The newest publicly traded genome editing company, Beam Therapeutics, has gotten one of its experimental sickle cell therapies to produce high levels of healthy hemoglobin in lab mice, it plans to announce at the (virtual) annual meeting of the American Society of Gene and Cell Therapy on Wednesday, paving the way for human trials.
Beam’s is one of more than two dozen sickle cell studies being presented at ASGCT. The intense activity is a dramatic turnaround: Although sickle cell disease is the most common inherited blood disorder, with an estimated 100,000 patients (mostly of African descent) in the U.S., for decades it received less scientific attention and funding support than other genetic diseases.
As scientists now evaluate multiple genetic strategies, “it’s a great problem to have, especially compared to the [R&D] desert we had,” said sickle cell expert Alexis Thompson of Children’s Hospital of Chicago.
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