Drug Development
The Inflation Reduction Act includes an exemption for orphan drugs for a single indication, but experts say this is far from sufficient to maintain momentum in the rare disease space.
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Vocal skeptics of COVID-19 vaccinations gave mRNA a bad name and government funding for mRNA research is now being cut. On the flip side, at least one CEO said the pandemic also provided “elevated acceleration” for the field, which also holds promise in therapeutics for cancer and rare diseases.
Beginning this week in Chicago, the American Association for Cancer Research’s annual conference will feature presentations that could have far-reaching implications for breast and blood cancers and more.
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.
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The Seattle-based company came to ASCO25 with new data on its neuroendocrine tumor–treating lead therapy, with big vibes and speedy speech.
Otsuka’s investigational antibody sibeprenlimab approximately halved levels of this key biomarker in patients with immunoglobulin A nephropathy.
Analysts at Truist Securities called the mid-stage data a “mixed bag,” also flagging gastrointestinal adverse events. However, the readout is unlikely to be “incremental” to Corcept’s overall stock narrative.
Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according to BMO Capital Markets analysts.
In this episode of Denatured, presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses how AI transformation can help organizations navigate a rapidly evolving regulatory environment with senior director of regulatory innovation and technology, Michelle Gyzen.
Analysts reacted positively to the news that uniQure is in alignment with the FDA on an accelerated approval pathway and on target for a Q1 2026 submission for its one-time gene therapy for Huntington’s disease—but patients have been here before.
The Platform Technology Designation, which predates the current FDA leadership, is designed to streamline the drug development and review process, particularly for rare diseases.
Disappointing results for iluzanebart come shortly after Vigil Neuroscience struck a buy-out deal with Sanofi, but analysts say the outcome is unsurprising and shouldn’t affect the deal.
J&J has a multi-year head start, but Gilead believes it can win market share by delivering a drug with better safety and at least as good efficacy.
Kura Oncology won FDA priority review for its drug the day before announcing new data at ASCO 2025 showing remission in about one-quarter of patients.