News

A new National Heart, Lung, and Blood Institute (NHLBI) challenge will award a first prize of $25,000 to a team of college or graduate students who develop an innovative tool to help spread evidence-based information about sickle cell disease (SCD). A total of $50,000 will be granted to the three…

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

Red blood cells in people with sickle cell disease (SCD) flow through blood vessels in a manner that damages the walls, which may explain blood vessel problems and increased inflammation in SCD, new research indicates. The study, “Flow-induced segregation and dynamics of red blood cells in sickle cell…

A recent manufacturing agreement between Aruvant Sciences and Lonza will advance the development of ARU-1801, an investigational gene therapy for the treatment of sickle cell disease (SCD). Under the agreement, Lonza will be responsible for producing Aruvant’s gene therapy for an upcoming pivotal clinical trial. The company already has…

The European Medicines Agency (EMA) is reviewing an application from Global Blood Therapeutics (GBT) that seeks full marketing approval for the oral therapy Oxbryta (voxelotor) for people with sickle cell disease (SCD), ages 12 and up. Oxbryta would be the first treatment targeting the root cause of…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

A clinical trial will assess a combination of MGTA-145 plus plerixafor as a new regimen for aiding in the collection of blood stem cells from adults and adolescents with sickle cell disease (SCD). The proof-of-concept trial, a collaboration between Magenta Therapeutics and Bluebird Bio, may establish this…

The U.S. Food and Drug Administration (FDA) has cleared the initiation of a Phase 1/2 trial investigating EDIT-301, Editas Medicine’s experimental gene editing cell therapy for  sickle cell disease (SCD). The planned open-label study, to be called RUBY, will assess the safety and efficacy of a…

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…

The investigational gene editing therapy GPH101 will be the first potentially curative treatment for sickle cell disease (SCD) to be tested in a Phase 1/2 clinical trial. The U.S. Food and Drug Administration (FDA) has cleared GPH101 for clinical testing. With this decision, the therapy’s developer, Graphite Bio,…