Cell and Gene Therapy
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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.
All six non-Hodgkin lymphoma patients on Legend Biotech’s CAR T therapy responded to treatment—findings that could make the biotech an attractive takeover target, according to analysts at Oppenheimer.
For Peter Pitts, a former associate commissioner at the FDA, the appointment to the board of BrainStorm Cell Therapeutics is an opportunity to fulfill a promise he made long ago to a patient with ALS.
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.
CREATE Medicines is working on a clinical-stage pipeline for cancer, while its autoimmune programs are still in preclinical testing.
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.
The FDA turned away Ebvallo in January, taking issue with the design of the registrational trial. In a recent meeting, however, the agency agreed that the study could in fact support the cell therapy’s approval. The news comes a week after the departure of controversial biologics Director Vinay Prasad.