Once-daily treatment with voxelotor, Global Blood Therapeutics’ lead therapy candidate, can effectively increase the amount of hemoglobin in about 58% of patients with sickle cell disease (SCD) in just 12 weeks, according to results from Part A of a Phase 3 trial. Supported by these positive results, the company…
Bluebird bio reported that its investigational gene therapy LentiGlobin showed promising results for severe sickle cell disease (SCD) patients in a Phase 1 clinical trial. Data from the ongoing HGB-206 Phase 1 multicenter study (NCT02140554) were revealed in an oral presentation, “Recent progress in gene therapy for severe…
The Sickle Cell Disease Association of America (SCDAA) is collaborating with Emmaus Life Sciences to increase patient education and awareness about the complications of sickle cell disease (SCD). The partners aim to improve national awareness so that information about the disease can reach broader audiences through online…
The Sickle Cell Disease Association of America (SCDAA) recently launched the first patient-driven registry for sickle cell disease (SCD), called Get Connected. The new registry was launched by the SCDAA on June 19, World Sickle Cell Day,…
MaxCyte, a company specialized in cell-based medicines, announced it has entered in a research agreement with the National Heart, Lung and Blood Institute (NHLBI) to develop potential therapies for sickle cell disease (SCD). Researchers will use MaxCyte’s gene-correction platform, Flow Electroporation Technology, in their work. The platform is…
The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to olinciguat (IW-1701) as a potential treatment of sickle cell disease (SCD), Ironwood Pharmaceuticals announced. Evidence suggests that the symptoms of sickle cell disease may be associated with nitric oxide deficiency in the blood. Nitric oxide…
The Vodafone Americas Foundation recently announced the winners of its 10th annual Wireless Innovation Project (WIP), which awarded $600,000 in three-year grants to tackle critical issues facing the global community. The first-place prize of $300,000 was awarded to a point-of-care platform called SMART, for its remote diagnostic testing targeting…
Sancilio Pharmaceuticals is reporting that its investigational oral compound Altemia (SC411, docosahexaenoic acid) showed an ability to safely treat sickle cell disease (SCD) in a Phase 2 study. Data from the Phase 2 SCOT trial (NCT02973360) trial were given at the 2018 American Society Pediatric Hematology and Oncology (ASPHO)…
The U.S. Food and Drug Administration placed a clinical hold on the investigational new drug (IND) application for CTX001, an experimental therapy for the treatment of sickle cell disease, according to a statement by CRISPR Therapeutics and Vertex Pharmaceuticals. The hold on the clinical development of CTX001 will…
As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…
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