Drug Development

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The prevalence of serious inflammatory safety issues such as cytokine release syndrome and immune effector cell–associated neurotoxicity syndrome limits the reach of these transformative cancer therapies.
After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told BioSpace, with both rare and chronic diseases headlining investor and R&D interest as JPM26 kicks off.
Recent breakthroughs and three decades of progress in treating Huntington’s disease
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AVROBIO is planning a Phase II/III registrational trial for its Gaucher Disease Type 3 (GD3) gene therapy candidate, AVR-RD-02, in 2023.
Neurocrine’s NBI-827104 failed to meet the primary endpoint in a Phase II study for epileptic encephalopathy with continuous spike-and-wave during sleep, the company announced Tuesday.
Eli Lilly and Boehringer Ingelheim declared another victory Wednesday for Jardiance – this one for children and adolescents with Type 2 Diabetes.
Findings from the Phase II ARTEMIS-UC and Phase IIa APOLLO-CD trials show that Prometheus’ PRA023 is potentially safe and effective for treating ulcerative colitis and Crohn’s disease.
Novartis announced that its Kisqali with endocrine therapy offered almost a year of progression-free survival (PFS) for an aggressive form of HER+/HER2- metastatic breast cancer.
Apogee Therapeutics sprang onto the immunology/inflammatory scene Wednesday heavy on capital but light on salient details of the pipeline.
Editas Medicine released positive proof of concept data Tuesday morning from the first patients dosed with its experimental CRISPR-based gene therapy for sickle cell disease.
Topline results from the Phase II TORREY study showed Gossamer Bio’s seralutinib met its primary efficacy endpoint in pulmonary arterial hypertension.
Mirati’s adagrasib demonstrated a solid safety profile in new data released Monday. Despite this, the company’s stock fell 8% in after-hours trading.
Monday, Verve Therapeutics revealed that the FDA has officially requested several buckets of additional safety information to lift the hold on its lead candidate, VERVE-101.