Both the U.K. MHRA and the U.S. FDA approved their first CRISPR-based gene therapy in 2023. Crispr Therapeutics AG and partner Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) was approved by the MHRA in November and the FDA on Dec. 8. The U.K. approval is for both severe sickle cell disease (SCD) and transfusion-dependent thalassemia (TDT). In the U.S., the approval is for severe SCD, with a PDUFA date for TDT coming up in spring 2024.
In their year-end list of top scientific achievements and the people who made them, both Science and Nature have included the fight against “the obesity epidemic.” Science named GLP-1 drugs as its Breakthrough of the Year, while Nature included Svetlana Mojsov in its 2023 list of the year’s most important investigators. Mojsov is research associate professor at The Rockefeller University and was an early contributor to understanding the metabolic role of GLP.
Spirits were high at the 2023 annual meeting of the American Society of Hematology (ASH), buoyed by the U.S. FDA approval of the first two gene therapies for sickle cell disease (SCD) the day before the conference kicked off in San Diego. The addition of gene therapy to the therapeutic arsenal for SCD is “phenomenal,” Adetola Kassim, director of the Adult Sickle Cell Disease Program and professor of medicine at the Vanderbilt-Ingram Cancer Center, told BioWorld. Nevertheless, at a Saturday, Dec. 9, session titled, “Improving Outcomes for Individuals with Sickle Cell Disease: Are We Moving the Needle?,” which Kassim chaired, the answer remained “maybe.”
Katy Rezvani received this year’s E. Donnall Thomas Prize for her work on natural killer (NK) cells at the annual meeting of the American Society of Hematology (ASH). It was not love at first sight, though.
Spirits were high at the 2023 annual meeting of the American Society of Hematology (ASH), buoyed by the U.S. FDA approval of the first two gene therapies for sickle cell disease (SCD) the day before the conference kicked off in San Diego. The addition of gene therapy to the therapeutic arsenal for SCD is “phenomenal,” Adetola Kassim, director of the Adult Sickle Cell Disease Program and professor of medicine at the Vanderbilt-Ingram Cancer Center, told BioWorld. Nevertheless, at a Saturday, Dec. 9, session titled, “Improving Outcomes for Individuals with Sickle Cell Disease: Are We Moving the Needle?,” which Kassim chaired, the answer remained “maybe.”
Spirits were high at the 2023 Annual Meeting of the American Society of Hematology (ASH), buoyed by U.S. FDA approval of the first two gene therapies for sickle cell disease (SCD) the day before the conference kicked off in San Diego.
Using microglia and an unbiased screening method, investigators have identified almost 60 previously unknown targets for γ-secretase. Investigators from KU Leuven and colleagues published their results in Molecular Cell on Nov. 16, 2023.
Investigators at the Chinese Academy of Sciences have generated a chimeric monkey by injecting an embryonic stem cell into the morula, which is an extremely early embryo consisting of 16 to 32 cells. The animal survived for only 10 days, and it is not the first live birth of a chimeric primate. But it is the first such chimera with contributions from an embryonic stem cell, and that stem cell contributed a far higher proportion of cells in the newborn than have been achieved in previous attempts at creating chimeras.
The gene for Huntington’s disease “was cloned in 1993, and everyone thought there was going to be a treatment right around the corner,” Sarah Tabrizi told the audience at the 2023 Annual Meeting of the Society for Neuroscience. Then, “it took 25 years for the first trial targeting the Huntington gene.”
Investigators at the Chinese Academy of Sciences have generated a chimeric monkey by injecting an embryonic stem cell into the morula, which is an extremely early embryo consisting of 16 to 32 cells. The animal survived for only 10 days, and it is not the first live birth of a chimeric primate. But it is the first such chimera with contributions from an embryonic stem cell, and that stem cell contributed a far higher proportion of cells in the newborn than have been achieved in previous attempts at creating chimeras.
