Recently, the Wu Xiaobing team at Genecradle Therapeutics, together with the Zhang Zaiqiang team at Beijing Tiantan Hospital and the Magdalena J. Koziol team at the Chinese Institute for Brain Researc

The Phase III confirmatory clinical trial of GC101 adeno-associated virus injection, independently developed by Genecradle Therapeutics, for the treatment of spinal muscular atrophy (5qSMA) has met it

Global First-in-Neonate UseIn September 2026, the first exploratory clinical data from a neonate was published online in preprint form for GC801, an engineered IdeS protease developed by Genecradle Th

PrologueIn August 2026, GSK announced the success of the Phase III clinical trial of its antisense oligonucleotide drug Bepirovirsen, achieving functional cure rates of 19%–26% in chronic hepatitis B,

On August 25, 2026, the initiation meeting for the "Single-Center, Randomized, Single-Blind, Placebo-Controlled, Single-Dose, Dose-Escalation Phase I Clinical Trial to Evaluate the Safety, Tolerabilit

Recently, the clinical research results of GC301 injection, an AAV gene therapy drug independently developed by Beijing Genecradle Therapeutics Co.,Ltd., for the treatment of infantile-onset Pompe disease (IOPD), were officially published as an original article in eClinicalMedicine, a sub-journal of The Lancet (part of The Lancet Discovery Science series).

GC101 + GC801: Making Universal Eligibility a Reality. The challenge of SMA gene therapy never lies solely in the drug itself, but also in a series of practical barriers surrounding treatment—among which pre-existing antibodies are a critical, once-overlooked link. Genecradle Therapeutics' parallel development of the gene therapy agent GC101 and the antibody degradation agent GC801 represents a systematic response across the full treatment chain: addressing the root cause of the disease on one hand, and removing pre-treatment barriers on the other.

Recently, the Beijing Key Laboratory of Precision Diagnosis and Innovative Translational Research for Pediatric Rare Diseases (hereinafter referred to as "the Laboratory"), co-established with the par

The 3-month follow-up results of the first sentinel patient in the RDGT-101 clinical study (ChiCTR2600121466), led by Professor Feng Zhichun’s team at the Seventh Medical Center of Chinese PLA General Hospital, have been officially announced. This is the world’s first human clinical study of AAV9-NAGLU for mucopolysaccharidosis type IIIB (MPS IIIB) administered via intracerebroventricular (ICV) injection, marking landmark preliminary evidence for this innovative therapy.