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2 months ago CAR-T

The World’s First! China’s Non-Viral Site-Specific Integrated PD1-CAR-T Therapy Brings New Hope for Systemic Lupus Erythematosus(SLE) Patients

**The World’s First! China’s Non-Viral Site-Specific Integrated PD1-CAR-T Therapy Brings New Hope for Systemic Lupus Erythematosus(SLE) Patients**

SLE

SLE

#PD1 #CART #SLE #SystemicLupusErythematosus #Lupus #CARTTherapy #BRL203 #Nature

In November 2024, Chinese gene and cell therapy company BRL Medicine announced that its self-developed non-viral site-specific integrated PD1-CAR-T therapy, “BRL-203,” has received clinical trial approval from the National Medical Products Administration (NMPA) of China. This groundbreaking therapy aims to treat moderate to severe refractory systemic lupus erythematosus (SLE) and is the world’s first non-viral site-specific integrated CAR-T product approved for autoimmune disease treatment. This innovation brings new possibilities in lupus treatment and offers global patients a safer and more effective treatment option.

### Disruptive Treatment Brings Hope to Lupus Patients

Systemic lupus erythematosus (SLE) is a severe autoimmune disease that can lead to irreversible organ damage due to relapse and progression, severely affecting patients’ quality of life and even life expectancy. In recent years, SLE has garnered widespread attention and discussion, highlighting the urgent need for new therapies to alleviate patients’ suffering and extend their lives. While existing treatments can control the disease to some extent, they still rely on long-term immunosuppressive drugs, with suboptimal effectiveness and a high risk of relapse.

BRL Medicine’s non-viral site-specific integrated PD1-CAR-T therapy, “BRL-203,” offers a new treatment option for SLE patients. As a novel CAR-T cell therapy, it is expected to open a new era in autoimmune disease treatment, providing patients with sustainable disease control and reducing the risk of relapse.

### Chinese Non-Viral CAR-T: Overcoming Safety and Efficiency Challenges of Traditional Therapies

BRL Medicine’s “BRL-203” utilizes self-innovated non-viral site-specific integration technology, employing CRISPR/Cas9 gene-editing to precisely target the PD1 site and insert CAR molecules that target CD19. This non-viral method avoids the potential tumorigenic risks of traditional viral vector production, significantly enhancing the safety of the product. Moreover, the therapy preparation takes only 3 days, greatly reducing the patient’s waiting time and lowering production costs, making it more accessible and affordable for patients.

Compared to traditional CAR-T products, which face challenges such as long production cycles, high costs, and potential risks, BRL Medicine’s non-viral site-specific integrated PD1-CAR-T platform not only enhances safety but also significantly improves clinical applicability. Previously, BRL Medicine’s other non-viral PD1-CAR-T therapy demonstrated excellent safety and efficacy in treating relapsed/refractory B-cell non-Hodgkin lymphoma (r/r NHL), with research results published in *Nature* magazine, earning widespread recognition in the medical community.

### Precise Targeting and Safe, Controllable Innovative Therapy

Unlike traditional random integration methods, China’s non-viral site-specific integration technology ensures that each CAR sequence is precisely placed at a designated location in the genome, reducing the risk of tumor formation during treatment. This breakthrough technology ensures product uniformity and significantly improves clinical safety. In early clinical trials, BRL Medicine’s PD1-CAR-T therapy achieved a 100% objective response rate (ORR), an 85.7% complete response rate (CR), with almost no high-risk side effects.

### Future Outlook: Diversified Options for Autoimmune Disease Treatment

The approval of BRL Medicine’s non-viral site-specific integrated PD1-CAR-T product is not only a boon for lupus patients but also a significant breakthrough in the global autoimmune disease treatment field. The company will accelerate the subsequent clinical progress of BRL-203, aiming to develop it into an efficient, safe, and accessible innovative therapy, bringing new hope to more patients.

🎉🎉To assess whether the condition is suitable for CAR-T therapy, you can submit pathology reports, treatment history, and discharge summaries to the Medical Department of <Advanced Medicine in China> for preliminary evaluation!

WhatsApp: Https://wa.me/+8613717959070

Email: doctor.huang@globecancer.com

#BRLMedicine #CAR_T #LupusTreatment #AutoimmuneDisease #NonViralTherapy #GeneTherapy #InnovativeMedicine #SLE #ClinicalTrials #MedicalBreakthrough


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2 months ago CAR-T

China’s CAR-T Technology Leads the World: A Miraculous Recovery Journey for Children with systemic lupus erythematosus (SLE)

**China’s CAR-T Technology Leads the World: A Miraculous Recovery Journey for Children with systemic lupus erythematosus (SLE)**

LUPUS

LUPUS

#SLE #lupus #systemiclupuserythematosus #CAR_Ttherapy #CancerSurvivor #ChineseHealthcare#PatientStory

In the field of medicine, the boundary between science and miracles is often blurred. This October, news from Zhejiang, China, shocked the world: 20 children suffering from refractory systemic lupus erythematosus (SLE) successfully discontinued the use of steroids and all immunosuppressants after receiving CAR-T cell therapy.

This global breakthrough not only brings hope to patients and their families but also demonstrates the immense potential of CAR-T therapy in the treatment of autoimmune diseases.

### Immune System Reset, New Life Begins

Systemic lupus erythematosus (SLE) is a complex autoimmune disease that many patients struggle to control despite long-term reliance on medications. This clinical trial, led by a team of renowned Chinese medical experts, marks the first time autologous CD19-targeted CAR-T cells have been applied to treat pediatric SLE patients. In just seven months, the symptoms of all 20 children improved significantly, and their quality of life greatly increased.

These children ranged in age from 6 to 19 years, with disease durations varying from four months to 11 years. Prior to receiving CAR-T treatment, they had been on an average of more than five different medications, with some taking as many as nine, yet the results were less than ideal. The introduction of CAR-T therapy became their last hope.

Although CAR-T therapy has already seen widespread application in the field of hematologic malignancies, this treatment for pediatric SLE represents a new frontier for CAR-T technology in autoimmune disease treatment, paving the way for innovative medical exploration.

### Clinical Data: Miracles in Reality

Among the 20 children, many showed remarkable recovery. For example, a 12-year-old girl from Shanghai, who had experienced recurrent lupus flare-ups, with a “butterfly rash” covering her face and massive proteinuria, saw a dramatic improvement after receiving CAR-T therapy. Within six months, she stopped all medications, her proteinuria decreased significantly, and her facial rash completely disappeared. By September, she had returned to school and was living almost normally.

Another 15-year-old girl from Shandong, despite having been diagnosed only two years prior, had progressed rapidly to kidney failure, requiring dialysis. After treatment, her blood creatinine level dropped from 700 μmol/L to 170 μmol/L, and her urine output increased from 50ml to 1000ml, allowing her to fully stop dialysis.

Moreover, all 20 children successfully discontinued steroids and immunosuppressants, and some patients’ disease activity scores dropped to zero. This means their immune systems have been reset, providing long-term control and, in some cases, a complete cure.

### Safety and Side Effects of CAR-T Therapy

Every revolutionary treatment carries risks, but the safety of China’s CAR-T therapy in this trial has been encouraging. CAR-T therapy works by genetically modifying T cells to recognize and attack specific target cells. Some patients experienced mild side effects, such as fever, nausea, and fatigue. Among the 20 children, only three exhibited mild neurological symptoms, which quickly improved with symptomatic treatment.

Research shows that CAR-T treatment in pediatric SLE patients comes with relatively mild side effects, and most patients did not experience any severe adverse reactions. This makes the future of CAR-T therapy in treating refractory lupus even more promising. This technology not only delivers significant therapeutic effects but also restores hope for a better future for these children.

### A Forward-Looking Treatment: From SLE to Other Diseases

Following the success of these 20 SLE cases, the research team plans to extend the application of China’s CAR-T technology to other autoimmune diseases, such as ANCA-associated vasculitis (AAV) and multidrug-resistant steroid-resistant nephrotic syndrome (MDR-SRNS). This means that in the future, more patients will have the opportunity to benefit from this revolutionary therapy.

🎉🎉To assess whether the condition is suitable for CAR-T therapy, you can submit pathology reports, treatment history, and discharge summaries to the Medical Department of <Advanced Medicine in China> for preliminary evaluation!

WhatsApp: Https://wa.me/+8613717959070

Email: doctor.huang@globecancer.com

#CancerTreatment #Immunotherapy #MedicalBreakthrough #AdvancedMedicine  #HealthRecovery


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4 months ago CAR-T

Chinese CAR-T Therapy: A New Frontier in Treating Systemic Lupus Erythematosus

Chinese CAR-T Therapy: A New Frontier in Treating Systemic Lupus Erythematosus

Systemic Lupus Erythematosus

Systemic Lupus Erythematosus

Recently, CSPC PHARMA’s CAR-T product, SYS6020, received approval for clinical trials from China’s National Medical Products Administration (NMPA). This groundbreaking development represents a significant milestone in cell therapy, especially for treating refractory active systemic lupus erythematosus (SLE). Notably, SYS6020 targets the BCMA antigen, potentially providing a novel and cost-effective treatment option for SLE patients worldwide.

**Innovative Approach and Promising Safety Profile**

SYS6020 is a pioneering mRNA-LNP-based CAR-T cell injection, marking it as the first of its kind to enter clinical trials. This innovative therapy specifically recognizes the BCMA antigen, attacking and eliminating immune cells responsible for elevated autoantibodies. This approach offers a unique, safe, and effective treatment option for SLE, distinct from traditional CAR-T therapies. Notably, SYS6020 exhibits high cell viability, a high CAR-positive rate, and reduced risks of genomic integration and cytokine release syndrome (CRS), common issues with conventional CAR-T treatments.

Preclinical studies have shown that SYS6020 effectively kills BCMA antigen-positive myeloma cells while maintaining a favorable safety profile. By employing LNP for T cell transfection, the therapy significantly reduces costs compared to using lentiviral vectors, lessening the financial burden on patients.

Lupus

Lupus

**Revolutionizing Treatment for Autoimmune Diseases**

SLE, a complex chronic autoimmune disease, is characterized by the immune system attacking its own tissues. According to the “Chinese Guidelines for Diagnosis and Treatment of Lupus Nephritis,” the prevalence of SLE in China is estimated to be between 30.13 and 70.41 per 100,000 individuals, translating to 422,000 to 986,000 patients.

Traditional SLE treatments, including steroids, often come with severe side effects and long-term health risks. In contrast, biological therapies have emerged as a game-changer by modulating immune responses, reducing the immune system’s attack on the body’s tissues, and decreasing dependency on steroids. However, only three biologics—belimumab, tildrakizumab, and anifrolumab—are currently approved globally.

CAR-T therapy has shown remarkable efficacy in treating refractory SLE, providing hope in a challenging therapeutic landscape. This therapy precisely targets “rogue” B cells responsible for autoantibody production, thereby minimizing organ damage and offering a more targeted approach than traditional treatments.

**Future Prospects and Ongoing Challenges**

While CAR-T therapy presents a promising new direction for SLE treatment, its long-term efficacy and safety remain under investigation. Larger clinical trials are needed to validate its effectiveness and safety, as current studies are limited to small samples.

Additionally, the potential long-term risks of CAR-T therapy, such as increased susceptibility to infections or malignancies, require thorough exploration. Despite these uncertainties, the active involvement of multiple domestic and international cell therapy companies underscores the global commitment to advancing CAR-T therapies for autoimmune diseases.

In summary, CAR-T therapy for SLE holds immense potential, with clinical trials offering hope for a safer and more effective treatment alternative. Continued research and positive trial outcomes could establish CAR-T as a viable therapeutic option for SLE patients, revolutionizing their treatment landscape.

🎉🎉To assess whether the condition is suitable for CAR-T or clinic therapy, you can submit Advanced Medicine in China for preliminary evaluation!
WhatsApp: +8613717959070
Email: doctor.huang@globecancer.com

#CAR_Therapy #SLETreatment #AutoimmuneInnovation #MedicalBreakthrough #LupusAwareness

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