Vertex Pharmaceuticals and CRISPR Therapeutics' gene-editing drug is the only sickle cell disease gene therapy on the market for young children.

The Phase I/II China-based trial will test the combination in patients with PD-L1-positive, EGFR mutated tumors.

In a Phase III trial, divarasib bested first-generation drugs Lumakras and Krazati, which the firm said could make it a new standard of care in KRAS G12C-mutated NSCLC.

The European Commission has approved Novartis' gene therapy Itvisma (onasemnogene abeparvovec) for patients at least two years old with 5q-associated spinal muscular atrophy (SMA) and biallelic SMN1 ...

Despite fears of an innovation slowdown, stoked by funding cuts and FDA personnel shakeups, the agency approved 17 ...

During its research day, the firm discussed a cell therapy effort in lupus, dubbed "007," and an AI platform for gleaning ...

The firm will submit a rolling NDA to the agency in early 2027, containing data from the randomized, controlled EMPEROR trial.

The firm has raised $78 million to date and is launching its first clinical trial to test a treatment for ...

The firm is preparing to test a gene replacement therapy that uses an ultrasound-mediated strategy to deliver a full-length ...

The T2EVOLVE association cements the work of a temporary Innovative Medicines Initiative-funded project that began in 2021.

The agency expects to issue decisions on Vyondys 53 and Amondys 45, which missed their primary endpoint in a confirmatory trial, by February 2027.

The program will help make genetic counselors available to any provider across different medical specialties in Tennessee and ...