The risk and benefit of Pfizer Inc.’s oral sickle cell disease drug Oxbryta (voxelotor) has flipped, prompted by what the company called new clinical data indicating “an imbalance in vaso-occlusive crises and fatal events” that need more study. Based on an EMA recommendation, Pfizer said it is voluntarily recalling all lots of Oxbryta from wherever it’s approved worldwide. Pfizer also is shuttering its Oxbryta clinical studies and expanded access programs.
Recent studies have shown that up-regulation of peroxisome proliferator-activated receptor γ coactivator-1α (PGC-1α) was able to induce fetal hemoglobin synthesis in human primary erythroblasts.
A recent study in PLoS One by researchers from Cincinnati Children’s Hospital Medical Center has evaluated the preclinical long-term safety of human A gamma-globin gene-carrying GbGM LV in wild-type mice.
Scientists at the Novartis Institutes for Biomedical Research (NIBR) in Cambridge have discovered a small molecule that could be used as a therapy for sickle cell disease (SCD). The molecular glue oral degraders of the WIZ transcription factor called dWIZ-1 and dWIZ-2, bind to cereblon (CRBN) and WIZ, marking it for degradation and inducing the expression of fetal hemoglobin (HbF).
Actinium Pharmaceuticals Inc. has received FDA clearance of an IND application to study Iomab-ACT for targeted conditioning prior to a bone marrow transplant (BMT) in patients with sickle cell disease.
The U.S. Department of Health and Human Services’ Office of the Inspector General disclosed an advisory opinion finding Bluebird Bio Inc.’s fertility support program for a gene therapy treatment could run afoul of federal anti-kickback statutes. That follows a similar opinion against Vertex Pharmaceuticals Inc., and its fertility program associated with gene-editing therapy Casgevy (exagamglogene autotemcel). Vertex subsequently filed a lawsuit.
Orum Therapeutics Inc. struck a potential $945 million (₩1.3 trillion) deal with Vertex Pharmaceuticals Inc. to discover novel degrader antibody conjugates (DAC) as targeted conditioning agents for use with gene editing, including Vertex’s gene therapy, Casgevy (exagamglogene autotemcel).
The success of a vaccine, a gene editing design for an untreated disease, or achieving cell engraftment after several attempts, comes from years of accumulated basic science studies, thousands of experiments, and clinical trials. Innumerable steps precede hits in gene and cell therapies before a first-time revelation, and most of them are failures at the time. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Baltimore last week, several groups of scientists presented achievements that years ago looked impossible.
Some gene therapies could be big winners under the changes the U.S. Centers for Medicare & Medicaid Services (CMS) is proposing to Medicare’s new technology add-on program (NTAP) for its fiscal 2025 inpatient prospective payment system.
The European Commission approved two therapies for progressive, genetic diseases: Biogen Inc.’s Friedreich’s ataxia drug, Skyclarys (omaveloxolone), and Crispr Therapeutics AG’s CRISPR/Cas9 gene therapy for sickle cell disease and transfusion-dependent beta-thalassemia, Casgevy (exagamglogene autotemcel, exa-cel).
