BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)--EdiGene, Inc., a global biotechnology company focused on translating gene-editing technologies into transformative therapies for patients with serious ...
CorrectSequence Therapeutics' CS-101 Successfully Cures One Patient of Transfusion-Dependent β-Thalassemia Compared with other CRISPR-based β-thalassemia gene editing therapies, CS-101 using tBE ...
― Robust and clinically meaningful absolute mean fetal hemoglobin (HbF) induction of 8.6% from baseline at 12 weeks of treatment; 7 of 16 patients achieved absolute HbF levels greater than 20% ― ― ...
Data demonstrate therapeutically relevant editing levels using a clinically validated strategy, supporting its development as a novel, in vivo approach to treating sickle cell disease and beta ...
The pathology of sickle cell disease is caused by polymerization of the abnormal hemoglobin S upon deoxygenation in the tissues to form fibers in red cells, causing them to deform and occlude the ...