Did You Know?

Two new gene therapies, Casgevy and Lyfgenia were recently approved to treat sickle cell disease. For many patients, these therapies can mean living free of painful crises and having hope for a cure.

A new half-matched bone marrow transplant is showing cure rates of 88–95% and costs less than one-quarter of gene therapy. This expands access to treatment for patients who don’t have a perfectly matched donor.

Sanofi is testing an oral drug called Rilzabrutinib that could reduce painful crises by targeting the immune system. It recently received FDA orphan drug status, showing promise for future care.

Scientists in India developed plant-based compounds that may safely reduce iron overload in sickle cell patients—cutting treatment costs by over 50%.