ATMP’s from idea to patient care: it’s all happening in Leiden!

| CIV Bio Sciences

Did you know that fifty years ago the first bone marrow transplant in Europe was performed at the Leiden University Medical Center (LUMC)? And did you know that the LUMC now has more than twenty years of experience in developing innovative cell and gene therapy applications?

Advanced Therapy Medicinal Products or ATMPs are advanced therapeutic drugs for cell and gene therapy. As the field of ATMP’s is rapidly developing worldwide, specifically in our Leiden Bio Science Park (LBSP), we simply had to dedicate a Life Long Learning session to this topic.

On February 29, CIV Bio Sciences partner LUMC organised a most educative programme consisting of talks and tours. The session was well-attended by a mix of people from the ATMP industry, service providers, and education. After the kick off by Sandra Migchielsen, programme manager CIV Bio Sciences  – explaining the why of the gathering (see below) – two presentations were held: on ATMP product development and on ATMP manufacturing.

Product development for the failing immune system

First Frank Staal, Professor of Molecular Stem Cell biology at the Department of Immunology, LUMC, illustrated his group’s ongoing work by discussing the preclinical and clinical development of a gene therapy treatment for babies with a rare inherited mutation in the RAG1 gene. These so called RAG1-SCID patients suffer from severe infections due to a failing immune system. In this therapy, the patient’s own blood stem cells are “repaired” in the lab using lentiviral vectors and transplanted back into the patient. The therapy has now reached a phase I/II clinical trial. The current two patients enrolled in the study are doing well.

[Later that afternoon Professor Frank Staal would receive the prestigious Rare Angel Award for his ongoing research on stem cell based gene therapies for hereditary immune deficiencies. He received the award on Rare Disease Day, February 29th, from the Dutch patient advocacy organization (VSOP).]

Center for Cell and Gene therapy manufacturing expertise

Next up was Pauline Meij, Head of the Center for Cell and Gene therapy (CCG). Pauline presented how over the years many ideas and results from LUMC’s research labs and clinical departments were translated into ATMP products, were produced in LUMC’s in-house Good Manufacturing Practice (GMP) facility and tested in a multitude of LUMC, national and international clinical trials. The CCG focusses on early product development and collaborates with many academic, not-for-profit as well as commercial partners. Pauline gave plenty of examples of medicinal products developed, covering disease areas such as immune disorders, cancer and diabetes. Along the way, many hurdles have been (and continue to be) overcome and many lessons learned in areas related to product development, manufacturing, patient access, market access, legislation and regulations. To this end, collaboration with industry and regulatory experts is crucial.