Cell and Gene Therapy
THE LATEST
Longeveron and Lexeo Therapeutics are working on CGT therapies to treat Alzheimer’s disease, but it’s not clear whether they have a better chance of success than traditional approaches.
Ultracompact CRISPR systems, which are in some cases one-third the size of Cas9, are being designed to be more specific and enable in vivo gene editing in difficult to reach tissues.
Approved under the regulator’s accelerated pathway, Tecelra is also the first new synovial sarcoma therapy in more than a decade, according to Adaptimmune Therapeutics.
Sarepta has been hit with another patent infringement lawsuit, this time from Sanofi and its subsidiary Genzyme alleging that the biotech used protected technology related to AAV vectors.
The advantages of using circular RNAs—including increased durability, enhanced protein expression and substantially lower manufacturing costs compared to linear mRNAs—have driven a spate of investment in this technology.
Fatalities are an unfortunate reality of clinical trials. How can companies best protect themselves?
After more than a decade devoid of therapeutic advancements, a first-in-class T cell receptor therapy could be on the immediate horizon for synovial sarcoma patients.
The company’s candidate, giroctocogene fitelparvovec, met its primary and key secondary objectives of superiority compared to the standard treatment of regular Factor VIII infusions.
The Department of Health and Human Services’ Office of the Inspector General found that bluebird bio’s fertility support program for its gene therapies could potentially violate federal anti-kickback statutes.
After a long and challenging journey for its stem cell therapy NurOwn, BrainStorm Cell Therapeutics has aligned with the FDA on the parameters of a Phase IIIb ALS trial that is expected to begin by the end of 2024.