NCT05577312 is a Phase 1, PHASE2 clinical trial of BRL-101 in Beta-Thalassemia, sponsored by Bioray Laboratories.

Who is sponsoring NCT05577312?

NCT05577312 is sponsored by Bioray Laboratories.

What drugs are being tested in NCT05577312?

Interventions in NCT05577312: BRL-101.

What condition does NCT05577312 study?

NCT05577312 studies Beta-Thalassemia.

Is NCT05577312 still recruiting?

NCT05577312 is currently enrolling by invitation. Last updated 13 January 2026.

Last reviewed 2026-01-13 · How we verify

NCT05577312

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia

ENROLLING BY INVITATION Phase 1, PHASE2 Last updated 13 January 2026

What this trial tests

Phase 1, PHASE2 trial testing BRL-101 in Beta-Thalassemia in 39 participants. Enrolling by invitation.

Timeline

1 November 2022

Primary endpoint
20 August 2026

10 July 2027

Quick facts

Lead sponsor	Bioray Laboratories
Phase	Phase 1, PHASE2
Status	ENROLLING BY INVITATION
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	39
Start date	1 November 2022
Primary completion	20 August 2026
Estimated completion	10 July 2027
Sites	4 locations across China

Drugs / interventions tested

BRL-101 — full drug profile →

Conditions studied

Beta-Thalassemia — all drugs for Beta-Thalassemia →

Sponsor

Bioray Laboratories — full company profile →

Who can join

Adults 3 to 35, any sex, with Beta-Thalassemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in subjects with Transfusion-Dependent β-Thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101)

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments.
Laurent M, Geoffroy M, Pavani G, Guiraud S. · · 2024 · cited 45× · PMID 38786024 · DOI 10.3390/cells13100800
Current trends of clinical trials involving CRISPR/Cas systems.
Zhang S, Wang Y, Mao D, Wang Y, et al · · 2023 · cited 35× · PMID 38020120 · DOI 10.3389/fmed.2023.1292452
CRISPR-Cas System: The Current and Emerging Translational Landscape.
Bhokisham N, Laudermilch E, Traeger LL, Bonilla TD, et al · · 2023 · cited 25× · PMID 37190012 · DOI 10.3390/cells12081103
Revolutionizing <i>in vivo</i> therapy with CRISPR/Cas genome editing: breakthroughs, opportunities and challenges.
Macarrón Palacios A, Korus P, Wilkens BGC, Heshmatpour N, et al · · 2024 · cited 23× · PMID 38362491 · DOI 10.3389/fgeed.2024.1342193
Gene Therapy Approaches for the Treatment of Hemophilia B.
Soroka AB, Feoktistova SG, Mityaeva ON, Volchkov PY. · · 2023 · cited 21× · PMID 37445943 · DOI 10.3390/ijms241310766
Recent advances in therapeutic gene-editing technologies.
Liu D, Cao D, Han R. · · 2025 · cited 17× · PMID 40119516 · DOI 10.1016/j.ymthe.2025.03.026
Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects.
Cetin B, Erendor F, Eksi YE, Sanlioglu AD, et al · · 2025 · cited 15× · PMID 40160040 · DOI 10.1017/erm.2025.10
Advancements in pre-clinical development of gene editing-based therapies to treat inherited retinal diseases.
Chirco KR, Martinez C, Lamba DA. · · 2023 · cited 8× · PMID 37210864 · DOI 10.1016/j.visres.2023.108257

Verify or expand the search:

Other trials of BRL-101

Trials testing the same drug.

NCT06300723 — Clinical Study of BRL-101 in Severe SCD · NA · enrolling by invitation
NCT06287099 — Clinical Study of BRL-101 in the Treatment of Sickle Cell Disease · NA · not yet recruiting

Other recruiting trials for Beta-Thalassemia

Currently open trials in the same condition.

NCT06364774 — ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia · Phase 1, PHASE2 · recruiting
NCT06568926 — Adherence of Beta Thalssemia Patients to Oral Chelation Therapy · NA · recruiting
NCT06539169 — FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases · recruiting
NCT06308159 — An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia · Phase 1, PHASE2 · recruiting
NCT06065189 — Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major · EARLY_PHASE1 · active not recruiting

Other Bioray Laboratories trials

Trials by the same sponsor.

NCT07248176 — Universal CAR-T Cell Therapy for MM · NA · recruiting
NCT07248163 — Universal CAR-T Cell Therapy for NHL · NA · recruiting
NCT06298630 — Long-term Follow-up Study of BRL-101 for TDT · not yet recruiting
NCT06633042 — The Safety and Efficacy of Universal CAR-T Cells Targeting BCMA in the Treatment of Refractory NMOSD · Phase 1 · not yet recruiting
NCT06300723 — Clinical Study of BRL-101 in Severe SCD · NA · enrolling by invitation

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT05577312 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Bioray Laboratories
Last refreshed: 13 January 2026

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05577312.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing