genetics

ARPA-H's new THRIVE program is funding seven teams to build reusable gene-editing platforms for rare pediatric diseases, aiming to move beyond one-patient-at-a-time therapies like the one made for infant KJ Muldoon.

University of Minnesota researchers built SpudCell, a 90-kilobase synthetic cell that feeds, grows and divides, though outside scientists say it is not yet alive.

Base editing knockout of NANOG in human embryos shows it is essential for the epiblast, the tissue that forms the body, arriving alongside a separate disease-gene editing study that has renewed debate over heritable genome editing.

Biallelic RNU2-2 variants cause a newly characterized neurodevelopmental disorder that researchers estimate accounts for roughly 10 percent of recessive NDD cases with a known genetic cause.

Early-phase data in 81 children with Dravet syndrome showed large seizure reductions and manageable safety issues as a phase 3 trial moves ahead.

Genome-wide analysis of 11,000-year-old remains from the Donghulin site near Beijing has identified a deeply divergent northern East Asian lineage with no match in any previously studied population, offering new evidence that cultural continuity does not guarantee genetic continuity.

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.