New research on CRISPR techniques that could provide treatments and improved diagnostics for several major viruses and other medical conditions is pushing valuations higher in the gene editing industry. Ailments like Cancer, HPV, and HIV are increasingly being targeted with an increasingly sophisticated CRISPR toolbox that has already managed to beat the street when commercialized by companies like Novartis.
At the end of October, MRP covered the latest developments in CRISPR “Prime Editing”, as well as the commercial successes of new CRISPR treatments being offered by Novartis and Spark Therapeutics. But the world’s foremost gene editing tool continues to heat up with new research and stock prices alike experiencing a breakout November.
The first attempt in the United States to use CRISPR against cancer seems safe in the three patients who have had it so far. The team of researchers from UPenn’s Perelman School of Medicine, backed by Tmunity Therapeutics, procured T cells from three cancer patients — two with multiple myeloma and one with sarcoma — through a blood draw, and genetically modified the cells’ DNA using CRISPR. They inserted a gene from a virus into the immune cells that causes the cells to target the protein NY-ESO-1, found on cancer cells, and silenced three genes within the cells that could interfere with their cancer-fighting ability. Two of the gene edits inactivate TCRα and TCRβ, causing T cell receptors to be removed. Without the receptors, the cells can more easily bind to cancer cells. The third edit disables PDCD1, a gene that can kill T cells. The researchers then infused the cells back into the patients, who have since had no significant adverse effects after six months.
In short, the treatment deletes three genes that might have been hindering these cells’ ability to attack the disease with minimal and manageable side effects. This study is not aimed at changing DNA within a person’s body. Instead, it seeks to remove, alter and give back to the patient cells that are super-powered to fight their cancer — a form of immunotherapy.
While the three patients have not yet improved as a result of the treatment, the study only sought to find if T-cells could indeed be safely edited and re-introduced to patients’ bodies, a first step in the process of developing the treatment. The patients were treated in January, April and August, with the first patient being followed for at least six months. The cells appear to be doing what scientists hoped they would do without producing any significant adverse effects, the researchers say…