Bluebird Bio extended a partnership with two divisions of Hitachi Chemical for the long-term development and manufacturing of LentiGlobin, its investigational gene therapy for sickle cell disease (SCD), and other potential and approved treatments. The two subsidiaries — Hitachi Chemical Advanced Therapeutics Solutions (HCATS) and Apceth Biopharma — will be in charge…
The Phase 2 STRONG-SCD trial evaluating olinciguat as a potential oral therapy for sickle cell disease is completely enrolled, Cyclerion Therapeutics said. Seventy patients with stable disease were recruited, and topline results are expected between July and September. The company is not expecting the supply of…
Global Blood Therapeutics (GBT) is giving $250,000 to support the U.S. sickle cell disease (SCD) community during the COVID-19 pandemic. Its newly created GBT Community Fund will award a total of $150,000 in grants to non-profit organizations supporting the needs of SCD patients and their families during…
High blood levels of two pro-inflammatory molecules, called interleukin-6 (IL-6) and interleukin-8 (IL-8), are linked to more frequent complications in people with sickle cell anemia, a study reports. The study, “High levels of proinflammatory cytokines IL-6 and IL-8 are associated with a poor clinical outcome…
The Institute for Clinical and Economic Review (ICER) says three recently approved therapies for managing sickle cell disease (SCD) are too costly based on early assessments of their effectiveness, and is recommending their prices be lowered. In a new evidence report, the independent watchdog agency on drug…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…
White blood cells, especially monocytes, may underlie the breathing difficulties affecting children with sickle cell disease (SCD), a small pilot study suggests. Asthma is estimated to impact from 17% to 28% of all children with SCD, its researchers noted, and obstructive lung disease — which affects exhaling — is…
The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation will each invest $100 million over the next four years to speed the development of affordable gene therapies for sickle cell disease (SCD) and the human immunodeficiency virus (HIV) on a global scale.
Enhanced parenting skills, including attentive listening and engaging in conversations, may help improve cognitive functioning in children with sickle cell disease (SCD), a study suggests. Parent stress was related to lower parenting responsiveness, which also may lead to lower cognitive function in children, the researchers said. The study, “…
Caraphenol A — a small molecule closely related to a natural compound called resveratrol, which is produced by red grapes — increased the efficacy of hematopoietic stem cell-based gene therapy, a new study shows. The molecule holds potential to shorten the time and cost of using these gene therapies…
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