News

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on FTX-6058, an investigational oral treatment for sickle cell disease (SCD) that’s being developed by Fulcrum Therapeutics. With the hold lifted, Fulcrum is planning to resume enrolling patients in a Phase 1b clinical trial…

The U.S. Food and Drug Administration (FDA) has cleared for use Trinity Biotech’s Premier Resolution System, an automated analyzer that can quickly and accurately detect and quantify hemoglobin variants. Hemoglobin is the protein that red blood cells use to carry oxygen through the bloodstream. Several hemoglobin variants identified…

A new, interactive application touted as the first of its kind can help people with sickle cell disease (SCD) learn about and communicate with their care team about their pain attacks. Called Pinpoint, the app is the product of a partnership between healthcare purchased services provider HPC…

Editas Medicine has expanded its partnership with Azzur Cleanrooms on Demand (COD) for the manufacturing of EDIT-301, its experimental cell-based gene-editing therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia. If approved, the scaling up of EDIT-301 needs to follow current good manufacturing practice (cGMP) standards — set…

Researchers have developed a new way of delivering gene-editing therapies for people with blood disorders such as sickle cell disease (SCD), which could improve treatment safety and lower costs if eventually adopted in the clinic. Instead of a standard method that requires a round of chemotherapy before externally-edited cells…

The European Commission has decided to revoke its conditional approval of Adakveo (crizanlizumab) for sickle cell disease (SCD) — meaning its developer, Novartis, will have to remove the antibody-based therapy from the market in all 27 countries that are part of the European Union. The decision came after…

A gene editing technology called adenosine base editing (ABE), wherein a single DNA building block is changed to increase fetal hemoglobin, may be more useful for treating sickle cell disease (SCD) than other gene editing techniques, a study suggests. “We showed that not all genetic approaches are equal. Base…

Pfizer has decided to end a treatment discovery program with Syros Pharmaceuticals that it inherited after acquiring Global Blood Therapeutics (GBT) last year, leaving Syros to seek another partner for the program, which targets two blood disorders, including sickle cell disease (SCD). The agreement termination was announced…

Genetic medicines company Scribe Therapeutics has expanded its collaboration with Sanofi to advance the development of a new wave of in vivo treatments for sickle cell disease (SCD) and other disorders. The collaboration leverages Scribe’s proprietary CRISPR X-Editing technologies and Sanofi’s targeted non-viral delivery technologies for the…

Researchers at Rice University in Texas have been awarded $2.6 million to further investigate questions surrounding the safety and efficacy of gene editing as a potentially curative treatment for sickle cell disease (SCD). The four-year grant from the National Institutes of Health (NIH) was awarded to Gang Bao, PhD,…