Sickle Cell Disease News

November 24, 2020November 24, 2020

Gates Foundation Funds Studies Into CRISPR/Cas9 Gene Editing for SCD

Intellia Therapeutics has received a grant from the Bill & Melinda Gates Foundation to develop sickle cell disease (SCD) treatments using the CRISPR/Cas9 genome editing technology, the company announced. The approach, ... Read more

November 19, 2020November 19, 2020

ARU-1801 Gene Therapy Granted Orphan Drug Status in EU

Sickle Cell Disease News

The European Medicines Agency (EMA) has granted the designation of orphan drug to ARU-1801, an experimental gene therapy being developed by Aruvant, for the treatment of sickle cell disease (SCD). The decision, based ... Read more

November 17, 2020November 17, 2020

Rifaximin, Oral Antibiotic, Named Orphan Drug for Potential to Ease Pain Crises

Sickle Cell Disease News

The U.S. Food and Drug Administration (FDA) has designated rifaximin, an oral antibiotic, an orphan drug as a potential treatment of pain crises due to sickle cell disease (SCD), according to a press release. A ... Read more

November 12, 2020November 12, 2020

FT-4202, Oral SCD Therapy in Clinical Testing, Named Orphan Drug in EU

Sickle Cell Disease News

FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD) by Forma Therapeutics, has been designated an orphan drug by the European Medicines Agency (EMA). The decision was based on a positive recommendation ... Read more

November 10, 2020November 10, 2020

NIH Funds New Project to Improve SCD Care for Children in Malawi

Sickle Cell Disease News

A team of researchers led by UNC Project-Malawi has been given a one-year, $500,000 grant to build clinical and research infrastructure that will improve diagnosis and treatment for children living ... Read more

November 5, 2020November 5, 2020

CSL889 Designated as Orphan Drug in Europe and US for Treating Sickle Cell Disease

Sickle Cell Disease News

The European Commission and the U.S. Food and Drug Administration have granted orphan drug status to CSL889, an investigational therapy for the treatment of acute vaso-occlusive crises (VOCs) in sickle ... Read more

November 3, 2020November 3, 2020

EU Approves Adakveo to Treat Pain Crises in SCD Patients, 16 and Older

Sickle Cell Disease News

The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent vaso-occlusive crises (VOCs) in people, 16 and older, with sickle cell disease (SCD). The approval covers ... Read more

October 27, 2020October 27, 2020

Phase 1 Trial of Oral FTX-6058 to Promote Fetal Hemoglobin Set to Open

Sickle Cell Disease News

FTX-6058, an investigational oral treatment being developed by Fulcrum Therapeutics for sickle cell disease (SCD) and beta-thalassemia, is entering a Phase 1 clinical trial in healthy volunteers, the company announced. The trial (NCT04586985), not ... Read more

October 22, 2020October 22, 2020

Siklos, Hydroxyurea-based Treatment for Pediatric SCD, Available in 100 mg Scored Tablets

Sickle Cell Disease News

Siklos (hydroxyurea) — indicated for the treatment of children 2 and older with sickle cell disease — is now available in 100 mg scored tablets, in addition to the 1,000 ... Read more

October 20, 2020October 20, 2020

Cyclerion Suspends Development of Olinciguat for Sickle Cell Disease

Sickle Cell Disease News

Cyclerion Therapeutics has decided to suspend the development of olinciguat, its investigational oral therapy for sickle cell disease (SCD), after the treatment failed to show adequate activity in a Phase ... Read more

October 15, 2020October 15, 2020

$7.1M NIH Grant Supports Study of Opioid Alternatives to Manage SCD Pain

Sickle Cell Disease News

The National Institutes of Health (NIH) has awarded a $7.1 million grant to University of Illinois Chicago (UIC) researchers to evaluate the effectiveness of two therapeutic strategies as alternatives to opioids in the management ... Read more

October 13, 2020October 13, 2020

C-Path Launches Consortium to Support Sickle Cell Therapy Development

Sickle Cell Disease News

A new consortium has been launched by the Critical Path Institute (C-Path) to support the development of safe and effective treatments for people with sickle cell disease. Called the Critical ... Read more

October 8, 2020October 8, 2020

Rivipansel Granted FDA’s Rare Pediatric Disease Designation for SCD

Sickle Cell Disease News

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to GlycoMimetics’ investigational therapy rivipansel (GMI-1070) for the treatment of sickle cell disease (SCD). This designation is given ... Read more

October 6, 2020October 6, 2020

Fulcrum’s FTX-6058 Shows Promising Efficacy as SCD Therapy in Disease Models

Sickle Cell Disease News

FTX-6058, an investigational treatment for sickle cell disease (SCD) and beta-thalassemia being developed by Fulcrum Therapeutics, showed efficacy at raising the levels of fetal hemoglobin in cellular and animal models ... Read more

October 1, 2020October 1, 2020

Medications Activating sGC Protein May Have Promise as SCD Therapy, Study Shows

Sickle Cell Disease News

Medications that activate the protein soluble guanylyl cyclase (sGC) may have promise in the treatment of sickle cell disease (SCD), a new study in cells and animal models indicates. The ... Read more