Immigrants who have lived abroad or grown up with stories of political chaos know that the most violent days always start out in an eerie quiet, as January 6, 2021, did in Washington, DC. By 1:10 pm that day, after then-President Donald Trump issued his call for thousands of supporters to march on the Capitol, anyone who was paying attention knew that something dangerous was about to take place.
Within a matter of minutes, at the other end of the National Mall, a Capitol police officer banged on the door of Rep. Ted Lieu (D-CA). “You need to evacuate immediately,” Lieu recalls the officer saying before urging him to remove the lapel pin that identified him as a member of Congress and directing him down five flights of stairs to a secure location.
CRISPR gene therapy for sickle cell disease approved by the FDA
Appeared in BioNews 1090
A clinical trial for a new gene therapy approach to treat sickle cell disease has been approved to proceed by the US Food and Drug Administration.
Patients with sickle cell disease have a mutation in the beta-haemoglobin gene, causing them to produce misshapen red blood cells that can block blood vessels leading to severe pain, anaemia and potentially life-threatening complications, such as organ damage and strokes. Currently, the only cure is a stem cell transplant from a healthy donor, but in the newly-approved trial, scientists from the University of California will use CRISPR/Cas9 genome editing to replace the faulty gene with a functional version.
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A novel bispecific protein significantly improved overall survival (OS) in metastatic uveal melanoma as compared with investigator s choice of treatment, including checkpoint inhibitors, a randomized trial showed.
Patients treated with tebentafusp had a median OS of 21.7 months versus 16.0 months for the control group. The benefit was consistent across all prespecified subgroups.
Grade 3/4 treatment-related adverse events (TRAEs) occurred infrequently, including a 1% incidence of severe cytokine release syndrome (CRS), reported Jessica C. Hassel, MD, of University Hospital Heidelberg in Germany, at the American Association for Cancer Research (AACR) virtual meeting. Tebentafusp showed a highly significant and clinically meaningful improvement in overall survival as first-line treatment of metastatic uveal melanoma, she said. It is the first investigational therapy in a phase III trial to improve overall survival in metastatic uveal melanoma, and this overal
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