Drug Development
As multiple companies vie to expand on Alnylam’s success in commercializing RNAi therapeutics, the pioneering company has set a goal of targeting small interfering RNA to any tissue by 2030.
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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.
AI is enabling the development of a next generation of drugs that can more precisely target cancer cells while sparing healthy tissues.
As the biopharma industry grapples with the uncertain macro environment brought on by the new administration, CEOs, regulators and many others speak out.
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Analysts were effusive about Merus’ new HNSCC data, writing that petosemtamab could “become the standard of care” in the first-line setting for this indication.
In this episode presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses the importance of target product profiles, particularly when navigating funding challenges, with Ian Fisher, head of development analytics.
From a higher bar for regulatory clearance to pricing limitations, drug development is more expensive than ever. This has led firms to make tough pipeline decisions early in the development process. The result may be costly for all of us.
Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to the most-rare conditions, there isn’t even a pipeline.
The late-stage results come in advance of pivotal data that Ionis expects to provide for its antisense oligonucleotide Tryngolza in the third quarter, building up toward a regulatory submission in hypertriglyceridemia by year-end.
In a year when eradicated diseases are on the uptick in America, how will American children survive RFK Jr.’s vaccine scrutiny and inconsistency? Two experts call on pharma and regulatory bodies to rebuild trust.
A new generation of checkpoint inhibitors is emerging, with some showing more promise than others. From recent TIGIT failures to high-potential targets like VEGF, BioSpace explores what’s on the horizon in immuno-oncology.
Govorestat failed to meet its primary endpoint in a Phase II/III trial for a rare form of Charcot-Marie-Tooth disease, a few months after the FDA rebuffed the same drug in a similar indication.
Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a personalized in vivo CRISPR editing therapy, which substantially eased the symptom burden in an infant.
The FDA and NIH recently announced plans to phase out animal testing requirements for some therapies. While organoid and AI providers celebrate, scientists warn that questions over safety, applicability and implementation remain.