5 Top Startups developing Central Nervous System Drugs

Curious about new technological advancements in the pharma industry? Explore our analysis of 270 global startups & scaleups developing central nervous system (CNS) drugs & learn how their solutions impact your business!

Staying ahead of the technology curve means strengthening your competitive advantage. That is why we give you data-driven innovation insights into the pharma industry. This time, you get to discover 5 hand-picked startups working on Central Nervous System drugs.

Global Startup Heat Map: 5 Top Startups Developing Central Nervous System Drugs

The 5 pharma startups you will explore below are chosen based on our data-driven startup scouting approach, taking into account factors such as location, founding year, and relevance of technology, among others. This analysis is based on the Big Data & Artificial Intelligence (AI)-powered StartUs Insights Discovery Platform, covering over 1.3 million startups & scaleups globally.

The Global Startup Heat Map below highlights the 5 startups our Innovation Researchers curated for this report. Moreover, you get insights into regions that observe a high startup activity and the global geographic distribution of the 270 companies we analyzed for this specific topic.

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CNS Pharmaceuticals works on the Blood-Brain Barrier (BBB)

Many disorders of the central nervous system, such as Alzheimer’s or Parkinson’s, have a vast amount of unmet clinical needs. For example, the blood-brain barrier is crucial for protecting the brain from pathogens. However, the barrier also presents a challenge for drugs targeting regions of the CNS. Pharma startups are therefore investigating drugs that cross the BBB for the treatment of CNS disorders.

CNS Pharmaceuticals is a US-based startup developing treatments for primary and metastatic cancers of the CNS. Berubicin, the startup’s novel therapy, is an anthracycline for the treatment of glioblastoma. The drug crosses the BBB and acts directly on tumor cells. The startup is also investigating drugs for the treatment of other CNS cancers such as pancreatic and ovarian cancers.

NovaGo Therapeutics develops Monoclonal Antibodies

Monoclonal antibodies are central to immunotherapy and find applications in the treatment of cancers as well as autoimmune disorders. However, their efficacy in the treatment of CNS diseases is limited by the blood-brain barrier too. That is why pharma startups are working on drugs that extend the therapeutic advantages of monoclonal antibodies to the treatment of neurological disorders.

Swiss startup NovaGo Therapeutics develops proprietary monoclonal antibodies for the treatment of CNS indications. The antibodies block Nogo-A, a potent nerve growth inhibitor. The startup is currently investigating its lead molecule for the treatment of diabetic retinopathy (DR), a major cause of blindness.

Amylon Therapeutics focuses on Antisense Oligonucleotides (ASOs)

Antisense oligonucleotides are a novel class of ribonucleic acid (RNA) targeting drugs that use synthetic deoxyribonucleic (DNA) fragments. They act in multiple ways, for example by inhibiting gene expression, inactivating microRNAs, or splicing messenger RNA (mRNA). Due to their specific molecular actions and pharmacological properties, ASOs are emerging as promising drugs for the treatment of CNS disorders.

Amylon Therapeutics is a Dutch startup that develops therapies for rare genetic disorders of the CNS. The startup’s RNA Modulation Technology uses ASOs to alter pre-mRNA splicing and reverse the effect of disease-causing mutations. AT-01, the startup’s lead therapy, is under development for the treatment of hereditary cerebral hemorrhage with amyloidosis-Dutch type (HCHWA-D). The startup is also targeting other spino-muscular and neurodegenerative indications.

Notogen creates a Regenerative Biologic Therapy

Many disorders of the CNS such as spinocerebellar ataxia and motor neuron diseases are degenerative in nature. Moreover, the ability of the CNS to regenerate from injury or disease is very limited. Regenerative biologic therapy offers promise for the treatment of neurodegenerative disorders. Pharma startups are working on a range of biomaterials and drug delivery solutions to enable regenerative biologic therapy for the treatment of CNS diseases.

Canadian startup Notogen develops a regenerative biologic therapy to treat degenerative disc disease (DDD). NTG-101, the startup’s lead drug candidate, is a protein-based biologic. The drug is administered via intradiscal injection and mediates the progression of DDD by inducing a regenerative effect. The drug contains a combination of recombinant human transforming growth factor-beta 1 (TGF-β1) and connective tissue growth factor (CTGF).

Priavoid offers Antiprion Drugs

Prions are misfolded proteins that mark several neurodegenerative diseases. There is no known treatment for prion diseases, largely due to how rare they are. Antiprion drugs are a new class of drugs that prevent the onset of prion disease. Pharma startups are testing several antiprion drugs for their efficacy in treating prion disease, among others.

Priavoid, a German startup, offers disease-modifying therapies for severe neurological disorders. The startup employs a mirror-image phage display to generate antiprion drugs. PRI-002, Priavoid’s lead drug candidate, is under clinical development for the treatment of Alzheimer’s disease. The startup’s pipeline also includes antiprion drugs for Parkinson’s, tauopathies, Huntington’s, and amyotrophic lateral sclerosis (ALS).

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