Crispr Therapeutics AG (CRSP)

[jondoeuk]

Slightly off-topic, but VRTX just published this (on an iPSC-derived islet-cell therapy) https://www.nejm.org/doi/full/10.1056/NEJMoa2506549

After twelve months, out of the twelve, ten no longer needed to inject insulin.

VRTX paid $100 million upfront for non-exclusive rights to CRSP's tech https://www.fiercebiotech.com/biotech/vertex-goes-back-crispr-well-paying-100m-upfront-exa-cel-partner-help-diabetes-work

CRSP is also working on its own iPSC-derived islet-cell therapy. Along with the core edits for immune evasion (MHC-I KO, PD-L1 KI and HLA-E KI) and cell fitness (TXNIP KO, TNFAIP3 KI and MANF KI) there will be additional ones (undisclosed).

TXNIP KO protects from oxidative and ER stress https://faseb.onlinelibrary.wiley.com/doi/10.1096/fj.08-111690

TNFAIP3 KI induces graft acceptance and protection from cytokine induced apoptosis (programmed cell death) https://insight.jci.org/articles/view/131028

MANF KI enhances proliferation and protection against inflammatory stress https://www.cell.com/cell-reports/fulltext/S2211-1247(14)00201-0