It is known that heterozygous mutations in the HBB gene, which encodes β-globin, are the cause of inherited β-thalassemia. A new case report describes a novel frameshift mutation in the HBB gene leading to a dominant form of β-thalassemia.
With a landmark U.K. approval in hand for Casgevy (exagamglogene autotemcel [exa-cel]) to treat sickle cell disease and transfusion-dependent beta thalassemia, Crispr Therapeutics AG and partner Vertex Therapeutics Inc. are turning their attention to the PDUFA dates set by the U.S. FDA for the treatment in both conditions.
Precision Biosciences Inc. uses its proprietary Arcus platform to develop in vivo gene editing therapies and has outlined new data from its wholly owned and partnered pipeline.
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.”
The positive opinion Jan. 27 from the EMA’s Committee for Medicinal Products for Human Use regarding Reblozyl (luspatercept) from Bristol Myers Squibb Co. to treat adults with non‑transfusion-dependent beta-thalassemia marked an advance in the space, where several developers are jockeying for position. Reblozyl, a first-in-class erythroid maturation agent, was first approved in November 2019 in the hands of Celgene Corp., acquired by Princeton, N.J.-based BMS the same year.
Mabwell Therapeutics Inc., a wholly owned subsidiary of Mabwell (Shanghai) Bioscience Co. Ltd., has entered into a license agreement with Disc Medicine Inc. for 9MW3011 (MWTX-001, MWTX-002 & MWTX-003) for hematologic diseases.
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%.
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.
Access to advanced therapies proved to be a major talking point at a conference in London, following the U.S. approval of Bluebird Bio Inc.’s Zynteglo (betibeglogene autotemcel) cell-based gene therapy for beta thalassemia and its $2.8 million price tag. Regulators in Europe backed Zynteglo in 2019 but Bluebird opted to withdraw the therapy in 2021 after deciding that the complex thicket of pricing bodies in Europe was too difficult to negotiate.
Bluebird Bio Inc. isn’t giving out much of the information on the margins for the cost of its newly approved cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia. The numbers will, the company said, come into better focus when another Bluebird drug is approved and launched.
