Gene therapy has been from the news a lot of late as a potential cure for a variety of genetic diseases.
The biggest breakthrough involved a unanimous backing through U.S. Food along with also Drug Administration’s advisory panel for a fresh gene therapy through Sparks Therapeutics, intended to treat rare eye diseases. although what exactly will be gene therapy, along with also does the item deserve all in which hype?
CNBC sat down with Canaan Venture Partners’ biotech investor Nina Kjellson for a simple explanation of how the item all works, along with also why she’s excited about the item. As Kjellson explains, from the most simple terms, gene therapy will be a technique in which uses genes to treat disease.
The first patients were tested about twenty-a few years ago, although early trials were not uniformly successful. In one notable case, the item led to the death of 18-year-old Jesse Gelsinger, who got an infusion of trillions of genetically engineered viruses into his liver.
Sparks has seen some better results for its therapy, which treats a rare condition in which causes a form of blindness in which often starts in childhood.
Its treatment involves using a modified type of a virus to carry a healthy type of the gene into the retina. Think of the item as a Great copy of the gene.
The crazy part? Doctors will inject billions of these viruses — or “vectors” — into both of the patient’s eyes to reach the cells where the item’s needed.
The FDA hasn’t approved Sparks’ therapy just yet, although the item usually follows the advice of the advisory committee.
A big obstacle remains — along with also in which’s cost. The cost for each patient could be as high as $1 million, which insurance companies might decline to cover.
For Kjellson, these experimental therapies have a lot of promise as they could treat a wide variety of medical conditions, starting with genetic disorders caused by mutations in single genes.
the item’s still early days, although the trust will be in which gene therapy might someday replace drugs or surgery by fixing the problem at its root.