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The use of gene therapy as an innovative approach to the treatment of rare kidney diseases will be the subject of the next scientific seminar at Biogem on Tuesday, March 24, at 12:00 p.m. in the "Emmanuele F.M. Emanuele" lecture hall. Dr. Antonio Miele will present the latest results of his research conducted in the Translational Nephrology laboratory.

Professor Lucia Altucci, Head of Biogem's Medical Epigenetics Laboratory, and Professor Vincenzo Carafa, co-head of the same laboratory, are among the leaders of a major breakthrough in the fight against Chronic Lymphocytic Leukemia: an international study coordinated by Professor Altucci has identified UVI5008, an innovative molecule capable of overcoming one of the main challenges to current treatments: drug resistance.

The research results were published in the Journal of Hematology & Oncology, one of the most important journals in the field of hematology-oncology.

How the New Molecule Works

Chronic Lymphocytic Leukemia is a form of blood cancer that affects B lymphocytes, key cells of the immune system. In recent years, therapies targeting a protein called BTK (Bruton's tyrosine kinase) have significantly improved patients outcomes. However, in many cases, the disease develops resistance mechanisms over time, making treatments less effective.

UVI5008 is the first compound in its class to act with a dual and innovative mechanism:

• It blocks the activity of the BTK protein, even when it has a specific mutation (C481S) responsible for resistance to currently used drugs.

• It reduces the production of the same protein at the genetic level, acting on the mechanisms that regulate gene expression in tumor cells.

This combined approach allows for a more comprehensive treatment of the disease than traditional inhibitors.

Promising Results

The study, conducted using integrated methodologies—from computers to cell models to tests on patients samples—demonstrated that UVI5008 is capable of selectively eliminating leukemia cells, evidencing superior efficacy compared to drugs already used in clinical practice, such as Ibrutinib, particularly in the most complex and resistant cases.

An international collaboration

The research is the result of a collaboration between several Italian and international academic and clinical institutions, including Biogem, the Luigi Vanvitelli University of Campania, the LUM University, the Ri.MED Foundation, the University Medical Center Groningen, the IRCCS San Raffaele Hospital, the University of Naples Federico II, the Santobono Pausilipon Hospital, the University of Vigo, and the University of Texas MD Anderson Cancer Center.

According to the researchers, UVI5008 opens the way to new therapeutic prospects for patients with chronic lymphocytic leukemia who no longer respond to available therapies. Further clinical studies will be necessary to confirm safety and efficacy in humans, but the results obtained so far indicate a potential breakthrough in the treatment of this disease.

For more information: https://link.springer.com/article/10.1186/s13045-026-01784-9

Biogem organizes a new event as part of a series of scientific seminars open to the public, both in person and online. On Monday, March 2nd at 12:00 p.m. in Biogem's “Emmanuele Emanuele” Aula Magna, there will be the seminar "Artificial Intelligence for Diagnostic Support in Inherited Kidney Diseases: Challenges and Opportunities." The special guest will be Professor Roser Torra, Head of Clinical Nephrology and Director of the Hereditary Kidney Diseases Clinic in the Nephrology Department at the Puigvert Foundation, Barcelona.

Professor Torra is a global authority in the field of nephrology: active in research for over 30 years, she combines her research with a strong clinical commitment. Her scientific authority is demonstrated by her multiple institutional roles, including the presidency of the European Renal Association (ERA).

Diabetes is a widespread disease with serious complications affecting multiple organs. Renal complications are among the most significant ones. In fact, about 40% of patients requiring dialysis are diabetic. Until a few years ago, there were no drugs capable of treating the renal complications of diabetes, so dialysis was the norm. Yet today there are a new series of therapies that can delay and, in some cases, eliminate the need for dialysis. This and much more will be discussed at the International Diabetology Conference scheduled in Naples from February 25 to 28, 2026. The event is organized by the Biogem Scientific Director, Professor Giovambattista Capasso, together with Dr. Silvio Settembrini.

In particular, during the conference, Professor Capasso has organized the second international conference on diabetic kidney disease, entitled "New Frontiers in Diabetic Kidney Disease: From Mechanisms to Patient Care." On Friday, February 27, from 9:00 a.m. to 6:15 p.m., some of the world's leading experts in kidney disease, diabetes, and their correlations will deliver their talks. The conference is being held under the patronage of Biogem and the ERA (European Renal Association), which will be represented by its current president, Professor Roser Torra, who will deliver the closing keynote address.

In addition to Professor Capasso, many members of the Nephrology and Dialysis Unit at the University of Naples L. Vanvitelli, which has enjoyed a strong clinical and scientific collaboration with Biogem for several years, will participate at the conference. Among others, Professor Francesco Trepiccione, head of Biogem's Nephrology Section, and Professors Alessandra Perna, Miriam Zacchia, Mariadelina Simeoni, and Giovanna Capolongo. The presence of Professor Vincenzo Carafa, co-head of Biogem's epigenetics laboratory, confirms that Biogem is at the forefront of international research about the effects of diabetes on kidney function.

For more information: https://www.napolidiabetologia.it/