Sickle Cell Disease News

June 21, 2022June 21, 2022

UConn Team Awarded Nearly $3M to Study Bone Loss in Sickle Cell

To mark World Sickle Cell Day, Marja Hurley, a University of Connecticut (UConn) physician-researcher, has been awarded nearly $3 million in research funding to investigate bone loss in people with ... Read more

June 16, 2022June 16, 2022

Exa-cel Continues to Prevent VOCs in Sickle Cell Patients: New Trial Data

Sickle Cell Disease News

Exagamglogene autotemcel (exa-cel), an experimental gene-editing cell therapy, increased fetal hemoglobin levels and prevented vaso-occlusive crises (VOCs) in 31 people with severe sickle cell disease (SCD), according to new data ... Read more

June 14, 2022June 14, 2022

App Will Track Newborns Diagnosed with SCD in 6 African Countries

Sickle Cell Disease News

Novartis is partnering with the American Society of Hematology (ASH) to roll out an app in six sub-Saharan African countries to track babies who receive a diagnosis of sickle cell ... Read more

June 9, 2022June 9, 2022

Supporters Set to Observe World Sickle Cell Day on June 19

Sickle Cell Disease News

From sporting the color red to “shining a light” on sickle cell, supporters are set to mark World Sickle Cell Day June 19 to call attention to the inherited blood ... Read more

June 7, 2022June 7, 2022

2 GBT Therapies Win FDA Orphan Drug, Rare Pediatric Designations

Sickle Cell Disease News

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to inclacumab and GBT601, two experimental therapies for sickle cell disease (SCD) being ... Read more

June 2, 2022June 2, 2022

UCSF’s Stad Center Will Cater to Pediatric Pain Management

Sickle Cell Disease News

The UCSF Benioff Children’s Hospitals in California has announced the addition of the Stad Center for Pediatric Pain, Palliative & Integrative Medicine, which is tailored for treating pain in children ... Read more

May 31, 2022May 31, 2022

2 Proteins That Silence Fetal Hemoglobin May Be Therapy Target

Sickle Cell Disease News

Interfering with two newly identified proteins — NFIA and NFIX — involved both directly and indirectly in silencing fetal hemoglobin in red blood cells, could be a new therapeutic approach ... Read more

May 26, 2022May 26, 2022

Hydroxyurea Use During Pregnancy Raises Risk of Miscarriage, Stillbirths

Sickle Cell Disease News

Treatment with hydroxyurea at the time of conception may be safe, but its use by women with sickle cell disease (SCD) during pregnancy was linked to twofold higher risks of ... Read more

May 24, 2022May 24, 2022

Phase 1 Trial Supporting GBT601 as Next-gen Therapy to Oxbryta

Sickle Cell Disease News

Multiple daily doses of GBT601, Global Blood Therapeutics (GBT)’s experimental oral therapy for sickle cell disease (SCD), were generally well tolerated and showed promising pharmacological and efficacy signals in six patients in ... Read more

May 19, 2022May 20, 2022

Lifetime Medical Costs for SCD Total $1.7M, Partly Out of Pocket: Study

Sickle Cell Disease News

The total lifetime medical costs of living with sickle cell disease (SCD) total about $1.6 million for women and $1.7 million for men younger than 65 who hold private health ... Read more

May 17, 2022May 17, 2022

ARU-1801 Gene Therapy Safely Limiting VOCs in 5 Treated Patients

Sickle Cell Disease News

ARU-1801, Aruvant Sciences’ experimental gene therapy for sickle cell disease (SCD), led to the production of fetal hemoglobin in red blood cells and significantly lowered the number of vaso-occlusive crises ... Read more

May 12, 2022May 12, 2022

Phase 3 Trials of CTX001, Gene-editing Cell Therapy, Set for Children

Sickle Cell Disease News

CRISPR Therapeutics and Vertex have launched two Phase 3 trials to assess the safety and effectiveness of CTX001, an experimental gene-editing cell therapy, one in children with sickle cell disease (SCD) and ... Read more

May 10, 2022May 10, 2022

Gazelle Upgrade Will Improve Measuring Fetal Hemoglobin Levels

Sickle Cell Disease News

Hemex Health’s portable point-of-care device, Gazelle, is being enhanced to more precisely monitor fetal hemoglobin levels. According to Hemex, this could be useful for monitoring people with sickle cell disease ... Read more

May 5, 2022May 5, 2022

FDA Grants Fast Track Status to SCD Gene-editing Therapy GPH101

Sickle Cell Disease News

Graphite Bio’s GPH101, an investigational gene-editing therapy that aims to correct the genetic mutation that causes sickle cell disease (SCD) and potentially cure the condition, has been awarded fast track ... Read more

May 5, 2022May 5, 2022

Organizations Rally to Help Ukrainian Rare Disease Patients

Sickle Cell Disease News

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ... Read more