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Biotech is increasingly financed, governed and regulated as though it were a mature pharmaceutical industry rather than a discovery system built around scientific uncertainty. Structural changes are needed to sustain the sector’s strategic innovation.
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Nusano will bring a massive new radioisotope facility in Salt Lake City online by the end of the year, establishing a supply of starting materials for the next generation of radiopharmaceuticals.
Last month, Revolution Medicines’ RAS inhibitor doubled survival in a Phase 3 pancreatic cancer trial. On the biotech’s heels are Immuneering, Actuate Therapeutics, Erasca and more, looking to improve on that result with increased tolerability—and more time for patients.
The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, BioSpace reviews genetic medicines in development for the central nervous system, retinal, cardiac and neuromuscular diseases.
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Read our takes on the biggest stories happening in the industry.
The Department of Health and Human Services is spinning its wheels, unable to establish steady leadership at three major divisions—the CDC and the FDA’s two primary review units.
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The selection of controversial TV personality Mehmet Oz to run the agency overseeing Medicare and Medicaid follows Trump’s pick of RFK Jr. to run its parent department, HHS.
Trump fingers Robert F. Kennedy Jr. to lead the HHS, lupus and ATTR-CM dominate headlines this week, bluebird bio has a cash gap to leap and RegenxBio eyes Sarepta in Duchenne muscular dystrophy.
A judge in the U.K. last month sided with Pfizer over GSK in a respiratory syncytial virus vaccine patent lawsuit, positioning both companies to compete for that market and laying down a marker for ongoing legal clashes in other parts of the world.
While the full impact of the Supreme Court decision remains unknown, the new regulatory landscape could be a net positive for drug developers.
A handful of billion-dollar deals in the rare disease space highlights the uptick in Big Pharma’s investment, but it’s still extremely low compared to the money flowing to more common indications.
After the unexpected success of their PHOENYCS GO study for dapirolizumab pegol in lupus earlier this fall, Biogen and UCB are planning a second late-stage trial by the end of the year to support a drug application.
Investigational CAR T therapies stole the spotlight at the American College of Rheumatology Convergence as data presented by Bristol Myers Squibb, Kyverna Therapeutics and more highlighted their potential to effectively treat lupus.
After failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells investors that it’s evaluating options for the program.
Analysts are split on whether the positive trial results will help Merck stem future Keytruda losses as the mega-blockbuster goes off patent in 2028.
Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for 2026—potentially posing a threat to Sarepta’s Elevidys.