New Chapter Unfolds: Overview of Over 10 Chinese CAR-T Pipelines Targeting Autoimmune Diseases from Companies like IASO Bio and JW Therapeutics

Jul 24, 2024 10:55 CST Updated 10:55

IASO Biotechnology

Cancer Treatment New Drug Developer

BRL Medicine

Cell and Gene Therapy Drug Developer

▎WuXi AppTec Content Team Report

Today (July 24), IASO Bio announced that its BCMA-targeted CAR-T therapy (Equecabtagene Autoleucel Injection) received approval from the U.S. FDA for a new drug clinical trial application to treat multiple sclerosis. Coincidentally, just this month...，BRL Medicine Develops Allogeneic Universal CD19-Targeted CAR-T Therapy (TyU19) for the Treatment of Autoimmune DiseasesPositive Research ResultsPublished in the prestigious international journal "Cell".These continuous advancements in CAR-T therapy for the treatment of autoimmune diseases have drawn widespread attention in the industry.Also brings new insights:Beyond cancer treatment,CAR-T Cell Therapy May Enter a New Chapter——Autoimmune Diseases。

Recently, an authoritative international journal, a subsidiary of "Nature"Nature BiotechnologyIt was also reported that current biotechnology companies are "equipping" CAR-T cells to destroy B cells, with the goal ofMultiple Sclerosis and a Range of Autoimmune Diseases。Looking globally,Indeed, many biopharmaceutical companies are developing CAR-T therapies for autoimmune diseases.

And in China, publicly available information shows that development is underway.The CAR-T product pipeline for the treatment of autoimmune diseases already has dozens.PaymentSome of these ongoing projects have entered the registration clinical stage, while others are still in investigator-initiated clinical studies (IIT).This article will be based on publicly available informationInventoryMore than 10Part of the CAR-T therapies under research for treating autoimmune diseases developed by Chinese companies(Not including R&D projects led by universities and research institutions), for readers' reference.

IASO Bio: Icaritin

Mechanism of Action: BCMA-Targeted CAR-T

Indications: MG,NMOSD, etc.

Equecabtagene Autoleucel is independently developed by IASO Bio.Fully Human Targeted BCMA Chimeric Antigen Receptor Autologous T-Cell Injection, which was approved in China in June 2023 for the treatment of patients with multiple myeloma. According to the official website of IASO Bio, the product treatsMyasthenia Gravis (MG), Neuromyelitis Optica Spectrum Disorder (NMOSD)、Multiple SclerosisHas been approved for registration of clinical research (IND) in China or the United States.In multiple autoimmune disease indications,Preliminary clinical results have been achieved with Idecabtagene Vicleucel.In March this year, IASO Bio announced the treatment of this productAutoimmune DiseasesTheTwo investigator-initiatedClinical Trial Data，Preliminary results show that the product has therapeutic effectsRefractory MGAndImmune-Mediated Necrotizing MyopathyThe potential. In July this year, researchers also announcedIdecabtagene VicleucelTreatmentCentral Nervous System Immune DiseasesTheClinical Data and Single-Cell Multi-Omics Analysis Results。

Ruishun Biologics: RJMty19 Injection

Mechanism of Action: CD19-CAR-DNT Cells

Indications: Systemic Lupus Erythematosus

RJMty19 Injection is an "off-the-shelf universal" CAR-DNT immunocyte therapeutic product without gene editing.Based on Humanized CD19-CAR Development。DNT Cells, Also Known as Double-Negative T Cells, Are a Subtype of T Cells in the Peripheral Blood of Healthy Individuals That Express the CD3 Molecule but Do Not Express the CD4 and CD8 Molecules.A highly promising off-the-shelf universal T-cell therapy for the clinical treatment of various tumors。According to publicly available information from RuiShun Biotech, this product has received IND approval in China for three indications, including two hematological malignancies and one autoimmune indication.Indications, used to treat refractorySystemic Lupus Erythematosus。

JW Therapeutics: Relma-cel Injection

Mechanism of Action: CD19-CAR-DNT Cells

Indications: Systemic Lupus Erythematosus

Relmacabtagene Autoleucel Injection is a CD19-targeted autologous CAR-T cell immunotherapy product that has been approved by China's NMPA for two indications, including large B-cell lymphoma and follicular lymphoma. In April 2023, the product was used to treat moderate to severeRefractory Systemic Lupus ErythematosusThe indication received the CDE's clinical trial implied permission. In May 2024, WuXi JWC announced at the 2024 European Congress of Rheumatology the release of Relma-cel Injection for the treatment ofPreliminary Clinical Data from IIT Studies in Chinese Adult Patients with Active Systemic Lupus ErythematosusData show that, even at a relatively low dose compared to hematological tumor indications, a single infusion of Relma-cel can still provide moderate to severe lupus erythematosus patients with deep and durable disease remission, demonstrating good safety.

BRL Medicine: TyU19, BCMA-UCART

Mechanism of Action: Next-generation universal allogeneic CD19 CAR-T therapy, BCMA-UCART

Indications:Diffuse cutaneous systemic sclerosis, multiple sclerosis, etc.

TyU19 is developed by BRL Medicine.Allogeneic universal CAR-T cell therapy product targeting CD19 antigenIn July this year, the research成果 on TyU19 for the treatment of autoimmune diseases was published in a top international academic journal."Cell"Above. The analysis shows that TyU19 is able toDepletion of B cells in 3 patients with refractory autoimmune diseases within two weeks, and reversal of extensive fibrotic damage in organs of two patients with diffuse cutaneous systemic sclerosis (SSc)According to the press release from BRL Medicine, TyU19 is the world's first allogeneic product to achieve positive results in the research for treating autoimmune diseases.Allogeneic Universal CAR-T Therapy。In addition, BRL Medicine is alsoIIT StudyEvaluation of BCMA-UCARTThe potential for autoimmune diseases,InvolvedMultiple Sclerosis, Myasthenia GravisAnd other diseases.

Hoyuncell: Naciaurel

Mechanism of Action: CD19-Targeted CAR-T

Indications:Refractory Autoimmune Diseases

Nazioleen (CNCT19) is a CAR-T cell therapy product targeting CD19, and also the first CAR-T cell therapy product in China's leukemia treatment field, which wasApproved for marketing in China in November 2023. At present, Heliocentric Biotech is also exploringThe Potential of NaKiOleCell in the Field of Autoimmune Diseases. According toClinicalTrialsOfficial Website,HeyuanBRL Medicine is conducting aCNCT19Treatment of Refractory Autoimmune DiseasesPhase 1Exploratory Clinical StudyTo evaluate the safety and tolerability of CNCT19 in patients with refractory systemic lupus erythematosus (lupus nephritis, immune thrombocytopenia), refractory ANCA-associated vasculitis, and refractory dermatomyositis.

Baiji Gene: CD19-Targeted CAR-T Product

Indications: Systemic Lupus Erythematosus

According toClinicalTrialsOfficial Website, Bio-Gene is collaborating as a partner in a Phase 1/2 clinical study to evaluate the CD19 CAR-T product for treatmentActive Systemic Lupus ErythematosusSafety and effectiveness.Studies have shown that the use of CD19-targeted CAR-T therapy in refractory SLE can improve patients' autoimmune symptoms, while allowing the discontinuation of immunosuppressants and hormones, maintaining long-term normalization of autoimmune indicators.

Precision Biologics: MC-1-50 Cell Preparation

Mechanism of Action: CAR-T Cell Therapy Targeting CD19

Indications:Refractory Autoimmune Diseases

MC-1-50 Cell Preparation isPrecision BiologicsDeveloped using the PRIMCAR platformThe Second-Generation CD19-Targeted CAR-T Cell Therapy. According toAccording to the official website of Precision Biotech, this productImplementation of a new type of faster and more efficient CAR-T preparation technology achieves lower dosage, fewer side effects, and longer-lasting results. Its...A phase 1 clinical trial for hematological tumor indications is currently underway. According toClinicalTrialsAccording to the official website, Precision Biologics is also conducting a Phase 1 clinical study aimed at evaluating CD19-targeted CAR-T therapy for variousRefractory Autoimmune DiseasesThe safety and efficacy, including systemic lupus erythematosus, Sjögren's syndrome, dermatomyositis and other diseases.

Fosun Kite: FKC-288

Mechanism of Action: Autologous Dual-Target BCMA/CD19-CAR-T Cells

Indications:Autoimmune Nephropathy, Light Chain (AL) Amyloidosis

According to the official website of Fosun Kite, FKC-288 is a product in its early-stage R&D pipeline, currently in the IIT-POC phase for hematological tumors and autoimmune diseases.According toClinicalTrialsAccording to the official website, researchers are conducting two exploratory Phase 1 clinical studies, respectively evaluatingAutologous Dual-Target BCMA/CD19-CAR-T Cell Therapy ProductFKC-288TreatmentAutoimmune Nephropathy, Light Chain (AL) AmyloidosisThe safety and efficacy of patients.

PRG Biotech: PRG-1801,PRG-2311

Mechanism of Action: BCMA-targeted CAR-T product,CAR-T Product Targeting CD19/BCMA

Indications: Lupus Nephritis, IgG4-Related Disease

According toClinicalTrialsAs can be seen from the official website inquiry,PRJ Biotech is currently collaborating on two Phase 1 clinical studies to evaluateCAR-T Cell Injection Targeting BCMAPRG-1801Safety and Efficacy of Treating Refractory Lupus Nephritis and IgG4-Related Disease, and EvaluationPRG-2311, a CAR-T cell injection targeting CD19/BCMASafety and Efficacy of Treatment for Relapsing or Refractory Lupus Nephritis and IgG4-Related Disease.

EdiGene:ATHENA CAR-T

Mechanism of Action:Allogeneic CAR-T Therapy

Indications:Systemic Lupus Erythematosus

According to the pipeline information on the Elixir Genomics website andClinicalTrialsOfficial WebsiteInformation, EdiGene'sAllogeneic CAR-T TherapyATHENA CAR-T isFor autoimmune diseases (Systemic Lupus Erythematosus) is in an investigator-initiated clinical study (IIT).

Shouge Bio:CD19-Targeted CAR-T Therapy

Indications: Relapsed/Refractory Autoimmune Diseases

According to the website of the Chinese Clinical Trial Registry, BRL Medicine is conducting an exploratory clinical study as a collaborator, which aims to evaluateCD19-Targeted CAR-T CellsSafety, Tolerability, and Feasibility of Treating Patients with Relapsed/Refractory Autoimmune Diseases. Indications Involved Cover Antiphospholipid Syndrome, Systemic Lupus Erythematosus, Systemic Sclerosis, Sjögren's Syndrome, ANCA-Associated Vasculitis, Idiopathic Inflammatory Myopathies, etc.

AccuraBio:CD7 CAR-T、CD19/BCMA CAR-T、CD19-BAFF CAR-T Cell Injection

Indications: SLE and other autoimmune diseases

According toClinicalTrialsAccording to the official website inquiry,As a collaborator, Yakobiotech is conducting several phase 1 clinical studies on CAR-T cell injection for the treatment of autoimmune diseases, includingCD7-targeted CAR-T product、CD19/BCMA-Targeted CAR-T Product、CD19-BAFF CAR-T Products, etc.，The indications involved include POEMS syndrome, amyloidosis, autoimmune hemolytic anemia, vasculitis, etc.At the 2023 ASH Conference, researchers also reported on Yako Bio'sCD19/BCMA CAR-T Cell Therapy for Refractory Systemic Lupus ErythematosusPhase 1 Clinical Study Safety and Preliminary Efficacy Data.Data show that CD19/BCMA CAR-T cell therapy is well-tolerated and can induce rapid and durable remission in severe refractory SLE.

CSPC Zhongqi Pharmaceutical:SYS6020

Mechanism of Action:CAR-T Therapy Targeting BCMA

Indications:SystemSystemic Lupus Erythematosus、Myasthenia Gravis

SYS6020 is an mRNA-LNP-based chimeric antigen receptor (CAR)-T cell injection, which was approved for the first time in China in June 2024 to treat relapsed or refractory multiple myeloma.ClinicalResearchAt that time, CSPC Pharmaceutical Group mentioned in its press release that SYS6020 has clinical development potential for treating autoimmune diseases, such as systemic lupus erythematosus and myasthenia gravis (MG).

Notably, there have been several recent advancements in CAR-T cell therapy for the treatment of autoimmune diseases globally. For example:Caribou BiosciencesThe IND application for CD19-targeted CAR-T therapy received FDA approval in April this year for lupus nephritis and extrarenal lupus.Atara BiotherapeuticsAnnounced in March its CD19-targeted CAR-T therapy forLupus NephritisThe IND application was approved by the FDA.Allogene TherapeuticsUsed forSystemic Lupus ErythematosusThe CD19/CD70-targeted CAR-T therapy is expected to enter Phase 1 clinical trials in early 2025.

References:

[1]CAR-Ts sweep into autoimmunity. Retrieved July 10, 2024 from https://www.nature.com/articles/s41587-024-02321-0

[2]Company official websites and public materials

[3]ClinicalTrials Official Website. Fromhttps://clinicaltrials.gov/

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