HN-2301: An Investigational In Vivo mRNA-Based CAR-T Cell Therapy for Autoimmune Diseases

I. Executive Summary

HN-2301 is an investigational therapeutic candidate being developed by Shenzhen MagicRNA Biotechnology Co., Ltd. It represents an innovative approach in the field of cell therapy, specifically designed as an in vivo generated Chimeric Antigen Receptor T (CAR-T) cell therapy. The core technology involves the delivery of messenger RNA (mRNA) encoding a CD19-targeting CAR, utilizing the company's proprietary Engineered Cell-targeted Lipid Nanoparticle (EnC-LNP) delivery platform. This method aims to transiently reprogram a patient's own T cells within their body to recognize and eliminate CD19-expressing B cells.

The primary therapeutic indications for HN-2301 are B-cell mediated autoimmune diseases, with initial clinical investigations focusing on Systemic Lupus Erythematosus (SLE) and Myasthenia Gravis (MG). Preclinical studies, including those in non-human primates and murine models of SLE, have reportedly demonstrated successful in vivo CAR-T cell generation and B-cell depletion, with therapeutic effects observed in disease models. HN-2301 is currently advancing through IND-enabling studies and into early-phase clinical trials in China.

This therapeutic modality holds the potential to overcome several limitations associated with traditional ex vivo CAR-T cell therapies, such as complex manufacturing processes, high costs, and the need for patient lymphodepletion. By leveraging transient mRNA expression and targeted LNP delivery, HN-2301 aims to offer a safer, more accessible, and potentially scalable treatment option for patients with severe autoimmune conditions. The ongoing and upcoming clinical trial results, along with further elucidation of its proprietary EnC-LNP technology, will be critical in determining the ultimate clinical utility and positioning of HN-2301 in the evolving landscape of autoimmune therapies.

II. Introduction to HN-2301

A. Nomenclature and Basic Identifiers

The investigational therapeutic agent central to this report is primarily designated as HN-2301. Alternative nomenclatures encountered in the available documentation include HN 2301 and HN2301.[1] For clarity and consistency, this report will predominantly use the designation HN-2301.

It is pertinent to address an initial discrepancy in the characterization of HN-2301. One database, Ozmosi, initially cataloged HN-2301 as an "IDH Inhibitor" with a "Nucleic Acid" modality.[1] However, this classification is superseded by more detailed, consistent, and developer-corroborated information. Shenzhen MagicRNA Biotechnology Co., Ltd., the developer, along with specialized pharmaceutical intelligence services such as AdisInsight, unequivocally identify HN-2301 as an in vivo generated CD19-targeting Chimeric Antigen Receptor T (CAR-T) cell therapy, which utilizes messenger RNA (mRNA) delivered via Lipid Nanoparticles (LNPs).[2] The mention of "IDH inhibitor" is therefore considered an error or refers to an unrelated entity, possibly due to the non-proprietary nature of the "HN-2301" identifier which has appeared in entirely different contexts, such as designations for LNG carriers.[6] Consequently, this report will focus exclusively on the profile of HN-2301 as a CAR-T therapeutic candidate developed by Shenzhen MagicRNA Biotechnology. The consistent and detailed information from the developer and specialized pharmaceutical databases provides a strong basis for this focused approach, ensuring the report accurately reflects the nature of the drug under investigation.

B. Developer: Shenzhen MagicRNA Biotechnology Co., Ltd.

HN-2301 is being developed by Shenzhen MagicRNA Biotechnology Co., Ltd., a biopharmaceutical company founded in 2021.[3] The company's strategic direction is centered on the research and development of nucleic acid-based medicines, with a pronounced focus on RNA therapeutics and sophisticated drug delivery mechanisms.[3] This is encapsulated in their stated mission: "Focusing on Nucleic Acid Drug Delivery, Benefitting Patients with RNA Therapies".[4]

A key technological asset of Shenzhen MagicRNA is its proprietary Engineered Cell-targeted Lipid Nanoparticle (EnC-LNP) platform.[3] This platform is integral to the company's strategy and is designed to enable the specific delivery of nucleic acid payloads to various target cell populations, including cells of the immune system, the central nervous system, and tumor cells.[3] The company's technological capabilities are built upon three core pillars: mRNA sequence design, efficient mRNA synthesis, and LNP-mediated efficient delivery.[3] Shenzhen MagicRNA has cultivated a proprietary library of ionizable lipids, which are critical components of LNPs influencing their delivery efficacy and safety profile. Notably, their core lipid, ILB-3132, is reported to be commercially available, suggesting a degree of maturity in their lipid chemistry expertise.[3]

The company was founded by Zha Gaofeng, Ph.D., who brings expertise in mRNA delivery and a multidisciplinary research background to the organization.[8] To support its research and development ambitions, Shenzhen MagicRNA was, as of early 2022, in the process of establishing a 2500 square meter R&D center and a GMP-grade pilot manufacturing facility. This infrastructure development indicates a commitment to integrated drug development and production.[4]

The rapid progression of Shenzhen MagicRNA from its establishment in 2021 to initiating clinical trials for HN2301 by early 2025 [3] is noteworthy. Such an accelerated timeline in the biopharmaceutical industry often suggests either a strong pre-existing technological foundation brought in by the founders or a particularly efficient and focused R&D engine. In this case, the company's explicit emphasis on its proprietary EnC-LNP platform and ionizable lipid library [3] points towards the delivery technology being a core strength that may enable such rapid advancement. This swift movement from concept to clinical testing underscores the potential of their platform but also highlights the importance of thorough evaluation of the preclinical data that supports this transition.

C. Therapeutic Class and Modality

HN-2301 is categorized within the therapeutic class of CAR-T cell therapies. Its composition involves lipids (as constituents of the LNP delivery system) and RNA (specifically, mRNA as the therapeutic payload).[2]

The modality of HN-2301 is that of an in vivo generated CAR-T cell therapy. This is a sophisticated and innovative approach that distinguishes it from conventional ex vivo CAR-T therapies. The therapy involves the administration of an mRNA sequence that encodes a CD19-targeting Chimeric Antigen Receptor. This mRNA is encapsulated within the company's proprietary EnC-LNPs, which are designed to specifically target and deliver their mRNA cargo to the patient's T lymphocytes in vivo.[3] Once the mRNA is internalized by the T cells, it is translated by the cellular machinery, leading to the transient expression of the CD19 CAR on the surface of these T cells. This process effectively reprograms the patient's own T cells into functional CAR-T cells directly within the body, obviating the need for external cell manipulation and expansion. The in vivo generation of CAR-T cells represents a significant potential advancement in the field, aiming to simplify treatment logistics, reduce costs, and possibly improve the safety profile of CAR-T therapy.

The company's name, "MagicRNA," and its explicit mission [4] firmly place it within the dynamic and rapidly advancing field of RNA therapeutics. HN-2301 is not merely a standalone product but also serves as a vanguard candidate for validating the capabilities of their EnC-LNP platform for in vivo cell engineering. The clinical performance of HN-2301 will therefore be a critical determinant for the future prospects of this platform technology across a wider array of potential RNA-based therapies. The choice of SLE and MG as initial target indications [2] is also strategically astute. These are serious autoimmune diseases with substantial unmet medical needs, and the role of B-cells in their pathogenesis is well-documented. Compared to the crowded oncology space for CAR-T therapies, these autoimmune indications may offer a more streamlined path to demonstrating clinical proof-of-concept and addressing specific, underserved patient populations.

D. Table: Summary of HN-2301 Key Characteristics

Characteristic	Description	Reference(s)
Official Name	HN-2301	1
Alternative Names	HN 2301, HN2301	1
Developer	Shenzhen MagicRNA Biotechnology Co., Ltd.	1
Therapeutic Modality	In vivo generated CAR-T cell therapy (mRNA-based)	2
Mechanism of Action (brief)	EnC-LNP delivery of CD19 CAR mRNA to T cells, leading to in vivo CAR-T generation and B cell depletion.	2
Key Delivery Technology	Engineered Cell-targeted Lipid Nanoparticle (EnC-LNP) platform.	3
Lead Target Indications	Systemic Lupus Erythematosus (SLE), Myasthenia Gravis (MG).	1
Current Highest Development Stage	Early Clinical (Phase 2 for MG, Investigator-Initiated Trial for SLE).	1

III. Mechanism of Action and Scientific Rationale

A. In Vivo Generation of CD19-Targeting CAR-T Cells

The fundamental mechanism of HN-2301 revolves around the in vivo production of CAR-T cells.[3] This is accomplished through the systemic administration of HN-2301, which consists of an mRNA sequence encoding a CAR specific for the CD19 antigen. This mRNA payload is encapsulated within Shenzhen MagicRNA's proprietary EnC-LNPs, which are engineered to selectively target and deliver the mRNA to the patient's T lymphocytes directly within the body.[3]

Upon successful delivery and internalization of the LNP by a T cell, the mRNA is released into the cytoplasm. The T cell's own translational machinery then synthesizes the CD19-CAR protein, which is subsequently expressed on the T cell surface. This process effectively transforms the patient's native T cells into CD19-directed CAR-T cells. These newly generated CAR-T cells are then capable of recognizing the CD19 protein, which is predominantly expressed on B cells. The interaction between the CAR and the CD19 antigen on B cells triggers the activation of the CAR-T cell, leading to the initiation of an effector immune response. This response culminates in the cytotoxic killing of the targeted CD19-expressing B cells.[2] AdisInsight describes the mechanism as "Immunologic cytotoxicity; T lymphocyte replacements," where "replacements" likely alludes to the in vivo generation or functional augmentation of T cells to become cytotoxic effectors.[2]

B. Rationale for Targeting CD19 in Autoimmune Diseases (SLE and MG)

CD19 is a transmembrane protein that is expressed on the surface of B lymphocytes throughout most stages of their development, from early pro-B cells to mature B cells and antibody-secreting plasma blasts, though it is typically absent on hematopoietic stem cells and long-lived plasma cells.[14] This expression profile makes CD19 an attractive target for therapies aimed at depleting the B cell lineage.

In a range of autoimmune diseases, including SLE and MG, B cells play a significant pathogenic role. Autoreactive B cells can produce autoantibodies that target self-antigens, leading to immune complex formation, complement activation, and tissue damage. Beyond autoantibody production, B cells also contribute to autoimmune pathology by presenting autoantigens to T cells and by secreting pro-inflammatory cytokines, thereby perpetuating the inflammatory cycle and contributing to disease manifestations.

The therapeutic strategy of HN-2301 is to leverage the in vivo generated CD19 CAR-T cells to achieve a comprehensive depletion of the CD19-expressing B cell population. By eliminating these B cells, HN-2301 aims to remove the primary source of pathogenic autoantibodies and disrupt the B cell-mediated inflammatory processes that drive these autoimmune conditions. The ultimate goal is to induce an "immune reset," a state where the pathogenic B cell compartment is eliminated, allowing for the potential repopulation of the immune system with new, naive B cells that are hopefully tolerant to self-antigens.[3] This concept of an immune reset via B cell depletion is a shared rationale among several companies developing CD19-targeted therapies for autoimmune disorders, including those working with ex vivo CAR-T cells.[15]

C. The EnC-LNP Delivery Platform

The EnC-LNP platform is the technological cornerstone of HN-2301, enabling the in vivo generation of CAR-T cells. The "EnC" designation likely signifies "Engineered Cell-targeted," highlighting the platform's capability for precise delivery of its mRNA payload.[3]

Shenzhen MagicRNA has developed this platform based on a proprietary library of ionizable lipids. Ionizable lipids are crucial LNP components that are typically neutral at physiological pH, which aids in stability and reduces toxicity during circulation. However, upon endocytosis into target cells, they become positively charged in the acidic environment of the endosome. This charge switch is thought to facilitate the disruption of the endosomal membrane and the release of the mRNA payload into the cytoplasm, where it can be translated. The company reports possessing the largest ionizable aminolipid library in China with independent intellectual property rights, and their core lipid, ILB-3132, is noted as being commercially available.[3]

The "cell-targeted" aspect of the EnC-LNP platform is critical. While standard LNPs often exhibit passive accumulation in the liver due to interactions with apolipoprotein E and uptake by hepatocytes, achieving specific targeting to other cell types, such as T lymphocytes, requires sophisticated engineering. This typically involves modifying the LNP surface with specific ligands (e.g., antibodies, antibody fragments, or small molecules) that bind to receptors expressed on the target T cells, thereby promoting selective uptake. The ability of the EnC-LNP platform to effectively and selectively deliver mRNA to T cells in vivo is fundamental to the mechanism of action and therapeutic potential of HN-2301. The preclinical NHP data showing B-cell depletion [3] provide initial evidence that this targeted delivery to T cells is functional, leading to the generation of effective CAR-T cells.

D. Advantages and Rationale of the In Vivo mRNA-LNP Approach for CAR-T in Autoimmunity

The development of in vivo mRNA-LNP CAR-T therapies like HN-2301 is driven by the prospect of overcoming several limitations associated with traditional ex vivo viral vector-based CAR-T cell therapies, particularly in the context of autoimmune diseases:

1. Transient CAR Expression and Enhanced Safety: mRNA is inherently a transient molecule within the cell; its expression typically lasts for several days to a week or two before it is degraded. Consequently, CAR expression driven by mRNA is not permanent, unlike CARs delivered by viral vectors that integrate into the host cell's genome.[15] This transient expression is often viewed as a significant safety advantage. For autoimmune diseases, where the goal is often to modulate or reset the immune system rather than achieve complete and permanent ablation of a cell lineage (as in cancer), prolonged CAR-T persistence and activity might be unnecessary or even detrimental. Transient CAR expression could reduce the risk of long-term B-cell aplasia and associated chronic immunosuppression, as well as potentially mitigate the severity and duration of side effects like CRS and ICANS.
2. Simplified Manufacturing, Cost-Effectiveness, and Accessibility: The in vivo approach eliminates the complex, patient-specific, and expensive ex vivo manufacturing process. This process involves apheresis, T-cell isolation, viral transduction, cell expansion, and cryopreservation, all of which require specialized facilities and significant time. HN-2301, as an LNP-encapsulated mRNA, could potentially be manufactured as an "off-the-shelf" product, dramatically reducing production costs, shortening the time from diagnosis to treatment, and making the therapy accessible to a broader patient population and in more diverse healthcare settings.[15]
3. Reduced Immunogenicity: LNPs are generally considered to have lower immunogenicity compared to viral vectors, which can elicit host immune responses that may limit efficacy or cause adverse effects.[16]
4. Potential for Re-dosing: The transient nature of mRNA-driven CAR expression and the potentially improved safety profile of LNP-based delivery may allow for re-dosing or multiple treatment cycles if required to achieve or maintain the desired therapeutic effect. This offers flexibility in tailoring treatment to individual patient needs.
5. Avoidance of Lymphodepleting Chemotherapy: A significant burden of ex vivo CAR-T therapy is the requirement for pre-treatment with lymphodepleting chemotherapy (e.g., fludarabine and cyclophosphamide). This conditioning regimen creates "space" for the infused CAR-T cells to expand and persist but is associated with significant toxicities, including myelosuppression and an increased risk of infections. In vivo CAR-T generation approaches, particularly those that can efficiently modify T cells without requiring prior lymphodepletion, would represent a major advancement in terms of safety and patient convenience.[15] While not explicitly stated for HN-2301, this is a general aspiration for the field.

The successful in vivo delivery of mRNA to T cells via the EnC-LNP platform is the linchpin of HN-2301's mechanism. Should this platform prove efficient and specific in clinical settings, it would not only validate HN-2301 but also open avenues for Shenzhen MagicRNA to develop other in vivo cell engineering therapies or RNA-based therapeutics by simply changing the mRNA payload or potentially the LNP's targeting moiety. This platform's potential for modularity is a significant strategic asset.

IV. Preclinical Development and Findings

A. Overview of Preclinical Program

HN-2301 has successfully navigated the preclinical candidate (PCC) selection process and, according to recent updates, is currently in the Investigational New Drug (IND)-enabling phase of development.[3] This progression indicates that HN-2301 has demonstrated a sufficiently promising profile in terms of in vitro and in vivo efficacy, preliminary safety, and manufacturability to justify its advancement towards human clinical trials. The IND-enabling stage involves a series of specific studies required by regulatory authorities to assess the safety and pharmacology of a drug before it can be tested in humans.

B. Efficacy in Animal Models

The preclinical development of HN-2301 has included evaluations in relevant animal models to establish proof-of-concept for its in vivo CAR-T generation mechanism and therapeutic potential in autoimmune settings.

• Non-Human Primates (NHPs): Studies in NHPs are a critical step in the preclinical validation of novel cell and gene therapies due to their physiological and immunological similarity to humans. For HN2301, NHP studies have reportedly demonstrated the successful in vivo reprogramming of T cells to express the CD19 CAR following administration of the EnC-LNP encapsulated mRNA. Crucially, these in vivo generated CAR-T cells were shown to induce B cell depletion not only in the peripheral blood but also within tissues.[3] The ability to achieve B cell depletion in relevant tissues (such as lymphoid organs or sites of inflammation) is a more stringent and compelling indicator of potential therapeutic efficacy for systemic autoimmune diseases compared to solely observing effects in circulation. This finding supports the notion that the EnC-LNP platform can effectively deliver its payload to T cells, and that the resultant CAR-T cells can traffic and exert their effector functions in tissue compartments.
• Systemic Lupus Erythematosus (SLE) Mouse Models: To assess efficacy in a disease-relevant context, HN2301 was evaluated in murine models of SLE. These studies have reportedly verified that administration of HN2301 led to therapeutic effects.[3] While the specific parameters demonstrating this therapeutic effect (e.g., reduction in autoantibody levels, amelioration of kidney damage, improved survival) are not detailed in the currently available information, the confirmation of a "therapeutic effect" is a positive preclinical milestone.

C. Key Presentations and Publications

A significant public disclosure of detailed preclinical data for HN-2301 is anticipated at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, scheduled for May 13-17, 2025, in New Orleans, LA.

• Shenzhen MagicRNA Biotechnology has announced the acceptance of an abstract for a poster presentation that will feature HN2301 data.[4]
• Poster Title: "A Vivo CAR T Producer (HN2301) for Autoimmune Disease Enables B cell Depletion in NHP Tissues and Performs Therapeutic Effect in an SLE Mouse Model".[5]
• Abstract Number: 794.[5]
• Session Date/Time: May 13, 2025, 6:00 PM – 7:30 PM (Central Time Zone, UTC-5).[5]

This presentation is poised to be a pivotal event for HN-2301, as it is expected to provide the scientific community with the first comprehensive look at the preclinical data package supporting its in vivo efficacy and mechanism of action. The details from this poster, once available, will be crucial for a more thorough assessment of HN-2301's preclinical promise and for contextualizing the design and expectations for its ongoing and upcoming clinical trials. The current information is largely high-level; the specifics regarding the magnitude and duration of B cell depletion, CAR-T cell kinetics, safety profile in NHPs, and quantitative measures of efficacy in SLE models will be of great interest. The fact that HN-2301 is already in the IND-enabling stage [3] while these results are being prepared for presentation suggests a degree of confidence within the company regarding the robustness of the preclinical findings.

D. Table: Summary of Key Preclinical Efficacy Data for HN-2301 (Anticipated from ASGCT 2025 Poster Abstract 794)

Note: The following table is predictive, based on the poster title and standard preclinical reporting. Actual data will depend on the content of the ASGCT 2025 presentation.

Parameter	Non-Human Primates (NHP)	SLE Mouse Model
HN2301 Intervention	Dose(s) and regimen to be detailed.	Dose(s) and regimen to be detailed.
CAR-T Generation	Confirmation of in vivo CD19 CAR expression on T cells. Kinetics and persistence.	Confirmation of in vivo CD19 CAR expression on T cells.
B Cell Depletion (Blood)	Extent (%) and duration of CD19+ B cell depletion in peripheral blood.	Extent (%) and duration of CD19+ B cell depletion in peripheral blood.
B Cell Depletion (Tissue)	Extent (%) of CD19+ B cell depletion in relevant tissues (e.g., lymph nodes, spleen).	Assessment of B cell infiltration in target organs (e.g., kidneys, spleen).
Efficacy Markers (SLE)	N/A	Reduction in autoantibody titers (e.g., anti-dsDNA), improvement in proteinuria, amelioration of glomerulonephritis, clinical score improvement, survival.
Safety/Tolerability	General health, cytokine release markers, liver function tests, hematological parameters.	General health, cytokine release markers, organ toxicity assessment.

V. Clinical Development Program

A. Overview

HN-2301 is transitioning from preclinical research into early-phase clinical development, with an initial focus on autoimmune diseases where B cell pathogenesis is well-established. Two key clinical trials have been identified for HN-2301: NCT06801119, targeting patients with Systemic Lupus Erythematosus (SLE), and NCT06965309, targeting patients with Myasthenia Gravis (MG). Both trials are investigator-initiated and are being conducted in China.[2]

B. HN-2301 for Systemic Lupus Erythematosus (SLE)

• Trial Identifier and Title: The trial is registered under NCT06801119. Its title is reported as "Efficacy and Safety of HN2301 in Refractory Systemic Lupus Erythematosus (SLE)" or, more descriptively, "Dose-escalation Study to Assess the Safety, Tolerability, and Preliminary Efficacy of HN2301 in Patients with Relapsing and Refractory Systemic Lupus Erythematosus".[10]
• Study Design: This is an investigator-initiated, prospective, open-label, single-group assignment, exploratory clinical trial. A key feature of the design is dose escalation, with three distinct dose groups planned to identify a safe and potentially effective dose range.[10]
• Objectives: The primary objectives are to evaluate the safety and tolerability of HN2301 injection in patients with relapsing and refractory SLE, and to gather initial data on its efficacy.[10] Specific primary and secondary endpoints (e.g., incidence of adverse events, changes in SLE Disease Activity Index 2000 (SLEDAI-2K), B cell counts, autoantibody levels, pharmacokinetic parameters) are not fully detailed in the provided snippets but would be standard for such a trial. Information from Synapse indicates the study aims to "Assess the Safety, Tolerability, and Preliminary Efficacy".[17]
• Patient Population: The trial aims to enroll adult patients (aged 18-69 years) diagnosed with SLE according to the 2019 European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) classification criteria. Eligible patients must have relapsing and refractory disease, defined by a SLEDAI-2K score of ≥6 points despite receiving stable standard-of-care treatment for at least 8 weeks.[11]
• Key Eligibility Criteria:
• Inclusion: Documented history of SLE for at least 6 months; positive serological markers (ANA, and/or anti-dsDNA, and/or hypocomplementemia); adequate organ function (bone marrow, coagulation, cardiopulmonary, liver, kidney).[11]
• Exclusion: Active significant infections (e.g., HBV, HCV, HIV, TB); history of major organ transplantation; pregnancy or breastfeeding; receipt of any other mRNA-LNP product or other LNP medications within the past two years; severe cardiovascular diseases; recent live vaccine administration.[11] The exclusion of prior LNP-based therapies is noteworthy and may reflect a desire to avoid potential confounding factors related to LNP immunogenicity or altered pharmacokinetics from previous LNP exposures.
• Planned Enrollment: Approximately 9 patients are planned for enrollment.[11]
• Intervention: HN2301 administered via parenteral injection.[2]
• Status and Timelines: As of early 2025, the trial status appears to be transitioning from "Not yet recruiting" to "Recruiting." HT Syndication reported a planned start date of February 2025.[10] AdisInsight noted clinical trial activity as of March 25, 2025.[2] Ozmosi and Synapse listed the trial as "Recruiting" in May 2025.[1] The estimated primary completion date is December 31, 2025.[1]
• Sponsor: Shenzhen MagicRNA Biotechnology Co., Ltd. is the sponsor.[10]
• Locations: The trial is being conducted in China.[2] Specific sites are not detailed in the provided snippets.

C. HN-2301 for Myasthenia Gravis (MG)

• Trial Identifier and Title: This trial is registered under NCT06965309 and is titled "Evaluating the Safety and Efficacy of HN2301 Injection in Patients with Refractory Myasthenia Gravis".[12]
• Phase: This is designated as a Phase 2 study.[12]
• Study Design: Similar to the SLE trial, this is a prospective, open-label, single-group assignment, exploratory clinical trial.[12] It is also investigator-initiated.[18]
• Objectives: The primary goals are to evaluate the safety and initial efficacy/effectiveness of HN2301 injection in treating patients with refractory MG.[12] Specific endpoints are not detailed but would likely include safety assessments, changes in MG-specific clinical scores (e.g., MG-ADL, QMG), B cell counts, autoantibody levels (AChR, MuSK), and PK/PD markers.
• Patient Population: The trial will enroll patients with a confirmed diagnosis of refractory generalized MG, who are positive for acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) antibodies, and have shown a poor response or lack of efficacy with standard therapies. Standard therapies include corticosteroids and immunosuppressants (e.g., azathioprine, mycophenolate mofetil, tacrolimus, cyclosporine A, cyclophosphamide), or rituximab.[12]
• Key Eligibility Criteria:
• Inclusion: Expected survival time greater than 12 weeks; adequate bone marrow, coagulation, cardiopulmonary, liver, and renal function.[12]
• Exclusion: History of significant cardiovascular conditions within 6 months prior to screening; $\geq$Grade 2 bleeding events within 30 days or requiring long-term anticoagulation; receipt of live vaccination within 30 days; severe central nervous system diseases.[12]
• Planned Enrollment: 9 participants are planned for this study.[12]
• Intervention: HN2301 administered via injection.[12]
• Status and Timelines: As of May 2025, the trial was reported as "Not yet recruiting" by Ozmosi and TrialScreen.[1] However, Veeva indicated it "Begins enrollment this month" (potentially May 2025) [18], and AdisInsight noted that Shenzhen MagicRNA plans a dose-escalation study for MG with an event date of May 11, 2025.[2] The estimated primary completion date is May 31, 2026 [1], with an overall estimated study completion date of December 1, 2026.[12]
• Sponsor: Shenzhen MagicRNA Biotechnology Co., Ltd..[18]
• Locations: Specific trial locations are not provided in the current snippets.

The concurrent initiation of these two early-phase trials in distinct but related B-cell mediated autoimmune diseases underscores Shenzhen MagicRNA's strategic focus on this therapeutic area for HN-2301. The small, exploratory nature of these trials, with a primary emphasis on safety and preliminary efficacy signals, is characteristic of first-in-human studies for novel therapeutic modalities. The dose-escalation design is crucial for identifying an optimal therapeutic window.

D. Safety and Tolerability (Overall Anticipated)

As HN-2301 clinical trials are in very early stages, no human safety and tolerability data are available in the provided research material. Preclinical safety assessments in NHP and mouse models are alluded to in the title of the ASGCT 2025 poster [5], but specific details are pending public disclosure.

Theoretically, the in vivo mRNA-LNP approach for CAR-T generation carries the potential for an improved safety profile compared to traditional viral vector-based ex vivo CAR-T therapies. These potential advantages include a reduced risk of severe Cytokine Release Syndrome (CRS) and Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) due to transient CAR expression, and the possibility of avoiding harsh lymphodepleting chemotherapy.[15] However, these benefits are currently theoretical for HN-2301 and must be rigorously evaluated in the ongoing clinical trials. Potential risks could include infusion-related reactions to the LNP formulation, on-target off-tumor effects (elimination of healthy B cells leading to hypogammaglobulinemia and infection risk), or unforeseen toxicities related to the specific LNP components or CAR construct.

E. Pharmacokinetics (Overall Anticipated)

No clinical pharmacokinetic (PK) data for HN-2301 are currently available. The assessment of HN-2301's PK profile – including parameters such as absorption, distribution, metabolism, and excretion of the LNP and mRNA, as well as the persistence of CAR expression on T cells – will be a key objective of the early-phase clinical trials. Understanding these PK/PD (pharmacodynamic) relationships will be essential for optimizing dosing regimens and predicting therapeutic effects.

F. Table: Overview of HN-2301 Clinical Trials

Feature	NCT06801119 (SLE Trial)	NCT06965309 (MG Trial)
Phase	Investigator-Initiated Trial (Exploratory, Dose-Escalation)	Phase 2 (Exploratory, Dose-Escalation)
Target Indication	Relapsing and Refractory Systemic Lupus Erythematosus (SLE)	Refractory Myasthenia Gravis (MG)
Primary Objectives (General)	Evaluate safety, tolerability, and preliminary efficacy of HN2301.	Evaluate safety, tolerability, and initial efficacy/effectiveness of HN2301.
Key Patient Population	Adults (18-69 yrs) with active SLE (SLEDAI-2K ≥6) despite standard treatment.	Adults (18-80 yrs) with refractory gMG (AChR or MuSK Ab+), poor response to standard therapies.
Planned Enrollment	~9 patients	~9 patients
Intervention	HN2301 injection (parenteral), dose escalation.	HN2301 injection (parenteral), dose escalation.
Current Status (as of mid-2025)	Likely recruiting or very recently initiated.	Likely initiating recruitment.
Sponsor	Shenzhen MagicRNA Biotechnology Co., Ltd.	Shenzhen MagicRNA Biotechnology Co., Ltd.
Est. Primary Completion Date	December 31, 2025 1	May 31, 2026 1

The data from these early trials will be critical. While safety and tolerability are paramount, any signals of efficacy, such as meaningful B-cell depletion, reduction in autoantibody levels, or improvements in disease-specific activity scores (e.g., SLEDAI-2K, MG-ADL), would provide important proof-of-concept for HN-2301 and the EnC-LNP platform in humans. The consistency in pursuing autoimmune indications with a B-cell depletion strategy suggests a coherent development plan.

VI. Regulatory Status and Intellectual Property

A. Current Regulatory Status

As of the latest available information, HN-2301 has completed the Preclinical Candidate (PCC) stage and is in the IND-enabling phase of development, according to Shenzhen MagicRNA Biotechnology's communications.[3] The initiation of investigator-initiated clinical trials (NCT06801119 for SLE and NCT06965309 for MG) in China implies that requisite local regulatory and ethical approvals have been secured to proceed with these human studies.[2] This typically involves review by hospital ethics committees and, depending on the nature and sponsorship of the IIT, may involve oversight from the National Medical Products Administration (NMPA) of China.

There is no information in the provided materials to suggest that HN-2301 has received IND approval from the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA) for company-sponsored trials. The Ozmosi database explicitly lists "FDA Designation: None" for HN-2301.[1] Achieving formal IND approvals from these major regulatory bodies for company-sponsored pivotal trials will represent a significant future milestone, indicating broader regulatory acceptance of the preclinical data package and manufacturing controls.

B. Intellectual Property

Shenzhen MagicRNA Biotechnology has placed a strong emphasis on developing proprietary technology, particularly concerning its EnC-LNP delivery platform. The company has reportedly applied for multiple invention patents in the field of LNP delivery, with at least two patents stated as granted.[3] A cornerstone of their IP strategy appears to be their extensive, proprietary ionizable aminolipid library, which they claim is the largest in China with completely independent intellectual property rights.[3] These ionizable lipids, including the commercially available ILB-3132, are critical for the performance of LNPs in delivering mRNA payloads.

Directly supporting these claims, Dr. Zha Gaofeng, the founder of MagicRNA, is listed as an inventor on Chinese patent application CN114105799B, which pertains to a method for the synthesis of cationic lipids.[19] This patent is tangible evidence of their innovation in lipid chemistry, a key component of LNP technology.

While a patent (WO2018126369A1) related to "Humanized anti-cd19 antibody and use thereof with chimeric antigen receptor" was identified [14], its listed assignees are not Shenzhen MagicRNA Biotechnology. This patent likely represents background intellectual property relevant to the broader field of CD19 CARs rather than being specific to HN-2301's unique mRNA-LNP composition or its proprietary delivery system. It is important not to misattribute such general patents to MagicRNA's specific product unless a clear link is established.

The core IP and potentially the most significant value proposition for Shenzhen MagicRNA lie in its EnC-LNP platform, especially the novel ionizable lipids and the engineering for cell-specific targeting. Effective and safe in vivo targeted delivery of RNA therapeutics remains a formidable challenge in the field, and robust IP protection for a successful solution would provide a substantial competitive advantage. This proprietary platform is not only crucial for HN-2301 but also for the company's broader pipeline aspirations in RNA medicines. The development of an in-house lipid library and associated IP reflects a strategic move to ensure freedom to operate and to avoid dependence on third-party LNP technologies, which can often entail restrictive licensing terms and costs.

VII. Competitive Landscape

A. Overview of In Vivo CAR-T Therapy for Autoimmune Diseases

The application of CAR-T cell therapy to autoimmune diseases is a rapidly emerging and highly innovative frontier in medicine.[15] Building on the transformative success of CAR-T therapies in hematological malignancies, researchers and biopharmaceutical companies are now exploring their potential to induce deep and durable remissions in severe autoimmune conditions. The primary strategy involves targeting pathogenic immune cells, most commonly B cells (via antigens like CD19 or BCMA), with the goal of achieving an "immune reset" – a profound depletion of autoreactive cells followed by the repopulation of a healthier, self-tolerant immune system.[15]

A key technological trend within this space is the development of in vivo CAR-T generation methods, particularly those utilizing mRNA delivery via LNPs. This approach aims to overcome significant limitations of traditional ex vivo CAR-T therapies, which include complex and lengthy manufacturing processes, high costs, the need for patient lymphodepletion prior to infusion, and potential long-term safety concerns associated with permanent genetic modification by viral vectors.[15] The field is still in its early stages, but the promise of a more accessible, potentially safer, and scalable CAR-T therapy for autoimmune diseases is driving considerable research and investment.

B. Key Players and Technologies

Several companies are actively pursuing in vivo generated or mRNA-based CAR-T therapies, with some focusing on autoimmune indications. The landscape is dynamic, with various approaches to CAR design, payload (mRNA, oRNA), and delivery (targeted LNPs, viral vectors for in vivo transduction).

• Capstan Therapeutics: This company is a prominent player focusing on in vivo CAR-T therapy for autoimmune diseases. Their lead candidate, CPTX2309, is an anti-CD19 CAR-T therapy generated in vivo by delivering mRNA via their proprietary CellSeeker™ targeted Lipid Nanoparticle (tLNP) platform, which aims to preferentially transfect CD8+ T cells. Preclinical data in NHPs have demonstrated B cell depletion without requiring lymphodepletion. Capstan plans to advance CPTX2309 into clinical trials in mid-2025 for autoimmune indications.[15] Their approach shares similarities with HN-2301 in terms of in vivo mRNA-LNP delivery and CD19 targeting for autoimmunity.
• Moderna: Known for its mRNA vaccine technology, Moderna is collaborating with Carisma Therapeutics to develop in vivo CAR-macrophage (CAR-M) therapies. This collaboration leverages Moderna's mRNA/LNP platform and has recently expanded to include two undisclosed autoimmune targets.[16] While targeting macrophages instead of T cells, this represents a significant effort in in vivo LNP-mRNA based cell engineering for autoimmune conditions.
• Cartesian Therapeutics: Cartesian is developing mRNA-based CAR-T therapies, but their current approach involves ex vivo electroporation of mRNA into T cells, rather than in vivo LNP delivery. Their lead candidates, Descartes-08 (autologous mRNA BCMA CAR-T) and Descartes-15 (next-generation autologous mRNA BCMA CAR-T), are being investigated for autoimmune diseases, including MG and SLE. Descartes-08 has shown positive Phase 2b results in MG, demonstrating efficacy with outpatient administration and no lymphodepletion.[26] While the delivery method differs, Cartesian is a competitor in the autoimmune space using transient mRNA CAR-T technology.
• Orna Therapeutics: Orna is developing in vivo CAR-T therapies using circular RNA (oRNA) delivered by LNPs. Their lead oncology candidate targeting CD19, ORN-101, is expected to enter clinical trials by 2026.[23] oRNA is designed for greater stability compared to linear mRNA, which could influence the duration of CAR expression. While initially focused on oncology, their LNP-oRNA platform could potentially be applied to autoimmune diseases.
• BioNTech: A leader in mRNA technology, BioNTech is developing CAR-T therapies, such as BNT211 (an autologous CLDN6 CAR-T combined with a CLDN6-encoding CAR-T cell amplifying RNA vaccine, CARVac) for solid tumors.[30] While BNT211 itself is not for autoimmune disease and uses ex vivo generated CAR-T cells amplified by an mRNA vaccine, BioNTech's deep expertise in mRNA and LNP technology makes them a formidable potential entrant or partner in the in vivo CAR-T space for various indications, including autoimmunity. Snippet [22] suggests that insights from mRNA vaccine development, where BioNTech was a pioneer, are influencing CAR-T advancements.
• Other Companies in In Vivo CAR Development: The broader in vivo CAR field includes companies like Umoja Biopharma (lentiviral in vivo CAR-T, UB-VV111 for CD19+ cancers, licensed to AbbVie), Sanofi (three in vivo CAR-Ts in discovery), Interius Biotherapeutics (lentiviral in vivo CAR-T, INT2104 for CD20+ lymphoma), and EsoBiotec (autologous, ex vivo anti-BCMA CAR-T with an in vivo project, ESO-T01/PRG2402, partnered with Pregene Biopharma).[16] While many of these are focused on oncology or use viral vectors, they contribute to the evolving landscape and technological advancements in in vivo cell engineering.

The ability to achieve efficient, specific, and safe in vivo delivery to T cells is a primary hurdle and a key differentiating factor in this competitive field. Success for HN-2301 will depend on demonstrating robust clinical data that supports the efficacy and safety advantages of its EnC-LNP platform and transient mRNA CAR approach over existing treatments and other emerging in vivo therapies.

C. Differentiating Factors for HN-2301

Based on the available information, potential differentiating factors for HN-2301 include:

1. Proprietary EnC-LNP Platform: Shenzhen MagicRNA emphasizes its engineered cell-targeted LNP technology, including a proprietary ionizable lipid library.[3] If this platform demonstrates superior T-cell targeting efficiency, transfection rates, and a favorable safety profile in vivo compared to competitors' delivery systems, it would be a significant advantage.
2. Focused Autoimmune Indications: The initial clinical development is squarely focused on SLE and MG, two B-cell mediated autoimmune diseases with high unmet medical needs. This clear focus allows for tailored trial designs and potentially faster proof-of-concept in these specific populations.
3. Rapid Development Pace: The relatively quick progression from company inception to early clinical trials [3] suggests agility and a potentially efficient development engine, which could provide a temporal advantage if early data are positive.

The remarkable efficacy shown by ex vivo CD19 CAR-T therapies in inducing drug-free remissions in some autoimmune patients [15] has validated CD19 as a target and created substantial enthusiasm for B-cell depleting CAR-T approaches in autoimmunity. This existing proof-of-concept de-risks the biological target for companies like Shenzhen MagicRNA. The innovation and competitive differentiation for HN-2301, therefore, lie primarily in its method of delivery (in vivo mRNA-LNP) and the potential benefits this method offers in terms of safety, accessibility, and cost-effectiveness compared to traditional ex vivo approaches.

D. Table: Comparative Overview of Selected In Vivo/mRNA-based CAR Therapies for Autoimmune Diseases

Company	Candidate	Modality	Target Antigen	Key Autoimmune Indication(s)	Delivery System (if specified)	Highest Development Stage (Autoimmune)	Reference(s)
Shenzhen MagicRNA Biotechnology	HN-2301	In vivo mRNA-LNP CAR-T	CD19	SLE, MG	EnC-LNP (T-cell targeted)	Early Clinical (Phase 2/IIT)	1
Capstan Therapeutics	CPTX2309	In vivo mRNA-LNP CAR-T	CD19	Broad Autoimmune (SLE, Myositis etc.)	CellSeeker™ tLNP (CD8 T-cell pref.)	Preclinical (Clinical trials planned mid-2025)	15
Moderna/Carisma Therapeutics	Undisclosed	In vivo mRNA-LNP CAR-Macrophage (CAR-M)	Undisclosed	Two Autoimmune Targets	Moderna mRNA/LNP platform	Preclinical/Discovery	16
Cartesian Therapeutics	Descartes-08	Ex vivo mRNA CAR-T (autologous)	BCMA	MG, SLE, Pediatric Autoimmune	mRNA electroporation	Phase 2b (MG), Planned (SLE, Pediatric)	26
Cartesian Therapeutics	Descartes-15	Ex vivo mRNA CAR-T (autologous, next-gen)	BCMA	Autoimmune (following myeloma trial)	mRNA electroporation	Phase 1 (Myeloma)	26

Note: This table focuses on therapies with a clear autoimmune indication and utilizing mRNA or in vivo generation. Some companies have broader platforms applicable to autoimmunity but may have lead assets in oncology.

VIII. Discussion and Future Outlook

A. Synthesis of Findings

HN-2301, developed by Shenzhen MagicRNA Biotechnology, is an investigational therapeutic agent representing a novel approach in the treatment of autoimmune diseases. It is designed as an in vivo generated CD19-targeting CAR-T cell therapy, utilizing the company's proprietary Engineered Cell-targeted Lipid Nanoparticle (EnC-LNP) platform to deliver CD19 CAR-encoding mRNA directly to a patient's T cells. This strategy aims to transiently reprogram these T cells to identify and eliminate pathogenic CD19-expressing B cells, which are key drivers in many autoimmune conditions.

Preclinical data, particularly from studies in non-human primates and murine models of Systemic Lupus Erythematosus (SLE), have indicated successful in vivo CAR-T cell generation, leading to B cell depletion in both blood and tissues, and demonstrating therapeutic effects in disease models.[3] Building on this preclinical foundation, HN-2301 is advancing into early-phase clinical trials in China, specifically targeting SLE (NCT06801119) and Myasthenia Gravis (MG) (NCT06965309).[1] These initial human studies are designed primarily to assess safety, tolerability, and preliminary efficacy, including dose-finding.

B. Potential of HN-2301

The potential of HN-2301 is multi-faceted:

1. Addressing Unmet Needs: For patients with severe, refractory SLE and MG, current treatment options are often limited by partial efficacy, chronic administration, and significant side effects associated with long-term immunosuppression. HN-2301 offers the prospect of a therapy that could induce deep and potentially durable remissions, possibly after a limited course of treatment.
2. Improved Therapeutic Profile: Compared to traditional ex vivo CAR-T therapies, the in vivo mRNA-LNP approach of HN-2301 has the potential for an improved safety profile (due to transient CAR expression and avoidance of viral vectors), significantly reduced manufacturing complexity and cost, and greater patient accessibility as an "off-the-shelf" product.
3. Platform Validation: Successful clinical development of HN-2301 would serve as crucial validation for Shenzhen MagicRNA's EnC-LNP platform. This could unlock the potential for developing a broader pipeline of RNA-based therapeutics targeting different cell types or delivering various genetic payloads for a range of diseases.

The development of HN-2301 is emblematic of a broader shift in cell and gene therapy towards strategies that simplify treatment administration and manufacturing, aiming to make these potent therapies as manageable and accessible as conventional biologic drugs. If the complexities of in vivo delivery and precise cell engineering can be mastered, it could herald a new era for treating not only severe autoimmune diseases but potentially a wider array of conditions.

C. Current Challenges and Opportunities

Despite its promise, the development of HN-2301 faces several challenges:

• Clinical Validation: The foremost challenge is to demonstrate robust safety and compelling efficacy in human clinical trials. The transition from animal models to human subjects can present unforeseen hurdles.
• Optimizing In Vivo CAR-T Therapy: Key questions remain regarding the optimal dosing regimen, the potential need for re-dosing to maintain therapeutic effects with transient mRNA expression, the achievable depth and duration of B cell depletion, and the long-term nature of the "immune reset."
• Manufacturing and Scalability: While simpler than ex vivo methods, GMP-compliant manufacturing of high-quality mRNA and precisely formulated EnC-LNPs at scale will be crucial for later-stage development and commercialization.
• Competitive Environment: The field of in vivo cell and gene therapy, and specifically CAR-T for autoimmune diseases, is becoming increasingly competitive. Differentiation based on technological superiority and clinical outcomes will be essential.
• Specificity and Off-Target Effects: Ensuring highly specific targeting of T cells by the EnC-LNPs in vivo is critical to maximize efficacy and minimize potential off-target transfection of other cell types, which could lead to unintended side effects.

Opportunities for HN-2301 include:

• First-in-Class/Best-in-Class Potential: If early clinical data are strong, HN-2301 could establish a leading position in the niche of in vivo mRNA-CAR-T therapies for specific autoimmune indications like SLE or MG.
• Platform Expansion: Positive validation of the EnC-LNP platform through HN-2301 could attract partnerships and enable the development of further RNA therapeutics.
• Addressing Refractory Populations: Focusing on patients who have failed multiple standard therapies provides a clear unmet need and a population where even modest, safe improvements could be clinically significant.

Shenzhen MagicRNA's strategy of initiating investigator-initiated trials (IITs) in China [11] may be a cost-effective and rapid way to gather initial human proof-of-concept data. Positive outcomes from these IITs would significantly de-risk further investment and facilitate engagement with major regulatory agencies for broader, company-sponsored clinical development programs.

D. Potential Impact on Treatment Landscape

If successful, HN-2301 and similar in vivo CAR-T therapies could fundamentally alter the treatment paradigm for severe B-cell mediated autoimmune diseases. Instead of relying on chronic, broadly immunosuppressive drugs with cumulative toxicities, these therapies offer the potential for inducing deep and durable remissions, possibly with a limited number of treatments. This could translate into a significant improvement in quality of life for patients, reducing the long-term burden of disease and treatment.

The long-term success of therapies like HN-2301 will critically depend on the durability of the immune reset they induce. While transient CAR expression offers safety benefits, the resultant B cell depletion must be sufficiently profound and sustained to break the cycle of autoimmunity and allow for the repopulation of a healthy, self-tolerant B cell compartment. Failure to achieve this could lead to disease relapse and necessitate frequent re-treatments, potentially diminishing the advantages over existing chronic therapies. Careful monitoring of B cell kinetics and long-term clinical outcomes in the ongoing trials will be essential to address this aspect.

IX. Conclusion

HN-2301, an investigational in vivo CD19 CAR-T cell therapy developed by Shenzhen MagicRNA Biotechnology, represents a promising and innovative approach to treating B-cell mediated autoimmune diseases such as SLE and MG. Its core technology, involving the delivery of CD19 CAR-encoding mRNA via a proprietary Engineered Cell-targeted Lipid Nanoparticle (EnC-LNP) platform, aims to offer a safer, more accessible, and potentially more cost-effective alternative to traditional ex vivo CAR-T therapies.

Preclinical studies in NHPs and murine SLE models have provided initial proof-of-concept, demonstrating in vivo CAR-T generation, B cell depletion, and therapeutic effects. The therapy is now advancing into early-phase clinical trials in China, which will be critical in evaluating its safety, tolerability, and preliminary efficacy in human subjects. The upcoming presentation of detailed preclinical data at the ASGCT 2025 meeting is keenly anticipated and will provide further insights into its foundational science.

The success of HN-2301 hinges on the clinical validation of its EnC-LNP platform's ability to efficiently and specifically target T cells in vivo, the safety profile of transiently expressed CARs, and the durability of the induced immune reset. If these aspects are favorably demonstrated, HN-2301 could significantly impact the treatment landscape for severe autoimmune diseases and position Shenzhen MagicRNA as a notable player in the field of advanced RNA therapeutics and in vivo cell engineering. Continued monitoring of clinical trial progress and forthcoming data disclosures will be essential to fully ascertain the therapeutic potential of HN-2301.

X. References

1 Ozmosi | HN-2301 Drug Profile.

2 AdisInsight | HN 2301.

11 Veeva Clinical Trials | Efficacy and Safety of HN2301 in Refractory Systemic Lupus Erythematosus (SLE).

3 Shenzhen MagicRNA Biotechnology Co., Ltd. | Official Website.

6 Lloyds Energy | Tech trio joins forces on advancing adoption of LNG as fuel.

12 TrialScreen | A Phase 2 interventional study of HN2301 in refractory myasthenia gravis.

11 Veeva Clinical Trials | Efficacy and Safety of HN2301 in Refractory Systemic Lupus Erythematosus (SLE). 11

8 Shenzhen MagicRNA Biotechnology Co., Ltd. | About Us - Our Mission.

4 Shenzhen MagicRNA Biotechnology Co., Ltd. | Official Website (Homepage details).

7 Tracxn | Hongxin Biotechnology - About the company. (Note: Hongxin Biotechnology appears to be an alternative name or closely related entity to Shenzhen MagicRNA Biotechnology)

9 SBS Genetech | MagicRNA Completed Angel Round of Financing.

10 HT Syndication | Clinical Trial: Efficacy and Safety of HN2301 in Refractory Systemic Lupus Erythematosus (SLE).

5 Shenzhen MagicRNA Biotechnology Co., Ltd. | ASGCT 28TH ANNUAL MEETING (HN2301 Poster Announcement).

4 Shenzhen MagicRNA Biotechnology Co., Ltd. | Official Website (News/Events section confirming ASGCT 2025).

16 AdvancingRNA | mRNA: Empowering Engineered In Vivo Cell Therapies.

11 Veeva Clinical Trials | Efficacy and Safety of HN2301 in Refractory Systemic Lupus Erythematosus (SLE). 11

10 HT Syndication | Clinical Trial: Efficacy and Safety of HN2301 in Refractory Systemic Lupus Erythematosus (SLE). 10

18 Veeva Clinical Trials | Efficacy and Safety of HN2301 in Refractory Myasthenia Gravis(MG).

33 MeiraGTx | MeiraGTx Announces Presentation of Four Posters at American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting. (General ASGCT, not specific to HN2301)

5 Shenzhen MagicRNA Biotechnology Co., Ltd. | ASGCT 28TH ANNUAL MEETING.5

13 Shenzhen MagicRNA Biotechnology Co., Ltd. | ASGCT 28TH ANNUAL MEETING.5

20 PMC | Cellular Immunotherapies for Autoimmune Diseases: A New Era in Treatment.

4 Shenzhen MagicRNA Biotechnology Co., Ltd. | Official Website (Pipeline and Technology). 3

5 Shenzhen MagicRNA Biotechnology Co., Ltd. | ASGCT 28TH ANNUAL MEETING.5

21 PMC | Mechanistic Evaluation of Anti-CD19 CAR-T Cell Therapy Repurposed for Systemic Lupus Erythematosus Using a Quantitative Systems Pharmacology Model.

4 Shenzhen MagicRNA Biotechnology Co., Ltd. | Official Website (Pipeline and Technology). 3

19 Tandfonline | Novel mRNA adjuvant ImmunER enhances prostate cancer tumor-specific immunity via STING-IFN-I pathway. (Mentions Zha Gaofeng patent)

13 Shenzhen MagicRNA Biotechnology Co., Ltd. | ASGCT 28TH ANNUAL MEETING.5

22 PMC | In Vivo Engineered CAR-T Cell Therapy: Lessons Built from COVID-19 mRNA Vaccines.

23 OncologyPipeline | In vivo CAR-T gains traction.

16 AdvancingRNA | mRNA: Empowering Engineered In Vivo Cell Therapies. 16

24 Fierce Biotech | Carisma, Moderna expand cell therapy collab to autoimmune area.

25 BusinessWire | Capstan Therapeutics to Participate in Upcoming Scientific Conferences and Present New Preclinical Data in Support of Lead Anti-CD19 In Vivo CAR-T Candidate, CPTX2309.

15 Capstan Therapeutics | Science & Platform.

26 NIH RePORTER | Manufacturing RNA-based CAR T cells to combat autoantibody-associated autoimmune disorders (AAAD). (Cartesian Therapeutics)

27 Cartesian Therapeutics | Pipeline.

14 Google Patents | WO2018126369A1 - Humanized anti-cd19 antibody and use thereof with chimeric antigen receptor.

4 Shenzhen MagicRNA Biotechnology Co., Ltd. | Official Website (Pipeline and Technology). 3

5 Shenzhen MagicRNA Biotechnology Co., Ltd. | ASGCT 28TH ANNUAL MEETING.5

34 DrugBank Online | Systemic Lupus Erythematosus (DBCOND0027960). (General SLE endpoints)

17 Synapse Patsnap | Shenzhen Hongxin Biotechnology Co., Ltd. (Lists NCT06801119)

11 Veeva Clinical Trials | Efficacy and Safety of HN2301 in Refractory Systemic Lupus Erythematosus (SLE). 11

28 Capstan Therapeutics | Capstan Therapeutics Presents Preclinical Data on Lead In Vivo CAR-T Candidate, CPTX2309, at American College of Rheumatology (ACR) Convergence 2024.

29 Synapse Patsnap Blog | Capstan Therapeutics Presents Preclinical Findings on CAR-T Candidate CPTX2309 at ACR Convergence 2024.

30 BioNTech Press Release | BioNTech Presents Positive Phase 1/2 Data Update for CAR-T Cell Therapy Candidate BNT211 in Advanced Solid Tumors at ESMO Congress 2023.

31 BioSpace | 5 CAR T Cell Therapies With Autoimmune Readouts in 2025. (General landscape)

4 Shenzhen MagicRNA Biotechnology Co., Ltd. | Official Website (Pipeline and Technology). 3

17 Synapse Patsnap | Shenzhen Hongxin Biotechnology Co., Ltd. (Lists NCT06801119, general endpoint context)

11 Veeva Clinical Trials | Efficacy and Safety of HN2301 in Refractory Systemic Lupus Erythematosus (SLE). 11

32 BioSpace | Capstan Therapeutics to Participate in Upcoming Scientific Conferences... (CPTX2309).

28 Capstan Therapeutics Press Release | Capstan Therapeutics Presents Preclinical Data... (CPTX2309 at ACR 2024).

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