Pipeline

nCode is advancing in vivo therapies designed by our AI platform, Synapse, to put the right protein in the right cell. Our two lead programs are shown below.

Program Indication selection Target ID Hit ID Lead selection Lead optimization In vitro and in vivo testing
In vivo CAR-T B-cell related autoimmune disease and hematology-oncology indications.
Lead optimization
Immunology: T-cell exhaustion rescue Immune oncology and CAR-T failure rescue.
Target ID

Both programs are preclinical, at pilot validation, with in vivo proof-of-concept data expected in 2027.

In vivo CAR-T

CAR-T therapy is powerful but held back by the cost and weeks-long process of engineering a patient's cells outside the body. nCode reprograms T cells inside the body with a single, off-the-shelf dose of Synapse-optimized mRNA, reaching more than fivefold CAR expression in target cells while staying quiet in off-target tissue such as the liver.

In vivo CAR-T: reprogramming T cells inside the body. Five steps — a single targeted dose; LNP plus Synapse mRNA; the T cell expressing CAR at more than fivefold expression; the T cell clearing its target cell; the mRNA self-clearing, transient and non-integrating. Cell-type selective by sequence: high expression in target T cells, suppressed in off-target tissue such as the liver.

Immunology: T-cell exhaustion rescue

In normal cancer defense and CAR-T indications, T-cells that should be fighting the disease become exhausted from over-stimulation. nCode is testing cell-type specific translating mRNA payloads to reactivate exhausted T-cells in vivo, restoring their disease modifying activity while avoiding anti-drug immunity and broad immune system activation.

T-cell exhaustion rescue: restoring immune function. Three steps — an exhausted, dim and dysfunctional T cell; a targeted mRNA-LNP reinvigorating the cell; the reinvigorated T cell clearing its target. Cell-specific mRNA acts only on the intended T-cell population, avoiding the toxicity of broad immune activation.