Where research meets care: inside SR-Tiget’s ecosystem 

Every gene therapy involves two parallel journeys. One belongs to the patient and their family, who must reach the clinical centre and navigate a lengthy, complex treatment process, often far from home. The other follows the biological samples analysed before and after treatment to assess its safety and long-term effects. 

At the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), these journeys bring together people with very different professional backgrounds. “Just Like Home” supports patients and families during their stay in Milan, while the SR-Tiget Clinical Laboratory (TCL) carries out the specialist analyses needed for clinical trials and therapy development. 

Just like Home’s tenth anniversary offers an opportunity to look more closely at this ecosystem, where research, clinical care and family support work together to turn scientific advances into treatments that patients can actually access. 

Just like Home was established in late 2016, following European Medicines Agency (EMA) approval and the introduction of a gene therapy for ADA-SCID to the European market, based on research carried out at SR-Tiget. Earlier gene therapy trials had involved relatively few patients, allowing clinical staff to manage the logistics themselves. As patient numbers grew, with many families travelling from abroad, a dedicated programme became essential. 

A treatment may be available, yet remain out of reach: obtaining a medical visa, paying for travel, finding accommodation or overcoming a language barrier can present formidable challenges for a family already coping with a rare disease diagnosis. 

Support begins before families arrive. Levi and the Just Like Home team join the initial meetings with clinicians, the doctor in the family’s home country and the parents to understand their circumstances and prepare for their stay. Assistance may cover visas, travel arrangements, transfers, accommodation, language and cultural mediation, practical help, and emotional and psychological support. Financial assistance with travel and living expenses is also available when needed. The programme does not cover clinical care or treatment costs. 

Most patients are children, and families spend an average of six months in Milan. Support therefore extends to the whole family. Care coordinators, administrative staff, research nurses, psychologists, mediators, professional carers, music therapists and other specialists work together to tailor the arrangements to each family’s needs: each family becomes “a small project within the wider programme”. 

In its first ten years, Just Like Home has welcomed and supported 227 patients from around 60 countries. In 2025 alone, 17 treatments were carried out and 133 follow-up visits coordinated. Families spent a combined 3,114 days in the accommodation provided, with 14 families staying for more than 100 consecutive days. These figures show how access to treatment also depends on making everyday life manageable during an extended stay away from home. 

As patients move through treatment, their samples follow a different route. They arrive at the SR-Tiget Clinical Laboratory (TCL) for highly specialised analyses that are generally unavailable in routine clinical laboratories. "Our laboratory is involved in clinical research, but it is not a research laboratory", explains its coordinator, Matias Soncini. The distinction matters. The TCL does not conduct independent research, and its staff do not choose which experiments to perform. Instead, they carry out the analyses specified in clinical protocols, following strictly defined procedures, schedules and quality requirements. Yet many tests involve little automation and demand the hands-on skills and technical expertise of an experienced wet-lab researcher. 

The TCL was established to bring methods developed by the research groups behind the therapies into a regulated setting. Before these methods can be used in clinical trials, they must be validated, standardised and documented to deliver reliable, reproducible results that can also support regulatory submissions. 

The analyses serve several purposes. Some assess safety, monitoring whether treatment is causing adverse effects. Others measure specific clinical endpoints or help deepen understanding of the therapy and the disease. One key test measures vector copy number: how many copies of the therapeutic gene are present in the patient’s cells and how this changes over time. Other analyses establish, for example, whether the product of the introduced gene is present in the expected cells and functioning as intended. 

Monitoring can continue for many years. Some clinical trials last a decade, and patients may be followed for as long as twenty years, either within or outside a trial. The TCL’s role therefore extends well beyond the immediate aftermath of treatment to assessing its long-term effects. 

Created by bringing together activities previously carried out by separate groups, the laboratory now has almost twenty staff. Its growth has prompted a new organisational structure, moving beyond teams dedicated solely to individual diseases towards areas of expertise such as molecular biology, cell biology and genetics. 

Just Like Home and the TCL do not work closely together on a daily basis, but both contribute to the same patient journey. Research nurses provide the main operational link, coordinating patients’ clinical schedules and informing the laboratory when samples will be collected and are expected to arrive. 

Plans must adapt to clinical realities. Many patients are babies or young children, and a blood sample may need to be postponed because of a fever, an infection or another unexpected issue. A sample expected in the morning may not arrive until the afternoon, requiring the laboratory to adjust its work. Testing schedules are therefore shaped not just by laboratory planning, but by patients’ actual needs. 

That connection can be harder to see for those spending their days in the laboratory. Technicians receive coded sample tubes and produce numerical results without necessarily meeting the people behind them. Soncini believes it is essential to remind staff why their work matters:

Institute-wide meetings, personal accounts and images of patients help make that connection visible again. Seeing a child running who would otherwise have progressively lost their abilities brings home the contribution of work that can sometimes feel repetitive and removed from clinical care.

Levi’s and Soncini’s careers illustrate the range of professional skills needed to make gene therapy possible. Levi studied international relations with the aim of working in the non-profit sector. After roles in communications, including work with pharmaceutical companies and patient organisations, and in child protection, she joined Fondazione Telethon in 2018. 

Her role calls for organisational and interpersonal skills, an understanding of health and social care networks, and careful attention to professional boundaries. Working closely with families does not remove the need for a clearly defined professional relationship. At the same time, supporting a child for months and seeing them return over the years inevitably builds a meaningful bond. “We first met some children when they were eight months old, and we continue to see them as they start school. Some have now finished school, passed their driving test or gone to university,” says Levi. 

Soncini’s background is in biotechnology research. Following a long academic career, including a project leader role, he moved into pharmaceutical quality control, gaining experience in managing regulated laboratories before joining SR-Tiget. The TCL looks for people with a researcher’s technical expertise who want a career outside academic research: one built around validated procedures, operational responsibilities and schedules driven by clinical needs. 

The work is demanding and often takes place behind the scenes, but it offers greater scope for stable employment and, above all, a direct contribution to patients’ treatment. The same combination of specialist expertise and a close connection to patients underpins Just Like Home. Within SR-Tiget’s ecosystem, turning research into care depends not only on scientists and doctors, but also on the professionals who make every step between the laboratory and patients’ lives work in practice.