Removing the spleen by keyhole surgery is a feasible option for children with sickle cell disease, according to a study that explored the procedure as the sole therapeutic intervention. But because the study described only short-term outcomes, researchers underscored that more research is needed to investigate how a splenectomy affects…
The recent signing into law by President Barack Obama of the 21st Century Cures Act, a bipartisan bill whose aims include speeding the development of treatments for patients with difficult medical conditions, like sickle cell disease (SCD), was welcomed by the biotech company Imara in a recent press release. The legislation, supported…
Scientists have created a risk profile that uses three genetic markers to determine whether there is a high or low chance of a sickle-cell-anemia (SCA) patient developing kidney disease. Preliminary results indicate that the combination does a better job of predicting kidney disease than markers used by themselves. The study,…
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,…
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