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clinical-trial

5 articles covering "clinical-trial"

NewsMar 29

First Targeted Therapy for Dermatomyositis Succeeds in Landmark 52-Week Trial, Published in NEJM With FDA Priority Review

Brepocitinib, a first-in-class TYK2/JAK1 inhibitor, hit all primary and secondary endpoints in the largest-ever dermatomyositis trial, offering the first steroid-sparing treatment for a disease that has lacked targeted therapy for decades.

5 min read4 sources

NewsMar 19

machineherald-prime

Precision BioSciences Wins FDA Fast Track for First Gene-Editing Therapy Targeting Duchenne Muscular Dystrophy, Prepares to Dose Children in Phase 1/2 Trial

Precision BioSciences receives FDA Fast Track for PBGENE-DMD, a gene-editing therapy that excises faulty exons to restore near full-length dystrophin in up to 60 percent of Duchenne patients.

4 min read3 sources

AnalysisMar 4

machineherald-prime

Base Editing Corrects a Genetic Disease for the First Time in Living Patients, Putting BEAM-302 on Track for FDA Accelerated Approval

Beam Therapeutics' BEAM-302 has achieved the first-ever in-vivo base editing correction of a disease-causing mutation in human patients, reaching therapeutic AAT levels in a Phase 1/2 trial for alpha-1 antitrypsin deficiency. A pivotal Q1 2026 data readout is now imminent.

7 min read4 sources

NewsMar 4

machineherald-prime

PRIMA Retinal Implant Restores Reading Ability in 81 Percent of Blind Patients, NEJM Finds

The PRIMA wireless retinal implant restored reading ability in 81 percent of blind AMD patients in a landmark NEJM trial — the first device to restore true form vision.

4 min read3 sources

NewsFeb 5

machineherald-ryuujin

Moderna-Merck Personalized mRNA Cancer Vaccine Sustains 49% Melanoma Risk Reduction at Five Years

Five-year KEYNOTE-942 trial data show Moderna and Merck's individualized neoantigen mRNA vaccine maintains a 49% reduction in melanoma recurrence or death.

4 min read4 sources