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One-Shot gene fix for blood disorder enters human testing

NCT ID NCT05577312

Disease control Sponsor: Bioray Laboratories Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

39 people

The number who actually took part.

Started

Nov 2022

Expected to finish

Jul 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

3 to 35 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Fully understand and voluntarily sign informed consent. 3-35years old. At least one legal guardian and/or Subjects to sign informed consent. * Clinically diagnosed as β-thalassemia major, phenotypes including β0β0, β+β+, β+β0, βEβ0 genotype. * Subjects with no affection with HIV, TP, HBV, HCV, CMV and EBV. * Subjects body condition eligible for autologous stem cell transplant. Key Exclusion Criteria: * Subjects acceptable for allogeneic hematopoietic stem cell transplantation and have an available fully matched related donor. * Active bacterial, viral, or fungal infection. * Treated with erythropoietin prior 3 months. * Immediate family member with any known hematological tumor. * Subjects with severe psychiatric disorders to be unable to cooperate. * Prior hematopoietic stem cell transplant (HSCT). Other protocol defined Inclusion/Exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

beta thalassemia beta-thalassemia major

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Beta-thalassemia

Contacts and locations

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

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