SpliceBio, a clinical-stage genetic medicines company pioneering protein splicing to address diseases caused by mutations in large genes, today announced that the first patient has been dosed in the ...

ElevateBio Life Edit (R) is the Company's gene editing technologies and R&D business, focused on building the world's largest gene editing toolbox to expand addressable diseases and open a new ...

LYNX-3, the second pivotal Phase 3 trial in keratorefractive participants with visual disturbances under mesopic, low-contrast conditions, is ongoing with topline results expected in the first half of ...

Positive interim efficacy data in 2025 underscore potential of ETX101 as a one-time, disease-modifying medicine for Dravet syndrome –– Internal GMP manufacturing capable of supporting pivotal trial ...

Precision and timing of gene expression is essential for normal biological functions and, when disrupted, can lead to many ...

Inducible promoters provide remarkable utility when sustained transgene expression compromises plant development or agronomic performance.

A giant functional atlas of immune cells in a Chinese cohort reveals crucial differences with European and Japanese data sets ...

The conservation of genome regulatory elements over long periods of evolution is not limited to vertebrates, as previously ...

Lexeo says the collaboration will accelerate the development of an unnamed preclinical cardiac gene therapy target.