Shanghai Saixinxin Biomedical Announces IND Approval in China for Global First-in-Class Intrathecal Treg Cell Therapy for ALS

On April 24, 2024, the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) officially approved the clinical trial application (acceptance number: CXSL2500094) for "Human Autologous Polyclonal Regulatory T-Cell Injection" by Shanghai Saierxin Biomedical Technology Co., Ltd. (NOVABIOTX). The indication is Amyotrophic Lateral Sclerosis (ALS, commonly known as Lou Gehrig's disease). As the world’s first innovative therapy using intrathecal injection of Treg cells to treat ALS, this breakthrough could potentially transform the survival outlook for hundreds of thousands of ALS patients globally.

▲ Image Source: CDE Official Website

Breakthrough Moment: Autologous Polyclonal Treg Cell Therapy Approved

ALS is characterized by progressive motor neuron death, with most patients succumbing within 2-5 years after diagnosis. Current medications only extend survival by a few months and fail to reverse nerve damage. The recently approved cell therapy precisely modulates the immune microenvironment within the CNS, targeting the core mechanism of the disease - reversing the Teff/Treg cell imbalance - offering the potential for functional recovery in patients.

Innovative Mechanism: Dual Pathways of Immune Modulation + Neural Repair

This therapy uses the patient's autologous Treg cells, which are expanded in vitro and then reinfused, through a dual-action pathway:

1. Immune Homeostasis Reconstruction: Inhibiting over-activated aggressive immune cells to prevent further damage to myelin and neuronal cells;

2. Nerve Repair Activation: Promoting myelin regeneration and repairing damaged neurons by leveraging the repair function of Treg cells, recruiting oligodendrocytes, or modulating the phenotypic transition of microglia.

Unlike traditional drugs that "delay progression," this therapy achieves a breakthrough from "neuroprotection" to "functional remodeling" for the first time.

Clinical Evidence: Dual Breakthroughs in Functional Improvement and Safety

According to the clinical data disclosed by Professor Zhang Yi from the First Affiliated Hospital of Zhengzhou University at the 9th Immuno-Gene & Cell Therapy Conference (IGC 2025):

• Functional Scale Breakthrough: ALSFRS-R Score Continuously Stable with Improvement

• Reversal of Motor Function: First Patient Showed Improved Finger Dexterity in 7 Days, Achieved Independent Feeding and Stair Climbing Within 4 Months

• System Function Stability: Grip strength value has improved compared to baseline, and pulmonary function indicators remain at baseline levels.

• Safety: 5 subjects showed good safety

Intrathecal injection delivers Treg cells directly to the site of nerve injury, which is the key to this innovative mechanism. This therapy has now entered the registered clinical trial phase, and the NOVABIOTX team plans to rapidly advance clinical trials to further verify its preliminary safety and efficacy. This approval fills a global gap in the field of intrathecal Treg cell therapy for ALS. If subsequent trials confirm its efficacy, it will propel ALS treatment worldwide from "slowing disease progression" into a new era of "functional restoration."