccine candidates and pandemic preparedness more broadly.
Vocal skeptics of COVID-19 vaccinations gave mRNA a bad name and government funding for mRNA research is now being cut. On the flip side, at least one CEO said the pandemic also provided “elevated acceleration” for the field, which also holds promise in therapeutics for cancer and rare diseases.
Beginning this week in Chicago, the American Association for Cancer Research’s annual conference will feature presentations that could have far-reaching implications for breast and blood cancers and more.