EurekAlert!

Investigators take first look at a second drug to combat sickle cell disease by turning up fetal hemoglobin

AUGUSTA, Ga. (May 11, 2023) – A class of drugs used for their ability to stop tumor cells from dividing is now under study for their potential to reduce the pain and damage caused by sickle cell ...
Monthly Prescribing Reference

Hemoglobin Elevating Agent Gets Orphan Drug Status for Sickle Cell Disease

AND017 is an investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor. The Food and Drug Administration has granted Orphan Drug designation to KIND Pharmaceutical’s AND017 for the ...
Medical Xpress

Reactivating silenced fetal hemoglobin genes could counter sickle cell–related diseases

Researchers from multiple institutions in China have found a way to use gene editing to reactivate dormant fetal oxygen-transporting proteins in adult blood cells to potentially reverse a wide range ...
FierceBiotech

Beam hits hemoglobin goal in first base editing clinical data, reports death tied to conditioning drug

Beam Therapeutics has presented the first clinical data on its base editing technology. All four sickle cell disease patients in the efficacy cohort had fetal hemoglobin levels above Beam’s target—and ...
GEN

Hypoxia-Fetal Hemoglobin Link May Lead to Novel Sickle Cell Therapies

Scientists at St. Jude Children’s Research Hospital and other colleagues say they have shown how a protein responsible for adapting to low oxygen conditions (hypoxia), causes increased expression of ...
News Medical

Targeting the hemoglobin problem in sickle cell disease using a new drug

A class of drugs used for their ability to stop tumor cells from dividing is now under study for their potential to reduce the pain and damage caused by sickle cell disease, investigators report. The ...
EurekAlert!

A link between hypoxia and fetal hemoglobin provides hope for sickle cell disease

(MEMPHIS, Tenn. – October 12, 2022) Scientists at St. Jude Children’s Research Hospital have shown how a protein responsible for adapting to low oxygen conditions (hypoxia), causes increased ...
Science Daily

Base editing shows potential superiority for curing sickle cell disease

Adenosine base editing restarted fetal hemoglobin expression in cells from patients with sickle cell disease. Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease ...
Business Wire

Long-term Follow-up Data From bluebird’s Gene Therapy Program in Sickle Cell Disease Support Durable, Potentially Curative Benefits Through Stable Production of Anti-Sickling ...

SOMERVILLE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or “bluebird”) today announced new and updated efficacy, safety and health-related quality of life (HRQoL) data ...
Case Western Reserve University

5 questions with... Olabimpe Olayiwola, sickle cell disease researcher

According to the Centers for Disease Control, approximately 100,000 Americans are affected by sickle cell disease (SCD), a group of inherited red blood cell disorders known to cause pain and other ...
The Boston Globe

After decades of neglect, sickle cell disease could soon be transformed with gene-based therapies

It’s one of the great paradoxes of medicine. Sickle cell disease was the first human disorder understood on a molecular level, its underpinnings explained in a landmark 1949 paper by a future two-time ...