Regeneron said it will reduce Medicaid and future U.S. drug prices in line with other developed countries, enable direct discounted purchases of Praluent through TrumpRx.gov, and continue investing in domestic R&D and...
The recommendation is backed by Phase III data showing that Novartis’ one-time gene therapy improved motor function in people aged two and older with SMA and could lessen reliance on ongoing chronic...
The agreement gives Adicet access to MaxCyte’s technology for developing scalable, non-viral gene-edited cell therapies.
Researchers are testing a novel CRISPR-based therapy for FSHD, with participants monitored for safety and long-term outcomes.
Klotho Neurosciences’ gene therapy is advancing plans for ALS clinical trials next year.
Researchers are exploring whether SIRT1 gene therapy can protect human retinal cells from damage linked to optic neuropathies.
With FDA approval secured, Ensoma prepares to advance its groundbreaking in vivo therapy into clinical trials targeting X-CGD patients.