Signal
28 articles covering "gene-therapy"
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.
UT Austin and Metagenomi researchers engineered Al3Cas12f RKK, a compact CRISPR enzyme that achieves over 80% editing efficiency while fitting inside AAV delivery vehicles.
The first once-weekly treatment for achondroplasia is now commercially available in the United States after Ascendis Pharma secured orphan drug exclusivity through 2033.
The Boston biotech closes a Series D round to advance ER-100, the first FDA-cleared therapy that uses partial epigenetic reprogramming to reverse cellular aging in humans.
A decade after the first lab-grown brain models, organoid research is crossing from laboratory curiosity to clinical reality, prompting calls for governance frameworks to match the pace of science.
The FDA has accepted Ultragenyx's resubmitted BLA for UX111, a one-time gene therapy for the fatal childhood disease Sanfilippo syndrome Type A, with a decision expected by September 19, 2026.
The RUBY trial of renizgamglogene autogedtemcel (reni-cel), a one-time CRISPR-Cas12a gene-editing therapy, showed that 27 of 28 patients with severe sickle cell disease had no painful crises after treatment, with hemoglobin levels normalizing to 13.8 g/dL by six months.
Rocket Pharmaceuticals' Kresladi becomes the first gene therapy approved for LAD-I, an ultra-rare immune disorder that leaves children vulnerable to fatal infections.
A two-vector system inserts a CAR transgene at the TRAC locus inside the body, generating functional cancer-killing T cells without ex vivo manufacturing or lymphodepletion.
Basecamp Research launches the Trillion Gene Atlas, a two-year initiative to expand known genetic diversity 100-fold by sequencing over 100 million species with partners Anthropic, Ultima Genomics, PacBio, and NVIDIA.
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.
A Nature paper describes INSTALL, a gene editing platform using circular single-stranded DNA to insert therapeutic genes without triggering fatal immune responses, keeping mice healthy at doses where conventional DNA proved lethal.