Chinese Base Editing Therapy ‘Clinically Cured’ Patient with β-Thalassaemia, Researchers Say

Shanghai-based biotech company CorrectSequence Therapeutics, in collaboration with Guangxi Medical University, has achieved a significant clinical milestone by successfully treating a young patient with transfusion-dependent β-thalassaemia using their base editing therapy, CS-101. The patient underwent treatment in October 2023 and, as of the reporting date, has maintained a haemoglobin level at or above 130 g/L without any adverse effects related to the administered therapy.

As per a company announcement, employing innovative transformer Base Editing (tBE) technology, CS-101 offers more efficient hematopoietic reconstruction and shorter timeframes for achieving transfusion independence compared to other CRISPR-based β-thalassaemia gene-editing therapies. It also minimizes safety risks such as large DNA fragment deletions or off-target mutations.

Μoreover, an investigational study using CS-101 to treat sickle cell disease (SCD) patients is currently underway.

The Thalassaemia International Federation (TIF) welcomes the progress and continuing advancements in associated curative therapies. While we remain optimistic about recent breakthroughs, we also remain mindful of these treatments still being in their infancy and not yet available to the majority.

As patient advocates, our primary objective is to support research that enhances clinical services and promotes innovation. Gene addition and gene therapy offer hope for the future, which we fully endorse. At the same time, we acknowledge that many patients endure significant challenges due to conventional treatments’ limitations.

Therefore, while promoting innovative curative approaches to address this genetic disorder’s complex challenges, TIF continues advocating for basic care provision to improve patients’ survival, wellbeing, quality of life, and social integration. In collaboration with the World Health Organization (WHO), governments, and communities of patients and healthcare professionals, TIF actively works to protect patients’ basic human rights and enhance access to innovative treatments that can transform lives.