Top 11 ALS startups in USA

Nov 14, 2025
1
Funding: $1.1B
Neurodegenerative diseases are one of the largest medical challenges of our time. Denali Therapeutics is dedicated to defeating neurodegenerative diseases through rigorous therapeutic discovery and development.
2
Funding: $634M
Annexon Biosciences a biotechnology company, discovers and developing novel therapeutics for neurodegenerative and autoimmune disorders.
3
Funding: $448.5M
Amylyx Pharmaceutical is a pharmaceutical company that specializes in providing solutions on Alzheimer’s and other diseases of the brain.
4
Funding: $374M
Voyager Therapeutics is a clinical stage gene therapy company developing life-changing treatments for severe diseases of the central nervous system (CNS). Gene therapy has the potential to transform the treatment of CNS and other diseases by providing a one-time therapy that dramatically improves patients' lives.
5
Funding: $181.2M
Skyhawk's is developing small molecule therapeutics that correct RNA expression.Their proprietary technology enables the rational design of small molecules that target the RNA splicing process.They are now developing drug candidates for cancer, neurological disease, and rare disease.
6
Funding: $135.5M
QurAlis is specifically focused on discovering & developing new therapies for amyotrophic lateral sclerosis (ALS), the most common form of motor neuron disease.
7
Funding: $134.1M
We use genomic data to find better drugs, faster. We find new cures for brain diseases 1000X more cheaply and quickly using our proven network algorithms.
8
Funding: $15.7M
28bio's Nerve-on-a-Chip platform facilitates prediction of both clinical neurotoxicity and efficacy in human neurodegenerative disease models earlier in the drug development pipeline.
9
Funding: $14.4M
Spinogenix is a developer of a new class of therapeutics for neurodegenerative and psychiatric diseases.
10

Iron Horse Therapeutics is developing treatments for amyotrophic lateral sclerosis (ALS) with compounds that modulate the activity of EphA4. Humans with EphA4 mutations that decrease its activity or reduced EphA4 expression levels exhibit prolonged survival and reduced disease severity. Similar results have been demonstrated in ALS mouse and rat animal models.
11
Funding: $120K
A bioinformatics company offering a unique platform for crowd-sourced brain research. It leverages cloud computing, big data and mobile technology to offer valuable personal insights and accelerate brain research globally.
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Editor: Jason Kwon
Jason Kwon is a senior editor for MedicalStartups. He has previously covered the pharmaceutical and medical research industries for FDAnews and worked as a head of marketing for medical startup Sonic Therapeutics. Before that, he co-founded a startup consulting business for emerging entrepreneurial hubs in Asia. Jason graduated from St. Bonaventure University’s journalism school. In his free time, Jason enjoys yoga, watching movie trailers, traveling to places where he can't get cell service. You can contact Jason at jaskwon(at)medicalstartups(dot)com