Recently, the European Hematology Association (EHA) released the EHA2024 Scientific Congress Report titled "EHA Perspectives on Emerging Technologies in Hematology". As the world's first base editing therapy for hemoglobinopathies, CS-101 of CorrectSequence Therapeutics (Correctseq) was included in the report for its safe and efficient base editing technology and the promising clinical results. The recognition of Correctseq’s CS-101 by the EHA2024 Scientific Congress Report indicates that it has the great potential to become the first-in-class base editing therapy and the best-in-class gene editing therapy for β-hemoglobinopathies.
EHA2024 Hybrid Congress was successfully held in Madrid, Spain from June 13 to 16, 2024. Dr. Lijie Wang, Head of Gene Editing Platform of Correctseq, gave an oral presentation titled "Treatment of Patients with Severe Transfusion-Dependent β-Thalassemia with CS-101, An Autologous, Ex Vivo Edited, CD34+ Hematopoietic Stem Cell Product Using Innovative transformer Base Editor (tBE)" on June 16, sharing the latest clinical data of CS-101, the tBE-based gene editing therapy for β-thalassemia.
▲ Dr. Lijie Wang, Head of Gene Editing Platform of Correctseq, gave an oral presentation at the EHA2024 Hybrid Congress
Upon completion of the conference, the EHA2024 congress published the annual scientific report highlighting the key perspectives on merging technologies in hematology. Dr. Wang's oral presentation on CS-101 was included in the EHA 2024 Scientific Congress Report in recognition for the innovative tBE technology as well as the superior clinical performance of CS-101. 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. The report details the design and mechanism of base editing of tBE. Through specific inhibitor (“lock-key”) system, tBE offers higher on-target editing efficiency and eliminates off-target mutations. The report also summarizes the latest clinical data of CS-101 and highlights CS-101 as “the only base editing therapy that has reached the clinical stage to date” , suggesting the potential of CS-101 as a global best-in-class therapy.
From EHA2024 Scientific Congress Report
▲ The editing process of tBE
The positive feedback from EHA2024 congress has brought great confidence for Correctseq to promote the clinical translation of innovative base editing technologies and bring the gene editing therapy to the world. Correctseq is rapidly and efficiently advancing the clinical trial of CS-101 for β-thalassemia. It is also preparing the clinical trial of CS-101 for sickle cell anemia and is launching the global recruitment program.
For more detailed information about the clinical trial and recruitment program, it is recommended to contact CT@correctsequence.com.
The EHA2024 Scientific Congress Report download link:
https://ehaweb.org/congress/eha2024-hybrid-congress/eha2024-sci-report
About CS-101
Correctseq’s CS-101 is a personalized treatment that begins with the collection of autologous hematopoietic stem cells from β-thalassemia patients. The transformer Base Editor (tBE) technology (Wang et al., Nat Cell Biol, 2021), developed by scientists at ShanghaiTech University, is then used to precisely edit the DNA sequence of the promoter region of the gene encoding γ-globin (HBG1/2) to mimic the naturally occurring single-nucleotide variant in the population with hereditary persistence of fetal hemoglobin, thereby reactivating the γ-globin expression to produce functional HbF. Finally, the edited hematopoietic stem cells are infused back into the patient, enabling the patient to continuously produce blood cells with intact hemoglobin and eliminating the need for frequent blood transfusions. While several gene editing strategies have been explored to restore normal hemoglobin levels in β-thalassemia patients, it has been shown that base editing of the BCL11A binding site at the HBG1/2 promoter by tBE – as in the CS-101 treatment – exhibited the highest level of γ-globin reactivation both in vitro and in vivo (Han et al., Cell Stem Cell, 2023).
Compared to traditional blood transfusion therapy and allogeneic hematopoietic stem cell transplantation, CS-101 offers several compelling advantages. One of the key benefits is its short preparation period. Another is eliminating the need for long waiting time for a matching donor since it utilizes the patient’s own hematopoietic stem cells. With these advantages, CS-101 has the potential to achieve a "single treatment, lifetime cure" within a few weeks.
In comparison to other CRISPR-based β-thalassemia gene editing therapies, CS-101 does not carry safety risks associated with large DNA fragment deletions, chromosomal translocation, or off-target mutations. This enhances the overall safety profile of the treatment and reduces potential complications.
About CorrectSequence Therapeutics
CorrectSequence TherapeuticsTM (CorrectseqTM), is a clinical-stage biotech company employing its proprietary transformer Base Editor (tBE) to pioneer next-generation gene editing therapies. Our leading pipeline candidate, CS-101, utilizing innovative base editor targeting HBG, curing β-hemoglobinopathies, has obtained IND approval from the China NMPA. Clinical data demonstrate its superior performance. Proof-of-concept (POC) data in mice for in vivo pipelines using tBE-editing therapies via lipid nanoparticle (LNP) delivery are available, including targets for hereditary angioedema (HAE), hypercholesterolemia, and hypertriglyceridemia. Ex vivo multiplex editing of T cells on multiple targets simultaneously preserved T cell growth and function in vivo compared to non-edited cells, establishing tBE as the ideal gene editing tool for the next-generation cell therapy development. We are developing multiple pipelines targeting genetic diseases, cancer immunotherapy, metabolic disorders, and cardiovascular diseases.
Please refer to our homepage for more information about the tBE and its therapeutics applications at www.xmyhtsm.com
