News

The U.S. Food and Drug Administration (FDA) is reviewing an application that seeks the approval of exagamglogene autotemcel, a CRISPR-based gene-editing therapy known as exa-cel, to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia. The rolling biologics license application was completed by Vertex Pharmaceuticals and CRISPR…

A protein called NLRP12 appears to act as a switch that turns on inflammation and tissue damage when it senses free heme, which is released from broken red blood cells during an infection and in other diseases like sickle cell disease (SCD). This protein could therefore be a potential…

The Committee for Medicinal Products for Human Use (CHMP) is recommending the conditional approval of Adakveo (crizanlizumab) for sickle cell disease (SCD) be revoked in Europe. According to the therapy’s developer Novartis, a final decision from the European Commission is expected in about two months. In the…

The 5th annual Kier’s Hope 5K Run and Fun Walk — a fundraiser launched by comedian Kier “Junior” Spates to support people with sickle cell disease (SCD) — is set to go this Saturday, June 3, in Dallas. It’s also Kier’s Hope Day in the City of Dallas.

People in Ghana with sickle cell disease (SCD) use prevention, monitoring, diagnosis, and treatment to self-manage their condition, but how this is done differs between adults and children, a study finds. People with SCD spoke more about doing things to stay healthy and treat problems that come up, rather…

Hillhurst Biopharmaceuticals announced that the final healthy volunteer has completed the study protocol in a Phase 1 clinical trial testing HBI-002, an oral carbon monoxide treatment being developed for sickle cell disease (SCD). Data are now being analyzed, and Hillhurst expects to announce top-line results before the end…

The Assistance Fund (TAF) has launched a new financial assistance program to cover out-of-pocket medical expenses for people with sickle cell disease (SCD). The program will cover copayments, health insurance premiums, costs related to ground travel needed to get treatment, and genetic testing, for people with SCD who…

Lobe Sciences has partnered with Pentec Health to distribute Altemia, an emulsified formulation of docosahexaenoic acid (DHA), a type of omega-3 fat, for sickle cell disease (SCD) patients in the U.S. and its territories. As part of the agreement, Pentec, which provides clinical nutrition and specialty care…

The investigational oral treatment FTX-6058 effectively raised the level of fetal hemoglobin in a sickle cell disease (SCD) patient to 24.9% in a clinical trial that’s now been placed on hold by regulators. According to the therapy’s developer, Fulcrum Therapeutics, these initial data had been obtained but not…

A Phase 1/2 clinical trial testing Beam Therapeutics’ gene-editing therapy BEAM-101 is continuing to enroll young adults with sickle cell disease (SCD). The study, called BEACON (NCT05456880), is active at five sites, with a waiting list for enrollment, according to Beam, which is sponsoring the trial.