The FDA has accepted Nanoscope Therapeutics’ BLA for MOGENRY, an optogenetic therapy designed to restore useful vision in severe retinitis pigmentosa regardless of the underlying mutation.
Arlocabtagene autoleucel met response and complete-response endpoints in patients already exposed to immunomodulators, proteasome inhibitors, anti-CD38 treatment and BCMA therapy, positioning GPRC5D as a possible next CAR T target after increasingly crowded myeloma treatment sequences.
Once-daily deucrictibant XR reduced hereditary angioedema attacks by 83% across the Phase 3 CHAPTER-3 population, giving Pharvaris a potential oral prophylaxis product alongside its separately developed immediate-release rescue formulation.
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.
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.