Author Archives: Steve Bryson PhD

FDA Grants Fast Track Status to SCD Gene-editing Therapy GPH101

Graphite Bio’s GPH101, an investigational gene-editing therapy that aims to correct the genetic mutation that causes sickle cell disease (SCD) and potentially cure the condition, has been awarded fast track designation by the U.S. Food and Drug Administration (FDA). The fast track process is designed to accelerate the…

Endari Oral Therapy Approved in UAE for SCD, Emmaus Announces

The United Arab Emirates (UAE) Ministry of Health has approved Endari (L-glutamine) for sickle cell disease (SCD), according to Emmaus Life Sciences, the therapy’s developer. During the five-month review process, which was originally expected to take up to one year, the oral therapy was available by early…

Eligible Patients in UK Granted Early Access to Oxbryta

Oxbryta (voxelotor), a daily oral therapy for hemolytic anemia associated with sickle cell disease (SCD), is now available to eligible patients in the U.K. under an early access program. It received a positive scientific opinion by the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) under the Early…

Pandemic Leading to Fewer Hospital Stays, but More Severe Crises

Fewer adults and children with sickle cell disease (SCD) have sought medical care for disease-related events during the COVID-19 pandemic, resulting in lower hospitalization rates, but longer hospital stays, according to data collected from a specialty center in Southern California that cares for a large number of SCD patients.

UK Sickle Cell Gene Therapy Research Awarded $3.1M Grant

Researchers at the University of York in the U.K. have been awarded a $3.1 million (£2.3 million) grant to accelerate research supporting stem cell gene therapy to treat sickle cell disease (SCD). The grant was awarded by the nonprofit Bill & Melinda Gates Foundation to track genetic changes in…