As fellow gene editing firm Crispr Therapeutics AG hosted an innovation day in which it confirmed plans for regulatory filings by year-end for an ex vivo gene editing therapy in sickle cell disease and beta-thalassemia, Precision Biosciences Inc. announced plans to develop an in vivo gene editing approach through a collaboration with Novartis AG that brings Precision an initial $75 million with up to $1.4 billion in potential milestones.
At the European Hematology Association's annual meeting in Vienna last week, companies reported impressive progress for the treatment of sickle cell disease.
At the European Hematology Association’s annual meeting in Vienna last week, companies reported impressive progress for the treatment of sickle cell disease.
Analysts have already started tagging Cogent Biosciences Inc.’s bezuclastinib as potentially best in class, after the company presented impressive, though early stage, data at the European Hematology Association Congress in Vienna demonstrating promising efficacy and a possibly differentiating safety profile for the selective KIT D816V inhibitor in advanced systemic mastocytosis.
Bluebird Bio Inc. became the latest in a spate of gene therapy firms to disclose restructuring plans, as the company aims to save $160 million over the next two years, saying goodbye to about a third of its workforce. It’s the other shoe to drop after Cambridge, Mass.-based Bluebird rattled Wall Street with phraseology in the firm’s fourth-quarter earnings report March 4 that expressed “substantial doubt” regarding whether operations could go on.
Fulcrum Therapeutics Inc. shares (NASDAQ:FULC) closed at $18.77, up $10.44 or 125%, on word of positive interim results from a phase I trial in healthy adult volunteers with oral FTX-6058 for sickle cell disease (SCD). The firm has “already achieved maximal target engagement [MTE] at all three doses,” said Christopher Morabito, the company’s chief medical officer. “I don’t think we’ll exceed that.”
LONDON – The World Health Organization (WHO) is to set up a channel for confidential reporting of illegal, unregistered, unethical or unsafe human genome editing research, as part of a new governance framework it is proposing to develop.
Princeton University spinout Neutigers Inc. is launching a study to explore the use of artificial intelligence (AI) and everyday wearables to flag early symptoms of sickle cell anemia vaso-occlusive crisis (VOC) before they get worse and land patients in the hospital. The aim is to reduce deaths and facilitate interventions to address the entire continuum of care for patients with the inherited red blood cell disorder, Adel Laoui, founder and CEO, told BioWorld.
Hemex Health Inc.’s newborn screening for sickle cell disease substantially reduces the labor involved for parents and providers in testing for the potentially fatal condition. The test had previously been able to test infants 6 weeks and older on the company’s Gazelle platform.
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.
