PRO-THYMUS: a groundbreaking solution to restore the immune system in AML patients, laureate of the Eurostars 3 program
Two Small and medium-sized enterprises (SMEs) and two world-leading institutes from Netherlands and France join forces to propose PRO-THYMUS, a novel groundbreaking solution combining cell and gene therapy to swiftly restore the immune system in patients with acute myeloid leukemia (AML) after blood (hematopoietic) stem cell transplantation (HSCT).
Led by Genewity B.V. (NL), reuniting Institut Imagine (Inserm, AP-HP, Université Paris Cité, FR), Smart Immune (FR) and Leiden University Medical Center (LUMC, NL), the PRO-THYMUS project is laureate of the third EUROSTARS program, which aims to support research and development performing SMEs, fostering their international collaboration capacities.
Tackling the unmet medical need for treatments that restores the immune system of AML patients
With a 5-year survival rate at 20%, AML is the most lethal cancer. It is also the most often occurring leukaemia affecting more than 20,000 new patients yearly in Europe. To reach cancer remission, several high-dose chemotherapy treatments are needed, which damage the patients’ thymus, the organ where T cells – a central component of the adaptive immune system – are produced. Around 40% of AML patients undergo a hematopoietic stem cell transplantation, the only potential cure available, but it can take up to two years for the immune system to be restored, and it may never fully recover. Thus, the only existing therapeutic option results in excessive patient suffering, high mortality and is linked with extremely high healthcare costs.
Reducing the burden on patients and healthcare system
PRO-THYMUS leverages groundbreaking cell therapy to restore both the thymus and T-cell population within weeks, instead of years, to greatly improve quality of life and life expectancy of AML patients. PRO-THYMUS is a unique opportunity for AML patients, as by accelerating the recovery of the immune system after transplantation, it will drastically lower the risk of opportunistic infections, occurring due to their weakened immune system, and disease relapse. Moreover, while improving life expectancy and quality of life of patients, PRO-THYMUS is also expected to lower the costs of patient care. The project will benefit from EUROSTARS funding to acquire pre-clinical in vitro and in vivo validation and accelerate the road to clinics and save thousands of lives in the future.
Few words from the coordinator:
At Genewity, it is our mission to save and improve lives with innovative cell- and gene therapies. As a recently founded company – in 2022- it is an absolute privilege to work with such excellent partners in a shared vision to reach impact for AML patients and others worldwide. We are excited to start this project and continue to be a bridge between excellent science and patient care.
Synergy of PRO-THYMUS partners
PRO-THYMUS is an ambitious collaborative project bringing together, for the first time, highly innovate. ve technologies from SMART IMMUNE and GENEWITY, and complementary expertise of its academic partners, Institut Imagine and LUMC.
GENEWITY and LUMC have a ground-breaking approach for regenerating the thymus, the organ essential for T-cell development, while SMART-IMMUNE’S ProTcells are unique T-cell progenitors produced according to a patented culture process, that will complete their differentiation directly in the thymus, leading to a very fast generation of a highly diverse and educated T-cells to fight tumors and infections. Combined in PRO-THYMUS, they will provide a unique opportunity to rapidly tackle both hurdles towards reconstitution of the immune system of AML patients’ after HSCT in a single therapy, greatly accelerating development of functional T-cells.
Dr. Isabelle André (Institut Imagine), is the original inventor of the ProTcell technology and has extensive expertise in T-cell development and immunology. As a co-founder together with Marina Cavazzana and Karine Rossignol, Dr. Isabelle André and the institute have a longstanding relation with SMART-IMMUNE, whose aim is to bring the technology that has been developed and invented at Institut Imagine to the market. Prof. Frank Staal (LUMC) has a long standing research interest in the thymus and has a strong relationship with GENEWITY to develop and commercialize this technology. LUMC provides access to state-of-the-art infrastructure for the in vitro development of the thymus cell and gene therapy and in vivo experiment facilities.
► About Eurostars:
A program developed by EUREKA and the European Commission within the framework of the European Partnership on Innovative SMEs, Eurostars is designed to support, as a priority, innovative SMEs with high growth potential and coordinating a collaborative original project.
► About GENEWITY®
Genewity B.V. is a recently founded biotech company based in Leiden, the Netherlands. Closely collaborating with the LUmc, we build on successes of our scientific and business teams in cell and gene therapies, including the first multi-centric clinical trial for gene therapy in the EU (the RAG1 SCID trial). Genewity has been founded to commercialize another potentially groundbreaking therapy for developing autologous thymus tissue de novo. Genewity is currently in a preclinical development stage and is engaged in raising a Series A to push the technology to the clinic.
► About Institut Imagine
Located on the campus of the Necker-Enfants malades hospital, the Institut Imagine is a world leader in research, care and teaching on genetic diseases. Its unique architecture, designed by Jean Nouvel and Bernard Valéro, brings together 1,000 researchers, physicians, teacher-researchers, engineers and health care personnel in a single location to work with patients, with the ambition of accelerating research and diagnosis and therapeutic innovation to change the lives of families affected by genetic diseases. The Institut Imagine has been certified “Institut hospitalo universitaire” (IHU), in 2011 and 2019 and a “Institut Carnot”, in 2020. It is supported by six founding members, including AP-HP, Inserm and Université Paris Cité, as well as by private partners and patrons. Every day in France, 64 babies are born with a genetic disease. Nearly 8,000 genetic diseases affect more than 3 million people, of which nearly one in two is undiagnosed and more than 8 in 10 have no dedicated treatment. Faced with this public health emergency, the challenge is twofold: to diagnose and to cure.
► About SMART-IMMUNE
Smart Immune is a clinical-stage biotechnology company developing ProTcell, a thymus-empowered T-cell therapy platform to fully and rapidly re-arm the immune system, enabling next-generation allogeneic T-cell therapies for all. The company is a spin-off from the Imagine Institute of Genetic Diseases, and was founded in 2017 by Karine Rossignol, PharmD-HEC, Marina Cavazzana, MD-PhD and Isabelle André, PhD to transform outcomes for patients with life-threatening diseases such as high-risk blood cancers and primary immunodeficiencies.
Smart Immune has clinical partnerships with leading institutions in the US and Europe. The ProTcell platform, already in Phase I/II clinical trials, enables the accelerated recovery of a complete immune repertoire in patients fighting cancer and infection. The ProTcell platform introduces potent, allogeneic T-cell progenitors which are then differentiated by the thymus into fully functional T-cells, to ultimately develop an ‘off the shelf’ T-cell medicine.
The company is headquartered in Paris, France, at Paris Biotech Santé.
► About Leiden University Medical Center
The Leiden University Medical Center (LUMC), housing the medical faculty of Leiden University, is an academic medical center for patient care, research and education, aiming to meet the challenges of tomorrow’s health care. Leiden University has been at the forefront of medical science and education for over 400 years. We believe that good education is a joint responsibility of students and professors and highly value a critical scientific attitude and curiosity.
LUMC is a modern university medical center for research, education and patient care with a high quality profile and a strong scientific orientation. Its unique research practice, ranging from pure fundamental medical research to applied clinical research, places LUMC among the world top. In addition, LUMC possesses state-of-the-art in vivo experiment facilities for translational research and pre-clinical studies in in vivo models. At LUMC, more than 8000 professionals are working towards patient care, novel therapies, diagnostics and performing tasks in research and biomedical education
As an innovator, LUMC stands for improving healthcare and people’s health. That is our mission. We do this from leading research and innovative education. Cooperation within and outside the LUMC is important for successful innovation. The LUMC therefore collaborates on a national and international level. We want to connect LUMC more strongly with our environment and pass it on better to future generations. We want to push boundaries to get better.