Sickle Cell Disease News

January 13, 2022January 13, 2022

Trial of Cell Therapy to Raise HbF Levels Showing Potential, But Sanofi Leaving

Sickle Cell Disease News

Sangamo Therapeutics is assuming all rights to SAR445136, an investigational gene-edited cell therapy co-developed with Sanofi and now in a Phase 1/2 trial in adults with severe sickle cell disease (SCD). Early trial ... Read more

January 11, 2022January 11, 2022

New Form of Oxbryta Suitable for Children Now Available in US

Sickle Cell Disease News

A new formulation of Oxbryta (voxelotor) that is suitable for treating children as young as 4 is now available in the U.S. at specialty pharmacies. Late last year, the U.S. ... Read more

January 6, 2022January 6, 2022

UK Sickle Cell Gene Therapy Research Awarded $3.1M Grant

Sickle Cell Disease News

Researchers at the University of York in the U.K. have been awarded a $3.1 million (£2.3 million) grant to accelerate research supporting stem cell gene therapy to treat sickle cell ... Read more

January 4, 2022January 4, 2022

Agios Launches Phase 2/3 Trial, RISE UP, for Mitapivat

Sickle Cell Disease News

Agios Pharmaceuticals has launched a Phase 2/3 trial called RISE UP to test its investigational therapy mitapivat (AG-348) in people with sickle cell disease (SCD). The trial (NCT05031780), which is ... Read more

December 30, 2021December 30, 2021

Prepare to Light Up Buildings for Rare Disease Day 2022

COVID-19 News, Sickle Cell Disease News

The National Organization for Rare Disorders (NORD) asks Americans to plan ahead to participate in the Light Up for Rare campaign to raise awareness of rare diseases. NORD is the ... Read more

December 28, 2021December 28, 2021

Q&A With New CEO of the SCDAA, Regina Hartfield

COVID-19 News, Sickle Cell Disease News

Regina Hartfield is set to become the president and CEO of the Sickle Cell Disease Association of America (SCDAA) on Jan. 14. She brings with her experience ranging from project ... Read more

December 23, 2021December 23, 2021

Lovo-cel Gene Therapy Program Suspended for Adolescents With SCD

Sickle Cell Disease News

A clinical program for lovotibeglogene autotemcel (lovo-cel), an investigational gene therapy for sickle cell disease (SCD), has been suspended for patients under 18 by the U.S. Food and Drug Administration ... Read more

December 21, 2021December 21, 2021

FDA Extends Use of Oxbryta to Children Starting at Age 4

Sickle Cell Disease News

The U.S. Food and Drug Administration (FDA) has agreed to expand the use of Oxbryta (voxelotor) — the first approved therapy targeting the underlying cause of sickle cell disease (SCD) ... Read more

December 16, 2021December 16, 2021

Higher FTX-6058 Doses Raise Hemoglobin in Healthy Adults in Trial

Sickle Cell Disease News

Higher doses of FTX-6058, an investigational oral treatment for sickle cell disease (SCD), effectively raised hemoglobin messenger RNA (mRNA) levels above those found at lower doses in healthy volunteers participating ... Read more

December 14, 2021December 14, 2021

Phase 1 Trial of TAK-755, Potential VOC-easing Therapy, Enrolling in US

Sickle Cell Disease News

A Phase 1 trial evaluating the safety and pharmacological properties of TAK-755 — Takeda’s experimental therapy for painful vaso-occlusive crises (VOCs) — in adults with sickle cell disease (SCD) is actively ... Read more

December 14, 2021December 14, 2021

Pain Crises New Main Goal in Ongoing Ardent Trial of IMR-687

Sickle Cell Disease News

Imara has announced that it will change the main goal of its ongoing Phase 2b Ardent trial, which is assessing the safety and efficacy of IMR-687 for the treatment of ... Read more

December 9, 2021December 9, 2021

IMR-687 Continuing to Lower VOCs and Hospitalizations in SCD Patients

Sickle Cell Disease News

IMR-687 (tovinontrine), an experimental oral therapy for sickle cell disease (SCD), reduced the rate of painful vaso-occlusive crisis (VOC), lowered hospitalization due to VOCs, and extended the time to a ... Read more

December 7, 2021December 7, 2021

RedMoon Project Hopes to Reduce Care Disparities for Black Community

Sickle Cell Disease News

Prompted by the disparities in care between children and adults with sickle cell disease (SCD) and inspired to raise awareness about rare diseases, Cory Lewis has made it his personal ... Read more

December 2, 2021December 2, 2021

Potential SCD Therapies by GBT Safe in Healthy People, Trials Show

Sickle Cell Disease News

GBT601 and inclacumab, experimental therapies from Global Blood Therapeutics (GBT) for sickle cell disease (SCD) and associated vaso-occlusive crises (VOCs), were generally safe and showed signs of biological activity in ... Read more

November 30, 2021November 30, 2021

Oral Oxbryta Safe, Effective Over Long Term and in Real World

Sickle Cell Disease News

Long-term treatment with Oxbryta (voxelotor) safely and effectively reduced anemia and red blood cell destruction (hemolysis), while keeping a low rate of painful vaso-occlusive crises (VOCs), in adolescents and adults ... Read more