EBMT 2026 | The need for regulatory innovation in the gene therapy field

The message is that really the technology is here. We have the ability to cure so many rare and ultra-rare diseases with the use of gene addition, with the improved lentivirus or even non-lentiviral vectors, or with the gene editing. And it is our ethical obligation as a physician and healthcare provider to advocate for these patients. But what we are seeing is that in immunodeficiency, in metabolic disease, many companies that have proven that this is clinically useful and effective, they don’t see a way for commercialization. So what I was excited to hear in the gene therapy plenary session, is that we need advances in regulatory frameworks. We need our regulatory bodies to think beyond the 21st century. They need to be able to match the advances in technology and medicine, and maybe they can approve gene therapy as a platform instead of per disease. Number two, it is really that we need also advances in the way it’s paid. So instead of having a company have to really get the R&D, get the commercialization, and probably incur costs of millions to be able to make it effective for them, there has to be a way for private and public partnerships. And a great example is the Telethon Foundation that was able to bring for Wiskott-Aldrich anemia a new gene therapy and get it not only to be provided for patients on compassionate use, but actually to get it approved commercially in the FDA and hopefully the EMA. So we need innovation beyond just the technology. We need innovation in the way we regulate this newer therapy and in the way we pay for these therapies.

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