Gene therapy, once dismissed as too dangerous, has made a comeback, with two products approved in the U.S. since December 2017 and hundreds more in the pipeline. STAT’s latest report takes a deep dive into a crucial component of these new treatments: the viral vectors used to deliver gene therapies to cells and organs.
As dozens of new gene therapies near the market, we spoke with academic experts, pioneers in the field, and executives with 18 companies, large and small, to identify the most important challenges surrounding the engineering of better vectors, their safety, effectiveness, efficiency, production, and cost — and how key players are thinking about overcoming those hurdles.
These engineered viruses are difficult to manufacture, particularly at the massive scale needed for some indications. Scientists are working hard to bring down the cost and speed up the process of making viral vectors, so that all the patients that could benefit from gene therapy will have access to it.
Beyond the introduction, this report has four major components:
— The basics of viral vectors and the history of their development;
— Major challenges in the development, manufacturing, and testing of viral vectors, and possible solutions;
— A close look at the status of gene therapies in 10 disease categories that are advancing through preclinical studies or are being tested in early-stage clinical trials;
— And perspective on the U.S. Food and Drug Administration’s approach to regulating viral vectors.
The report — “The STAT guide to viral vectors, the linchpin of gene therapy” — is intended for anyone with a strong interest in gene therapy, including biotech executives, investors, scientists, lawyers, policymakers, and patients and families interested in learning more. Our aim is to make the problems, stakes, and possibilities clear to everyone.
To buy the full report, please click here.