Genetically engineered immune cells target cancer cells that may be responsible for leukemia relapse
Genetically modified immune cells successfully target specific cancer cells that may be responsible for the relapse of acute myeloid leukemia (AML). In a study published on 28 April in Nature Communications, the researchers developed a CAR T cell therapy (UCART123) targeting CD123, which is found on leukemia stem cells and enables T cells to seek out […]
PD-1 Blockade in Mismatch Repair–Deficient, Locally Advanced Rectal Cancer | NEJM
Breakthrough findings were presented at the 2022 ASCO Annual Meeting and published in The New England Journal of Medicine by researchers at Memorial Sloan Kettering Cancer Center (MSK) confirming a clinical complete response in all 14 patients who received the immunotherapy treatment dostarlimab as a first-line treatment for mismatch repair-deficient (MMRd) locally advanced rectal cancer. This new approach of “immunoablative” […]
Gene therapy’s comeback: how scientists are trying to make it safer – Nature
It is a pity that non integrating lentiviral vectors are unknown or underused. This is not THE solution but in several cases, I think their use would be more relevant than AAVs use in term of immune response. The advancements of gene therapy will go through the design and development of complementary vector platforms.
CRISPR/Cas9 deletions induce adverse on-target genomic effects
In a recent study, a team of researchers used a CRISPR/Cas9 system to evaluate the use of tRNA by deleting two tRNA genes from the genomes of hyperhepatocellular carcinoma and human chronic myeloid quasi-haploid leukaemia cells. The authors discovered numerous unexpected genomic changes at the target region using an improved droplet-based target enrichment approach followed […]
UCSF researchers hope to cure sickle cell disease through CRISPR gene editing
With a $17 million grant, UCSF Benioff Children’s Hospital in Oakland will launch a four-year trial evaluating CRISPR/Cas9 gene editing as a potential cure for sickle cell disease. The trial is the first to evaluate the use of non-viral CRISPR gene editing tools to correct a harmful mutation in the beta-globin gene in a patient’s […]
FDA grants fast track designation to CD33-directed CAR-T for acute myeloid leukemia
The FDA has granted accelerated designation to PRGN-3006 for the treatment of adults with relapsed or refractory acute myeloid leukemia. PRGN-3006 (Precigen Inc.) is a CAR T cell therapy that targets the CD33 protein on the surface of cancer cells. This investigational therapy, designed to reduce manufacturing time to 1 day, is based on Precigen’s […]
Nasal Spray Sends CRISPR to the Brain
Mucopolysaccharidosis I is caused by mutations in the gene for the enzyme alpha-L-iduronidase (IDUA). This enzyme is required for the breakdown of substances called glycosaminoglycans (GAGs) that are by-products of chemical reactions in the body’s cells. In 2018, a team of researchers was able to demonstrate the feasibility of CRISPR gene editing in vivo in […]
Rapid manufacturing of non-activated potent CAR T cells | Nature Biomedical Engineering
In the process of engineering CAR T cells, if the patient’s T cells are out of the body for too long, they can lose their ability to replicate, which is critical to their effectiveness as a live drug. Thus, a research team has developed a new approach that reduces the time needed to modify patients’ […]
Bioinstructive implantable scaffolds for rapid in vivo manufacture and release of CAR-T cells | Nature Biotechnology
Many people are excluded from CAR T cell-based treatments because of its cost. One reason for the high cost is that the manufacturing process is complex, time-consuming and must be individually tailored to each cancer patient. So to address this challenge, the researchers created a biotechnology called Multifunctional Alginate Scaffolds for T cell Engineering and […]
Researchers develop new precision medicine technology for cancer immunotherapy
CAR T-cell therapy and antibody treatments are two types of targeted immunotherapies that have revolutionized the fields of cancer care. However, there are still significant challenges in identifying cancer cell surface proteins as targets for immunotherapies. A research group at Lund University in Sweden is well on their way as they have developed a new […]
