News

The first patient with severe sickle cell disease (SCD) who received a single infusion of CTX001, an experimental gene-editing cell therapy, remains free of vaso-occlusive crises (VOCs) nine months after treatment, a Phase 1/2 clinical trial shows. Findings from the trial, “(S280) Initial Safety…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

Supporters are poised to mark World Sickle Cell Day, observed each June 19 to raise awareness about the red blood cell disorder, and to raise funds to fight it. Across the globe, supporters will participate in activities such as wearing red for sickle cell disease (SCD), sponsoring a…

Bluebird Bio’s investigational gene therapy LentiGlobin safely prevents serious vaso-occlusive crises and acute chest syndrome in adolescents and adults with severe sickle cell disease (SCD), according to recent data from a Phase 1/2 clinical trial. The findings, “Outcomes in patients treated with LentiGlobin…

Global Blood Therapeutics (GBT) will ask the U.S. Food and Drug Administration (FDA) to allow children with sickle cell disease (SCD), ages 4 to 11, to also be treated with Oxbryta (voxelotor). The company announced plans to file an application requesting extended approval after a Type B meeting with FDA…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.

The Sickle Cell Society of the U.K. has created a survey to gather feedback from people with sickle cell disease (SCD) and their caregivers during the COVID-19 pandemic. Overall, the society aims to use data collected to assess if patients and those caring for someone with this disease have…

IMR-687 safely increases the levels of fetal hemoglobin and the number of fetal hemoglobin-producing cells in people with sickle cell disease (SCD), according to early data from an ongoing Phase 2a clinical trial. The findings support the continuation of IMR-687’s clinical development as a potential disease modifying…

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

A smartphone app that measures hemoglobin levels using pictures of a person’s inner eyelid may help assess anemia in people with blood disorders such as sickle cell disease, a new study reports.