News

2 Common SCD Mouse Models Not Suited for Gene Editing Studies

Two mouse models commonly used to study sickle cell disease (SCD) are not suitable for testing certain gene editing treatment approaches, including those for fetal hemoglobin production, because of their specific genetic makeup, a study reported. “This work provides a reminder for scientists to carefully consider the genetics of…

Alliance Launches to Advocate for US Policies to Improve Patient Care

A public policy alliance has launched to advocate for federal policies that address the “crisis of care” facing the approximately 100,000 sickle cell disease (SCD) patients in the U.S. The ad-hoc alliance, called the Sickle Cell Disease Partnership, comprises more than a dozen groups representing patient advocates, healthcare…

Survey Reveals Shortfalls in SCD Care, Economic Burden

Lowering the risk of long-term complications, including organ damage and hemolytic anemia — driven by the destruction of red blood cells — are common goals for sickle cell disease (SCD) patients, caregivers, and healthcare professionals, according to data from Global Blood Therapeutics’ international SHAPE survey. Survey results also…

GBT Launches Phase 2/3 Study for Next-gen Version of Oxbryta

Global Blood Therapeutics (GBT) has begun a Phase 2/3 trial to study the safety, tolerability, effectiveness, and pharmacological properties of its experimental oral therapy GBT601 in people with sickle cell disease (SCD). Initial data from the study (NCT05431088) are expected before the end of the year, according…

Precision, Novartis Partner on New Gene Therapy for Blood Disorders

Precision BioSciences and Novartis are teaming up to develop and commercialize a custom gene therapy as a potential treatment approach for certain inherited blood disorders, including beta thalassemia and sickle cell disease (SCD). Under their new exclusive license and collaboration agreement, the companies will combine Precision’s proprietary ARCUS genome…

UConn Team Awarded Nearly $3M to Study Bone Loss in Sickle Cell

To mark World Sickle Cell Day, Marja Hurley, a University of Connecticut (UConn) physician-researcher, has been awarded nearly $3 million in research funding to investigate bone loss in people with sickle cell disease (SCD). A four-year $2.21 million endowment comes from the National Institutes of Health (NIH) to Hurley…

Exa-cel Continues to Prevent VOCs in Sickle Cell Patients: New Trial Data

Exagamglogene autotemcel (exa-cel), an experimental gene-editing cell therapy, increased fetal hemoglobin levels and prevented vaso-occlusive crises (VOCs) in 31 people with severe sickle cell disease (SCD), according to new data from the CLIMB-SCD-121 trial. Vertex Pharmaceuticals and CRISPR Therapeutics, which are jointly developing the therapy, recently launched…