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ETX101 shows promising early signals in Dravet syndrome

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Recent interim Phase 1/2 POLARIS results suggest ETX101, an investigational one-time gene therapy for children with SCN1A-positive Dravet syndrome, may reduce seizures and support developmental progress. The findings are promising but preliminary; ETX101 is not an established treatment. In an update reported in September 2026, participants who completed 52 weeks had median reductions in monthly countable seizure frequency of about 79% at dose level 3 (3 participants) and 89% at dose level 4 (5 participants). Across participants at those dose levels with longer or shorter follow-up, median reductions were about 76% and 60%, respectively. For children treated before age 2, cognitive assessment scores continued to rise, with reported developmental trajectories approaching the range expected for neurotypical children. Improvements were also reported on adaptive-behavior measures, including communication and daily living skills. These comparisons do not establish that ETX101 caused deve...

Sanofi rules out major restructuring as it prepares for Dupixent’s patent loss

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FDA approves camizestrant combination for ESR1-mutated advanced breast cancer

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Recent Alzheimer’s developments across France, Germany, Italy and Spain

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Actio’s remaining drug pipeline moves to Setara after Jazz buyout

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Lilly raises 2026 outlook as GLP-1 demand and Medicare Bridge access boost sales

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AstraZeneca targets $1.5B in annual savings to reinvest in its pipeline

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Sanofi weighs selective “moonshot” deals as it plans for the Dupixent patent cliff

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Dren Bio raises $350 million to advance rare blood cancer drug

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Flagship combines three biotech platforms to launch AI drug-discovery company Etcelera

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Aspen raises $141M and Bambusa brings in $169M in biotech IPOs

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FDA draft proposes a streamlined route for some patient-specific gene-editing therapies

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Oura and UC San Diego Health launch smart-ring study of obesity treatment outcomes

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