Infrastructure initiatives Call 2021-2

GMP process and product development of cell and gene therapies

The call Infrastructure initiatives 2021-2 focuses on the development of infrastructure for GMP process and product development of cell and gene therapies.  

Cell and gene therapies are a promising new group of medicinal products for the treatment of cancer. To translate developments from the laboratory to the clinic (translational research), new innovations from the laboratory setting have to be developed into products that are manufactured according to Good Manufacturing Practice (GMP). This step requires to design, optimize, and qualify a manufacturing process and GMP-grade end product. The technical development of manufacturing processes and GMP-grade end products, next to scientific evidence of preclinical safety and efficacy, is an essential step to start clinical trials with cell and gene therapies.

Problem statement

Yet, GMP process and product development is one of the largest bottlenecks for translational research. The complex product characteristics versus regulatory requirements, and the high manufacturing costs versus the limited resources of medical centers for GMP process and product development create a bottleneck for translational research and further development towards clinical practice. Academic developers of these new medicines are often unaware of the challenges and have limited means to find colleagues with the relevant expertise. Furthermore, a step-by-step approach, instead of an approach that is working towards an end product that is suited for implementation in clinical practice, can lead to upscaling issues when explorative clinical outcomes are promising. Altogether, these limitations and issues lead to delays or failures to develop novel cell and gene therapies with potentially high benefits for patients.  

Scope

The call Infrastructure initiatives 2021-2 focuses on the development of infrastructure that supports and facilitates GMP process and product development of cell and gene therapies and/or new relevant technologies (e.g. gene editing). Intended initiatives and platforms can consist of different resources, such as facilities, equipment and other process technology, supply chains, IT systems and staff.  

Below we provide a few intended initiatives as an example:

• Central platform (hub) for GMP process and product development for one or multiple subtypes of cell and gene therapies (e.g. Tumor Infiltrating Lymphocytes, CAR-T cells or Natural Killer cells), for different indications. This platform could consist of a facility that runs according to GMP, but operates separate from GMP cleanrooms, and/or consist of a facility with closed systems for manufacturing that can be used for process and product development of one or multiple cell and gene therapies for different indications.
• The development of process technologies that facilitate manufacturing of larger batches or that enable more automated manufacturing (e.g. closed manufacturing systems) for one or multiple subtypes of cell and gene therapies.
• Central technology hub for manufacturing of GMP-grade materials (e.g. viral vectors or other materials for genetic modification) for a non-profit price, and/or process development for new technologies for genetic modification (e.g. gene editing).
• Partnerships with international technology hubs and/or qualified vendors that are supported with a common supply chain, in order to create access to GMP-grade materials and/or products for a non-profit price across the Netherlands.  

Criteria

• Initiatives aim to create tangible and/or virtual infrastructure that accelerates and optimizes GMP process and product development of promising cell and gene therapies for the treatment of cancer.
• Initiatives that are aimed at connecting medical centers or production facilities, without the creation of tangible and/or virtual infrastructure, are out of scope for this call.
• Initiatives facilitate further development of cell and gene therapies by realizing sustainable infrastructure that mitigates the risks for individual product development trajectories.
• The infrastructure supports researchers nation-wide. Infrastructure that is confined to one medical center or production site is accessible to researchers from other institutes. Consortia and other partnerships are encouraged. International collaboration is possible.
• KWF expects that when funding has ended, the initiative is self-sustainable in order to continue and to be implemented further. Proposals include a strategy for sustainable financing of the initiative.

Criteria for public-private partnerships

If proposals consist of partnerships with private partners, these proposals need to adhere to the guidelines of Health~Holland. Public-private partnerships (PPS) are subsidized with PPS allowance by the Dutch government to stimulate basic research and entrepreneurship. KWF has access to this PPS allowance. For the pre-proposals the following guidelines are of importance:

• The collaboration consists of at least one academic center and one private entity.
• IP rights of the academic institution, company or joint IP are documented in a consortium agreement (upon funding decision).
• Initiatives consist of basic-, industrial-, or combination research, with a maximum PPS allowance of 75%, 50% and 25%, respectively.
• The contribution of the research organization is at least 10% of the total budget.
• The contribution of the private entity is at least 15% of the total budget.

A select number of project leaders are invited to submit a full proposal based on the anticipated impact of the pre-proposed initiative. KWF may approach project leaders to advise on eligibility for the PPS allowance and/or to suggest to merge multiple pre-proposals to one full proposal.

Indicative Budget

The indicative budget is 5 million euro. The budget may be higher due to additional PPS allowance if applicable.

Timeline 

Further Information

Please consult the KWF Guidelines 2021 for further information on the process, characteristics and eligibility terms for the Infrastructure Initiatives funding type.