Sickle Cell Disease News

September 22, 2020September 22, 2020

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

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. The ... Read more

September 17, 2020September 17, 2020

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

COVID-19 News, Sickle Cell Disease 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 DNA ... Read more

September 15, 2020September 15, 2020

Study Identifies FDA-approved Medications With Potential for Treating SCD

Sickle Cell Disease 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 findings ... Read more

September 11, 2020September 11, 2020

Activities Underway for Newborn Screening Awareness Month

Sickle Cell Disease News

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. Baby’s ... Read more

September 10, 2020September 10, 2020

Genetic Differences Seen to Mirror Sickle Cell Severity in African Study

Sickle Cell Disease 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 cell ... Read more

September 7, 2020September 8, 2020

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

Sickle Cell Disease 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 people ... Read more

September 3, 2020September 3, 2020

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

COVID-19 News, Sickle Cell Disease 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 Africa. ... Read more

September 2, 2020September 3, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

Sickle Cell Disease News

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 “A ... Read more

September 1, 2020September 1, 2020

Sickle Cell Awareness Month Events Underway Across US

Sickle Cell Disease 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 hosting ... Read more

August 27, 2020August 27, 2020

European Commission Grants Orphan Drug Status to Imara’s IMR-687 for Sickle Cell Disease

Sickle Cell Disease News

The European Commission has granted orphan drug designation to IMR-687, Imara’s potential new therapy for sickle cell disease (SCD). In the European Union, this designation is given to investigative therapies ... Read more

August 25, 2020August 26, 2020

EDIT-301, Experimental Cell Therapy for SCD, Earns Rare Pediatric Disease Designation

Sickle Cell Disease News

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation to EDIT-301, a potential cell therapy for the treatment of sickle cell disease (SCD). This designation is given ... Read more

August 20, 2020August 20, 2020

Medical Marijuana Reduces Pain and Hospital Visits in SCD Patients

Sickle Cell Disease News

Use of medical marijuana to ease pain is associated with fewer visits to the hospital in people with sickle cell disease (SCD), a study suggests. Patients given medical marijuana felt ... Read more

August 18, 2020August 18, 2020

First Sickle Cell Patient Dosed in Phase 2b Trial of Imara’s IMR-687

Sickle Cell Disease News

A Phase 2b clinical trial that is investigating the safety and effectiveness of IMR-687 in people with sickle cell disease (SCD) has dosed its first participant, according to the potential therapy’s ... Read more

August 17, 2020August 18, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Sickle Cell Disease News

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The ... Read more

August 13, 2020August 14, 2020

Sickle Cell Oral Treatment Xromi Approved in Scotland for Ages 2–9

Sickle Cell Disease News

The Scottish Medicines Consortium (SMC) has approved the use of Xromi (hydroxycarbamide), a liquid form of hydroxyurea, for treating children with sickle cell disease (SCD). Xromi is expected to be used ... Read more