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Susan Mou: Presenting Long-Term Clinical Outcomes in SCD and TDT
Susan Mou/LinkedIn
Oct 4, 2026, 01:03

Susan Mou: Presenting Long-Term Clinical Outcomes in SCD and TDT

Susan Mou, Chief Executive Officer at CorrectSequence Therapeutics, Expert at IFPMA, shared CorrectSequence Therapeutics‘s post on LinkedIn:

“We proudly present long-term clinical readouts in sickle cell disease (SCD) and patients from different regions of the world with TDT.”

CorrectSequence Therapeutics shared a post on LinkedIn about a recent article by et al., published in Cell Stem Cell, adding:

“Cell Stem Cell: tBE-mediated Therapy Achieves Durable Clinical Remission in Sickle Cell Disease and β-Thalassemia Across Different Genetic Backgrounds

Following 100% transfusion independence in Chinese TDT patients, new study confirms tBE is equally safe and effective for African SCD patients and TDT patients from South or Southeast Asia.

Susan Mou

On September 7, 2026, Shanghai, Cell Stem Cell published online clinical research from CorrectSequence Therapeutics (Correctseq) in collaboration with multiple institutions in a paper titled ‘Clinical base editing for β-hemoglobinopathies across different genetic backgrounds’, demonstrating that CS-101/CS-206—base-editing therapy developed with the transformer Base Editor (tBE)—achieved consistent efficacy and safety in β-hemoglobinopathy patients of diverse genetic origins.

This follows the team’s prior clinical report on five Chinese transfusion-dependent β-thalassemia (TDT) patients treated with CS-101, all achieving transfusion independence (Lai et al., Nature, 2026).

The new study extends treatment to four additional patients from Nigeria, Laos, Malaysia, and Pakistan—one with sickle cell disease (SCD) and three with TDT.

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.

Susan Mou: Presenting Long-Term Clinical Outcomes in SCD and TDT

Figure 1. As of August 2026, the SCD patient had sustained freedom from VOCs for 18 months after receiving Correctseq’s CS-206 treatment.

Broad Applicability Across Various Ethnicities and Mutations

β-hemoglobinopathies are among the most common monogenic disorders, with SCD affecting over 300,000 and TDT over 40,000 newborns annually worldwide. Pathogenic mutations vary significantly across populations.

The team previously developed the ultra-high-precision tBE (Wang et al., Nat Cell Biol, 2021) to precisely edit the HBG1/2 promoter region in autologous HSPCs collected from patients, reactivating γ-globin expression.

The four patients’ genotypes in the current study encompassed βS/βS SCD and three TDT genotypes—β⁰/βᴱ, β⁰/β⁰ with a large deletion, and β⁰/β⁰ with single-nucleotide insertion—validating the strategy’s universal applicability.

Clinical Data: Rapid Engraftment, Durable Response, Complete Transfusion Independence, and No Off-target Mutations

The SCD patient (21-year-old female from Nigeria), who experienced more than four VOCs during the year prior to enrollment, achieved neutrophil and platelet engraftment on days 13 and 21 post-infusion.

Total hemoglobin level increased from 7.7 g/dL at baseline to 12.9 g/dL at month 3, remaining above 11 g/dL; HbF level increased from 3.5% to 62.2%, while HbS level decreased from 76.1% to 31.6%, stabilizing at a approximately 6:4 ratio. At 15.5 months follow-up, no VOCs occurred.

Susan Mou: Presenting Long-Term Clinical Outcomes in SCD and TDT

Figure 2. Clinical outcomes of the SCD patient

  1. The expression levels of total Hb, HbF, HbA, HbS, and HbA2 over time in the patient 1, n is 1. Baseline percentages were determined at the time of studyenrollment (month 0).
  2. Percentage of F-cells over time in the patient 1, n is 1. F-cell baseline percentages were determined at the time of study enrollment (month 0).
  3. The occurrence of VOC events after tBE-edited HSPCs infusion.
  4. The occurrence of RBC transfusion events after tBE-edited HSPCs infusion. All RBC transfusions after transplantation were performed according to the studyprotocol. The wash-out period refers to the 60 days after the last RBC transfusion.

The three TDT patients (ages 3-29, from Laos, Malaysia, and Pakistan) achieved median neutrophil engraftment at 13 days and platelet engraftment at 27 days. Mean total hemoglobin concentration reached 11.6 plus or minus 1.2 g/dL and mean HbF concentration increased to 9.8 g/dL at month 3.

At median follow-up of 17.5 months, all achieved sustained transfusion independence. No off-target edits or product-related adverse events were detected.

Susan Mou: Presenting Long-Term Clinical Outcomes in SCD and TDT

Figure 3. Clinical outcomes of the TDT patients

  1. The expression levels of total Hb, HbF, HbA, HbE, and HbA2 over time in the patients 2–4, n is 1. Baseline percentages were determined at the time of studyenrollment (month 0).
  2. Percentage of F-cells over time in the patients 2–4, n is 1. F-cell baseline percentages were determined at the time of study enrollment (month 0).
  3. The occurrence of RBC transfusion events after tBE-edited HSPCs infusion. All RBC transfusions after transplantation were performed according to the studyprotocol. The wash-out period refers to the 60 days after the last RBC transfusion.

Comparison with Nuclease-Based Gene Editing Therapies: Faster Engraftment, Higher Expression, Better Safety

In SCD clinical trials, tBE achieved superior neutrophil engraftment (13 days) compared to Cas9 (27 days) and Cas12a (23 days), and superior platelet engraftment (21 days) versus Cas9 (35 days) and Cas12a (25 days). tBE sustained HbF more than 60% of total hemoglobin, markedly outperforming Cas9 and Cas12a regimens (less than 50%).

Unlike nucleases that rely on DNA double-strand breaks (DSBs), tBE enables precise base conversion without cutting DNA, avoiding p53 activation, apoptosis, large deletions, and chromosomal rearrangements. Its dual gRNA and ‘lock-and-key’ design further minimizes off-target risks. Through a cleavable ‘lock’, tBE becomes active only at on-target sites to induce highly efficient editing. When binding at off-target sites, tBE was ‘locked’ to avoid triggering off-target mutations.

Global Progress and Regulatory Pathway

To date, CS-101 and CS-206 have treated more than 30 patients across China, Africa, Southeast Asia, and South Asia, with 100% of patients achieving transfusion independence or freedom from VOCs, accompanied by sustained, high-level hemoglobin expression. CS-101, the world’s first ongoing base-editing therapy candidate to enter clinical development, with the first patient dosed in October 2023, has completed Phase I and is now being evaluated in pivotal trials. All patients treated in Phase I have maintained transfusion independence for more than one year, with the longest duration approaching almost three years.

Professor Chen Jia, founder of Correctseq and Director of the Gene Editing Center at ShanghaiTech University, stated:

‘This Cell Stem Cell paper validates tBE’s broad applicability across diverse genetic backgrounds, completing the translational journey from bench to global clinical application. Our team is also exploring RNA editing, prime editing, and mitochondrial DNA editing for other therapeutic areas.’

Dr. Mou Xiaodun, CEO of Correctseq, added:

‘The data demonstrate tBE as a global Best-in-Class platform. We are also expanding into metabolic and cardiovascular diseases including hypertriglyceridemia or familial chylomicronemia syndrome (FCS), ASCVD or hyperlipoproteinemia, homozygous familial hypercholesterolemia (HoFH), and metabolic dysfunction-associated steatohepatitis (MASH). We are accelerating multiple pipelines toward global IND submission to bring China-originated gene editing to more patients worldwide.’

About CorrectSequence Therapeutics (Correctseq), is a clinical-stage biotech company employing its proprietary transformer Base Editor (tBE) to pioneer next-generation gene editing therapies.

The company has developed multiple state-of-the-art base-editing systems that offer exceptional precision, minimize off-target effects, and enhance ex vivo and in vivo editing efficiency. Its robust pipeline spans genetic disorders, metabolic diseases, and cardiovascular conditions, with several programs already advancing toward clinical development.”

Title: Clinical base editing for β-hemoglobinopathies across different genetic backgrounds

Authors: Rongrong Liu, Yongrong Lai, Lijie Wang, Xiaowen Qian, Xu-Kai Ma, Yaliang Li, Gaohui Yang, Lingling Shi, Zifeng Li, Jia Wei, Zhenbin Wei, Hongsheng Wang, Xuemei Zhou, Ling Yu, Bei Yang, Xiaowen Zhai, Xiaodun Mou, Li Yang, Jia Chen

Susan Mou: Presenting Long-Term Clinical Outcomes in SCD and TDT

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Africa ASCVD Bei Yang Blood Cell Stem Cell Chen Jia China double-strand breaks DSBs Familial Chylomicronemia Syndrome FCS Gaohui Yang Hb HBA HbE HbF Health Hematology Hemoglobin Hemostasis Hemostasis News Hemostasis Today HoFH Homozygous Familial Hypercholesterolemia Hongsheng Wang Jia Chen Jia Wei Laos Li Yang Lijie Wang Ling Yu Lingling Shi Malaysia MASH Medicine metabolic dysfunction-associated steatohepatitis Nigeria Pakistan Rongrong Liu SCD Sickle Cell Disease Susan Mou tBE TDT transformer Base Editor VOCs Xiaodun Mou Xiaowen Qian Xiaowen Zhai Xu-Kai Ma Xuemei Zhou Yaliang Li Yongrong Lai Zhenbin Wei Zifeng Li β-Thalassemia
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