Cell and Gene Therapy
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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.
Be Biopharma’s terminated trial is the latest setback in the hemophilia space, where companies like Pfizer and BioMarin have opted to pull their respective products from the market after weak traction.
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.