The European Commission has granted orphan drug designation to IMR-687, Imara’s potential new therapy for…
Articles by Joana Carvalho, PhD
The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation to EDIT-301, a potential…
A Phase 2b clinical trial that is investigating the safety and effectiveness of IMR-687 in people with sickle…
An umbilical cord blood transplant, coupled with a reduced intensity conditioning (RIC) regimen, can safely and effectively treat children with…
Mitapivat (AG-348) safely increased the amount of hemoglobin and changed the levels of biomarkers of red blood damage…
The first patient with severe sickle cell disease (SCD) who received a single infusion of CTX001, an experimental…
The Sickle Cell Society of the U.K. has created a survey to gather feedback from people with sickle cell disease…
IMR-687 safely increases the levels of fetal hemoglobin and the number of fetal hemoglobin-producing cells in people with…
Agios Pharmaceuticals announced that, despite the current COVID-19 outbreak, it remains on track to decide by mid-year next steps for mitapivat…
The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to CTX001, an…