TARGETED GENOMIC MESSENGER PLATFORM

A New Delivery Engine
for Solid Tumor Oncology

KOP's proprietary TGM platform pairs a tumor-seeking peptide carrier with antisense oligonucleotide (ASO) payloads — achieving intracellular mRNA knockdown of oncogenes that have historically been undruggable, in solid tumors beyond the liver.

TGM

Platform Name

Targeted Genomic Messenger

ASO

Payload Class

Antisense oligonucleotide

Extra-hepatic

Delivery Profile

Solid tumor targeting

Modular

Architecture

Swap ASO for new targets

HOW IT WORKS

Three-Step Mechanism of Action

The TGM platform operates through a precise sequence: targeted delivery, cellular uptake, and oncogene silencing.

01
🎯

Target — Tumor-Selective Peptide Finds the Cell

A tumor-selective peptide carrier binds receptors overexpressed on cancer cells. Primary address: V1/VC1 peptide — demonstrated in vitro across breast and prostate tumor types with selectivity over low-receptor controls, backed by human clinical imaging precedent. Second address: IR peptide — a breast-selective targeting peptide with direct evidence of peptide-mediated ASO cargo uptake.

02
🔬

Deliver — Multivalent Scaffold Carries the Payload In

A cleavable conjugate carries the ASO payload into the cell. The platform deploys a tunable multivalent scaffold engineered to reward receptor binding with cytosolic payload release. Productive internalization and endosomal escape are measured directly by a head-to-head assay with strict go/no-go criteria — in collaboration with Galenvs Sciences.

03
🧬

Silence — ASO Knocks Down the Oncogenic Driver

The ASO payload hybridizes to the target oncogene mRNA — CCND1 (cyclin D1), BIRC5 (survivin), or other hard-to-drug drivers — triggering RNase H-mediated degradation. Up to 95% knockdown achieved in preclinical models. The modular architecture means both the targeting ligand and the payload are interchangeable: a platform, not a single molecule.

KOP Targeted Cancer Cell Therapy Platform Pipeline

KOP Targeted Cancer Cell Therapy — Platform Pipeline Overview

WHAT WE'RE BUILDING

A Modular Oncology Delivery Platform — In Vivo by Q1 2027

KOP's TGM platform is a receptor-targeted peptide-ASO conjugate system: a defined chemical entity pairing a tumor-selective targeting peptide with a gene-silencing ASO payload through a cleavable linker. Both the targeting ligand and the payload are interchangeable — a platform, not a single molecule. The platform has established tumor-selective targeting, peptide-mediated cargo uptake, and validated payload potency. The next milestone: one to two in vivo-ready conjugates into PK/PD studies by Q1 2027, in collaboration with Galenvs Sciences.

Targeted Delivery — Two Receptor Addresses

Primary: V1/VC1 peptide with in vitro proof across breast and prostate. Second: IR peptide with direct evidence of peptide-mediated ASO cargo uptake in breast cancer cells

Multivalent Scaffold — Engineered for Internalization

Tunable valency scaffold deploys multivalency behind the receptor that rewards it, with cytosolic payload release. Endosomal escape measured by head-to-head assay with strict go/no-go criteria

Optimized Payloads — Up to 95% Knockdown

ASO leads finalized with conjugate design complete. CCND1 and BIRC5 targets validated. Lead candidate selection converging on 1–2 in vivo-ready conjugates for PK/PD studies

ONCOGENE KNOCKDOWN — PRECLINICAL RESULTS

Next-Gen Sequence95%
CCND1-01693%
CCND1-01591%
CCND1-00974%
CCND1-02268%
Screen Avg.41%

In vitro: 24-hr screen, mean across 3 cancer cell lines. In vivo: Charles River PC3 xenograft.

COMPETITIVE LANDSCAPE

Why Existing Platforms Fall Short

Current RNA delivery technologies are constrained by liver-first biodistribution and systemic toxicity. KOP's TGM platform is engineered specifically for solid tumors.

PlatformDelivery RouteSolid Tumor AccessToxicity ProfileModular Targets
GalNAc ConjugatesHepatocyte-selective✕ Liver onlyLow (hepatic)✕ Limited
LNP / Lipid NanoparticlesSystemic IV⚠ PoorInflammatory / immunogenic⚠ Complex
Naked ASOSystemic⚠ LimitedOff-target effects✓ Yes
KOP TGM PlatformReceptor-targeted✓ Extra-hepaticNo observed (preclinical)✓ Yes
THE PATH FORWARD

From Lead Optimization to In Vivo — Q1 2027

A single forward path: optimize, select, and advance the lead conjugate into in vivo PK/PD studies. In collaboration with Galenvs Sciences.

NOW

Lead & Construct Optimization

Finalize ASO leads and conjugate design

NEXT

Delivery & Valency Selection

Head-to-head uptake assay to pick receptor address + valency

THEN

Candidate Selection

Converge on 1–2 in vivo-ready conjugates

Q1 2027

In Vivo PK/PD Studies

Advance lead candidate(s) into animal PK/PD studies

Milestone: One to two in vivo-ready conjugates into PK/PD studies by Q1 2027 — Galenvs Sciences collaboration

PIPELINE EXPANSION

Modular Architecture Enables Rapid Target Expansion

By swapping the ASO payload while retaining the peptide delivery vehicle, KOP can address new oncogene targets and tumor types without redesigning the platform.

Prostate Cancer

Target: CCND1 / BIRC5

In Vivo PoCLead Program

Breast Cancer

Target: CCND1

In VitroActive

Brain Cancer (GBM)

Target: CCND1

In VitroActive

Ovarian Cancer

Target: CCND1

In VitroActive

A De-Risked, Mechanism-Led Delivery Platform

Established tumor-selective targeting, validated payload potency, and an engineered multivalent scaffold. One to two in vivo-ready conjugates into PK/PD studies by Q1 2027.

KOP Therapeutics

Advancing tumor-targeted RNA therapeutics toward IND-enabling development, partnership, and commercialization.

Preclinical Stage

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