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As fellow gene editing firm Crispr Therapeutics AG hosted an innovation day in which it confirmed plans for regulatory filings by year-end for an ex vivo gene editing therapy in sickle cell disease and beta-thalassemia, Precision Biosciences Inc. announced plans to develop an in vivo gene editing approach through a collaboration with Novartis AG that brings Precision an initial $75 million with up to $1.4 billion in potential milestones.
New and updated preclinical and clinical data presented by biopharma firms at the European Hematology Association Congress, including: Abbvie, Affimed, Agios, Ascentage, Canbridge, Crispr, Daiichi, Disc, Genmab, Iaso, Innovent, Mustang, Novartis, Vertex, XNK.
At the European Hematology Association's annual meeting in Vienna last week, companies reported impressive progress for the treatment of sickle cell disease.
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.
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