Praxis Precision Medicines bypassed non-selective sodium channel toxicity in SCN2A and SCN8A DEEs by targeting persistent current. Now the FDA is examining its pivotal trial.
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KRAS sat on the undruggable list for decades. Silexion's SIL204 is built to silence the mutation earlier in the cell, and the Phase 2/3 program is now moving into patients.
FDA approved Lilly's Inluriyo plus Verzenio for ESR1-mutated ER+/HER2- metastatic breast cancer after endocrine progression, doubling median PFS to 11.1 months in EMBER-3. The clinical gain comes with 86% diarrhea and neutropenia rates, challenging early liquid biopsy switching models.
Ultragenyx's AAV9 gene therapy is the first treatment for Sanfilippo syndrome Type A. Full approval on 17 treated children against a natural history control now collides with a diagnostic window most families still miss.