REGENXBIO said the FDA will consider existing longer-term data for NAVSUNLI under the accelerated approval pathway without requiring new studies, allowing the company to target a BLA resubmission in Q3 2026.
The company is working on iPSC-derived, AAV, and small molecule therapies for neurodegenerative, orthopedic, and blood-based disorders, with the help of OpenAI's models which seem to be accelerating life science research.
The funding will support Latus Bio’s lead gene therapy programs in Huntington’s and CLN2 disease while advancing its low-dose AAV delivery platform designed to make gene therapies safer, more scalable, and accessible...
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.
