Date: 2017-03-10
Type of information: Research agreement
Compound: novel T cell therapies
Company: Crispr Therapeutics (Switzerland - UK) Neon Therapeutics (USA - MA)
Therapeutic area: Cancer - Oncology
Type agreement: R&D - research
Action mechanism: gene editing technology/cell therapy. “CRISPR” refers to Clustered Regularly Interspaced Short Palindromic Repeats that occur in the genome of certain bacteria, from which the system was discovered. Cas9 is a CRISPR-associated endonuclease known to act as the “molecular scissors” that cut and edit, or correct, disease-associated DNA in a cell. A guide RNA directs the Cas9 molecular scissors to the exact site of the disease-associated mutation. Once the molecular scissors make a cut in the DNA, additional cellular mechanisms and exogenously added DNA will use the cell’s own machinery and other elements to specifically ‘repair’ the DNA. This technology may offer the ability to directly modify or correct the underlying disease-associated changes in the human genome for the potential treatment of a large number of both rare and common diseases.
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