GenSight Biologics gets early access nod for candidate gene therapy in Israel

Applications include detailed scientific rationale and supporting clinical evidence

Biohaven reports Phase 2 depression trial results

Despite some promising subgroup signals, the company indicated it will not pursue further psychiatric trials

INOVIO’s DNA therapy for rare throat disease gets FDA review

INOVIO remains confident in the therapy’s benefits and intends to meet with the FDA to discuss maintaining eligibility under this pathway

Senhwa Biosciences launches groundbreaking trial for HER2-positive tumours

CX-5461 is “the world's first and most advanced G-quadruplex (G4) stabilizer in development

SN Bioscience’s SNB-101 wins FDA orphan drug designation for gastric cancer

The drug, currently in a phase 1b/2 clinical trial for small cell lung cancer, is a polymer nanoparticle formulation of SN-38

ENHERTU gains first-ever nod in China for metastatic breast cancer

The approval follows results from the DESTINY-Breast06 phase 3 trial, which showed ENHERTU significantly extended progression-free survival compared to chemotherapy

US House passes Bill reauthorizing FDA rare disease incentive program

InnoCare Pharma gets green light for Phase II trial of oral lupus drug

CLE, a common form of lupus erythematosus, primarily causes diverse skin lesions

Symbiosis rolls out first 10,000 vial batch at Stirling Facility

This production milestone positions Symbiosis at the forefront of meeting rising global demand for life-saving medicines

FDA okays AQVESME as first treatment for anemia in alpha- & beta-thalassemia

The approval makes AQVESME the only FDA-approved therapy for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia