Expected Outcome: This topic aims at supporting activities that are enabling or contributing
to one or several expected impacts of destination 5 “Unlocking the full potential of new tools,
technologies and digital solutions for a healthy society”. To that end, proposals under this
topic should aim for delivering results that are directed, tailored and contributing to all of the
following expected outcomes:
Competent authorities, researchers and developers use assays for the valorisation and/or
assessment of efficiency, delivery, safety, potency or mode of action of novel advanced
therapeutic interventions based on either pluripotent stem cells, genome editing or RNA,
that are aligned with regulatory standards.
Clinicians, researchers and developers test several new advanced therapies based on
pluripotent stem cells, gene editing or RNA ready through clinical trials meeting the
Scope: The recent development of advanced therapies has been hampered by the lack of
robust research on certain key parameters e.g. safety, upscaling, immunity, potency assays,
cost-effectiveness, and early on in development. This topic aims to ensure that the next wave
of advanced therapies, based on either pluripotent stem cells, gene editing or RNA, are
established in a timely fashion and in accordance with the appropriate regulatory standards for
further clinical testing. It will support preclinical research platforms for disorders with high
prevalence and burden 162 that tackle the bottlenecks currently encountered in the field,
ensuring that promising advanced therapies can reach the market within the next decade.
Applicants should justify the disease or disorder to be targeted with its prevalence level, the
related burden and unmet needs. Applicants should propose activities in one or several of the
following areas, taking into consideration the Oviedo Convention, eligible actions and ethical
principles as defined by the regulation:
Method development for the production and differentiation of pluripotent stem cells
(defined as cells that can give rise to cells from all three embryonic germ layers), to
include defining appropriate potency assays. Complementary activities to assess mode of
action, safety, in vivo validation or upscaling procedures could be considered.
Development and validation of biological assays and methods that can demonstrate
efficacy, delivery, specificity, and safety (including off-target effects) of genome editing
products in targeted cells and tissues (e.g. base editing, prime editing, transcription
activator-like effector nucleases, zinc-finger nucleases, clustered regularly interspaced
short palindromic repeats). Complementary activities to assess in vivo validation or
upscaling procedures could be considered.
Development and validation of novel RNA-based therapeutics targeting noncommunicable
diseases. Complementary activities to assess mode of action, delivery,
safety, in vivo validation and/or upscaling procedures could be considered.
Study, analysis and tackling of different immune responses, taking into account factors
like sex and age, generated by any of the above-mentioned advanced therapies in vivo,
facilitating regulatory approval for next phase of research and development.