Clinical Trial

Disease: Sickle Cell Disease, SCD (NCT05456880)

Disease info:

Sickle cell disease is a group of disorders that affects haemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder have atypical haemoglobin molecules called haemoglobin S, which can distort red blood cells into a sickle or crescent shape.

The production of haemoglobin A, which is the principle type of haemoglobin in humans, is governed by 3 genes: HBA1, HBA2, and HBB. Each haemoglobin A molecule consists of two alpha and two beta chains, and mutations in either of the HBA or the HBB genes may result in abnormal haemoglobin molecules with reduced or diminshed function. Sickle cell diseaase arises from a single point mutation in the 6th codon of the beta-globin gene (HBB), which results in a valine instead of a glutamic acid in the haemoglobin beta-chain.

Abnormal haemoglobin ultimately leads to anaemia as well as other symptoms, depending on the exact mutations present. Diseases caused by defective haemoglobin fall into a larger category of diseases known as the "haemoglobinopathies" which also include the thalassemias, a related group of diseases that are characterised by reduced or deficient rather than abnormal haemoglobin. 

Sickle cell disease affects approximately 100,000 individuals in the USA and more than 3 million worldwide
Official title:
A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) to Increase Fetal Hemoglobin (HbF) Production in Patients With Severe Sickle Cell Disease


Medical Information

Phone: 857-327-8641



United States, Massachusetts

Boston Children's Hospital, Boston, Massachusetts, United States, 02215


United States, Minnesota

University of Minnesota, Minneapolis, Minnesota, United States, 55455


United States, Wisconsin

Medical College of Wisconsin, Milwaukee, Wisconsin, United States, 53226

Study start:
Aug. 30, 2022
Gene editing method:
Base editing
Type of edit:
Gene enhancement
Delivery method:
Electroporation - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


BEAM-101 induces single base changes seen in individuals with hereditary persistence of foetal haemoglobin (HPFH).

Using adenine base editors (ABEs), BEAM-101 recreates HPFH by installing base edits in the gamma globin gene promoters, HBG1 and HBG2, that disrupt repressor binding and lead to increased expression of gamma globin.

Last updated: Sep. 22, 2023
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