Pipeline
In vivo CAR-T
CAR-T therapy is an advanced cell therapy that introduces a chimeric antigen receptor (CAR) into a patient’s immune T cells to precisely target and eliminate cancer cells. Ex vivo CAR-T involves isolating T cells from a patient’s blood, modifying them through complex genetic engineering, and reinfusing them. It has dramatically raised survival rates for pediatric acute lymphoblastic leukemia to around 60%, earning the name “the dream cancer therapy.” Yet significant challenges remain to be addressed.
MVRIX is developing an in vivo CAR-T therapy, MIC-001, based on its proprietary Navibody platform—an mRNA@LNP targeted delivery technology that precisely targets CD3 expressed on T cells.
Our goal is to significantly expand the therapeutic reach of CAR-T and improve accessibility beyond the limits of conventional ex vivo treatments.
Current challenges and the breakthrough
CAR-T therapy is an innovative cancer treatment that offers the possibility of cure for patients with relapsed or refractory blood cancers. By using the patient’s own T cells, it minimizes immune rejection, while residual CAR-T cells in the body continue to monitor for cancer cells and help prevent relapse. However, the current ex vivo approach faces major limitations in treatment accessibility. With in-vivo CAR-T technology, we aim to overcome these challenges and bring the benefits of CAR-T therapy to more patients.
Challenges that Ex-vivo CAR-T face
Long and costly treatment
Complex development requiring dedicated facilities
Patient risks
(lymphodepletion and other side effects)
Ex vivo CAR-T involves isolating a patient’s T cells, inserting the CAR gene, and expanding the modified cells outside the body before reinfusion. This process requires dedicated manufacturing facilities and long development timelines, resulting in high treatment costs and limited accessibility that place significant physical and financial burdens on patients.
In vivo CAR-T
Short treatment time and 1/10 the cost
Simple process, customization
Reduced risk (Combination therapy and repeat dosing)
In vivo CAR-T therapy generates CAR genes directly inside the body, enabling true off-the-shelf treatment.
This approach shortens treatment time and reduces costs, allowing patients to receive care at local hospitals without the need for specialized facilities. It also lowers patient burden, making combination therapy and repeated dosing more feasible.
Limitations of Conventional Ex vivo CAR-T
Complex, facility-intensive manufacturing
The 'harvesting → engineering → reinfusion' process of ex vivo CAR-T faces logistical limitations
Months-long "vein-to-vein" time
The complexity of the process and logistical constraints can cause critical patients to miss their therapeutic window
Prohibitive costs (~500M KRW/treatment)
The high treatment cost limits access to only a small number of patients
Serious safety concerns
Unpredictable side effects such as CRS and ICANS
Advantages of In-vivo CAR-T Therapy
Simplified development and administration
Single-injection administration of standardized drug enables treatment to begin within days
Immediate treatment
'Off-the-shelf' availability for on-demand treatment, allowing critical patients to begin treatment without delay
Significant cost reduction
Scalable manufacturing enables lower pricing and broader patient access
Improved safety profile
Reduced side effects via gradual in vivo activation
How to improve in vivo CAR-T
The true potential of in vivo CAR-T begins with precise delivery.
We are developing clinically viable in vivo CAR-T therapies through safe, scalable LNP-based delivery technology.
LNP
Safety
Low immunogenicity
No risk of genomic integration
Scalability
Scalable manufacturing(CMC)
Streamlined QC
Repeat dosing
Enables repeat dosing
Control and enhancement of effect
Viral Vector
Safety
Potential immunogenicity
Risk of insertional mutagenesis
Scalability
Complex manufacturing process
High cost and limited scalability
Repeat dosing
Limitations due to antibody formation
Potential for reduced efficacy
How to improve in vivo CAR-T
We consider the following factors essential for the commercialization of in-vivo CAR-T therapeutics.
T-cell targeting specificity
Since genetic material is administered directly into the body, precise delivery exclusively to T cells is essential.
Genome vector selection
We use LNPs for their high delivery efficiency, low immunogenicity, and suitability for large-scale production.
Genome selection
mRNA has a shorter expression duration compared to DNA, but offers greater safety with a significantly lower risk of secondary malignancies.
Repeat dosing safety
To enable personalized in-vivo CAR-T therapy, the design must allow for repeat dosing and other flexible strategies.
Precision targeting of in vivo CAR-T
In-vivo CAR-T with the Navibody platform is specialized for targeted delivery. Targeted delivery is an essential requirement for the commercialization of in-vivo CAR-T therapeutics.
Maximized therapeutic efficacy
The efficiency and durability of tumor clearance are proportional to the precision of targeted delivery.
Minimized side effects
If CARs are delivered and edited in non-target cells, they may cause unpredictable toxicity or immune disruption.
Prevention of cumulative toxicity
Genome vectors such as LNPs allow transient expression and repeat dosing, enabling more flexible regimens with improved safety.
Development Roadmap
With the Navibody platform, our technology can be applied across a broad range of therapeutics,
supported by proven versatility, stability, and scalable productivity.