Doctors at Great Ormond Street Hospital London have reported the first successful use of base-edited donor T cells, in the treatment of refractory T-cell lymphoblastic lymphoma. The case of a 13-year-old girl named Alyssa, whose disease had not responded to chemotherapy and a bone marrow transplant, was presented at the American Society of Hematology meeting in New Orleans on Dec. 11, 2022.
After gaining U.S. FDA priority approval for the first gene therapy to treat hemophilia B, CSL Ltd. reported long-term data from the pivotal HOPE-B trial that showed a single infusion of Hemgenix (etranacogene dezaparvovec-drlb) generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding. Presented at the American Society of Hematology (ASH) annual meeting on Dec. 10, data showed 24-month results reinforced the safety of treatment, with no serious treatment-related adverse effects.
Affimed NV said the latest data with its lead innate cell engager (ICE) known as AFM-13, disclosed at the American Society of Hematology (ASH) meeting, have caused the focus to shift from monotherapy to combination regimens.
Vega Therapeutics Inc. emerged from stealth in advance of a podium presentation at this year’s American Society of Hematology meeting in New Orleans. The company, a spin-out from Star Therapeutics Inc., will disclose preclinical data on its lead drug candidate VGA-039, a first-in-class antibody in development for von Willebrand disease.
The U.S. FDA gave its go-ahead for Hemgenix (etranacogene dezaparvovec-drlb), Uniqure NV’s one-time gene therapy – the first for the treatment of adults 18 and older living with hemophilia B. Patients have been waiting “maybe beyond two decades” for a new therapy, Uniqure CEO Matthew Kapusta said. Hemgenix emerged from pioneering work by St. Jude Children’s Research Hospital and the University College London.
Merck & Co. Inc. is adding to its hematology assets in the takeover of Imago Biosciences Inc. for $36 per share, an arrangement with an equity value of about $1.35 billion. Shares of Imago (NASDAQ:IMGO) took flight as Wall Street learned of the deal, ending the day at $35.59, up $18.19, or 104%. Imago is working on new drugs for myeloproliferative neoplasms and other bone marrow diseases. Lead candidate bomedemstat (IMG-7289), an orally available inhibitor of the epigenetic enzyme lysine-specific demethylase 1 (LSD1), is undergoing phase II trials for the treatment of essential thrombocythemia, myelofibrosis and polycythemia vera, along with other indications.
Galecto Inc.’s late-September news from its ongoing phase IIa trial with GB-2064 in myelofibrosis hiked shares of the Boston-based firm and piqued interest in the lysyl oxidase-like 2 (LOXL2) target, where such names as Gilead Sciences Inc. and Pharmaxis Ltd. also have been busy.
X4 Pharmaceuticals Inc.’s recent stock-boosting phase Ib news with lead candidate mavorixafor fueled already-strong interest in finding an alternative therapy for neutropenia and deepened esteem for the drug’s long-known mechanism of action.
After winning the backing of European regulators, Biomarin Pharmaceutical Inc. is returning to the U.S. FDA with its hemophilia A gene therapy, valoctocogene roxaparvovec, following a rejection in August 2020. It's armed with data it hopes will assuage concerns about long-term safety and benefits.
CRISPR-based cell therapies continued to gain steam Sept. 27 with the announcements of a potentially valuable big pharma collaboration and an ambitious global regulatory push.
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