In 2021, a study by The Lancet Neurology showed that more than 3 billion people worldwide had a neurological condition. That’s over one in three!
Access to treatment varies widely, with high-income countries having up to 70 times more neurological professionals per 100,000 people than low and middle-income countries. Furthermore, the complexity of these conditions often leads to misdiagnosis and delayed care, exacerbating symptoms and increasing the burden on patients and caregivers.
Gene therapy is emerging as a powerful therapeutic strategy for neurodegenerative disorders, including Alzheimer’s disease (AD), Parkinson’s disease (PD), and Huntington’s disease (HD). It can treat the disease-causing mutation by supplying a functional copy of the mutated gene or editing the genomic sequence.
It addresses the root cause of these disorders by modifying or replacing faulty genes, making it mostly a one-time treatment that provides lifelong benefits. This can be more cost-effective in the long run than other therapies requiring ongoing treatment.
This article explores five startups developing innovative gene therapy solutions for neurological disorders in 2024.
1. CavGene Therapeutics uses a modified virus to treat dyskinesia
| Founding Year | 2022 |
| Headquarters | Michigan, United States |
| Total Funding Amount | Not disclosed |
| Last Funding Round/Amount | Pre-Seed round |
| Website | cavgene.com |
CavGene Therapeutics utilizes gene therapy to treat levodopa-induced dyskinesia (LID) in patients with Parkinson’s Disease.
This condition is a frequent side effect of a drug commonly prescribed for Parkinson’s patients. The drug, levodopa, can alleviate the tremors, stiffness, and other motor symptoms of Parkinson’s.
However, its long-term use can cause dyskinesia, the involuntary and uncontrolled movement of hands, the head, and other body parts that negatively impact the quality of life.
CavGene uses a harmless virus to deliver genetic material into the brain. This reduces existing dyskinesia and prevents its development.
Its work is built on decades of research led by Kathy Steece-Collier, a professor of Translational Neuroscience in the College of Human Medicine at Michigan State University. This makes them a pioneer in this specific area of neurodegenerative disease treatment.
Its CEO, Dr. Fredric Manfredsson, is a former Chair of the American Society for Gene & Cell Therapy Neurological & Ophthalmological Committee and the past President of The American Society for Neural Therapy and Repair. Dr. Manfredsson earned his doctorate in neuroscience from the University of Florida and has expertise in gene therapy for Parkinson’s disease and other neurodegenerative diseases.
CavGene Therapeutics has funding from a Pre-Seed round raised on Feb 14, 2023. The specific amount is undisclosed.
2. Mana.bio uses AI to create lipid nanoparticle systems
| Founding Year | 2021 |
| Headquarters | California, United States |
| Total Funding Amount | $29.5M |
| Last Funding Round/Amount | Seed Round / $19.5M |
| Website | www.mana.bio |
Mana.bio is an AI-based drug delivery startup focusing on gene therapy, including DNA and RNA-based therapeutics and vaccines. Their technology is powered by machine learning and nanotechnology to identify formulations of lipid nanoparticle systems.
The startup’s main product is its unique AI-based lipid nanoparticle (LNP) delivery platform, which accelerates the development of nucleic acid-based therapeutics and vaccines. By integrating machine learning and high-throughput screening into its development process, Mana.bio LNPs can deliver RNA payloads to specific tissues more safely and effectively.
Their CEO, Yogev Debbi, has over a decade of entrepreneurial experience. He previously co-founded Genome Compiler Corp, which Twist Bioscience successfully acquired.
Mana.bio has raised a total of $29.5M in funding. Their latest funding came from a Seed round on October 10, 2023.
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3. Celosia Therapeutics is treating Alzheimer’s with gene therapy
| Founding Year | 2022 |
| Headquarters | Sydney, Australia |
| Total Funding Amount | $2M |
| Last Funding Round/Amount | Seed round/$2M |
| Website | celosiatx.com |
Celosia Therapeutics is using gene therapies to treat motor neuron disease (MND), epilepsy, and Alzheimer’s disease. Their approach arises from advances in gene therapies and a greater understanding of the molecular pathways contributing to neurodegenerative diseases.
The startup modifies the neuronal microenvironment to combat neurodegenerative diseases. It has exclusive access to a portfolio of gene therapies and is actively developing a therapeutic pipeline, including various supporting technologies.
Its CEO, Dr. Brenton Hamdorf, has 20 years of experience in molecular biology, microbiology, and biochemistry, working across agricultural, environmental, and medical research. He holds a Ph.D. in Biochemistry from the University of New England.
Celosia Therapeutics has raised a total of $2M in funding. This was a Seed round raised on Nov 28, 2022.
4. Epeius Pharma can deliver therapy directly to the central nervous system
| Founding Year | 2019 |
| Headquarters | HaMerkaz, Israel |
| Total Funding Amount | $814K |
| Last Funding Round/Amount | Seed round |
| Website | www.futurx.co.il/portfolio/epeius-pharma |
Epeius Pharma is developing next-generation biological vehicles for delivering therapeutic proteins into the central nervous system (CNS). This is particularly challenging in neurological diseases due to the blood-brain barrier (BBB), which tightly regulates the transport of molecules to the brain and blocks the transport of most large, charged, and hydrophilic molecules, which are common features of therapeutic agents.
The lack of efficient CNS delivery methods limits protein replacement therapy and remains one of the greatest unmet needs for neurological disorders treatment. Epeius Pharma’s platform allows for an effective and scalable strategy for neurological disease medication using direct neuron protein replacement treatments.
They are leveraging the mechanisms used by certain organisms, such as the protozoan parasite Toxoplasma gondii, which have independently evolved to breach the BBB and secrete proteins into their host’s cells.
Its Chief Scientific Officer, Dr. Doron Levin, holds a PhD from the Weizmann Institute of Science (Rehovot, Israel) and has vast expertise in molecular biology, biochemistry, biophysics, cell biology, and cell signaling.
Epeius Pharma Ltd. raised funding from a Seed round on Jan 15, 2020.
5. NEUmiRNA Therapeutics is treating epilepsy with modified RNA
| Founding Year | 2020 |
| Headquarters | Copenhagen, Denmark |
| Total Funding Amount | $3.12M |
| Last Funding Round/Amount | Grant/$720K |
| Website | www.neumirna.com |
NEUmiRNA Therapeutics is a Danish pharmaceutical startup developing ribonucleic acid therapies to treat neurological disorders.
The company’s main offerings include disease-modifying treatments designed to address the underlying causes of various neurological conditions. Their core technology is an RNA drug platform that targets specific neurological disorders by utilizing anti-microRNA (anti-miRNA) oligonucleotides to target dysfunctional microRNA (miRNA) molecules.
NEUmiRNA Therapeutics’ primary focus is on epilepsy, where their drug candidate, NMT.001, has shown a promising disease-modifying effect. This could revolutionize epilepsy treatment, particularly for those with treatment-resistant forms, potentially reducing the need for frequent treatments. They are also exploring projects in Parkinson’s disease and pain management.
Their CEO, Janine Erler, has 23 years of experience in academic research, including 16 years leading an independent research group. She helped secure the €2 million grant funding for the company.
The company’s most recent funding, totaling $720K, came from the fourth grant round on 29 February 2024.
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Author: Hemanth Shenoy, Market Research
Related Read: The Role of RNA Therapeutics in Neurological Disorders Treatment
