AI-driven Extrahepatic delivery platform

Engineer a patient's T cells into a cancer therapy — without taking them out of the body.

Aptavex's platform incorporates novel, AI-optimized cell-targeting binders into mRNA-lipid nanoparticles (mRNA-LNPs), so a single infusion finds circulating T cells directly and arms them in place — no apheresis, no ex vivo manufacturing, no cell-therapy cold chain.

StagePreclinical
ModalityBinder-LNP · mRNA
Target cellCirculating T lymphocytes
Funding pathSBIR, Grants, and Strategic Partnerships
Mechanism — single IV dose t = 0h
LNP + binder T cell (in vivo) CAR-T (armed) tumor cell
Systemic dose Selective uptake Transient arming
AI molecular design

AI-Driven Molecular Engineering Platform

Designing and optimizing targeting binders and therapeutic cargo sequences for next-generation precision RNA therapeutics. Aptavex is built as an AI-native gene delivery design company — the models are not an add-on to the chemistry, they are how the chemistry gets designed.

01 / Binder design

Generative binder design

Generative sequence models propose candidate binders and score them for predicted 3D structure and target affinity before a single molecule is synthesized, replacing slow empirical selection with computational search.

02 / Cargo optimization

mRNA cargo optimization

Codon usage, UTR architecture, and secondary structure are computationally tuned for translational efficiency, stability, and reduced innate immune activation — for every construct the platform delivers.

03 / Design-test-learn

Closed-loop iteration

Experimental binding and expression data feed back into the models after every design cycle, continuously improving prediction accuracy and compounding the platform's advantage over time.

The constraint

Ex vivo CAR-T works — the manufacturing doesn't scale.

Every approved CAR-T product is built the same way: collect a patient's T cells, ship them to a facility, engineer and expand them outside the body, then infuse them back. That process is what limits access.

01 / apheresis

A collection procedure, first

Every patient starts with leukapheresis before any therapy can be made — an added clinical step before treatment even begins.

02 / lead time

Weeks between draw and dose

Cells travel to a manufacturing site, are engineered and expanded in batch, then travel back — during which a fast-moving cancer keeps moving.

03 / logistics

A single-patient supply chain

Cryopreservation, chain-of-identity tracking, and dedicated manufacturing slots make each dose its own bespoke operation.

Aptavex Therapeutics — APM-LNP Extrahepatic Delivery Platform
The platform

Skip the manufacturing step. Arm the T cells where they already are.

Aptavex pairs a T-cell-selective cell-free novel binder with an mRNA-LNP carrying a CAR construct. The binder is the address label; the LNP is the delivery vehicle; the mRNA does the engineering — all inside the patient.

01

Select the targeting binder

An AI-designed, engineered binder sequence is optimized to adopt a stable three-dimensional structure that specifically recognizes a T cell surface marker. Acting as a molecular address label, the binder can be manufactured by chemical synthesis instead of cell culture, enabling faster, more scalable, and cost-effective production.

02

Incorporated into an mRNA-LNP using a proprietary labeling technology

The binder is displayed on the surface of a lipid nanoparticle encapsulating CAR-encoding mRNA, redirecting the particle's natural tropism away from the liver and toward circulating lymphocytes.

03

Dose intravenously, systemically

A single infusion circulates like any biologic. The targeting ligand concentrates uptake in the intended cell population instead of relying on passive, liver-dominant distribution.

04

Arm T cells in place

Once internalized, the mRNA is translated transiently, and the T cell expresses a chimeric antigen receptor on its own surface — becoming a tumor-seeking effector cell without ever leaving the bloodstream.

Beyond CAR-T

The same platform, one new targeting ligand away.

The mechanism doesn't depend on T cells specifically — it depends on the binder. Swap the targeting ligand for one selective to a different cell surface marker, and the same mRNA-LNP chassis can be redirected to other cell populations and other therapeutic payloads.

01 / HSC editing

In vivo HSC editing

Retarget the binder to hematopoietic stem cell markers to deliver gene-editing payloads directly, without myeloablative conditioning or ex vivo cell handling.

02 / Brain delivery

Brain delivery

A binder selected against a blood-brain-barrier transport receptor could carry the same mRNA-LNP payload across the BBB non-invasively, without intrathecal dosing.

03 / CNS diseases

Neurodegenerative disease

Paired with neuronal or glial cell-surface targeting, brain delivery extends the same systemic-dosing approach to CNS-directed mRNA therapeutics.

04 / Immune cells

Other immune effector cells

NK cells, macrophages, and other lymphocyte populations are addressable the same way, opening a path to additional in vivo engineered-cell therapies.

Why a cell-free novel binder, not antibodies

The targeting ligand is what makes systemic dosing feasible.

Antibody-conjugated LNPs have shown selective delivery is possible. Our cell-free novel binder keeps that principle while changing the economics and immunology of the ligand itself.

Antibody-conjugated ligand

  • Produced in mammalian cell culture
  • Batch-to-batch glycosylation variability
  • Can trigger anti-drug immune responses
  • Conjugation chemistry adds a bioprocess step
  • Large size can limit LNP loading density
  • Complex, capital-intensive manufacturing scale-up

Aptavex binder ligand

  • Can be produced using a cell-free synthesis system
  • Defined sequence, lot-to-lot consistency
  • Low intrinsic immunogenicity
  • Controllable targeting ligand incorporation
  • Small footprint, high ligand density per particle
  • Streamlined, highly scalable manufacturing
The team

Formulation science, RNA biology, and clinical strategy in one founding team.

Sophia T., PharmD

Co-Founder & CEO · Head of Strategy

Sets clinical and regulatory strategy and leads company operations, translating platform biology into a fundable, executable development plan.

Eric Tam, PhD

Co-Founder & Scientific Lead

Over 12 years of experience leading lipid nanoparticle (LNP) formulation, process development, and targeted delivery initiatives across academia, biotech, and industry, with deep expertise in LNP-based therapeutics, next-generation drug delivery platforms, protein therapeutics, and RNA biology.

Where things stand

Early-stage, hypothesis-driven, funded to derisk the core mechanism.

Program stage
Preclinical — lead binder-LNP candidates in formulation and screening
Funding path
Pursuing non-dilutive SBIR funding and other strategic funding opportunities to accelerate the clinical translation of the technology
Focus indication
HSC editing, in vivo CAR-T generation, and neurodegenerative diseases
Get in touch

Interested in the science, a partnership, or funding this work?

We're speaking with collaborators, non-dilutive funding programs, and scientific advisors who work in RNA therapeutics, nanomedicine, or cell engineering.

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