3 Startups Redefining Neurological Disease Treatment with RNA Technology

The original article is published here with additional information.

Neurological disorders, ranging from Alzheimer’s disease to Parkinson’s and multiple sclerosis, present significant challenges in the medical field due to their complex nature and the brain’s intricate structure. One of the main obstacles in treating these disorders is the blood-brain barrier (BBB), a selective semipermeable border that protects the brain from foreign substances.

While this barrier is crucial for maintaining the brain’s internal environment, it also restricts the passage of most therapeutic agents, including large and small-molecule drugs. These molecules often fail to reach therapeutic concentrations in the brain, rendering them ineffective in treating central nervous system (CNS) disorders.

Unlike traditional drugs, RNA therapeutics can be designed to target the genetic underpinnings of neurological disorders specifically. They work at the molecular level to modulate gene expression by silencing harmful genes or promoting the expression of beneficial proteins. This specificity enhances the therapeutic potential and minimizes the risk of off-target effects. RNA therapeutics can also cross the blood-brain barrier (BBB) through various mechanisms.

These 3 startups are researching RNA therapeutics for neurological disease.

1. NEUmiRNA

NEUmiRNA is a pioneering startup harnessing the power of antisense oligonucleotides to develop innovative treatments for neurological conditions such as epilepsy. Their cutting-edge technology involves delivering precisely designed antisense oligonucleotides to the brain, where they can effectively inhibit problematic enzymes like Adenosine Kinase (ADK).

By blocking these enzymes, NEUmiRNA’s approach aims to correct the underlying molecular imbalances contributing to neurological diseases. This novel drug delivery method overcomes one of the major challenges in treating brain disorders — the inability of many conventional drugs to cross the blood-brain barrier and reach their intended targets.

NEUmiRNA’s antisense oligonucleotides are engineered to penetrate this protective barrier, ensuring that the therapeutic agents can act directly on the affected brain regions. With this technology, the startup is paving the way for more effective and targeted treatments, thus offering new hope to patients suffering from debilitating neurological conditions like epilepsy.

In Oct 2023, NEUmiRNA collaborated with Motac Neuroscience to develop groundbreaking RNA therapies poised to revolutionize Parkinson’s Disease treatment.

Motac offers specialized preclinical services to pharma companies, supporting the discovery and development of therapeutics targeting neurological and psychiatric disorders.

This collaboration is another proof that NEUmiRNA’s solution is promising.

2. Crucible Therapeutics

Motor Neuron Disease (MND) and Frontotemporal Dementia (FTD) are neurodegenerative disorders that currently lack curative treatments. MND progressively leads to the degeneration of motor neurons, resulting in paralysis and mortality within a few years of onset.

FTD, conversely, selectively targets the frontal and temporal lobes of the brain, manifesting as personality changes, language impairments, and movement disturbances. Both conditions represent significant unmet medical needs, as there is a critical dearth of effective therapeutic options.

Crucible Therapeutics, a spin-out company from the University of Sheffield, has emerged as a promising player in the quest to develop treatments for MND and FTD cases caused by the C9orf72 genetic mutation.

This particular mutation disrupts the production of functional RNA molecules and generates harmful RNA species that contribute to neuronal degeneration and cellular damage within the brain.

Crucible Therapeutics’ innovative approach addresses this root cause by targeting the SRSF1 protein. This protein can bind to and remove deleterious RNA molecules from the cell nucleus, potentially mitigating cellular damage. This strategy holds the potential to offer a novel treatment modality for MND and FTD by directly addressing the underlying genetic etiology rather than merely managing the symptoms.

In September 2023, the startup raised ￡5M from Northern Gritstone and Argobio Studio to fund essential development, leading to its first clinical trial.

3. Vico Therapeutics

VICO Therapeutics tackles neurological disorders with a new approach: RNA modulation.

Our genetic code is transcribed into RNA, a blueprint for protein production. In neurological diseases, mutations can disrupt this process, creating abnormal proteins that damage nerve cells.

VICO Therapeutics’ weapon of choice is antisense oligonucleotides (AOs), tiny synthetic RNA molecules designed to target specific RNA sequences.

Their VICOMER? platform offers an arsenal of ways to manipulate RNA. AOs can block the translation of harmful proteins, essentially preventing their creation. They can also correct RNA splicing, ensuring proper protein assembly or even editing mutations within the RNA.

Additionally, the platform allows for RNA degradation, eliminating harmful molecules or even activation, boosting the production of beneficial proteins.

This versatility allows VICO Therapeutics to tailor the RNA modulation approach to each patient’s genetic defect. By targeting the root cause of the disease at the RNA level, this personalized medicine approach can potentially be more effective and safer than traditional therapies.

The startup has raised $90 Million in funding. Their latest round of funding was Series B, in which they raised €54M from Ackermans & van Haaren.

Ackermans & van Haaren is a diversified company operating in 5 sectors. Out of 11 investments, It was a lead investor in only three companies, including VICO Therapeutics.

Future Outlook

The future of RNA therapeutics is promising, especially in addressing complex neurological disorders. Startups and research institutions are at the forefront, driving innovations that could revolutionize treatment methodologies.

Developing RNA therapeutics has historically been challenging for pharma companies due to high costs and technical complexities. However, the emergence of startups specializing in RNA therapy development is paving the way for more efficient and cost-effective solutions.

Nevertheless, the current landscape is fragmented, with different startups and research groups focusing on distinct aspects of the RNA therapeutic development pipeline. Identifying and integrating the most promising solutions from these diverse sources can be daunting for those seeking to develop RNA therapeutics for neurological diseases.

This is where startup scouting companies like GreyB play a crucial role, offering expertise in pinpointing the startups and academic institutions making headway in targeted areas of RNA therapy.

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