In the largest report on acute stroke in sickle cell disease to date, researchers at the Medical University of South Carolina and elsewhere provide the first systemic evidence for the safe use of tPA in stroke patients with sickle cell disease. Adult patients with sickle cell disease (SCD) who experience a stroke caused by a clot (i.e., ischemic strokes or IS) can be treated safely with tissue plasminogen activator (tPA) if they qualify, report investigators at the Medical University of South Carolina (MUSC) and elsewhere in the March 2017 issue of Stroke.

Last week, 100,000 Americans with sickle cell disease and millions more around the world got encouraging news. Investors gave a vote of confidence, in the form of a $120 million IPO, to Global Blood Therapeutics (NASDAQ: GBT), a four-year-old biotech working on a pill that could become the biggest medical advance ever for the disease. That pill, which just began its first human trial in January, is a once-a-day medicine that a patient would have to take for life. If approved, the drug would be the only sickle cell treatment to potentially halt the effects of the disease. The lone drug currently on the market for the disease only addresses some of its symptoms.

Imara Inc. today announced it will report additional preclinical and Phase 1 clinical data from the company’s lead compound, IMR-687, a once-daily, oral therapy designed to address the underlying pathology of sickle cell disease (SCD). In preclinical studies, IMR-687’s mechanism of action was shown to positively impact both red and white blood cell pathologies associated with the disease. The company has previously reported the ability of IMR-687 to induce fetal hemoglobin (HbF) in murine and human cells at ASH in 2017. Importantly, the data from a recent Phase 1 clinical trial in healthy volunteers demonstrated IMR-687 to be safe and well-tolerated at the target dose that exceeded the effective doses in cell and animal models of SCD, thereby clearing a path to a Phase 2 study in adult patients with SCD. The new data will be presented at the 6th Annual Sickle Cell Therapeutics Conference in New York.

Researchers at Mississippi State University (MSU) are using tiny polymer chips to build models to help them better understand sickle cell disease. Research on the models, which can also be used to study heart disease, might lead identification of molecules or factors that new treatments can target to lessen organ damage caused by the disease.

Introducing a “natural” genetic mutation into the blood cells of people with sickle cell and like diseases, using CRISPR gene editing, was seen to restore the production of fetal hemoglobin and offer a way to treat these disorders, researchers report. Their study was published in the journal Blood, in an article titled “KLF1 drives the expression of fetal hemoglobin in British HPFH.” Sickle cell disease (SCD) patients have a genetic defect that leads them to produce hemoglobin S, or sickle hemoglobin, instead of normal hemoglobin. This impairs the ability of red blood cells to bind to oxygen molecules in the lungs, and consequently to deliver oxygen to tissues throughout the body.

Gamida Cell, a leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases, announced today that it has been awarded a grant of $3.5 million from the Israel Innovation Authority (IIA) of the Israeli Ministry of Economy and Industry. The mission of the IIA is to encourage innovation and entrepreneurship in various industries, including science and technology, while stimulating economic growth. The non-dilutive funding, combined with the $40 million financing round, will support Gamida Cell's ongoing research and development efforts, including its Phase 3 registration study of NiCord for hematological malignancies, such as leukemia and lymphoma, its clinical trials of CordIn for sickle cell disease and thalassemia, and its NK cell therapy for blood and solid cancers.

Computer models developed by Brown University mathematicians show new details of what happens inside a red blood cell affected by sickle cell disease. The researchers said they hope their models, described in an article in the Biophysical Journal, will help in assessing drug strategies to combat the genetic blood disorder, which affects millions of people worldwide. The model uses detailed biomechanical data on how sickle hemoglobin molecules behave and bind with each other to simulate the assembly of a polymer fiber. Prior to this work, the problem had been that as the fiber grows, so does the amount of data the model must crunch. Modeling an entire polymer fiber at cellular scale using the details of each molecule was simply too computationally expensive.

Global biotherapeutics leader CSL Behring announced today that it has agreed to acquire Calimmune, Inc., a biotechnology company focused on the development of ex vivo hematopoietic stem cell (HSC) gene therapy with R&D facilities in Pasadena, California and Sydney, Australia for an upfront payment of $91 million. The acquisition will provide CSL Behring with Calimmune's pre-clinical asset, CAL-H, an HSC gene therapy for the treatment of sickle cell disease and β-thalassemia, which complements CSL Behring's current product portfolio and deep expertise in hematology. Additionally, CSL Behring will acquire two unique proprietary platform technologies, Select+ and Cytegrity. These technologies are designed to address some of the major challenges currently associated with the commercialization of stem cell therapy, including the ability to manufacture consistent, high-quality products, and to improve engraftment, efficacy and tolerability. Both technologies have broad applications in ex vivo stem cell gene therapy.

Bioverativ Inc. (NASDAQ: BIVV), a global biotechnology company focused on the discovery, development and commercialization of innovative therapies for hemophilia and other rare blood disorders, and Bicycle Therapeutics Ltd., a biotechnology company pioneering a new class of therapeutics based on its proprietary bicyclic peptide product platform, today announced a research collaboration focused on the discovery, development and commercialization of innovative therapies for hemophilia and sickle cell disease. The collaboration will seek to identify and develop Bicycles to treat rare blood disorders. Bicycles are a new therapeutic modality that combines attributes of antibodies, small molecules and peptides within one molecule, enabling high selectivity and affinity while simultaneously being able to penetrate and bind to the target(s) of interest within the body.