2019 CRACK IT challenges: CleanCut
This Challenge aims to develop an in vitro model to replace in vivo tumourigenicity studies for safety assessment of genome edited human haematopoietic stem cells (hHSCs).
Phase 1: six months, Phase 2: Up to three years
Phase 1: Up to £100k, Phase 2: Up to £1 million
Novartis, Bayer and Takeda
Diseases such as sickle cell anaemia, haemophilia, thalassemia and severe combined immunodeficiency result from modifications in a single gene occurring throughout the cells of the body. In the European and East Mediterranean area alone, at least 2,000 births a year are affected 1,2,3. The only curative treatment is an allogenic haematopoietic stem cell (HSC) transplant, which involves transferring HSCs from a healthy donor to the patient’s body after high-intensity chemotherapy or radiation. These treatments are expensive, (the cost of the transplant is ~ $275,000) 4, require patients to undergo lifelong pharmacological immunosuppression (costs ~ $12,000/year/patient 5, and appropriate donors need to be identified to prevent adverse immune responses.
A number of blood-related monogenic diseases have the potential to be cured with genome edited human HSCs (GE-hHSCs) that have been modified by inserting, deleting, modifying or replacing genetic material. This has created a significant amount of research and development to penetrate a market expected to reach $8.1 billion by 2025 6. Eight clinical trials based on the use of GE-hHSCs are ongoing (https://clinicaltrials.gov/), and the number is expected to increase over the next decade.
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