Genomics

Bezuclastinib hit a 65% objective response rate in advanced systemic mastocytosis; Cogent plans to file its NDA in June 2026.

OraSure's Colli-Pee Dx kit, cleared for use with Roche cobas systems, enables self-collected urine testing for four STIs from home.

Novartis says its AOC therapy del-brax hit biomarker endpoints in the FORTITUDE study, with Phase III now enrolling 200 FSHD patients.

The Barcelona biotech has dosed its first participants in a 30-patient study of TrkB modulator CTH120, the only compound in late-stage development for

The genomic medicine company is weighing a sale or partnership as it advances a BLA-ready Fabry gene therapy and two CNS programmes.

The Cambridge biotech has initiated its pivotal NORTHSTAR study of oral gildeuretinol in Stargardt disease, with no approved treatment currently available.

The designation is backed by Phase 1b data showing motor function gains in children who had a suboptimal response to prior gene therapy.

Blood publication of KOMET-007 data shows deep, durable responses for ziftomenib plus venetoclax/azacitidine in NPM1-mutated relapsed/refractory AML.

Phase 2 CRDF-004 data presented at ASCO 2026 support a registrational Phase 3 trial after FDA End-of-Phase 2 alignment.

Artios Pharma has started its randomised Phase 2 POLKA study of Pol-theta inhibitor ART6043 plus olaparib in gBRCA-mutated HER2-negative breast cancer.

Madrigal's MASH therapy Rezdiffra has crossed blockbuster sales thresholds as the company expands its pipeline with a licensed siRNA targeting PNPLA3.

Madrigal Pharmaceuticals has licensed ARO-PNPLA3 from Arrowhead, targeting a common genetic driver of MASH prevalent in Hispanic patients.

Peer-reviewed results show buntanetap improved cognition in pTau217-positive early AD patients, with biomarker reductions suggesting potential disease-modifying activity.