Barrie Carter, president of the American Society of Gene and Cell Therapy, and owner and manager of Carter BioConsulting, hails the resurgence of gene and cell therapy in an Xconomy piece. One reason there is a renewed acceptance after some high-profile failures a few years ago: improved delivery methods.
"Currently, the gene delivery systems are being modified to decrease the risk of clonal expansion leading to leukemia in some cases that is an undesirable side effect," Carter writes. "Several groups tested gene therapy for an inherited eye disease caused by a mutant gene leading to retinal degeneration and blindness. A viral vector placed under the retina gave substantial gains in retinal sensitivity and improvement in visual mobility. These advances in patients with rare, inherited genetic diseases provide proof of concept for gene therapy."
Carter cites successes in gene and cell therapies for complex multigenic and acquired diseases such as cancer, in addition to heart failure and Parkinson's disease. Development of technologies such as siRNA delivery continues, as well.
One point Carter makes near the beginning is that gene and cell therapies are right on schedule when it comes to standard biotech clinical and developmental timelines. Most technologies take 15 to 20 years of research development and clinical trials before they're ready for prime time.
"With the exciting results accruing in the clinic and the strong interest from Big Pharma and biotech players, hopefully investors will not be far behind," Carter writes.
- read the piece in Xconomy