Neuroscience
Sangamo, which has been having cash problems, will receive $18 million upfront in licensing fees for its AAV capsid that in preclinical studies has shown the ability to cross the blood-brain barrier.
After some high-profile crashes, the one-time biotech darling is inching toward success with its Hunter syndrome treatment, which today began a rolling BLA for accelerated approval.
Biopharma leaders react to the forced resignation of CBER Head Peter Marks as RFK Jr.’s promised job cuts begin at the FDA; Novo Nordisk presents mixed results from oral semaglutide in cardiovascular disease; the EU’s Committee for Medicinal Products for Human Use declines to recommend Eli Lilly’s Alzheimer’s drug; and pharma R&D returns grew in 2024.
The European Union’s CHMP said that the benefits of the drug, already approved in the U.S., do not outweigh the risk of potentially fatal brain swelling and bleeding.
WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II FORWARD-53 trial. Wave plans to file for accelerated approval of the candidate in 2026.
Eisai’s new fiscal 2027 forecast for Leqembi is roughly 50% lower than its projections a year ago.
President Donald Trump continues to warn of tariffs on the pharmaceutical industry; Susan Monarez replaces Dave Weldon as CDC director nominee; Novo Nordisk joins the triple-G race; Alnylam wins approval for Amvuttra in ATTR-CM; and Cassava Sciences ends development of simufilam in Alzheimer’s.
After years of controversy and allegations of doctored data, Cassava is moving on from Alzheimer’s.
The British pharmaceutical giant is working with the U.K. Dementia Research Institute to exploit a “natural randomization” experiment to determine whether 65- and 66-year-olds who received GSK’s shingles vaccine Shingrix have reduced dementia risk.
The gene therapy world is in turmoil, but Arbor, armed with more than $1 billion in partnerships and raises, is going forward.
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