News

The U.S. Food and Drug Administration approved the start of a first clinical trial of CRISPR_SCD001, the first non-viral and CRISPR/Cas9-based gene editing therapy for sickle cell disease (SCD). Both the therapy and the upcoming Phase 1/2 trial — planned to start this summer — are the result of a…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

Imara is opening higher dose arms in two Phase 2b trials assessing the safety and efficacy of IMR-687, its potential therapy for sickle cell disease (SCD) and beta-thalassemia, in patients with these inherited blood disorders, the company announced. The decision, which applies to the Ardent (NCT04474314) and…

Graphite Bio has raised $150 million in Series B funding to expand and advance the clinical development of its pipeline of investigational gene-editing therapies — including GPH101, a potentially curative treatment for sickle cell disease (SCD). In addition…

Adding Cell Source’s special class of immune cell, Veto T-cells, to blood stem cells from an unrelated donor led to a successful transplant and to the production of normal red blood cells in a mouse model of sickle cell disease (SCD), the company announced. The preclinical Cell Source-sponsored…

Global Blood Therapeutics (GBT) has opened its grant program — the Access to Excellent Care for Sickle Cell Patients (ACCEL), now in its third year — to proposals supporting programs aiming to improve access to high-quality healthcare for people with sickle cell disease (SCD). GBT will donate up…

Registration is open for the 8th annual “Walk with the Stars,” one of the largest fundraisers organized by the Sickle Cell Disease Association of America (SCDAA) that supports the development of better treatments and a cure for sickle cell disease (SCD). All proceeds from the July 10 event…

LentiGlobin, Bluebird Bio’s investigational gene therapy for sickle cell disease (SCD), is unlikely to be the cause of a blood cancer that arose in one of the patients who received the therapy in an early clinical trial, according to new analyses. In February, Bluebird paused two clinical…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

The opioid crisis, along with the stigma of suspected drug use, and racial bias all contribute to make treatment access more difficult for people with sickle cell disease (SCD). That was the take-home message of the…