Fiercebiotech.com

The calling card of Alzheimer’s disease is a buildup of misfolded amyloid protein in the brain, but many medicines struggle to penetrate the barrier protecting the mind from outside interference. A new study suggests that crossing the blood-brain barrier might not be necessary at all to clear amyloid plaques—and that a one-time gene therapy could one day prove an effective treatment for the devastating neurodegenerative disease.

Leerink Partners analysts slashed their sales forecast for enGene Therapeutics’ bladder cancer candidate after phase 2 data dented their belief that the biotech can match rivals, including Johnson & Johnson.

Odyssey Therapeutics has become the latest biotech to voyage to the public markets via an upsized $304 million IPO this morning.

Not long after the departure of former CEO Usman Azam, a new leader is taking her Cue in the form of Shao-Lee Lin, M.D., Ph.D. Lin steps into Cue’s president and chief exec spot after previously serving as founder and CEO of eye disease specialist Acelyrin, which later merged with Alumis.

Banyan BioInnovations is debuting with more than $100 million aimed at developing NewCos around promising clinical assets, plus a partnership with global contract research organization Icon.

BeOne Medicines has shaken up its early pipeline, dropping five phase 1 cancer programs and stopping a phase 2 arthritis trial based on new data.

Ascendis Pharma has ended internal development of its IL-2 candidate, pivoting away from oncology and calling time on an asset it once tipped for accelerated approval.

For people living with exocrine pancreatic insufficiency due to cystic fibrosis, the only treatment option is a regimen that can include up to 40 pills a day. With a $250 million investment from Blackstone Life Sciences, Anagram Therapeutics hopes to reduce that pill burden to just three pills daily.

Entrada Therapeutics’ Duchenne muscular dystrophy data have fallen well short of expectations, crashing the biotech’s stock as investors evaluated the fallout from a key readout expected to validate its platform.

The recent run of first-quarter earnings results marked a shift for Big Pharmas away from AI hype and toward explaining how the technology is being applied in practice.