Vertex pharmaceuticals and molecular templates establish collaboration to discover and develop novel targeted conditioning regimens to enhance hematopoietic stem cell transplants

Vertex pharmaceuticals incorporated and molecular templates, inc. announced that the two companies have entered into a strategic research collaboration to discover and develop novel targeted conditioning regimens that may enhance the hematopoietic stem cell transplant process, including transplants conducted as part of treatment with ex vivo crispr/cas9 gene editing therapies such as ctx001.  ctx001 is currently being evaluated in two ongoing phase 1/2 studies in patients with transfusion-dependent beta thalassemia and severe sickle cell disease, where a hematopoietic stem cell transplant is required as part of treatment with ctx001.  the collaboration will seek to discover a new conditioning regimen utilizing mtem’s engineered toxin body (etb) platform, which is designed to specifically target and remove specific cells to enable successful engraftment of new cells.
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