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In a complete response letter published by the FDA on Monday, the agency said a resubmission for REGENXBIO’s Hunter syndrome gene therapy should provide evidence of normalized or improved biomarker levels or neurodevelopmental outcomes.
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Generate:Biomedicines has hit the public markets as the world begins to question the usefulness of AI technology. CEO Mike Nally says biology is the key to unlocking the technology’s full potential.
The CDC’s changes threaten to cut vaccine sales for makers including Pfizer, Moderna, Merck and more, but a legal expert suspects affected manufacturers will stay on the sidelines rather than back a push to declare the revised schedule unlawful.
This week’s Capitol Hill meetings come on the heels of rejections of ultra-rare disease drugs developed by Biohaven and Saol Therapeutics. Physicians and patient groups implored the FDA to expedite these treatments.
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Eli Lilly’s win in a head-to-head trial drove Novo Nordisk’s market cap to pre-Wegovy levels not long after the victor became the first pharma company to top a $1 trillion valuation. It seems one company can do no right, while the other can do no wrong.
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The FDA approval of Alnylam’s Amvuttra sets up a three-way race with Pfizer and BridgeBio, which both market transthyretin stabilizers for transthyretin amyloid cardiomyopathy.
Paratek Pharmaceuticals is betting that OptiNose’s chronic rhinosinusitis treatment will be a partner to its antibiotic treatment Nuzyra.
After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent Project Muscular Dystrophy stepped up—as they always do.
The deal is the latest in a series of Sanofi investments in its immunology portfolio. According to Sanfoi, DR-0201 can achieve deep B cell depletion, giving it the potential to reset the immune system.
The Supreme Court last year blocked a previous settlement proposal from Purdue, arguing that the plan would afford the Sackler family too much protection.
A group of medical experts expressed concern about growing “disinformation” and “misinformation,” calling for adherence to recommended vaccine schedules.
Democratic senators from Georgia, Oregon, Maryland and New Mexico called the Trump administration’s decision to terminate hundreds of CDC staffers reckless and unfair.
Learn about making the most of interview feedback, navigating bonus clawbacks and networking for niche roles.
Nearly half of BioSpace poll respondents recently took a biopharma job they were overqualified for, a finding that didn’t surprise a talent acquisition expert, who said it’s become much more likely to happen.
After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.