Modifying a patient’s DNA is no longer just for science fiction novels. The CRISPR gene editing technique developed by Jennifer Doudna and Emmanuelle Charpentier only took 10 years to reach the market as Casgevy (exagamglogene autotemcel/exa-cel, Vertex Pharmaceuticals Inc.), treating congenital pathologies such as β-thalassemia and severe sickle cell disease. But science does not stop.
Researchers have identified a new class of antibiotics that works by blocking the transportation of lipopolysaccharide (LPS) to the outer membrane of the gram-negative bacterium Acinetobacter baumannii. The most advanced member of the class, zosurabalpin (RG-6006, Roche AG), was effective against multiple A. baumannii strains, including carbapenem-resistant and multidrug-resistant strains.
In July, Leqembi (lecanemab, Biogen Inc./Eisai Co. Ltd.) became the first amyloid-targeting drug to win traditional approval from the U.S. FDA, after getting accelerated approval in January based on the surrogate endpoint of plaque removal.
Modifying a patient’s DNA is no longer just for science fiction novels. The CRISPR gene editing technique developed by Jennifer Doudna and Emmanuelle Charpentier only took 10 years to reach the market as Casgevy (exagamglogene autotemcel/exa-cel, Vertex Pharmaceuticals Inc.), treating congenital pathologies such as β-thalassemia and severe sickle cell disease (SCD). But science does not stop.
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.
In November, investigators at the Chinese Academy of Sciences reported generating a chimeric monkey by injecting an embryonic stem cell into the morula, which is an extremely early embryo consisting of 16 to 32 cells.
Researchers have used explainable artificial intelligence (explainable AI) to find structurally new antibiotics with minimal toxicity. They reported their findings online in Nature on Dec. 20, 2023. In animal testing, compounds identified via the method showed that they had activity against drug-resistant gram-positive bacteria including methicillin-resistant Staphylococcus aureus (MRSA), one of the most serious bacterial public health threats.
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.”
