At the European Hematology Association’s annual meeting in Vienna last week, companies reported impressive progress for the treatment of sickle cell disease.
New and updated preclinical and clinical data presented by biopharma firms at the European Hematology Association Congress, including: Abbvie, ADC, Apellis, Aruvant, Caribou, Cogent, CTI, Cytovia, GBT, Genentech, GT, Imago, Janssen, Keros, MEI, Morphosys, Oryzon, Roche, Ryvu, Sobi, Vincerx, Vor.
At the 2022 Annual Congress of the European Hematology Association, researchers from the Spanish National Cancer Institute reported how high levels of the RNA-binding protein hnRNP-K could lead to bone marrow failure.
Analysts have already started tagging Cogent Biosciences Inc.’s bezuclastinib as potentially best in class, after the company presented impressive, though early stage, data at the European Hematology Association Congress in Vienna demonstrating promising efficacy and a possibly differentiating safety profile for the selective KIT D816V inhibitor in advanced systemic mastocytosis.
After surprising Wall Street by unanimously voting in favor of the gene therapy elivaldogene autotemcel (eli-cel) for early active cerebral adrenoleukodystrophy from Bluebird Bio Inc., the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee met again June 10, this time to examine the risk-benefit profile of the company’s betibeglogene autotemcel (beti-cel) for people with transfusion-dependent beta-thalassemia.
The Cellular, Tissue and Gene Therapies Advisory Committee scrutinized Bluebird Bio Inc.’s gene therapy elivaldogene autotemcel (eli-cel) for early active cerebral adrenoleukodystrophy (CALD) in patients without a matched sibling donor.
Pointing to an anomalously high placebo response rate at eastern European trial sites in its Forward phase III trial testing fostamatinib in warm autoimmune hemolytic anemia (wAIHA), Rigel Pharmaceuticals Inc. executives remained confident there could be a path forward for the SYK inhibitor in treating the rare blood disorder. The disappointing top-line data, however, sent company shares (NASDAQ:RIGL) falling more than 60% June 8 to close at 70 cents, a penny above its same day 52-week low.
Wall Street took in stride mixed FDA briefing documents with regard to the upcoming adcom review of Bluebird Bio Inc.’s two gene therapy prospects, and shares of the company (NASDAQ:BLUE) closed at $3.61, up 63 cents, or 21%.
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