Flagship Pioneering-founded genome engineering biotech Tessera Therapeutics today announced an investment of up to $50 million from the Bill & Melinda Gates Foundation to develop curative, globally accessible in vivo genetic therapies for sickle cell disease.
Tessera is developing Gene Writers for sickle cell disease designed to enable a true correction of the sickle mutation via one-time intravenous administration of the therapy.
This in vivo approach is unlike recently approved genetic therapies for sickle cell disease that require complex stem cell mobilization, ex vivo cell manipulation, or toxic chemotherapy conditioning prior to infusion – necessary steps that carry additional risks to patients, can require lengthy hospitalization and represent a significant burden for patients and their families.
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