Cognigenics

Cognigenics is revolutionizing brain health with intranasal RNA therapeutics, targeting root causes of anxiety, cognition, and psychosis for safe, durable, and scalable CNS treatments.

Preclinical POC shows 94% improvement in memory and 34% reduction in anxiety in multiple rodent models and species via 5-HT2A silencing in hippocampal and subcortical circuits. No local or systemic toxicity was observed.

Lead program COG-301: siRNA targeting the 5-HT2A receptor for Parkinson’s psychosis — the first clinical application and regulatory validation of the platform.

Customer Description

• Primary Customer: Patients with Parkinson’s Disease Psychosis (PDP) and treatment-resistant anxiety spectrum disorders.
• Location: Initial focus on the U.S. and EU5 markets (the largest CNS drug markets with established orphan drug pathways), followed by global expansion.
• Target Age Group:
• PDP: Predominantly 65+ years, given Parkinson’s disease demographics.
• Anxiety Disorders: Broad, with peak prevalence in 25–55 years.
• Other Characteristics: Patients underserved by current therapies—seeking safe, durable, at-home treatments without daily pills or hospital infusions.

Market Size & Dynamics
• Parkinson’s Disease Psychosis (orphan entry point): ~1M Parkinson’s patients in the U.S., ~50% develop psychosis → ~500,000 potential patients U.S./EU, with ~$2–3B annual market.
• Generalized Anxiety Disorder & Anxiety Spectrum: ~300M affected globally (~40M in the U.S.), anxiety drugs >$5B global market, expected CAGR ~5–7%.
• Cognitive Impairment / Neurodegeneration: Alzheimer’s & related disorders represent >$10B markets with strong growth.

Market Development
• Current Market: Strong demand for CNS treatments, but existing drugs are inadequate (daily compliance, limited efficacy, systemic toxicity).
• Trend: Rising prevalence of psychiatric and neurodegenerative disorders due to aging populations and post-pandemic mental health surge.
• Future Market: Precision RNA therapeutics for the CNS do not yet exist, but indirect indicators (growth in RNA therapies, intranasal delivery adoption, CNS drug market expansion) suggest a future multi-billion-dollar category.

The Opportunity: Brain disorders represent one of the largest and fastest-growing areas of unmet medical need, affecting over a billion people worldwide. Yet today’s approaches fall short: small molecules act broadly and cause systemic side effects; viral vectors raise safety concerns and cannot be redosed; and intrathecal oligonucleotides and neuro-modulation remain invasive, costly, and poorly scalable.

The Gap: No existing modality provides a non-invasive, re-dosable, and tunable solution for direct gene-level modulation of brain circuits.

Cognigenics is pioneering the first platform for non-invasive, tunable, and re-dosable genetic medicines for the brain. By uniting programmable RNA payloads, optimized carriers, and device-enabled precision, we are establishing intranasal RNAi (iRNAi) as a new therapeutic class for psychiatry, neurology, and cognitive disorders.

Competitors and Existing Alternatives: Current treatments for Parkinson’s Disease Psychosis and anxiety rely on antipsychotics, SSRIs, SNRIs, and hospital-based infusions (e.g., clozapine, pimavanserin, ketamine, esketamine). These drugs require daily or frequent dosing, often with limited efficacy, systemic toxicity, and poor patient compliance. In PDP, the only FDA-approved drug (pimavanserin) shows modest benefit and carries black-box warnings. In anxiety, SSRIs and benzodiazepines dominate a $5B+ market, yet many patients remain resistant or discontinue due to side effects.

Cognigenics differentiates itself by introducing a first-in-class intranasal RNA therapeutic that bypasses the blood–brain barrier and directly modulates validated CNS targets. Unlike existing options, our approach is non-invasive, durable, reversible, and designed for convenient once-weekly at-home use. While competition exists across psychiatry and neurology, no current therapies combine precision RNA-based silencing with intranasal delivery, positioning Cognigenics to create and lead an entirely new category of CNS treatments.

Revenue Streams: Cognigenics’ initial revenue will come fr om high-value orphan CNS indications, beginning with Parkinson’s disease psychosis (PDP), wh ere pricing aligns with specialty neurology drugs. As clinical validation grows, the iRNAi platform expands into broader psychiatric and neurodegenerative markets such as generalized anxiety and Alzheimer’s, unlocking multi-billion-dollar opportunities. Additional revenue will be generated through licensing partnerships, co-development deals, and potential applications of intranasal RNA delivery across the CNS and beyond.

Cost Structure: Near-term costs are driven by R&D and clinical development, including preclinical toxicology, IND-enabling studies, and Phase I–III trials. Manufacturing and delivery rely on scalable siRNA synthesis, lipid nanoparticle formulation, and low-cost intranasal devices. As programs advance, expenses shift toward regulatory filings, reimbursement strategy, and targeted commercialization via specialist sales. This lean, staged cost structure enables efficient progression from high-margin orphan indications to larger, scalable psychiatric markets.

Cognigenics is developing a new class of CNS therapeutics using its iRNAi platform, which delivers siRNA intranasally to bypass the blood–brain barrier. Our lead asset, COG-301, silences the 5-HT2A receptor for Parkinson’s psychosis and anxiety with once-weekly, at-home dosing. We begin in orphan CNS indications for rapid approval and premium pricing, then expand into broader psychiatric and neurodegenerative markets. Long term, we aim to establish RNA-based brain medicine as a scalable, transformative category.

The majority of the capital will be allocated to R&D and clinical development, including the completion of preclinical toxicology studies, IND-enabling studies, and Phase I/II clinical trials for our lead candidate, COG-301. Investment will also support the manufacturing scale-up of siRNA and lipid nanoparticle formulations, as well as the development of a reliable intranasal delivery device. Additional funds will cover regulatory submissions, intellectual property expansion, and strategic partnerships to accelerate the development process. As programs advance, spending will shift toward commercial readiness, including market access planning and targeted outreach to specialists.

Cognigenics offers investors the opportunity to participate in the development of a new therapeutic category: intranasal RNA-based medicines for the brain. By targeting validated CNS receptors with precision siRNA delivery, we address massive unmet needs in psychiatry and neurology, beginning with Parkinson’s Disease Psychosis and anxiety spectrum disorders.

Investors gain early access to a scalable platform technology with multiple pipeline opportunities, orphan-drug advantages (including accelerated approval and premium pricing), and clear expansion into large psychiatric and neurodegenerative markets. With strong IP, a differentiated delivery route, and a de-risked entry strategy, Cognigenics is positioned to generate both high clinical impact and significant financial returns.

Risks: As with any emerging therapeutic platform, Cognigenics faces several risks. Scientific and clinical risk includes the possibility that preclinical efficacy may not fully translate in human trials, or that safety and tolerability concerns arise with intranasal siRNA delivery. Regulatory risk involves the novelty of RNA-based CNS therapies, which may lead to longer review times or additional FDA/EMA requirements. Manufacturing and delivery risk stems fr om scaling lipid nanoparticle–siRNA formulations and ensuring consistent intranasal biodistribution.

On the business side, market adoption risk exists if physicians or patients are slow to embrace RNA-based CNS treatments, particularly in psychiatry, wh ere prescribing habits are entrenched. Competitive risk arises from both existing drugs (e.g., antipsychotics, SSRIs) and emerging CNS technologies (e.g., gene therapies, psychedelic-assisted treatments). Finally, financing risk reflects the capital-intensive nature of drug development, which requires sustained funding through key clinical milestones. Cognigenics mitigates these risks with a staged development strategy, an orphan indication entry point, validated CNS targets, and a scalable delivery platform.

2025 One Mind Accelerator
https://onemind.org/what-we-do/one-mind-accelerator/

• Intranasal Delivery of siRNA via Optimized Lipid Nanoparticles
• Lipid Nanoparticle-Delivered siRNA for Targeted Knockdown of 5-HT2A
• Receptors to Treat Parkinson's Disease Psychosis
• Method for Treating Neurological Conditions and Improving Human Cognition
• Systems and Methods for an Intranasal Drug Delivery System
• Compositions and Methods to Enhance Memory by Modulating 5-HT2A
• Methods to Enhance Memory by Modulating 5-HT2A Receptor Expression
• Methods to Enhance Cognition by Modulating 5-HT2A Receptor Expression
• System and Method for Sensor Monitoring of RNA Therapies
• Intranasal Delivery of shRNA to Knockdown the 5HT-2A
• Receptor Enhances Memory and Alleviates Anxiety
• Compositions and Methods for Modulating Cognition With Interfering RNA
• Systems and Methods for an RNA-Adjunct Psychedelic Therapy
• Systems and Methods for an Intranasal Drug Delivery System
• System and Method for Sensor Monitoring of RNA Therapies
• Intranasal Delivery of HTR1A mRNA for Neuronal Hyperactivity Management
• Intranasal Delivery of siRNA Via Optimized Lipid Nanoparticles for Targeted Transport to Subcortical Brain Regions
• Intranasal Delivery of HTR1A mRNA for Neuronal Hyperactivity Management
• Lipid Nanoparticle-Delivered siRNA for Targeted Knockdown of 5-HT2A Receptors to Treat Parkinson's Disease Psychosis
• Compositions and Methods for Intranasal Delivery of siRNA-Lipid Nanoparticles Targeting Muscarinic Autoreceptors for the Treatment of Cognitive Disorders