Widely used as a genome editing tool, the CRISPR-Cas9 system allows researchers to precisely induce frameshift mutations in specific genes or insert foreign nucleic acid sequences into a cell’s DNA.

The BRILLIANCE study of EDIT-101 in Leber congenital amaurosis type 10 (LCA10) – an inherited form of blindness – was reported to be the first 'in vivo' CRISPR/Cas9 medicine to be administered ...

VERVE-102 targets the PCKS9 gene as VERVE-101 does. However, it uses a different lipid nanoparticle delivery method. Verve is arguably the riskiest of these five CRISPR stocks. However ...

CRISPR is a technology that can be used to edit genes and, as such, will likely change the world. The essence of CRISPR is simple: it’s a way of finding a specific bit of DNA inside a cell.

LETI-101 has demonstrated therapeutically relevant reductions of mutant huntingtin (HTT) protein, exceeding 80%, while preserving essential ...

ElevateBio is seeking out partnerships for its lead gene therapy after the construct reduced the levels of a toxic protein in ...

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CRISPR Therapeutics and Vertex Pharmaceuticals ... BEAM is developing its lead pipeline candidate BEAM-101 in SCD indication in the phase I/II BEACON study. Beam Therapeutics is also advancing ...