About the technology
Optimeos is developing a solution suite to overcome crucial formulation challenges and address unmet medical needs. Optimeos’ formulation platform navigates diverse obstacles in complex biologics delivery, ranging from the short half-lives of peptide therapeutics to the poor cell specificity of non-viral gene therapies. Our delivery platform has four key points of differentiation:
1) Unmatched capability for multiple payload delivery: Co-encapsulation of numerous APIs such as mRNA, siRNA, tRNA, DNA, ASO, proteins, peptides, or adjuvants
2) Enhanced tissue targeting: Modular assembly for tunable properties and inclusion of targeting moieties to direct cargo to different tissues and avoid liver clearance
3) Superior dosing profile: Higher cargo content and precise control over release for more efficient disease modification
4) Tunable immunogenicity: Immune-silent non-viral platform for repeat dosing of therapies, or adaptation with adjuvants for enhanced immunity
These delivery solutions are achieved through two formulation types:
CINCs (Coated Inverse NanoCarriers) are nanoparticles optimized for the targeted intracellular delivery of mRNA, DNA, and other therapeutic cargo.
LINCs (Long-acting Inverse NanoCarriers) are microparticles optimized for the long-acting extracellular delivery of peptides and other therapeutic cargo over weeks to months.
Pipeline
Rare Diseases:
Hepatic Gene Replacement
Our immune silent, non-viral gene replacement therapy is intended to provide the functional Arginosuccinate Synthetase enzyme that is deficient in Citrullinemia Type 1. We are leveraging the CINC technology to provide mRNA and DNA approaches to functionally curing this disease in both the pediatric and adult settings.
About Citrullinemia Type 1
This devastating urea cycle disorder results in elevated blood ammonia that causes severe neurologic damage and even death if left untreated. Existing therapies are either highly invasive (e.g. liver transplants) or inadequate (e.g. ammonia scavengers and severe diet restrictions). New therapies that provide monthly to yearly benefits after a single infusion are critically required in this and related urea cycle disorders.
Immunology:
In Vivo T Cell Engineering
The modularity of the CINC platform allows co-delivery of RNA, DNA and protein cargo with high cell specificity throughout the body. Optimeos is utilizing this unique capability to deliver cargo to T cells, enabling in vivo cell engineering for both autoimmune and cancer indications, without the need for ex vivo processing or conditioning regimens.
Obesity and Metabolic Diseases:
Long-acting GLP-1 depot
Our investigational GLP-1R agonist LINC depot is designed to exhibit zero-order pharmacokinetics over 30-60 days following a simple subcutaneous injection. By providing constant GLP-1 exposure, we eliminate the high plasma levels that are associated with gastrointestinal side effects for improved patient experience.
Optimeos’s Leadership
Shahram Hejazi, PhD
Bob Prud'homme, PhD
Gregory Harriman, MD
Jason Smith, PhD
Rob Pagels, PhD
Chet Markwalter, PhD
Mark Esposito, PhD
Partnering
Optimeos is actively seeking collaborations in key focus areas:
Long–acting injectables: Partners with therapeutic peptides or proteins that are dosed daily-to-weekly that would benefit from >30 day dosing while exhibiting improved, zero order pharmacokinetics.
Example: Transforming daily dosing of rapidly cleared GLP-1R agonists into a constant exposure LINC product with dosing every 60 days, removing Cmax-driven GI toxicity and reducing total exposure.
Cell–specific Intracellular delivery: Partners with novel DNA, RNA or protein therapeutics (or combinations of any) that require extrahepatic delivery to key target cell types. Cell types must be accessible from standard injection routes (e.g. blood, intrathecal, intramuscular).
Example:: Delivery of protein-RNA complexes in a CINC product to circulating CD3-positive T cells for the treatment of Type 1 diabetes
If you are interested in collaborating with Optimeos, please contact our business development team at info@optimeos.com.