The first patient has been enrolled in a Phase 1 clinical trial evaluating the safety and tolerability of Rigel Pharmaceuticals’ fostamatinib — a medication approved in the U.S. for treating an autoimmune condition — in people with sickle cell disease (SCD). The Phase 1 study (NCT05904093) is…

A small proof-of-concept clinical trial evaluating motixafortide for stem cell mobilization — regimens essential for gene therapies — in sickle cell disease (SCD) has dosed its first patient. Stem cell mobilization regimens currently available can cause serious side effects in SCD patients. This Phase 1 study (NCT05618301),…

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…

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 safety and feasibility of using motixafortide for stem cell mobilization in people with sickle cell disease (SCD) will be tested in a Phase 1 clinical trial. Stem cell mobilization is an essential step for gene therapies, but currently used mobilization regimens can cause serious side effects in SCD…

FTX-6058, an investigational oral treatment being developed by Fulcrum Therapeutics for sickle cell disease (SCD) and beta-thalassemia, is entering a Phase 1 clinical trial in healthy volunteers, the company announced. The trial (NCT04586985), not yet enrolling, aims to recruit 88 adults at its single Altasciences Clinical Kansas site. The…

Bluebird bio reported that its investigational gene therapy LentiGlobin showed promising results for severe sickle cell disease (SCD) patients in a Phase 1 clinical trial. Data from the ongoing HGB-206 Phase 1 multicenter study (NCT02140554) were revealed in an oral presentation, “Recent progress in gene therapy for severe…

An upcoming Phase 1 clinical trial was recently awarded $5.74 million to evaluate an innovative blood stem cell transplant procedure for adults with severe sickle cell disease (SCD). The California Institute for Regenerative Medicine (CIRM) granted the funding to City of Hope, where the research will be conducted, and…

Imara, a biotechnology company that is working towards developing new therapies against sickle cell anemia and other blood disorders, announced that the first healthy volunteer has been dosed in a Phase I clinical trial testing the safety and tolerability of their product candidate called IMR-687.