News

The U.S. Food and Drug Administration (FDA) has cleared Beam Therapeutics to start clinical testing of BEAM-101, the company’s investigational gene editing cell therapy for sickle cell disease (SCD). “BEAM-101 has the potential to offer a one-time treatment for patients with sickle cell disease, and this clearance enables…

Enrollment is underway for a Phase 1/2 trial evaluating the safety and efficacy of EDIT-301, an experimental gene editing cell therapy for people with sickle cell disease (SCD). The trial, called RUBY (NCT04853576), had been cleared for launch by the U.S. Food and Drug Administration earlier this…

Oxbryta (voxelotor), Global Blood Therapeutics’ first-in-class oral therapy for sickle cell disease (SCD), has won the 2021 Prix Galien USA Award for best biotechnology product. The award is among the health industry’s most prestigious honors, and recognizes an outstanding product that has improved patient well-being and impacted human…

In support of its work to develop a rapid test that can be used to diagnose sickle cell disease (SCD), a team of undergraduate researchers at Lehigh University has received a $15,000 award as part of the National Institutes of Health’s Healthcare Technologies for Low-Resource Settings Prize. “I was…

A point-of-care, low-cost device called Gazelle — that’s both portable and easy to use — can accurately diagnose sickle cell disease (SCD) in less than 15 minutes, according to a study done in India. As noted by researchers, this device has the potential to be adopted as a…

Emmaus Life Sciences is seeking the approval of Endari (L-glutamine), an oral treatment for sickle cell disease (SCD), in the the United Arab Emirates (UAE). “We look forward to working with the U.A.E Ministry of Health to bring this important medication to our patient population in the…

Scientists Kenneth Ataga, MD, and Santosh Saraf, MD, have received a $3.2 million grant to continue investigating the potential of machine learning as a method to help identify sickle cell disease (SCD) patients at high risk of developing progressive kidney disease. The grant awarded by the…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

GPH101, Graphite Bio’s investigational gene editing therapy, is able to correct the disease-causing mutation in blood stem cells of people with sickle cell disease (SCD), new preclinical data shows. Graphite also announced it remains on track to start enrolling participants in a Phase 1/2 trial testing the gene…

Health Canada has expanded its approval of Chiesi Global Rare Diseases’ iron-binding oral treatment Ferriprox (deferiprone) to patients who have blood transfusion-induced iron overload due to sickle cell disease (SCD) or other anemias. The decision comes about five months after the U.S. Food and Drug Administration (FDA) approved…