One-Shot gene fix for blood disorder enters human testing
NCT ID NCT05577312
First seen Jun 27, 2026 · Last updated Jul 22, 2026 · Updated 2 times
Summary
This early-stage trial tests a one-time gene therapy called BRL-101 for people with transfusion-dependent beta-thalassemia, a severe blood disorder. The treatment uses the patient's own stem cells, edited with CRISPR to boost healthy hemoglobin production. The goal is to see if a single infusion can safely reduce or eliminate the need for regular blood transfusions. Only 39 participants aged 3–35 are being enrolled.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- BRL-101 (gene-edited stem cells)
- What this could lead to
- If successful, this could free patients from lifelong blood transfusions by enabling their body to produce healthy red blood cells.
- What could go wrong
- This is an early-phase trial with only 39 participants, so results may not apply to everyone. Gene editing carries risks like failed engraftment or long-term side effects that are still unknown.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for BETA-THALASSEMIA are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300000, China
-
Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510510, China
-
No. 923 Hospital of Joint Support Force of Chinese People 's Liberation Army Hospital
Nanning, Guangxi, 530021, China
-
Shenzhen Children 's Hospital
Shenzhen, Guangdong, 510006, China
-
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen Universit
Guanzhou, Guangdong, 510006, China
-
The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, 530021, China
-
Xiangya Hospital of Central South University
Changsha, Hunan, 510510, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Gene therapy aims to free patients from lifelong blood transfusions
- How does a blood disorder drug perform in everyday practice?
- Newborn screening study aims to catch rare diseases at birth
- Talking therapy could help thalassemia patients take their meds
- Drug cocktail may cut transfusions for kids with thalassemia
- Scientists track lifespan of transfused blood in sickle cell kids