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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.
The FDA has introduced models intended to accelerate rare disease drug development, but recent reversals of guidance from the agency speak to a lack of clarity in its implementation. AI can help focus this process.
European pharma companies splashed billions of dollars into the U.S. biopharma sector in a matter of days, but there are differing views on whether the activity represents the rise of a new buyer class or a quirk of timing.
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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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As part of cost-cutting efforts, Alector is letting go of about 25 people as it focuses on advancing its preclinical and research pipeline. Alector is also continuing clinical-stage work on programs for frontotemporal dementia and Alzheimer’s disease.
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 9% average salary increase from 2023 to 2024 was the largest for life sciences professionals since 2021. Several factors could be behind the spike, including companies providing higher pay because bonuses and stock compensation went down.
Led by alums from Takeda and Boston Pharmaceuticals, Hillstar Bio is working on treatments that remove harmful immune cells to relieve disease.
Merck joins a growing list of companies targeting lipoprotein(a), high levels of which are associated with an elevated risk of adverse cardiovascular outcomes.
President Donald Trump’s tariffs on pharmaceuticals “to come at some point,” per CNBC, as companies promise to build infrastructure in the U.S.
The Eylea franchise was in need of a win after an appellate court last week denied the pharma’s bid to block Amgen’s biosimilar from the U.S.
If confirmed, Susan Monarez will have her work cut out for her, facing a measles outbreak that has already killed two people—the first measles-related deaths in the U.S. since 2015.
The company, launched with help from ex-Novartis executives, is targeting glutamate signaling in the brain to help treat alcohol- and cocaine-use disorders, among other indications.