Part of the reason for CAR T cells’ astonishing success in B-cell cancers is that B cells are astonishingly easy to replace. CAR T cells are specific, yes. But they are not specific to tumor cells. They are specific to their target antigens. In the case of Yescarta (axicabtagene ciloleucel, Gilead Sciences Inc.) and Kymriah (tisagenlecleucel, Novartis AG), the first two clinically approved T cells, that target is CD19, which is expressed on B-cell precursors. And when it is successful, the treatment leaves patients without any B cells at all.
Disc Medicine Inc.’s positive phase II data from an ongoing, open-label trial called Beacon with oral bitopertin in erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) whetted investor appetite for the results of the other mid-stage Disc experiment known as Aurora with the compound, due early next year.
Barring truly major surprises, exagamglogene autotemcel (Exa-cel, Vertex Pharmaceuticals Inc.) is on track to become the first approved CRISPR-based gene editing therapy. It is partly in expectation of Exa-cel’s approval that the European Hematology Association (EHA) and the European Society for Bone Marrow Transplantation hosted a session on “transplantation versus gene therapy in sickle cell disease.”
Researchers have ameliorated both monogenic and complex inflammation-driven diseases through transplantation of hematopoietic stem cells with an inserted IL-1 receptor antagonist (IL-1RA) gene. The team showed that in animal models the transplanted cells worked better than monoclonal antibodies to reduce symptoms in systemic autoinflammatory diseases (SAIDs), a group of childhood-onset, lifelong diseases that vary in severity depending on the underlying mutation, but can be life-threatening.
Pfizer Inc. has positive phase III data for its hemophilia treatment as it wades deeper into an indication that already has plenty of competition and at least one company with earnings of more than $1 billion. Pfizer’s marstacimab in treating hemophilia could lead to the first once-weekly subcutaneous treatment for hemophilia B and could end up being the first treatment administered as a flat dose for treating hemophilia A or B.
Varmx BV has raised €30 million (US$32 million) in an extension to its series B to fund preparations for the pivotal trial of VMX-C001, a modified recombinant factor X that acts as a universal antidote to reverse the effects of direct oral anticoagulants. The drug is intended for use in patients suffering severe spontaneous bleeding or needing emergency surgery.
After long years of endeavor, Scarlet Therapeutics Ltd. believes it is poised to realize the potential of red blood cells (RBCs) as drug delivery vehicles. The company recently closed a seed round and is now laying the ground for its first clinical trials in which cultured and genetically engineered RBCs will be used to deliver enzyme replacement therapies in the treatment of rare inherited forms of two metabolic disorders.
Homology Medicines Inc. has reported data on an adeno-associated viral (AAV) vector-based therapy, HMI-104, an AAV treatment intended to induce hepatic expression of a complement C5 monoclonal antibody, named as C5mAb, for the potential treatment of paroxysmal nocturnal hemoglobinuria (PNH) as well as other complement-driven pathologies. C5mAb is thought to bind to C5 and inhibit the C5-mediated hemolysis observed in PNH.
After Keros Therapeutics Inc.’s first-quarter earnings report, the Wall Street spotlight turned its beam toward additional data due soon from the phase II studies with KER-050, an ActRIIA-Fc fusion protein in myelodysplastic syndrome (MDS) and myelofibrosis.
Swedish Orphan Biovitrum (Sobi) AB is offering a hefty 95% premium to shareholders in CTI Biopharma Corp., as it tables a $9.10-per-share cash offer for the company, which implies a total equity valuation of $1.7 billion. Shares in Seattle-based CTI (NASDAQ:CTIC) had closed May 9 at $4.82 but surged 85% to close at $8.93 during trading May 10. The premium calculation is based on CTI’s 30-day volume-weighted average trading price of $4.67 prior to the deal announcement.
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