News

CTX001, an experimental gene-editing cell therapy, safely and effectively increased the levels of fetal hemoglobin and prevented vaso-occlusive crises (VOCs) in three people with severe sickle cell disease (SCD), according to updated data from the CLIMB-SCD-121 Phase 1/2 trial. CTX001 is being jointly developed by CRISPR…

Emmaus Life Sciences has launched a program that will allow eligible sickle cell disease patients in the U.S. to access its Endari (L-glutamine) medication at low cost or free of charge. Patients who currently lack insurance coverage or financial means to afford treatment may reach out to Endari’s…

Treatment with Oxbryta (voxelotor) may be a feasible alternative to blood transfusions for sickle cell disease (SCD) patients who are experiencing respiratory distress and pneumonia related to COVID-19, a case report suggests. These findings are particularly important during the ongoing pandemic in the U.S., in which rising blood…

Starting treatment with rivipansel (GMI-1070) shortly after vaso-occlusive crisis (VOC) onset significantly shortened hospital stays and the time to opioid discontinuation in children and adults with sickle cell disease (SCD), new analyses of the RESET clinical trial show. The data further support “the potential benefits of…

Global Blood Therapeutics (GBT) has launched an early access program for voxelotor in Europe and other regions outside the U.S. to allow people with hemolytic anemia associated with sickle cell disease (SCD) to have access to treatment. The early access program will be open to those…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

Hemex Health was awarded third place and $100,000 in a National Institutes of Health (NIH) competition for its collaborative noninvasive diagnostics system for disorders such as sickle cell disease (SCD). The aim of the contest, called the NIH Technology Accelerator Challenge, was to spark development of…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

Intellia Therapeutics has received a grant from the Bill & Melinda Gates Foundation to develop sickle cell disease (SCD) treatments using the CRISPR/Cas9 genome editing technology, the company announced. The approach, to be refined within living organisms (in vivo studies), aims to deliver a functional copy of…

The European Medicines Agency (EMA) has granted the designation of orphan drug to ARU-1801, an experimental gene therapy being developed by Aruvant, for the treatment of sickle cell disease (SCD). The decision, based on a positive recommendation by the EMA’s Committee for Medicinal Products for Human Use,…