Clinical research Unit

Clinical research Unit


Head of Unit

Alessandro Aiuti



The SR‐Tiget Clinical Research Unit (CRU) was established in 1999 to create an infrastructure with dedicated staff and in‐house expertise for conducting advanced therapies clinical trials for genetic diseases according to Good Clinical Practices (GCP) standards. The Unit was originally established through a dedicated grant of Fondazione Telethon to SR‐Tiget and is led by Alessandro Aiuti.

The mission of the CRU is the application of research discoveries and novel gene and cell therapy approaches to the treatment of genetic diseases focusing on primary immunodeficiencies, lysosomal storage disorders and hemoglobinopathies. It provides a suitable environment to translate the results of basic and clinical research into clinical practice and vice versa, while maintaining high quality standards of clinical trials. The CRU has been recognized as a center of excellence for the treatment of patients affected by genetic disorders, coming from various countries.


The Unit is composed by a Clinical Hematology Research Unit and a Pediatric Clinical Research Unit. The activity of the Unit is supported by the SR-Tiget Clinical Trial Office, the SR-Tiget Clinical Lab and the “Just like home” program dedicated to patients and their families. All together the staff of the CRU has the required expertise for carrying out in-house advanced therapies-based clinical trials according to GCP. The CRU acts in strict cooperation with the Pediatric Immuno‐Hematology Unit of Ospedale San Raffaele and the San Raffaele Stem Cell Program:

  • The Pediatric Immuno-Hematology Unit, directed by Alessandro Aiuti, takes care of the diagnosis and treatment of pediatric patients affected by hematological, immunological and metabolic diseases, with a particular focus on genetic disorders;
  • The San Raffaele Stem Cell Program, directed by Fabio Ciceri, is the largest Italian hematopoietic stem cell transplant center. Special areas of interest are stem cell transplantation, cell and gene therapy and high-risk hematological malignancies.

The approach to the patients is holistic, taking care of all the medical, nursing and psychological aspects thanks to a multidisciplinary team.

Research activity

Clinical research activities aim at facilitating clinical translation of basic science and platform innovations deriving from SR-Tiget experimental research programs into novel experimental protocols for the treatment of inherited immunodeficiencies (Adenosine Deaminase-Severe Combined Immunodeficiency and Wiskott-Aldrich Syndrome), hematological diseases (β-thalassemia) and lysosomal storage disorders (Metachromatic Leukodystrophy and Mucopolysaccharidosis Type I Hurler). In addition, the Unit is performing clinical research to improve the knowledge of rare genetic diseases. SR-Tiget has implemented several successful gene therapy clinical trials, which have already treated >120 patients and led to the filing and approval of 2 novel advanced therapy medicines (out of the 11 gene therapy drugs approved in total in the European Union).

For more detailed information about research activity of Clinical research Unit click here.