News

Casgevy improved quality of life for severe SCD patients in trial

People with severe sickle cell disease (SCD) have reported significant improvements in their quality of life after receiving the approved gene-editing therapy Casgevy (exagamglogene autotemcel). That’s according to a new analysis of data from patients involved in a clinical trial that had tested the SCD treatment in individuals…

Gene-editing therapy CS-101 used in SCD patient for first time

Correctsequence Therapeutics’ experimental gene-editing therapy CS-101 has been used to treat a person with sickle cell disease (SCD) for the first time. Prior to treatment with CS-101, the patient — a 21-year-old woman from Nigeria — had been experiencing frequent vaso-occlusive crises (VOCs), or painful episodes caused by…

Blood drive, walks planned as part of Sickle Cell Awareness Month

Fundraising walks, a golf tournament, and a virtual blood drive are among activities planned to raise awareness of sickle cell disease (SCD) in September, National Sickle Cell Awareness Month. The campaign aims to raise funding for research into the inherited blood disorder and to support the approximately 100,000 U.S. residents…

Inclacumab fails to reduce pain crises vs. a placebo in SCD trial

Pfizer’s investigational therapy inclacumab failed to outperform a placebo in reducing the rate of vaso-occlusive crises — episodes marked by painful inflammatory responses — in people with sickle cell disease (SCD), according to new data from the Phase 3 THRIVE-131 study. “We recognize this news is disappointing…

Gene-edited cell therapy for severe SCD lands new FDA designation

The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to Beam Therapeutic’s BEAM-101, a one-time, gene-edited cell therapy for severe sickle cell disease (SCD). RMAT status is granted to therapies that have shown potential for treating serious or life-threatening conditions. It provides a…