December 1, 2025, CorrectSequence Therapeutics Co., Ltd. (Correctseq), a clinical stage biotechnology company aiming to use the innovative gene editing technology to help people with severe diseases, announced a poster presentation at the 67th ASH Annual Meeting and Exposition, taking place December 6-9, 2025, in Orlando, Florida, and online.
Publication Number: 6090
Presentation Title: Rapid, efficient and durable fetal hemoglobin production following CS-101 treatment in transfusion-dependent β-thalassemia participants: An autologous, ex vivo edited CD34+ stem cell product using the innovative transformer base editor (tBE)
Presentation Time: December 8, 2025, 6:00 PM - 8:00 PM(EST)
Format: Poster presentation
Session Name: 801. Gene Therapies: Poster III
Room: OCCC - West Halls B3-B4
For more information about the meeting, visit: https://www.hematology.org/meetings/annual-meeting
About CorrectSequence Therapeutics
CorrectSequence TherapeuticsTM (CorrectseqTM) (www.correctsequence.com) 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. CS-101 enabled over 10 transfusion-dependent β-thalassemia patients to achieve transfusion independence, and the first sickle cell disease (SCD) patient to remain free of vaso-occlusive crises (VOCs). Clinical data demonstrates its superior performance, indicating significant potential for CS-101 to become a best-in-class gene editing therapy for curing patients with β-hemoglobinopathies.
CS-121, an in vivo tBE-based gene-editing therapy targeting APOC3 via lipid nanoparticle (LNP) delivery for chylomicronemia / hypertriglyceridemia, has successfully treated the first patient.
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.
tBE offers significant advantages in controlling off-target effects and enhancing in vivo editing efficiency, making it ideal for both multiplex editing and precise single-target editing. It is compatible with various delivery system, including ex vivo editing in hematopoietic stem cells (HSCs) and T cells, as well as in vivo editing via LNPs or adeno-associated viruses (AAVs).
We are developing multiple pipeline programs targeting genetic diseases, metabolic disorders, and cardiovascular diseases.
Our focus on biotechnology innovation, research and development underscores our commitment to providing efficient, reliable and safe solution for unmet medical needs.
