Orion Therapeutics is a delivery platform-enabled RNA therapeutics company. We pair a proprietary lipid nanoparticle GENESYS™ delivery platform that entirely excludes ionizable cationic lipids with a vascular RNA therapy pipeline. Our safe, versatile, scalable, and targeted delivery platform can be leveraged across a broad range of disease applications to advance RNA therapies to market internally and in partnership.
RNA technology thus has the potential to revolutionize medicine with life-saving and even curative treatments for previously unmet clinical needs, including cancers, autoimmune disorders, and neurodegenerative diseases. The primary bottleneck in the translation of the 1300+ early-stage RNA programs in existence has been the biological delivery system, not the RNA drug payload itself, due to toxicity, immunogenicity, and the inability to target specific cells, tissues, and organs, leading to off-target effects. All RNA drug payloads require a nanoscale delivery system to Package, Protect, and Target those payloads to their site of action in the body. We pair a proprietary lipid nanoparticle delivery platform ( GENESYS™) that entirely excludes ionizable cationic lipids with a vascular RNA therapy pipeline. Our incorporation of lipopeptides resolves many of the challenges faced by ionizable lipids – including the toxicity and immunogenicity issues that most recently halted the Verve Phase 1b trial. We can also modify our platform for active delivery to resolve a significant gap in the market – the ability to target outside the liver. We have demonstrated this with our lead program for restenosis, with additional pipelines towards CNS indications and CAR-T cell therapy applications.
Our team has leading expertise in the non-viral delivery and formulation of RNA drugs.
- Trey Fisher, PhD, has over a decade of experience in preclinical and clinical trial development of non-viral delivery systems.
- Michael McCaman, PhD, has 35+ years experience in pharmaceutical development of biological therapeutics. His specialties range from formulation and process development to CMC and commercialization. His program at Intellia was the first gene editing drug to enter late phase clinical trials.
- Deidra Mountain, PhD, is a professor at UTMC whose work focuses on novel therapeutic modalities to treat vascular disease and injury.
- Leanne Minall, PhD, has managed R&D for several RNA-based therapies and prior was a senior scientist in the novel delivery technologies group at Pfizer.
- Jennifer Zachry, PhD, MBA brings experience in business development and product management in biotech startups.
Given our strong expertise, we are well-equipped to accelerate early-stage RNA companies and pipelines.
Orion’s patented, GENESYS delivery platform will be leveraged towards RNA developers for advancing early-stage RNA therapeutic pipelines and technologies by providing a safe, versatile, and targeted biological delivery system. We completed 100+ customer discovery interviews as part of the NSF I-Corp program and refined our customer segments within the scope of early-stage RNA programs to:
- small-to-midsize biotechs
- big pharma
- RNA Contract Development & Manufacturing Organizations (CDMOs).
We support partnerships opportunities through co-development projects and collaborative agreements for the development of new exclusive intellectual property (IP) based on our delivery platform. These projects can be initiated through development and option (D&O) agreements or initial fee-for-service agreements. The use of initial fee-for-service agreements helps de-risk our delivery platform, allowing clients to engage in technology evaluations of the Orion delivery platform with their RNA constructs. We focus on reaching preclinical and IND-enabling milestones for subsequent clinical development and commercialization. Our collaborative model involves clients providing validated RNA constructs with their target of interest while our experienced team at Orion performs Proof of Concept (POC) studies using relevant in vitro and in vivo models. Based on the outcomes, we then facilitate the negotiation of licensing agreements that grant our pharmaceutical partners exclusive access to the developed delivery IP. The licensing agreements may be tailored to offer target-restricted, disease-restricted, or unrestricted access, ensuring a customized approach that meets the specific needs of our partners.
We have already initiated technology evaluations with three clients for three different disease indications. We aim to advance RNA developer clients into Development & Option (D&O) Agreements with Orion Therapeutics. The ultimate goal is out-licensing of the delivery system, with associated fees, milestones, and lifetime royalties. We anticipate that any of these licensing deals producing a drug that progresses all the way to the market would generate $110M+ in revenue. There are several advantages to this strategy – we provide further validation of the Orion GENESYS platform and our partners bear the regulatory burden for advancing an RNA drug through clinical trials with our support.
The ultimate goal of these activities is to provide validation and meet data milestones that can be leveraged into a Co-Development deal or Strategic Partnership with a large pharmaceutical partner, many of whom we have already had conversations with, that gives Orion access to multiple of their therapeutic pipelines. As recent examples in the space, Entos, a delivery platform company, signed a deal with Eli Lilly for $50 million upfront with $400 million in milestones per target and a 5% lifetime royalty. Nanovation recently completed a deal with Novo Nordisk for $600M in upfront, milestones, and royalties.
We are seeking $5M in seed capital to advance the Orion GENESYS™ delivery platform towards key platform and asset milestones over the next 24 months. These activities will help Orion to secure a Series A raise to advance our restenosis RNA drug asset to IND-enabling studies, IND, and Phase I clinical trials. Our restenosis RNA drug asset is currently supported by a Phase I NSF STTR grant that we will transition to a Phase II in Q2 of 2025. Seed round funds will be utilized to advance the Orion GENESYS™ delivery platform towards proof of concepts in multiple disease indications internally and in partnership (around BBB delivery, vaccine applications, and CAR-T cell therapy). We will initiate NHP toxicology studies of our platform. Additionally, Orion has a library of 180+ novel lipopeptide structures that will be screened through established Design of Experiments protocols to identify lipid nanoparticle candidates with improved potency and safety profiles. We will also test in vivo proof of concepts for targeted delivery (3 extra-hepatic sites) to expand the utility of our platform across disease indications. Alongside these activities, we have budgeted funds and identified partners for the stability and scalability studies necessary to produce engineering batches. At the end of 24 months, we will have leveraged these activities into a multiple licensing agreements and a strategic partnership or co-development deal with a large pharmaceutical partner.
The Orion GENESYS™ delivery platform uses novel proteolipids to encapsulate RNA payloads and avoids synthetic ionizable/cationic lipids associated with dose-limiting adverse events in humans. Our technology platform has been validated on multiple fronts. We received a Phase I National Science Foundation STTR in April of 2022 (Award Number (FAIN): 2309031) to advance our RNA drug asset in treatment of restenosis. We have recently demonstrated a proof of concept for our lead assset in a rodent model of a balloon angioplasty. This pilot pogram provides validation for the GENESYS delivery platform. We received the NIH Targeted Genome Editor Challenge award to target our lipid nanoparticle delivery system to previously inaccessible areas of the body which could be leveraged across 20+ disease indications. In addition, we won the Eli Lilly Genetic Grand Medicine Challenge for $100,000 for our delivery platform. We have IP protection across multiple fronts. We have a published patent for our formulation and process (US Published Patent: US20210244665A1, NEUTRAL LIPOSOMES CONTAINING BIOLOGICALLY ACTIVE AGENTS; published: August 18, 2021). We have a second provisional patent (US PCT: PCT/US2024/014622) MULTIFUNCTIONAL LIPID NANOPARTICLES AND USES THEREOF). We have a third provisional patent that covers composition-of-matter of 180+ novel lipid nanoparticle formulations (PCT63/571,942). We are currently conducting technology evaluations with several clients and conducting proof of concept studies.
NSF Phase I STTR: $275K + $100K LaunchTN Match Eli Lilly Grand Medicine Challenge: $100K NIH Targeted Genome Editor Challenge Phase I Winner: $75K $250,000 pre-seed SAFE; friends & family $300,000 Bridge Round; InvestTN, University of Tennessee Research Foundation Accelerate Fund
