About four years after launch, Orna Therapeutics Inc. signed its second major deal, this time validating the lipid nanoparticle delivery technology it acquired through its Renagade Therapeutics Inc. buyout in May 2024, with Vertex Pharmaceuticals Inc. seeking next-generation approaches for hemoglobinopathies.
The U.S. CMS has negotiated outcomes-based agreements with Bluebird Bio Inc. and Vertex Pharmaceuticals Inc. to make their costly sickle cell gene therapies the first treatments to become available through the voluntary Medicaid Cell and Gene Therapy Access Model.
Alongside the release of abstracts related to the American Society of Hematology meeting next month in San Diego, and as part of the firm’s third-quarter update, Beam Therapeutics Inc. disclosed that one patient died in the phase I/II trial testing BEAM-101 in sickle cell disease (SCD).
Although more and more gene therapies are getting the FDA stamp of approval, concerns persist about their potential long-term risks. U.S. lawmakers have proposed several pieces of legislation over the past few years to address some of the uncertainties. Now the Congressional Research Service (CRS) is suggesting other requirements Congress may want to consider to improve the regulatory landscape for gene therapies, especially those intended to treat blood disorders.
Editas Medicine Inc. has achieved in vivo preclinical proof of concept of hematopoietic stem and progenitor cell (HSPC) editing and fetal hemoglobin (HbF) induction in humanized mice engrafted with human hematopoietic stem cells and lacking their own hematopoietic cells.
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.
