Gene therapy
The FDA approved the expansion of Casgevy, which had previously been greenlit for patients 12 and up, into a younger pediatric population under the agency’s Commissioner’s National Priority Voucher program.
Teams at facilities being developed by Eli Lilly, Regeneron and other companies will receive early technical guidance and additional perks from the FDA.
A few short days after announcing an FDA pivot on a separate asset, REGENXBIO is planning to test the agency’s apparent newfound rare disease outlook on another late-stage gene therapy.
If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
Instead of using viral vectors, SonoThera’s genetic medicines are delivered through an ultrasound-mediated technology that could help sidestep key safety issues with conventional delivery methods.
Analysts homed in on Duchenne muscular dystrophy and myotonic dystrophy type 1 assets during first quarter earnings as major players like REGENXBIO and Novartis as well as Dyne, Wave, Solid and Sarepta near the regulatory finish line.
Eli Lilly joins hands with Engage Bio, acquiring the DNA delivery platform developer in hopes of bolstering its genetic medicines portfolio.
The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, BioSpace reviews genetic medicines in development for the central nervous system, retinal, cardiac and neuromuscular diseases.
Shares of REGENXBIO declined 37% on a mixed data readout and other updates from the company’s first quarter earnings call Thursday.
In this episode of Denatured, you’ll be hearing from Miguel Forte, president of the International Society for Cell and Gene Therapy (ISCT), and Jon Ellis, co-founder & CEO of Trenchant Bios, speaking live from the ISCT annual meeting. We dive into mesenchymal stem cells and induced pluripotent stem cells, exploring the science behind them, the manufacturing challenges, and the potential for scalable, engineered next-generation therapies.
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