The FDA has lifted clinical holds on four studies from Bluebird Bio Inc., following recent similar actions with other gene therapy programs. Two of the studies concern phase I/II and phase III clinical trials of the gene therapy Lentiglobin (BB-1111) in treating sickle cell disease. The remaining two studies are phase III clinical trials of betibeglogene autotemcel gene therapy, which share a vector with Lentiglobin, for treating transfusion-dependent beta-thalassemia.
Advances lately in the genome-editing space include Beam Therapeutics Inc. publication in The CRISPR Journal details of its work with inlaid base editors, which the firm is applying in the BEAM-102 program for sickle cell disease. IBEs’ predictable, shifted editing window lets researchers go after disease-causing mutations that canonical base editors cannot reach, Beam said, and do the job with high efficiency and few off-target effects on the genome. The hottest news due in the near-term future from the sector will spill from Intellia Therapeutics Inc., of Cambridge, Mass., which is due to roll out first-in-human data with a systemic CRISPR-based genome editing therapy, NTLA-2001, in hereditary transthyretin amyloidosis.
In biotech and biopharma’s third-largest ever up-front development and commercialization deal, Crispr Therapeutics AG will receive an initial $900 million in an amended deal with Vertex Pharmaceuticals Inc. to lead the development, manufacturing and commercialization of gene editing therapy CTX-001 for sickle cell disease and transfusion-dependent beta-thalassemia.
Just weeks after two unexpected cases of blood cancer landed trials of its investigational gene therapies for sickle cell disease (SCD) and beta-thalassemia on FDA-issued clinical holds, Bluebird Bio Inc. said it's talking to regulators about their resumption after what RBC analyst Luca Issi called a "partial exoneration" of the BB-305 lentiviral vector shared between the medicines.
Shares of Bluebird Bio Inc. (NASDAQ:BLUE) fell 37.8% to $28.44 on Feb. 16 as the company temporarily suspended two trials of its experimental gene therapy for sickle cell disease, Lentiglobin (BB-1111), while investigating one unexpected case of acute myeloid leukemia (AML) and another of myelodysplastic syndrome among participants in a phase I/II study of the candidate, called HGB-206. A second patient experienced MDS in 2018.
Imara Inc. reported a mixed bag of phase IIa study data for its lead candidate, IMR-687, for treating sickle cell disease in adults, bringing an end to a study that the company found cumbersome and that eventually stung its stock.
Despite challenges tossed at the phase III study of mitapivat from Agios Pharmaceuticals Inc. by COVID-19, top-line data showed the oral therapy hit its primary endpoint in treating adults with pyruvate kinase (PK) deficiency who don’t receive regular transfusions.
Shares of Bluebird Bio Inc. (NASDAQ:BLUE) sank 16.6%, or $9.72, to close at $48.83 as Wall Street reacted to news that the U.S. regulatory filing for Lentiglobin in sickle cell disease (SCD) will be delayed. Previously expected in the second half of next year, the filing won’t happen until late 2022.
Cyclerion Therapeutics Inc.’s phase II blowup with sickle cell disease (SCD) candidate olinciguat ended its development, and attention turned to the Cambridge, Mass.-based firm’s earlier-stage effort with IW-6463, a drug in the same class for central nervous system (CNS) disorders.
The CDC estimates that sickle cell disease affects well over 100,000 Americans, with the disease occurring most often in African Americans. September has been designated as National Sickle Cell Awareness month designed to focus attention on the ongoing research in this field and the need for new treatments.
