News

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.

Despite improvements in newborn screening for sickle cell disease (SCD) in Brazil, the mortality rate for children and young adults with the disease remains high, according to a recent study that highlights the burden of the disease and the need for improved healthcare for these patients. Researchers found that the…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to EDIT-301, an experimental cell-based gene-editing therapy given as a one-time infusion for the treatment of sickle cell disease (SCD). The designation is given to spur the development of therapies for diseases affecting fewer than 200,000 people…

A new gene-editing approach called prime editing was able to selectively correct the genetic deficit underlying sickle cell disease (SCD) in stem cells collected from patients, a recent study reported. “We were able to directly fix the sickle cell mutation in human hematopoietic stem cells (HSCs) … which we…

Bluebird Bio is seeking approval from the U.S. Food and Drug Administration (FDA) for its gene therapy candidate lovotibeglogene autotemcel — lovo-cel for short — for adults and children, ages 12 and older, with sickle cell disease (SCD). The company has filed a biologics license application (BLA) to…

Children with sickle cell disease (SCD) might be at risk for severe obstructive sleep apnea (OSA) even if they’re a normal weight, according to a study. This contrasts with obesity being a widely known risk factor of OSA in the general population. OSA occurs when the natural relaxation of…