Results from the Phase 2 SUSTAIN clinical trial demonstrated that crizanlizumab (SEG101, Selexys Pharmaceuticals, Novartis), an anti-P-selectin antibody, reduced by 45.3% the median annual rate of sickle cell-related pain crises (SCPC), compared to placebo in patients with or without hydroxyurea therapy, a drug used to reduce the rate…
Imara recently presented positive preclinical results of its lead drug candidate IMR-687, a potent PDE9 inhibitor for the treatment of sickle cell disease (SCD). The presentation, which was previewed in a November article, took place at the 2016 American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego,…
The UT Physicians Comprehensive Sickle Cell Center has expanded its facilities in response to the needs of an increasing number of people living with sickle cell disease (SCD) who suffer from chronic pain, the university announced in a press release. The new center will offer sub-specialty, social…
Imara, a biotechnology company that is working towards developing new therapies against sickle cell anemia and other blood disorders, announced that the first healthy volunteer has been dosed in a Phase I clinical trial testing the safety and tolerability of their product candidate called IMR-687.
Karolinska Development’s Modus Therapeutics has been issued two U.S. patents covering its proprietary drug sevuparin, a candidate for the treatment of sickle cell disease. Specifically, the two patents have been granted claims for the substance sevuparin and a manufacturing method for producing the drug (U.S. Patent 9480701) and for…
Researchers were able to mend the mutated gene that causes sickle cell disease, and successfully transfer the corrected stem cells into mice. Their work could have enormous implications for millions worldwide, as it is proof-of-concept that such a treatment might also work in humans. The study, “CRISPR/Cas9 β-globin gene targeting in human haematopoietic…
Imara is scheduled to present preclinical efficacy and safety data on IMR-687, its lead product candidate for the treatment of sickle cell disease (SCD), at the American Society of Hematology (ASH) 58th Annual Meeting and Exposition in San Diego, Calif., Dec. 3-6. The oral presentation, titled “A Novel,…
The U.S. Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) from Emmaus Life Sciences for its orally administered pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell disease. If the FDA approves this new treatment, it would be the first FDA-approved medicine available…
The U.S. Food and Drug Administration (FDA) has approved the use of Immucor’s PreciseType HEA test to screen blood donors for sickle cell trait (SCT), an inherited blood disorder that affects 1 million to 3 million Americans. SCT is not a disease, but people with the disorder…
Researchers have cured mice with a genetic blood disease by correcting mutated genes in blood cell-producing stem cells. The method offers a new therapeutic approach for treating blood disorders, such as sickle cell disease and thalassemia. The gene-editing technique can be administered easily into living animals and notably decreases off-target effects…
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