Cell therapy
With promising early results, cell and gene therapies are making headway against both rare and common ocular and auditory diseases.
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.
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.
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.
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.
Cell therapy biotech Artiva Biotherapeutics plans to use the funds raised to support the development of its AlloNK therapy for systemic lupus erythematosus.
To improve its reviewers’ understanding of cell and gene therapy manufacturing, the agency has launched a program that will involve a tour of manufacturing facilities and daily workshops for its staffers.
Interius BioTherapeutics has received approval from Australia’s Human Research Ethics Committee to begin the first-in-human trial of an investigational in vivo CAR-T therapy designed to treat B-cell malignancies.
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