Top 11 ALS startups in USA
Dec 02, 2025
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15
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.
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.
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.
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.
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 develops small molecule therapeutics that modulate RNA expression for treatment of cancer, neurological diseases and rare diseases. The company's flagship RNA-targeting drug for Huntington's disease is currently in Phase III clinical trials. Skyhawk's platform consists of: SKYSTAR - system for identifying priority RNA targets, SKYLIBRARY - library of compounds for RNA targets, SKYSEQ - multiplex screening system that simultaneously tests dozens of important targets across various RNA motifs to identify compounds selective for a specific disease target and SKYAI - machine learning model that integrates data from SKYSTAR, medicinal chemistry, animal studies, toxicology studies and clinical programs to develop the next generation of novel RNA splicing modulators.
Skyhawk develops small molecule therapeutics that modulate RNA expression for treatment of cancer, neurological diseases and rare diseases. The company's flagship RNA-targeting drug for Huntington's disease is currently in Phase III clinical trials. Skyhawk's platform consists of: SKYSTAR - system for identifying priority RNA targets, SKYLIBRARY - library of compounds for RNA targets, SKYSEQ - multiplex screening system that simultaneously tests dozens of important targets across various RNA motifs to identify compounds selective for a specific disease target and SKYAI - machine learning model that integrates data from SKYSTAR, medicinal chemistry, animal studies, toxicology studies and clinical programs to develop the next generation of novel RNA splicing modulators.
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.
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.
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.
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.
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.
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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