News

September 24, 2020September 24, 2020

Gene-editing Therapy CTX001 Granted Priority Medicines Designation for Severe SCD in EU

News

CTX001, an investigational gene-editing cell therapy, has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of severe sickle cell disease (SCD). The announcement was ... Read more

September 22, 2020September 22, 2020

Graphite Bio Raises $45M to Move New Way of Gene Editing Into Sickle Cell Trial

News

Graphite Bio, a startup biotech company, has raised $45 million in an initial financing round to advance its gene editing technology to treat sickle cell disease and other genetic conditions. ... Read more

September 17, 2020September 17, 2020

GreenLight Wins $3.3M Grant to Pursue mRNA-based Gene Therapies

News

The Bill & Melinda Gates Foundation has awarded a $3.3 million grant to GreenLight Biosciences to develop gene therapies revolving around messenger RNA (mRNA) — the intermediate molecule between ... Read more

September 15, 2020September 15, 2020

Study Identifies FDA-approved Medications With Potential for Treating SCD

News

Using a newly developed assay, researchers identified seven U.S. Food and Drug Administration (FDA)-approved medications that may be effective, with minimal safety issues, in treating sickle cell disease (SCD). Their ... Read more

September 11, 2020September 11, 2020

Activities Underway for Newborn Screening Awareness Month

News, syndicated

As it does each September during Newborn Screening Awareness Month, Baby’s First Test is sharing information and stories that highlight efforts throughout the U.S. to bring attention to newborn testing. ... Read more

September 10, 2020September 10, 2020

Genetic Differences Seen to Mirror Sickle Cell Severity in African Study

News

Genetic analyses of African patients identified several genes and biological pathways that are associated with disease severity and longevity, and that might serve as new therapeutic targets in sickle ... Read more

September 7, 2020September 8, 2020

‘Brain-Gut Axis’ May Be What Links Stress and Crises in SCD Patients

News

Microbes in the gut play a key role in inducing vaso-occlusive crises (VOCs) in response to stress, a study in mice suggests. Stress is known to trigger VOCs in ... Read more

September 3, 2020September 3, 2020

Grant Expands SCD Patient Registry of COVID-19 Infections to Include Africa

News

The SECURE-SCD registry, which is collecting de-identified data on individuals with sickle cell disease (SCD) who develop COVID-19, will be expanding to include information on children and adults in ... Read more

September 2, 2020September 2, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

News, syndicated

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme ... Read more

September 1, 2020September 1, 2020

Sickle Cell Awareness Month Events Underway Across US

News

September is National Sickle Cell Awareness Month, set aside annually to bring attention to the blood disorder and the needs of the community. From securing local government proclamations and ... Read more