Drugs / CS-101
Trials 6
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 03 trials | ||||||
| Phase 0 | NCT06328764 | Mar 2024 → Jul 2026 overdue | beta thalassemia | CorrectSequence Therapeutics Co., Ltd | Enrolling by invitation | No outcome recorded |
| Phase 0 | NCT06065189 | Nov 2023 → Aug 2025 | beta-thalassemia major | Children's Hospital of Fudan University | Completed | No outcome recorded |
| Phase 0 | NCT06024876 | Aug 2023 → Jul 2025 | beta thalassemia | CorrectSequence Therapeutics Co., Ltd | Completed | No outcome recorded |
| Phase 11 trial · 1 met primary | ||||||
| Phase 1 | NCT06291961 | Apr 2024 → Nov 2025 | beta-thalassemia major | CorrectSequence Therapeutics Co., Ltd | Completed | Met primary |
| Phase 21 trial | ||||||
| Phase 2 | NCT07489196 | Apr 2026 → Jan 2028 expected | beta-thalassemia major | CorrectSequence Therapeutics Co., Ltd | Not yet recruiting | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT06479616 | Jun 2024 → Dec 2026 expected | beta thalassemia | Children's Hospital of Fudan University | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 14
| Date | Issuer | Release |
|---|---|---|
| 2026-09-07 | CorrectSequence Therapeutics Co., Ltd | Results Cell Stem Cell: tBE-mediated Base Editing Therapy Achieves Durable Clinical Remission in Sickle Cell Disease and β-Thalassemia Across Different Genetic Backgrounds correctsequence.com ↗
All achieved rapid hematopoietic reconstitution, sustained high-level pan-cellular HbF expression, complete transfusion independence or freedom from vaso-occlusive crises (VOCs), with no detectable off-target edits, or product-related adverse events. |
| 2026-07-24 | CorrectSequence Therapeutics Co., Ltd | Results ASH Clinical News | Base Editing Contributes to Meaningful HbF Production in Beta Thalassemia correctsequence.com ↗
the investigators report that an infusion of autologous CD34+ cells modified using a tBE at clinical scale (CS-101) can lead to rapid and sustained increases in both total hemoglobin and fetal hemoglobin (HbF) levels in patients with beta thalassemia, as well as early and enduring transfusion independence. |
| 2026-04-24 | CorrectSequence Therapeutics Co., Ltd | Results China Daily | Chinese Researchers Debut Breakthrough DNA Repair Tool for Genetic Disorders correctsequence.com ↗
All five patients in the trial received CS-101, a treatment developed by Chinese researchers, and achieved fast hematopoietic reconstruction. |
| 2026-04-09 | CorrectSequence Therapeutics Co., Ltd | Results Xinhua Net | Chinese Scientists Report First Clinical Success Using Base Editing to Treat Severe Blood Disorder correctsequence.com ↗
The study, published Wednesday in the world-class science journal Nature, reveals that five patients with transfusion-dependent beta-thalassemia were able to discontinue their grueling monthly blood transfusions after receiving a one-time infusion of an experimental therapy called CS-101. |
| 2026-04-09 | CorrectSequence Therapeutics Co., Ltd | Results Landmark Trial Published in Nature: China's Novel Base-editing Therapy Brings Hope of Cure for Thalassemia Patients correctsequence.com ↗
Jointly conducted by CorrectSequence Therapeutics, the First Affiliated Hospital of Guangxi Medical University, ShanghaiTech University, Fudan University, and Shanghai Clinical Research and Trial Center, the early-stage clinical trial of the base-editing drug CS-101 injection reports that all treated transfusion-dependent patients rapidly achieved transfusion independence and regained healthy hematopoietic function. |
| 2025-08-26 | CorrectSequence Therapeutics Co., Ltd | Results High-Precision Base Editing Clinical Treatment for Sickle Cell Disease — CorrectSequence Therapeutics' CS-101 Achieves Clinical Cure correctsequence.com ↗
The patient demonstrated a significant and sustained increase in fetal hemoglobin (HbF) levels, accompanied by a marked reduction in sickle hemoglobin (HbS). |
| 2025-05-23 | CorrectSequence Therapeutics Co., Ltd | Results China Daily | Chinese treatment cures Pakistani girl correctsequence.com ↗
After undergoing gene-editing therapy, her dependency on blood transfusions ended and she has returned to living a normal life, doctors said. |
| 2025-05-21 | CorrectSequence Therapeutics Co., Ltd | Results SHINE | Innovative treatment developed in China saves 4-year-old correctsequence.com ↗
The hospital has cured four children so far with CS-101, the other three were Chinese. |
| 2025-03-19 | CorrectSequence Therapeutics Co., Ltd | Results CorrectSequence Therapeutics’ CS-101 Successfully Treats Malaysian β-Thalassemia Patient correctsequence.com ↗
A Malaysian β-thalassemia patient who received base editing therapy (Correctseq’s CS-101) in China has been transfusion-free and returned to normal life. |
| 2024-12-10 | CorrectSequence Therapeutics Co., Ltd | Results Locally-Developed Gene Editing Technology Cures Boy with Serious Blood Disorder correctsequence.com ↗
The boy no longer needed blood transfusions just two weeks after receiving treatment. |
| 2024-07-22 | CorrectSequence Therapeutics Co., Ltd | Results The First Clinical Gene Editing Therapy to Treat An Overseas Patient in China correctsequence.com ↗
The patient has achieved a sustained transfusion-free status for over two months, with the hemoglobin level stabilized at above 120 g/L. |
| 2024-07-19 | CorrectSequence Therapeutics Co., Ltd | Results CS-101 Clinical Result Included in EHA2024 Scientific Congress Report correctsequence.com ↗
According to the report, as the world's first base-editing therapy for hemoglobinopathies, Correctseq’s CS-101 has successfully cured several patients with β-thalassemia. |
| 2024-04-02 | CorrectSequence Therapeutics Co., Ltd | Regulatory CorrectSequence Therapeutics’ Base Editing Drug "CS-101 Injection" Obtains IND Approval from the NMPA correctsequence.com ↗ |
| 2024-01-08 | CorrectSequence Therapeutics Co., Ltd | Results The World’s First Clinical Base Editing Therapy for Hemoglobinopathy correctsequence.com ↗
Eight weeks after CS-101 treatment, the patient’s fetal hemoglobin (HbF) level has increased to ~95 g/L, accounting for ~81% of total hemoglobin. |
All press releases naming this drug 15 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 7 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | CS-101 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06024876 ↗ |
| Action | Restore | “which can re-induce the production of γ-globin chain” NCT06024876 ↗ |
| Modality | Cell therapy | “CS-101 is an autologous CD34+ cell suspension, edited by ex vivo base editing technology” NCT06024876 ↗ |
| Target | HBG1 | “The aim was to target the binding motif of the transcription repressor BCL11A in the HBG1 and HBG2 promoters7 to reactivate fetal haemoglobin (HbF) production.” PMID 41951736 ↗ Apr 2026 |
| Target | HBG2 | “The aim was to target the binding motif of the transcription repressor BCL11A in the HBG1 and HBG2 promoters7 to reactivate fetal haemoglobin (HbF) production.” PMID 41951736 ↗ Apr 2026 |