Feb 20, 2023
Sandy Macrae, CEO of Sangamo Therapeutics, talks about the promise and limitations of genomic medicine to find the most effective and affordable treatments for patients with autoimmune and inflammatory diseases. Their CAR-Treg cell therapy platform allows T cells to be tracked throughout the body and activated to release chemicals and signals to reduce inflammation. Small molecule oral pills will always be used to treat diseases and vaccines to prevent them. The goal of genomic medicine is to eradicate the disease.
Sandy explains, "We co-opted what's a very natural process and attached a whole series of things to it. The zinc finger gets us to the right bit of the DNA, and then we can either cut the DNA. We can turn it down, turn it off, replace a base, or replace a chunk of it. That's the advantage of the zinc fingers. It's natural. It's small. It's easily delivered and has a whole range of functions that allows us to choose the right tool for the right disease."
"But there's going to be a growing space for diseases where there is a clear genetic influence or genetic participation in the disease, where a simple injection of a virus that takes the editing technology to the DNA can change the patient's course of their disease. I'm thinking of Sickle Cell Disease, where patients who were going into the hospital 10, 12, or 15 times a year are now not having any of those painful episodes. I'm thinking of Hemophilia, where they used to have to inject two or three times a week and have bleeding episodes, and they can now walk free and not worry about their bleeding."
"But really, the excitement within Sangamo is the next-generation projects, the Tregs that we just spoke about, and a whole platform of brain-active conscription factors that we're working on."
@SangamoTx #GenomicMedicine #GeneTherapy #CellTherapy #ZincFingers #Tregs #Tcells