Gene therapy: The breakthrough that inspired Spider-Man | Euronews Tech Talks
Although an inspiration for science fiction movies, gene therapy is real. Here is how it works and what could be improved about it.
Gene therapy, a medical approach that targets genetic causes of diseases, has recently made its way into the Marvel superhero film, Spider-Man: Brand New Day. In the movie, Spider-Man experiences DNA mutations leading to new and uncontrollable powers. To manage these abilities, he utilizes RNA interference, a real-world gene therapy technique that suppresses particular gene activity.
To delve deeper into the workings and limitations of gene therapy, Euronews Tech Talks conducted an interview with Alberto Auricchio, director of the Telethon Institute of Genetics and Medicine in Naples and president of the European Society of Gene & Cell Therapy.
Auricchio explained that gene therapy has been primarily utilized to treat monogenic diseases, and is now being studied to find treatments for various illnesses, such as cancer and HIV. The therapy functions by introducing nucleic acids, such as DNA and RNA, into target cells and tissues for therapeutic purposes. These nucleic acids can be administered either in vivo, within the body, or ex vivo, in cells extracted from an organism and subsequently transplanted back into the body.
Delivery methods can be achieved using either viral or non-viral vectors, and gene therapy can be categorized into gene addition and gene editing, with gene silencing and gene correction being further subtypes.
Although gene therapy is no longer limited to clinical trials, Auricchio noted that there are already approved gene therapy products available to patients. The first in vivo gene therapy approved in Europe is Glybera, which treats severe or multiple pancreatitis attacks due to lipoprotein lipase deficiency, and the first ex vivo gene therapy approved in Europe is Strimvelis, used to treat severe combined immunodeficiency due to adenosine deaminase deficiency.
Gene therapy is considered promising due to its durability, as it is designed as a once-in-a-lifetime treatment, with decades-long stability in therapeutic effects for patients.
However, despite its scientific potential, the main limitation of gene therapy lies in its high cost, making it economically challenging. Auricchio highlighted that these drugs are expensive to develop and often come with high prices, some reaching millions of euros. For example, Strimvelis costs €594,000 excluding VAT, posing a significant challenge for pharmaceutical companies, especially when targeting rare diseases, which are not typically attractive markets for traditional drug developers.
Written by urgent.news from Euronews's reporting — not their text. Machine-written — may contain errors; check the original before relying on it.
