After long years of painstaking work, the commercialization of cell and gene therapies picked up pace in 2022, with multiple approvals. More progress is expected in 2023, with several firsts in the offing and products for larger patient populations reaching the market.
Parties to Astrazeneca plc’s potential $1.8 billion takeover of Cincor Pharma Inc. are keeping mum about contingent value rights included in the deal for baxdrostat, the phase III-bound aldosterone synthase targeter in the works to treat hypertension and chronic kidney disease. Another acquisition that captured headlines as the J.P. Morgan Healthcare Conference launched in San Francisco: the $1.4 billion-plus agreement whereby Chiesi Farmaceutici S.p.A. will take ownership of Amryt Pharma plc. And there was more, as Ipsen SA pledged $952 million to make Albireo Pharma Inc. its own.
Trex Bio Inc., which kicked off 2022 with a big pharma partnership, is back at it again, starting the new year with a potential $1.1 billion agreement with original backer Eli Lilly and Co. targeting immune-mediated diseases. Under the terms, Trexbio gets $55 million up front, with Lilly picking up an exclusive worldwide license for candidates from three programs.
New and updated clinical data presented by biopharma firms at the American Society of Hematology annual meeting and exposition, including: Amgen, Beigene, Novartis, Pharming.
New and updated clinical data presented by biopharma firms at the American Society of Hematology annual meeting and exposition, including: Abbvie, Actinium, Adicet, Affimed, Agios, Alphamab, Aptose, Argenx, Ascentage, Bellicum, Blueprint, BMS, Cellcentric, Cogent, CSL, CTI, Ellipses, Excellthera, Fate, Genentech.
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.
New and updated clinical data presented by biopharma firms at the San Antonio Breast Cancer Symposium, including: Ambrx, Astrazeneca, Celcuity, Treadwell, Zymeworks.
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