Pulmovant’s mosliciguat produced unusually large improvements across pulmonary vascular resistance, exercise capacity and cardiac strain in PH-ILD, giving Roivant a strong Phase 2 hand as the much larger PHrontier study tests whether those effects hold.
Pivot has FDA-cleared software upgrades and national PBM access. Modular Medical now has to prove that simpler insulin delivery converts users.
Zanvastro finally gives Alexander disease a targeted therapy. Its next challenge is finding patients and funding a $1.14 million annual regimen.
Etcamah can be started before visible progression when ESR1 resistance emerges. The bigger change may be how often breast cancer is tested.
An investigator-led U.S. trial will combine elranatamab with Starton Therapeutics’ investigational controlled low-dose lenalidomide in relapsed or refractory multiple myeloma.
CorrectSequence’s transformer base-editing platform produced sustained fetal hemoglobin above 60% and freedom from vaso-occlusive crises in a Nigerian sickle cell patient, while three additional thalassemia patients became transfusion independent.
Rznomics researchers report an optimized self-targeting and splicing system that doubled circularization efficiency versus conventional PIE for a roughly 7.8 kb Factor VIII RNA payload.
Isomab’s review of 11 historical therapeutic-angiogenesis studies found similar malignancy rates with pro-angiogenic treatment and placebo, supporting the safety rationale for preclinical ISM-001 without proving the new antibody is safe.
Teleflex is highlighting BIONETIC-TRA data showing 100% procedural success with the Dynetic-35 stent through radial access in 25 patients with iliac artery disease.
Aptar Pharma’s Nanopharm will develop the formulation and inhalation strategy for Aceso Therapeutics’ preclinical ACT-101 antisense drug, which aims to stabilize CFTR mRNA rather than edit DNA.