KOP Therapeutics is advancing a pipeline of peptide-guided ASO programs across four solid tumor indications — all built on the same TGM delivery platform, enabling capital-efficient expansion from a single validated mechanism.
All programs are in preclinical development. IND-enabling studies are the next milestone for the lead program.
KOP-101
Prostate Cancer
Target: CCND1 / BIRC5
Mechanism: CCND1 / BIRC5 ASO Knockdown
Lead program. In vivo proof-of-concept completed at Charles River Laboratories. Statistically significant TGI confirmed (p=0.049). 96-hr kill study (ED50/ED90) underway at Galenvs Sciences. Next: lead & construct optimization — finalize ASO leads and conjugate design, then advance 1–2 in vivo-ready conjugates into PK/PD studies by Q1 2027.
In Vitro Screen
In Vivo PoC (Charles River)
96-hr Kill Study (ED50/ED90) — Galenvs Sciences
Lead & Construct Optimization + Delivery Valency Selection
In Vivo PK/PD Studies — Q1 2027
IND-Enabling Studies
KOP-102
Breast Cancer
Target: CCND1
Mechanism: CCND1 ASO Knockdown
In vitro knockdown confirmed across breast cancer cell lines. CCND1 overexpression is a validated driver in ER+ and HER2+ breast cancer subtypes.
In Vitro Screen
In Vivo PoC
96-hr Kill Study
IND-Enabling Studies
KOP-103
Brain Cancer (GBM)
Target: CCND1
Mechanism: CCND1 ASO Knockdown
Glioblastoma multiforme (GBM) represents a high unmet need with limited treatment options. CCND1 overexpression is implicated in GBM proliferation and resistance.
In Vitro Screen
In Vivo PoC
96-hr Kill Study
IND-Enabling Studies
KOP-104
Ovarian Cancer
Target: CCND1
Mechanism: CCND1 ASO Knockdown
Ovarian cancer has high CCND1 expression and limited targeted therapy options. KOP's peptide carrier targets LHRH receptors overexpressed on ovarian tumor cells.
In Vitro Screen
In Vivo PoC
96-hr Kill Study
IND-Enabling Studies
Completed
In Vitro Validation
Up to 95% knockdown · 95 constructs screened · 4 cancer types
Completed
In Vivo PoC (Charles River)
Statistically significant TGI · p=0.049 · PC3 xenograft · no observed toxicity
Active
96-hr Kill Study — Galenvs Sciences
ED50 / ED90 · Galenvs Sciences CRO · Montreal, QC · underway
Next
Lead & Construct Optimization
Finalize ASO leads · conjugate design · delivery valency selection · head-to-head uptake assay
Target: Q1 2027
In Vivo PK/PD Studies
1–2 in vivo-ready conjugates · PK/PD animal studies · candidate selection
Target
IND-Enabling Studies
GLP tox · CMC · FDA pre-IND meeting
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