Coming off a tough year for raising money, Cadrenal Therapeutics Inc. is leading the charge into a new year by completing 2023’s first U.S. IPO. The company closed its IPO of 1.4 million shares at $5 each for $7 million. The stock (NASDAQ:CVKD) began trading Jan. 20 and closed 9.37% downward on Jan. 25 at $2.90 per share.
Just in time for the Chinese New Year, Mabwell Bioscience Co. Ltd. announced that its U.S. subsidiary, Mabwell Therapeutics Ltd., closed a licensing deal with Disc Medicine Inc. worth up to $412 million for preclinical candidate 9MW-3011, a humanized monoclonal antibody (MAb) that regulates iron metabolism.
The serious adverse event in the first phase I/II patient dosed with nulabeglogene autogedtemcel (nula-cel) – a gene editing, autologous hematopoietic stem cell therapy for sickle cell disease (SCD) – may have involved the quality of the stem cells and not the conditioning regimen or characteristics of the patient at baseline. But researchers won’t know until more work is done.
Jasper Therapeutics Inc. may have found a way around toxicity with current approaches in sickle cell disease (SCD) conditioning approaches, if phase I/II data with briquilimab stay consistent – and the drug already has proved itself across a range of indications. Wall Street liked the prospect, sending the Redwood City, Calif.-based firm’s shares (NASDAQ:JSPR) on a wild ride to close at $2.74, up $2.26, or 476%.
Little more than a month after the U.S. FDA approved the first gene therapy for adults with hemophilia B, Uniqure NV’s Hemgenix, strong phase III data have come from Pfizer Inc. The Pfizer results show fidanacogene elaparvovec, a vector containing an AAV capsid and a high-activity human coagulation factor IX (FIX) gene for treating adult men with moderately severe to severe hemophilia B, hit the primary endpoint in the phase III Benegene-2 study. The one-time therapy is designed to allow those living with hemophilia B to be able to produce FIX instead of receiving regular, ongoing doses of exogenous FIX.
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.
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