Gene Therapy: Luigi Naldini receives Phacilitate Lifetime Achievement Award 2024

Luigi Naldini, the director of the San Raffaele-Telethon Institute for Gene Therapy, has been honored with the Phacilitate Lifetime Achievement Award 2024 for his research on lentiviral vectors

The winner of the Phacilitate Lifetime Achievement Award 2024 is Luigi Naldini, director of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan. The award was granted in recognition of Naldini’s pioneering role in the field of gene therapy, particularly as the “father” of lentiviral vectors – therapeutic tools derived from the HIV virus that now play crucial roles in clinical applications. The ceremony took place on January 18th in Miami, Florida, as part of the Advanced Therapies Week.

Naldini: “Naldini: "I will continue to seek solutions based on gene and cell therapy"

Phacilitate Advanced Therapies Award recognizes innovations in the field of advanced therapies. The judging process involved a two-phase evaluation by an international panel of experts representing research institutions, universities, and the biotechnology industry. Established in 2023, the award was initially presented to Carl June for his contributions to cellular therapy with CAR-T.

Naldini expressed his gratitude upon receiving the award, stating, “I am grateful to my mentors and fantastic colleagues, as well as excellent students who have accompanied me on this extraordinary journey from the lab bench to the patient’s bedside, spanning nearly three decades, sharing inspiration, challenges, frustrations, and, finally, successes. I will continue to work with renewed energy and enthusiasm in the pursuit of gene and cell therapy solutions for the many patients in need of innovative treatments for diseases once deemed fatal.”

Sven Kili, president of the Phacilitate award jury, remarked, “Professor Naldini perfectly embodies the concept of an ‘innovator.’ His unwavering dedication in developing therapies for the benefit of patients has driven him and his team to not be discouraged by early setbacks but instead to develop better and increasingly safer therapies. Without his dedication and perseverance, gene therapy would not be at the stage it is today, capable of changing the lives of patients with approved drugs for genetic diseases such as ADA-SCID and metachromatic leukodystrophy.”

Lentiviral vectors, a treatment for severe genetic diseases

Naldini’s research on lentiviral vectors has significantly contributed to the treatment of severe genetic diseases. At San Raffaele Hospital, almost a hundred patients with conditions such as metachromatic leukodystrophy, Wiskott-Aldrich syndrome, beta-thalassemia, and mucopolysaccharidosis type 1 have benefited from this innovative therapy, involving stem cells corrected through lentiviral vectors.

Over 400 individuals worldwide have received treatments based on engineered blood stem cells using lentiviral vectors, addressing 19 different pathologies. Approximately 3,500 people have benefited from such therapies, and four gene therapies have been authorized for market release.

From blood stem cells to genetic editing

Naldini’s ongoing experimental research aims to introduce innovative solutions to expand the applications of gene therapy and enhance its effectiveness and safety. Recent efforts include a gene therapy strategy with anti-tumor action based on engineering blood stem cells. This approach, currently in clinical trials for treating glioblastoma at San Raffaele and Besta Hospitals in Milan, activates targeted immune responses within tumors, sparing the rest of the body from side effects.

The study is sponsored by Genenta Science, an innovative startup founded by Naldini in collaboration with San Raffaele and is currently listed on the Nasdaq.

Naldini’s laboratory has also been instrumental in the early development of targeted gene editing using CRISPR technology, paving the way for new and revolutionary applications in the field.


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