TURBO-ZM

A Novel Approach
to Modulating the Ribosome

Ribosomes interact with messenger
and transfer RNA to translate genetic information into a protein.

Modulating the interaction between the ribosome and messenger RNA has been established as a therapeutic approach to addressing a number of diseases.

Rare Genetic Diseases

Cystic fibrosis (CF), familial adenomatous polyposis (FAP),
epidermolysis bullosa (EB), Duchenne muscular dystrophy (DMD)

Inflammatory Disorders

Crohn’s Disease

Cancers

Colon cancer, pancreatic cancer

Currently Available Molecules
That Target the Ribosome
Have Significant Limitations

platform

Zikani’s TURBO-ZM™ (Tuning the Ribosome with Zikani Molecules) is a proprietary platform that allows rapid synthesis of novel compounds that can be optimized to target the human ribosome in a disease specific manner. With TURBO-ZM™, we can design novel oral ribosome modulating agents (RMAs) that can be tuned to deliver specific results. Zikani has an expanding library of these novel oral RMAs that have demonstrated strong activity across a range of diseases.

Zikani’s TURBO-ZM™ platform yields highly specific ribosomal modulators, expanding druggable space

platform

Zikani’s RMAs are
Designed To:

mouth

Be orally delivered
at low efficacious
doses for humans

risk

Minimize safety
risks
of other
readthrough agents

target

Target specific diseases

The Zikani RMA Library

Zikani has established the TURBO-ZM™ platform PoC
(activity and druggability) across multiple disease areas.

Zikani is currently developing RMAs for the following rare diseases:

Class 1
Cystic Fibrosis

(CF)

APC mutant familial
adenomatous polyposis

(FAP)

APC mutant
colorectal cancers

(CRC)

Recessive dystrophic
epidermolysis bullosa

(RDEB)