With sickle cell disease, the mutations in a person’s two copies of the HBB gene cause red blood cells to become rigid and crescent-shaped, resembling a sickle, rather than the usual round ...

Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene ...

This protection against malaria has kept the sickle cell gene mutation in the population. Sickle cell trait is found in up to ...

Cyclosporin H boosts a new lentivirus-delivered gene therapy with enhanced anti-sickling properties in blood stem cells from ...

It may be possible to collect nearly three times as many stem cells for sickle cell gene therapy with motixafortide than standard plerixafor.

ST. LOUIS — All morning, nurses had been coming in, taking vitals, offering blankets, pressing buttons to silence alarms. It ...

Beam’s medicine uses a technology known as base editing to activate a gene in stem cells collected from people with sickle cell disease, an inherited blood condition that can cause debilitating pain ...

Blood stem cells develop through different stages to become fully mature red blood cells. This fundamental biologic process ...

This fundamental biologic process is defined by a series of complex metabolic processes, which are often dysregulated in ...

Excitement around the sickle cell treatment market has been palpable. Around the beginning of the year, two new breakthrough gene editing medications — including the first FDA-approved CRISPR ...

Agios expects to report topline results from this 52-week Phase 3 study in late 2025. Sickle cell disease is an inherited, lifelong blood disorder caused by mutations in hemoglobin, the major protein ...

CAMBRIDGE, Mass., Oct. 23, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare ...