The first participant has been enrolled in the Phase 1/2 clinical trial CEDAR evaluating Graphite Bio’s investigational gene editing therapy GPH101, which is designed to directly correct the genetic mutation that causes sickle cell disease (SCD). “We are thrilled that our first patient is now enrolled in our CEDAR…
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…
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…
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…
The National Institutes of Health (NIH) has awarded a $10.5-million grant to support several research projects seeking to improve care for people with sickle cell disease (SCD). Research will focus on children and young adults, and on combating racism and other barriers to proper healthcare. SCD disproportionately affects Black…
The Sickle Cell Consortium, a U.S.-based nonprofit, is hosting a virtual event in late August that will focus on facilitating conversations among people with sickle cell disease (SCD) and their caregivers. “We’re bringing these patients together to talk about what they’re experiencing, strategies for navigating the realities brought…
Endari (L-glutamine), an approved oral treatment for sickle cell disease (SCD), has now been added to the Preferred Drug List in Texas. The move will make Endari more accessible to people in the state who are insured through Medicaid, the government program that provides health insurance to low-income…
Emmaus Life Sciences has submitted an application seeking the approval of its oral medication Endari (L-glutamine) to treat sickle cell disease (SCD) in Kuwait. The company announced that the Kuwait Drug and Food Control administration, which is responsible for registering pharmaceutical products in the country, has accepted…
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