Programmable immune medicines.Powered by mRNA.Delivered in vivo.
A programmable mRNA platform that creates immune medicines directly inside patients—eliminating the need for ex vivo cell manufacturing while enabling therapies across multiple diseases.
MSVClinical highlights & pipeline
3/3 responses in 1L HCC (MT-303). Additional patients dosing · initial data Q4 2026.
CRT-402 HREC-approved · FPI Q3 2026 · proof of mechanism Q3–Q4 2026 · global Ph2 SLE 2027.
No Gr3+ CRS · no DILIs · transient uncomplicated cytopenias · repeat dosing without steroid premeds.
Pipeline
Focus · Asset · TargetHREC-approved in Australia · FPI Q3 2026 · early efficacy Q3–Q4 2026. Complete lymph-node B-cell depletion at 0.25 mg/kg in NHP.
Extends to plasmablast-driven disease (SLE flare, ANCA vasculitis). IIT by YE26, Ph1 2027–28.
3/3 responses in 1L HCC — first in vivo CAR responses in a solid tumor. Additional data Q4 2026.
First multi-lineage in vivo CAR in a single product · CRT-401 FPI 1H27, data 2H27.
Displaces lentivirus with one-and-done mRNA-based gene delivery. In vivo POC Q1 2027.
Platform pillars
A single stack — mRNA, orientation-controlled tLNP, cell-selective CARs — powered for rapid clinical translation.
- Tunable depletion
- Dose-dependent depth of B-cell depletion enables controlled immune reset.
- Repeat dosing
- >20 infusions in a single patient; weekly regimen feasible in humans.
- Clinically validated
- mRNA and lipids with proven human safety across 51 dosed patients.
- No reactogenicity
- Engineered mRNA avoids IL-1β / TNF / IFN-α — no steroid premeds required.
- Orientation-controlled tLNP
- Site-specific DARPin conjugation drives 10× mRNA delivery at lower doses.
- Global GMP scale
- 5,000 m² in-house facility · 5 suites · pDNA, mRNA, LNP, fill/finish.
How it works
- 1Engineered mRNA — linear, non-pseudo-U; expresses CAR > 7 days without triggering IL-1β / TNF / IFN-α.
- 2Modular tLNP targeting — swap the site-specific DARPin to hit CD5, CD7 or CD8; same stack, new cell type, 10× uptake at lower dose.
- 3In vivo CAR — transient, tunable, redosable; complete B-cell depletion including lymph node.
Best-in-class in vivo dose
Dose for deep depletion (mesenteric LN), NHP — lower is better.
MT-303 — 1L HCC Data
GPC3 in vivo CAR-Myeloid — first in vivo CAR responses in a solid tumor in humans.
- Target · modality
- GPC3 · in vivo CAR-Myeloid (mRNA + tLNP)
- Setting
- 1L HCC in combination with atezolizumab + bevacizumab
- Dosing
- 0.015–0.1 mg/kg IV every two weeks · avg 4 doses (max 20)
- Safety
- No Grade 3+ CRS · no DILIs · transient uncomplicated neutropenia / thrombocytopenia
- Next
- 2–3 additional patients dosing by end of year · initial data Q4 2026
RetroT — Stable in vivo CAR
A next-generation, all-RNA genome-integration platform that enables durable, site-specific CAR insertion without double-strand breaks, viral vectors, or DNA templates — the one-and-done complement to our redosable mRNA CAR system.
Mechanism
- 1All-RNA payload — mRNA encodes CAR cargo plus engineered LINE-1 machinery, delivered by CD8-targeted tLNP.
- 2DSB-free integration — CRISPR-guided nickase + LINE-1 target-primed reverse transcription insert the transgene at a defined genomic locus.
- 3Stable CAR expression — engineered T cells retain antigen-dependent cytotoxicity, sustained activation, and durable tumor control.
Clinical translation
- Advantage
- Displaces lentivirus with one-and-done mRNA-based gene delivery
- Preclinical proof
- NSG leukemia xenograft: single infusion reduced tumor burden
- Safety profile
- Precise junction fidelity, stable transgene copy number, no partial insertions
- Milestone
- In vivo POC Q1 2027