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.”
It is known that heterozygous mutations in the HBB gene, which encodes β-globin, are the cause of inherited β-thalassemia. A new case report describes a novel frameshift mutation in the HBB gene leading to a dominant form of β-thalassemia.
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.
CAR T-cell therapies have worked well at curing blood malignancies, but a group out of the University Hospital of Erlangen have repurposed the technology as a treatment for autoimmune diseases. The expansion into new diseases has required cooperation between multiple departments, with CAR T experts taking the lead on treatment and potential side effects, and rheumatologists measuring the outcomes of the treatment.
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.”
Wall Street’s measure of how Cogent Biosciences Inc.’s KIT D816V inhibitor bezuclastinib (often shortened to bezu) might fare in mastocytosis against U.S. FDA cleared Ayvakit (avapritinib), the tyrosine kinase inhibitor from Blueprint Medicine Corp., caused the former’s stock (NASDAQ:COGT) to tumble, closing Dec. 11 at $4.06, down $4.58, or 53%. Data from the ongoing phase II Summit trial testing bezu in non-advanced systemic mastocytosis rolled out during the American Society of Hematology (ASH) meeting in San Diego. Waltham, Mass.-based Cogent’s prospect turned up a rapid and continuing improvement in patient symptoms, with a 57% median best improvement on Mast Cell Quality-of-Life.
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.
Following a strategic transaction with Graphite Bio Inc., Kamau Therapeutics is emerging from stealth with sickle cell treatment nulabeglogene autogedtemcel (nula-cel). Kamau received an option to acquire all of Graphite’s genome editing assets, including a platform technology that integrates precision DNA repair using homology directed repair and CRISPR/Cas9, as well as the autologous CRISPR/Cas9 gene corrected CD34+ cell product nula-cel, which offers a potential cure for sickle cell disease derived from the patient's cells.
Both Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) and Bluebird Bio Inc.’s Lyfgenia (lovotibeglogene autotemcel, lovo-cel) received U.S. FDA approval Dec. 8, providing 16,000 American sickle cell patients who have recurring vaso-occlusive events with access to the first cell-based gene therapies.
The U.S. FDA has approved Novartis AG’s Fabhalta (iptacopan) as the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria, a rare blood disease that impairs blood cell production. This is the only factor B inhibitor of the immune system’s complement pathway and is expected to be on the market before December ends. Fabhalta has plenty of competition from already-approved therapies and more treatments are in development.
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