50 Million euro investment for LUMC spin-off RougeTx advances therapies for inherited bleeding disorders.
reNEW Principal Investigator Associate Professor Ana Hidalgo Simon published three papers on successful translation and clinical application of advanced therapies. The publications collect current best practices, discuss the alternative routes to patients of orphan gene therapies and identify core competencies necessary for translation of Advanced Therapy Medicinal Products (ATMPs).
Comprehensive resource for translation
The first paper, published in Stem Cell Reports, gathered current best practices for translation of stem cell-derived products from the laboratory to the clinic. A challenging task, since such translation is a multidisciplinary effort for which required skills and knowledge are often highly fragmented.
The International Society for Stem Cell Research (ISSCR) therefore brought together experts in a taskforce to fulfill this task. Hidalgo Simon, one of these experts, explains: “Together, we developed a comprehensive and interactive document that guides academic and industrial parties in developing stem cell-derived therapies. The resource comprises information not only on scientific and technical aspects but also provides a global perspective on the regulatory landscape. Our hope is that the resource will contribute to more stem cell-derived therapies, such as those developed within reNEW, reaching the clinic.”
ATMPs for orphan diseases
ATMPs have revolutionized the treatment landscape for rare diseases. However, despite often life-changing, these medicines face unique challenges after market approval, including commercial withdrawal and limited patient access.
“Many ATMPs for orphan diseases originate in an academic setting, where resources and knowledge that ensure continuous access for patients through commercialization are often scarce. As a result, many of these therapies currently struggle despite regulatory approval,” said Hidalgo Simon.
In a perspective, published in Blood ICT, Hidalgo-Simon and Claire Booth (University College London, UK) discuss the future of ATMPs for orphan diseases and explore novel models that ensure these therapies continue reaching patients in need. Among other things, the researchers discuss new models for commercialization, such as those that include charitable foundations. They also touch upon the various regulatory initiatives in the European Union that support this special class of ATMPs.
“Our paper highlights the need for coordinated multistakeholder action. The lessons learned over the last years may very well chart new pathways to ensure that products stay in the market to reach the patients who need them,” concludes Hidalgo Simon.
What does it take?
At the basis of success for these best practices and models for translation are highly skilled professionals that possess the right set of competencies. In the final manuscript, published in Frontiers in Digital Health, mapped these competencies for the first time. To do so, they combined a targeted literature review with a structured expert consensus survey.
“ATMPs are relatively new and it has become clear that their translation requires a specific set of competencies,” said Guillen, first author of the paper. “Through our approach, we identified 16 fundamental competencies needed for success. These cover strategy, manufacturing, scalability, quality management, clinical translation and cross-disciplinary collaboration.”
These ATMP-specific competencies can serve as a basis for targeted training programs, that will help to build a new generation of professionals capable of navigating the complex and evolving translational needs for ATMPs.
