RuiTherapeutics' Off-the-Shelf CAR-NK Therapy KN5501 Receives CDE IND Clearance for Three Autoimmune Indications

On March 31, Rui Therapeutics (hereinafter referred to as "Rui Therapeutics") announced significant good news: the company's independently developedKN5501 Cell InjectionThe Investigational New Drug (IND) application for a novel drug targeting three refractory autoimmune diseases has officially received tacit approval from the Center for Drug Evaluation (CDE) under the National Medical Products Administration (NMPA). The approved indications precisely cover three major autoimmune disease areas with urgent clinical needs: primary Sjögren's syndrome unresponsive to prior standard treatments, systemic sclerosis showing no relief or rapid progression despite adequate treatment, and refractory lupus nephritis. This marks a new phase of clinical development for this globally leading CD19-targeted CAR-NK cell therapy, opening a new chapter in autoimmune disease treatment.

Precise Targeted Immune Reconstruction: Solving Treatment Challenges at the Root 

The core pathogenesis of autoimmune diseases lies in the abnormal activation of B cells, which leads to the immune system's "failure to distinguish between self and non-self," resulting in attacks on the body's own tissues and organs. KN5501, a CD19-targeted CAR-NK cell therapy drug independently developed by Rui Therapeutics, precisely identifies and deeply eliminates CD19-positive B cells within patients, blocking abnormal immune responses while inducing the reconstruction of the patient’s own immune system. This achieves profound or even complete disease remission from the root cause, helping patients completely摆脱长期依赖激素、免疫抑制剂的治疗困境, significantly reducing drug side effects, and markedly improving long-term quality of life.

Breakthrough in Universal Off-the-Shelf Technology Reshapes the Accessibility of Cell Therapy

Unlike the personalized customization model of "one drug for one person" in traditional autologous CAR-T cell therapy, KN5501 is based on Rui Therapeutics' globally pioneeringHTAS-RV™ Delivery System and CAR-NK Cell Expansion Cryopreservation Technology, achieving core technology breakthroughs in "generalization" and "off-the-shelf" capabilities.

Three Major Indications Precisely Address Unmet Clinical Needs

This time, KN5501 has been approved for three INDs of autoimmune diseases at one time, precisely meeting multiple unmet clinical needs in this field:

1. Primary Sjögren's Syndrome (pSS) with poor response to prior standard treatment: pSS is one of the most common autoimmune diseases globally, with patients experiencing dry mouth and dry eyes as core symptoms. Long-term treatment can only provide symptomatic relief, lacking curative means targeting the root cause. The immune reconstitution mechanism of KN5501 is expected to improve patient symptoms from the source, achieving long-term disease remission.

2. Systemic sclerosis (SSc) that shows no relief or rapid progression after adequate treatment）: Also known as "scleroderma," it is a rare autoimmune disease characterized by progressive fibrosis of the skin and internal organs. Patients in the advanced stage have a very poor prognosis, and the effectiveness of existing treatments is limited. The development of KN5501 will fill the treatment gap for patients with this rare disease.

3. Refractory Lupus Nephritis (LN): As the most severe organ complication of SLE, lupus nephritis is the leading cause of end-stage renal disease and mortality in patients. Current treatment methods struggle to achieve long-term remission, and the clinical exploration of KN5501 will bring a therapeutic turning point for these high-risk patients;

Internationally Leading R&D Progress: Ushering in a New Era of Cell Therapy for Autoimmune Diseases

Currently, no CAR-NK cell therapy drugs targeting the same site have been approved for marketing globally, and the development progress of KN5501 is at the forefront internationally. The approval of the three INDs for autoimmune indications this time is not only an important milestone for Rui Therapeutics in the field of cell therapy but also fills the global gap in off-the-shelf CAR-NK treatments for autoimmune diseases, opening up a new path for the treatment of autoimmune conditions.

According to the introduction, the clinical trial approved this time is an open, single-arm, dose-escalation Phase I clinical study targeting three indications. It will separately evaluate the safety, tolerability, pharmacokinetic characteristics, and preliminary efficacy of KN5501 in subjects with different indications, laying a solid foundation for subsequent pivotal clinical development.

References:

1. CDE official website, other publicly available online information

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