Tag: gene therapy
Marty Makary and Vinay Prasad embrace a “more fluid concept of evidence” at the FDA
Last Thursday during a roundtable on stem cell therapies, new FDA Commissioner Marty Makary referred to EBM levels of evidence as an artificial and dogmatic construct. Apparently Dr. David Katz's "more fluid concept of evidence" now reigns at the FDA—selectively.
Personalized CRISPR Gene Editing Therapy
While the medical world is melting down from the absolute apocalypse that is RFK Jr., it’s good to celebrate that (at least for now) medical progress continues to march on. Recently published in the NEJM is a case report of a breakthrough that we may look back on as a milestone in medicine. Patient-Specific In Vivo Gene Editing to Treat a Rare...
Deciding Which Risks to Take
No medical treatment is risk-free. Paul Offit's new book covers the history of innovations that went awry and advises how to balance the risks of new medical innovations with the risk of not treating.
The latest antivax false claim: mRNA vaccines against COVID-19 are not vaccines but “medical devices” or “gene therapy”
There's a new antivaccine talking point in town, and it's just as much disinformation as other antivaccine talking points. It's the claim that mRNA COVID-19 vaccines are not really vaccines but "medical devices," "gene therapy," or "experimental biologics" and that they were falsely classified as vaccines in order to bypass safety testing. Here, we discuss why this claim is utter nonsense based...
Would you pay $1 million to enroll in a phase 1 clinical trial of an “anti-aging” gene therapy?
Libella Gene Therapeutics, LLC made the news last week for announcing a "pay-to-play" trial of its telomerase-based anti-aging gene therapy. What was shocking about the announcement was not that it was a "pay-to-play" trial, given that such trials have become all too common, but rather the price of enrollment: $1 million. Worse, the trial is being conducted in Colombia; the therapy doesn't...
The Gene: An Intimate History
Six years ago I reviewed Siddhartha Mukherjee’s book The Emperor of All Maladies: A Biography of Cancer. It was hands-down one of the best books I have ever read on a medical topic. Now he’s done it again. His new book is titled The Gene: An Intimate History. Mukherjee is a superb writer. Much of what I said about his first book...