gene-therapy

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.

The FDA has cleared Intellia Therapeutics to resume its MAGNITUDE Phase 3 trial of nexiguran ziclumeran for ATTR cardiomyopathy, five months after a patient death and subsequent clinical hold prompted a safety overhaul.

MagicRNA's mRNA lipid nanoparticle therapy reprogrammed patients' own T cells inside the body, wiping out disease-driving B cells in refractory lupus patients without conventional cell manufacturing.

The FDA rejected uniQure's Phase I/II data for AMT-130 and now demands a sham surgery-controlled Phase 3 trial, reversing its own prior guidance and sending shares down 40 percent.

UNSW and St. Jude researchers demonstrate that removing methyl tags from DNA can switch genes back on, opening safer paths for treating sickle cell disease.