WP 1

Establishment of robust protocols for correction of the IL2RG gene in SCID-X1.
The objectives are:

  1. Selection of the best performing nucleases for functional correction of the IL2RG
  2. Develop a robust and scalable protocol for targeted integration in NSG-mouse long-term repopulating human HSC
  3. Proof-of-efficacy of disease correction in SCID-X1 HSPCs
WP 2

Establishment of robust protocols for TCR gene editing in T lymphocytes for the treatment of leukemias.
The objectives are:

  1. Creation of a TCR addition vector library to introduce reactivity against a large number of tumor antigens
  2. Characterization of the optimal T-cell subset to be utilized in TCR editing
  3. Clinical development of TCR editing protocol for subsequent evaluation in clinical trials
  4. Determine efficacy of TCR editing in gene modified cells
WP 3

Development and scale-up of manufacturing of HSPC and T-cell products.
The objectives are:

  1. Develop processes for production of suitable quality mRNA and LV to support pre-clinical studies
  2. Scale-up and optimization of gene targeting protocols in HSPC and T-cells for the two proposed gene therapy applications
  3. Preparation of one full-scale lot of modified cells for each proposed gene therapy application
  4. Perform preclinical GLP studies of toxicity and biodistribution of both types of cell product in NSG mice
WP 4

Innovation, Exploitation, Dissemination.
The objectives are:

  1. Perform all required actions for IP Management and protection of innovations in SUPERSIST
  2. Establish strategic roadmaps to ensure the seamless translation of project outcomes toward products
  3. Prepare the exploitation of project results via establishing a short business plan with the necessary steps to reach the market
  4. Dissemination and promotion of the results
WP 5

Coordination, Management, Ethics.
The objectives are:

  1. Coordinate all actions for the achievement of SUPERSIST objectives on time and on budget
  2. Ensure that all budgetary actions are performed effectively and according to the rules and regulations established by the EC and the consortium agreement ensuring that the received funds are correctly distributed and accounted for, ensure that reporting is performed on a regular basis, according to the EC guidelines
  3. Monitor project progress and provide with all relevant information for the project