Gene therapies are no longer a dream

In a recent article, Biopharma Reporter magazine highlights the significant advances that have occurred in recent years in the field of gene therapy, transforming it from a futuristic promise to a concrete reality with treatments already available. These represent a light of hope to combat diseases that, until now, were considered incurable. However, the biggest challenge we face is how to administer these therapies effectively.

Prof. Luigi Naldini, Director of the San Raffaele Telethon Gene Therapy Institute in Milan, Italy, who has been at the front line of gene therapy research for 25 years, emphasizes that it is crucial to develop precise delivery systems. These must ensure that treatments safely and efficiently reach the target cells, which would optimize therapeutic benefits and minimize the risks of side effects. Among others, nanoparticles stand out as one of the most promising candidates, although they still have a way to go in the development process.

The article also highlights the importance of close collaboration between the biotechnology sector and large pharmaceutical companies to overcome obstacles related to regulation, production and distribution. Only through these joint efforts can we ensure that innovative treatments reach the patients who need them.

It should be noted, regarding this topic, that Fundacion Noelia is working on three projects to develop different gene therapy administration systems, aware that this is one of the main obstacles we face. In particular, one of them focuses on nanoparticles, placing us in a leading position in the research of treatments for rare genetic diseases such as DMC-Col6.

You can read the entire article (in English) at

Luigi Naldini: Gene therapy no longer a ‘dream’ but big pharma must step up

07-Mar-2024 By Isabel Cameron

In the realm of modern medicine, few advancements have inspired and challenged the industry quite like gene therapy.https://www.biopharma-reporter.com/article/2024/03/07/luigi-naldini-gene-therapy-no-longer-a-dream-but-big-pharma-must-step-up