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After a patient safety signal and then death, the FDA in October 2025 placed holds on two of the company’s CRISPR programs for hereditary transthyretin amyloidosis.
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Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.
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The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
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While at SCOPE 2025, Sam Srivastava, CEO at WCG Clinical discusses the challenges and responsibilities of the life sciences industry in building public trust amidst growing anger towards healthcare.
ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, Niemann-Pick disease type C, metachromatic leukodystrophy and more.
As it did during the COVID-19 pandemic, mRNA technology offers an efficient way forward in developing products for diseases that lack approved treatments.
The companies were two years into a four-year, $400 million agreement aimed at developing and marketing gene therapies together.
One of the lowest paid CEOs in pharma—and one of the only woman leading a top-tier giant—is set to receive up to $27.2 million in 2025.
As they navigate a competitive job market, biopharma professionals are making four key interview mistakes, according to two talent acquisition experts. They discuss those errors and offer tips for how to get those critical conversations right.
In this episode of Denatured, BioSpace’s Head of Insights Lori Ellis and Miruna Sasu, CEO of COTA, discuss life sciences investment and the potential for disruption.
While Kallyope’s drugs are mechanistically unique, the biotech is competing in a crowded space, with other therapies that appear to elicit superior weight-loss.
The company will push through with an accelerated approval application for odronextamab in follicular lymphoma, leaving diffuse large B cell lymphoma behind.
The move comes after President Donald Trump warned Big Pharma leaders that he would impose tariffs on them if they refuse to reshore their manufacturing operations.