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2019 CRACK IT challenges: CleanCut

Funding details
Registration opens
12 Sep 2019, 00:00
Registration closes
06 Nov 2019, 00:00
Closes
06 Nov 2019, 00:00
Award
Up to £100k in Phase 1
Organisation
NC3Rs
Summary

Overall aim

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).

 

Duration

Phase 1: six months, Phase 2: Up to three years

 

Budget

Phase 1: Up to £100k, Phase 2: Up to £1 million

 

Sponsor(s)

Novartis, Bayer and Takeda

 

Background

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.

 

For more information, click here.

 

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