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On March 31, Rui Therapeutics (hereinafter referred to as "Rui Therapeutics") announced significant good news:Rui Therapeutics' self-developed KN5501 cell injection has officially received tacit approval from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) for Investigational New Drug (IND) applications targeting three refractory autoimmune diseases.The indications approved this time precisely cover three major areas of autoimmune diseases with urgent clinical needs, namely: primary Sjögren's syndrome with unsatisfactory efficacy after previous standard treatments, systemic sclerosis without remission or with rapid progression despite sufficient treatment, and refractory lupus nephritis. This marks that this world-leading CD19-targeted CAR-NK cell therapy drug officially enters a new phase of clinical development, opening a brand-new chapter in the treatment of autoimmune diseases.
Precise Targeted Immune Reconstruction: Solving Treatment Challenges at the RootThe core pathogenesis of autoimmune diseases is the "failure to distinguish self from non-self" triggered by abnormal activation of B cells, which leads to 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 in patients, blocking abnormal immune responses while inducing the reconstruction of the patient’s immune system. This achieves deep or even complete remission of the disease at its root, helping patients completely摆脱长期依赖激素、免疫抑制剂的治疗困境,大幅降低药物副作用，显著提升长期生存质量。Breakthrough in Universal Off-the-Shelf Technology Reshapes Accessibility of Cell TherapyUnlike the personalized customization model of "one drug for one person" in traditional autologous CAR-T cell therapy, KN5501, supported by Rui Therapeutics' globally pioneering HTAS-RV™ delivery system and CAR-NK cell expansion and cryopreservation technology, has achieved core breakthroughs in "universalization" and "off-the-shelf" capabilities.Three Major Indications Precisely Address Unmet Clinical NeedsKN5501 has been granted three IND approvals for autoimmune diseases at once, precisely addressing 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 root, 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 kidney 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 DiseasesCurrently, 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 international forefront. The approval of 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 treatment for autoimmune diseases, opening up a new path for the treatment of autoimmune diseases.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 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.Source: Rui Therapeutics
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