Gene therapy goes beyond rare diseases: an interview with Alessandra Biffi

From an accidental start to world-class science

Alessandra Biffi came to gene therapy by chance: while she was training to become a doctor, a professor of haematology noticed her and invited her to write her thesis in his laboratory. Today she is one of the world's leading experts in gene therapy — an approach in which a person's DNA is altered to treat or prevent certain diseases. She previously led the gene therapy programme at the Dana-Farber Cancer Institute in Boston, and now heads the division of paediatric haematology, oncology and stem cell transplantation at the university hospital in Padua, Italy. 'I have an enormous passion for what I do, and it is renewed every day — that is the real driving force,' she said in an interview with Euronews Next as part of the podcast Euronews Tech Talks.

Therapy not only for rare diseases

Although gene therapy sounds like science fiction, it is an actively studied technique used to treat a wide range of diseases. It was originally used against monogenic disorders — conditions caused by a mutation in a single gene — but it is now also applied to some forms of cancer. 'The spectrum of conditions that can be treated with this approach is expanding to include non-inherited diseases,' Biffi noted. She is currently working on the Immunostem clinical trial, which is studying how gene therapy could prevent the full onset and progression of type 1 diabetes. The first phase of the clinical trial began a few months ago, and Biffi is hopeful about the results: in her words, it could provide further proof that gene therapy is applicable not only to ultra-rare but also to widespread diseases — exactly the kind of opportunity the whole field is now looking for.

The first patient

Years of working with severe conditions left Biffi with one patient she cannot forget. He was the first person she ever treated: the fourth child in a family whose previous children had suffered from the same genetic disease and died from it. The patient was diagnosed with metachromatic leukodystrophy — a rare genetic disorder that gradually robs a person of the ability to walk, speak and see. He was saved by a drug developed by Biffi and her colleagues at the San Raffaele Telethon Institute for Gene Therapy in Milan, where she worked earlier in her career. The drug, called Libmeldy, is now available on the market. 'He now lives in Italy and leads a very healthy and successful life. He goes to school and is growing up. And of course I am very, very emotionally attached to him,' Biffi said.

AI in the development of new drugs

Gene therapy is developing rapidly, and — like other areas of science — it is being influenced by artificial intelligence. At the end of September, the company Anthropic announced that its Claude model had helped discover a new enzyme system, which CEO Dario Amodei described on X as a possible 'new mechanism for gene editing'; experts, however, are sceptical about the real significance of this discovery. Biffi takes a broadly positive view of the use of AI in gene therapy: 'It needs to be properly regulated and governed, but I genuinely believe that in the area of discoveries needed to develop new drugs, including gene therapy, AI can make a big difference.' In her view, AI can help scientists design new molecules needed for editing DNA. 'In any situation, AI can be a great aid to progress,' she added.