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AIRNA’s lead candidate AIR-001 works by correcting the most common pathologic mutation driving the rare disease alpha-1 antitrypsin deficiency.
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With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery breakthroughs are needed to propel RNA editing to the next stage.
Morale is low at the FDA, which was hit with layoffs this week following RFK Jr.’s confirmation. Biopharma leaders and agency insiders fear further workforce cuts could delay new medicines.
2023 marked the most bankruptcies in biopharma in more than a decade, with 14 companies filing for Chapter 11 protection. The number remained high in 2024.
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Emma was a 28-year-old nursing student when she was diagnosed with triple-negative breast cancer.
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Read our takes on the biggest stories happening in the industry.
Like they say about the weather in Iceland, if you don’t like an action taken by the new administration, wait five minutes; it’ll probably change. The markets, it seems, don’t react kindly to that kind of policymaking.
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The French pharma is getting Black Diamond’s de-prioritized molecule for non-small cell lung cancers with specific mutations whose development had been paused to save money.
Roche’s up to $1 billion investment will provide access to Oxford BioTherapeutics’ antibody-drug conjugate platform for undisclosed cancer targets.
The biotech is exploring opportunities for a reverse merger or other business combinations. CFO and now interim CEO Anup Radhakrishnan will take charge of these negotiations.
The CDC budget cuts could pose headwinds for HIV drugmakers like Gilead and Merck but are unlikely to severely cripple their HIV divisions, according to analysts.
Layoffs leave more than empty desks—they leave uncertainty, guilt and anxiety. Three simple steps will help you regain control of your work, well-being and career.
Sarepta will update Elevidys’ label after a patient died following treatment; the FDA issues flu vaccine recommendations without advisor input; Trump CDC nominee Dave Weldon pulled at last minute; and FDA decisions expected for Alnylam’s Amvuttra in ATTR-CM and Milestone’s etripamil in tachycardia.
The explosion of GLP-1 weight loss drugs is reminiscent of the early days of PD-1 inhibitors, but key market differences suggest history may not repeat itself.
The ongoing conflicts between Ukraine and Russia, as well as Israel and Palestine, have sent ripples across various industries, including pharma. Medical science liaisons can help.
After a patient taking the Duchenne muscular dystrophy gene therapy Elevydis died of liver injury, Sarepta will update the label to reflect the safety signal.
Mirador debuted last year with a massive $400 million and the goal of developing game-changing therapies for inflammatory and fibrotic diseases. The company aims to enter the clinic this year.