Signal
23 articles covering "gene-therapy"
Scribe's ELXR platform silences PCSK9 in the liver without cutting DNA; a single low dose sustained LDL reductions for roughly 18 months in primates.
Eli Lilly acquired Engage Bio and its Tethosome non-viral DNA delivery platform for up to $202 million, aiming to overcome the immune and redosing limits of viral gene therapies.
Fractyl Health received authorization in the Netherlands to run the world's first clinical trial of an AAV-based GLP-1 gene therapy for type 2 diabetes, targeting patients who cannot sustain daily injections.
An AAV9 gene-regulation therapy delivered durable, dose-dependent seizure control and early signs of neurodevelopmental rescue through 52 weeks, just as the pivotal study begins dosing.
Regenxbio's gene therapy RGX-202 met the primary endpoint of its pivotal Phase III AFFINITY DUCHENNE trial, with 93% of participants reaching the microdystrophin expression threshold.
Regeneron's one-time intracochlear gene therapy for OTOF-related deafness cleared the FDA 61 days after BLA filing, the first gene therapy product approved under the Commissioner's National Priority Voucher pilot program.
Intellia Therapeutics will report topline Phase 3 results for lonvoguran ziclumeran in hereditary angioedema on April 27, 2026 — the first Phase 3 readout for any in vivo CRISPR candidate.
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.