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CRACK-IT

Crack-it is a challenge-led competition that funds collaborations between industry, academics and SMEs to solve business and scientific Challenges which will deliver 3Rs benefits, either by improving business processes or developing a commercial product

 

Manuela Raimondi of Politecnico di Milano leads one of the teams awarded with funding to carry out Phase 1 (proof-of-concept studies) of the CleanCut challenge: ‘Establishing an in vitro model to assess the safety of genome edited human haematopoietic stem cells’

 

Sponsored by Bayer, Novartis and Takeda, this Two Phase Challenge aims to develop an in vitro model to replace in vivo tumourigenicity studies for the safety assessment of genome edited human haematopoietic stem cells (hHSCs).

A number of blood-related monogenic diseases, such as sickle cell anaemia and haemophilia, have the potential to be cured with genome edited hHSCs. Gene editing of hHSCs using designer nucleases (e.g. CRISPR) offers a high level of precision but can cleave undesired genomic regions causing off-target medications, which could generate cells with oncogenic potential. Currently, in vivo studies to assess the tumourigenicity of genome edited products are a regulatory requirement. These studies are lengthy, costly, require the use of large numbers of animals (e.g. mice) and are not always predictive of the risk to human safety.

Creation of an in vitro assay through this Challenge could replace these in vivo tumourigenicity studies.