Drug Development
Infigratinib topped “even the most optimistic expectations” for efficacy and safety in the late-stage PROPEL 3 study in achondroplasia, Truist Securities analysts said Thursday.
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Analysts, investors and scientists are eager for Biogen’s 2026 BIIB080 readout. Even if successful, executives warn that there are many more steps before the Alzheimer’s therapy could reach the market.
With a clutch of key data and planned regulatory applications this year from Avidity Biosciences, REGENXBIO and Capricor Therapeutics, CureDuchenne CSO Michael Kelly sees “momentum” in the Duchenne muscular dystrophy pipeline, as Sarepta’s Elevidys leaves the door open.
After advancing in lockstep through the pandemic, the fortunes of the biotechs have diverged as their use of COVID-19 windfalls has taken shape.
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The Bespoke Gene Therapy Consortium intends to bring AAV-based gene therapies to patients whose diseases are often ignored by commercial interests.
In the latest HEALEY trial readout, Clene’s CNM-Au8 significantly reduced plasma neurofilament light chain levels in ALS patients, which could be a strong and reliable signal of its clinical benefit.
In a unanimous 21-0 vote, an FDA advisory committee recommended an updated monovalent formulation of the COVID-19 vaccine targeting the now-dominant XBB.1.5 subvariant.
After an FDA advisory committee unanimously recommended Leqembi’s full approval, questions linger around amyloid-related imaging abnormalities and a potentially cumbersome patient registry.
The Japanese biopharma is diving deeper into targeted protein degradation, paying $35 million upfront for access to Cullgen’s uSMITE platform.
Eli Lilly has secured the rights to Verve Therapeutics’ gene editing approach, a “one-and-done” method that the companies hope will lower the cardiovascular risk factor lipoprotein(a).
Following cases of serious bleeding in patients, five of which were fatal, the regulator has put Mersana’s investigational antibody-drug conjugate UpRi on partial clinical hold in two ovarian cancer trials.
Jubilant Therapeutics’ differentiated, orally bioavailable molecules address both validated and novel therapeutic targets in oncology and immunology.
The biotech company is collaborating with Israel’s NeuroSense to study how the latter’s amyotrophic lateral sclerosis candidate affects plasma neurofilament levels in patients.
The company announced Monday with its second-quarter earnings that the regulator has officially halted the Phase I study after a child with acute myeloid leukemia died following treatment.