Gene and Cell Therapies

Earlier this month we attended the 2022 World Medical Innovation Forum presented by Mass General Brigham ("MGB"), one of the best research hospitals in the world. This year's conference focused on the very exciting field of Gene and Cell Therapies ("GCTs").

What are GCTs?

Put simply, GCTs are treatments for diseases that use edited genes, modified cells, or both. Gene therapies aim to treat disease by replacing, inactivating, or introducing genes into cells, while cell therapies endeavor to do the same with cells that are first cultivated or modified (typically in a lab) and then delivered to the relevant part of the patient's body. Some therapies can use both techniques.

GCTs hold much promise. You may have heard us talk about how, in the future, various diseases - such as many forms of cancer - might be cured without the need for invasive surgery. Instead, patients might receive a cocktail of modified white blood cells or viruses which have been engineered to target the undesirable cancerous cells (and only those cells). Other technologies are being explored as paths to potential cures, but GCTs are at the forefront of the life sciences revolution. This short video summarizes GCTs and might very well be the best use of three minutes you will have all day. What might GCTs lead to? A conference panel called the Disruptive Dozen focused on twelve disruptive technologies that make use of GCTs. The twelve speakers were selected by a panel of one hundred MGB senior faculty (MGB is affiliated with Harvard) as pursuing innovation that is most likely to have a significant impact on healthcare. Here are three of our personal favorites. The links are to short, two-minute videos summarizing each research project. 1. Regrowing Cells in the Inner Ear to Treat Hearing Loss Converting existing ear cells into so-called hair cells, which are damaged with age and exposure to loud sounds. The human body is unable to repair hair cells on its own, which in turn leads to progressive (and currently irreversible) hearing loss. 2. Harnessing the Power of RNA to Treat Brain Cancer Using RNA, best known for its role in the Covid 19 vaccines, to potentially cure glioblastoma. This is a highly lethal brain tumor that has killed two of our former bosses as well as various famous individuals like Ted Kennedy and John McCain. Interestingly, this form of cancer may be more common in highly educated or high-functioning individuals. 3. Cell Therapies to Conquer Common Forms of Blindness Restoring sights to blind patients by replacing cells that have been lost or injured with retinal stem cells from healthy donors or reprogrammed corneal stem cells from the patient himself. You can see the other finalists here.