The landscape of gene therapy companies tackling neurodegenerative disorders is brimming with innovative solutions. These companies operate within the biotechnology and pharmacological industry, developing groundbreaking gene therapies. Their revolutionary products and therapies aim to address genetic diseases like Parkinson's, Huntington's, and Alzheimer's by manipulating the patients' genes. This cutting-edge technology offers hope for longer, healthier lives for individuals with these debilitating conditions. The industry continues to advance, with firms exploring CRISPR/Cas9 gene editing therapies, gene therapy solutions, and adeno-associated virus (AAV) gene therapy. The momentum in this industry suggests a future defined by the discovery of more effective and innovative treatments for neurodegenerative disorders.


Top 25 Neurodegenerative Disorder Gene Therapy Companies

1. Intellia Therapeutics, Inc.

  • Website: intelliatx.com
  • Headquarters: Cambridge, Massachusetts, United States
  • Founded: 2014
  • Headcount: 201-500
  • Latest funding type: Post Ipo Equity
  • LinkedIn

Intellia Therapeutics is a biotechnology company focused on developing CRISPR/Cas9 gene editing therapies. They are dedicated to transforming the lives of patients suffering from genetic diseases. Intellia Therapeutics is committed to using their expertise in genome editing to create innovative treatments.


2. Asklepios BioPharmaceutical, Inc. (AskBio)

  • Website: askbio.com
  • Headquarters: Research Triangle Park, North Carolina, United States
  • Founded: 2001
  • Headcount: 501-1000
  • Latest funding type: Acquired
  • LinkedIn

AskBio is a gene therapy company focused on advancing genetic technology and life-saving AAV gene therapy. They offer AB-1005, an adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of mild to moderate Parkinson’s disease. They are committed to investigating the therapeutic potential of GDNF gene therapy for Parkinson’s disease and other related conditions.


3. REGENXBIO Inc.

  • Website: regenxbio.com
  • Headquarters: Rockville, Maryland, United States
  • Founded: 2008
  • Headcount: 201-500
  • Latest funding type: Post Ipo Equity
  • LinkedIn

REGENXBIO is a company dedicated to discovering and developing innovative gene therapies to address severe and debilitating diseases with unmet needs. They aim to improve treatment options and provide curative potential through their gene therapy solutions. With their NAV® Technology Platform, they are driving progress towards making gene therapy a reality for patients.


4. Prevail Therapeutics

Prevail Therapeutics is dedicated to improving treatment outcomes for patients with neurodegenerative diseases through the development of disease-modifying gene therapies.


5. Axovant Gene Therapies, Ltd

  • Website: axovant.com
  • Headquarters: New York, New York, Switzerland
  • Founded: 2014
  • Headcount: 51-200
  • LinkedIn

Axovant is a clinical-stage company focused on developing gene therapies for rare neurodegenerative diseases like GM1 gangliosidosis and Tay-Sachs/Sandhoff disease.


6. Locanabio, Inc.

  • Website: locanabio.com
  • Headquarters: San Diego, California, United States
  • Founded: 2016
  • Headcount: 51-200
  • Latest funding type: Series B
  • LinkedIn

Locana is a biotechnology company that focuses on developing RNA-targeting gene therapies for genetic diseases. They offer a powerful platform called CORRECTx™, which has shown promise in reducing disease-causing repeat expansions in a model of Amyotrophic Lateral Sclerosis. The company's mission is to advance genetic medicines and improve patient outcomes.


7. Neurogene Inc.

  • Website: neurogene.com
  • Headquarters: New York, New York, United States
  • Founded: 2018
  • Headcount: 51-200
  • Latest funding type: Series A
  • LinkedIn

Neurogene is a biotech company focused on developing gene therapies for rare neurological diseases. They are dedicated to addressing the high unmet need in this field and aim to revolutionize medical treatment through innovation and scientific execution. With a strong sense of purpose, Neurogene aims to develop life-changing genetic medicines for individuals and families affected by devastating neurological conditions.


8. GenSight Biologics

GenSight Biologics is a leader in gene therapy against blindness, focusing on the development of therapies to preserve or restore vision in patients suffering from neurodegenerative ophthalmic diseases.


9. Aspen Neuroscience, Inc.

  • Website: aspenneuroscience.com
  • Headquarters: La Jolla, California, United States
  • Founded: 2018
  • Headcount: 11-50
  • Latest funding type: Debt Financing
  • LinkedIn

Aspen Neuroscience is a leading company that specializes in personalized cell therapy for Parkinson's disease. They aim to safely reverse the effects of the disease by leveraging stem cell biology and genomics.


10. Myrtelle

  • Website: myrtellegtx.com
  • Headquarters: Wakefield, Massachusetts, United States
  • Founded: 2020
  • Headcount: 11-50
  • Latest funding type: Series Unknown
  • LinkedIn

Myrtelle is a gene therapy company focused on transforming the treatment of neurological diseases. They are committed to developing innovative approaches to hundreds of disorders involving myelin. Currently, they are conducting a clinical trial for the treatment of the rare monogenetic disorder, Canavan disease.


11. AviadoBio

  • Website: aviadobio.com
  • Headquarters: London, England, United Kingdom
  • Founded: 2019
  • Headcount: 11-50
  • Latest funding type: Series A
  • LinkedIn

AviadoBio is a biotech company specializing in gene therapy for the treatment of neurodegenerative disorders. With a team of experienced scientists and clinicians, AviadoBio aims to develop innovative gene therapies using adeno-associated viral gene vectors. Their focus is on ALS and FTD genes, and they have generated stem cell and transgenic mouse models for research and gene testing purposes. The company has garnered recognition for their contributions to the field of neurodegenerative disorders.


12. NeuExcell Therapeutics

  • Website: neuexcell.com
  • Headquarters: King Of Prussia, Pennsylvania, United States
  • Founded: 2016
  • Headcount: 11-50
  • Latest funding type: Seed
  • LinkedIn

NeuExcell Therapeutics is a gene therapy company focused on neurodegenerative diseases. Their disruptive AtN technology has the potential to regenerate neuronal tissue in a damaged area caused by various neurodegenerative conditions. Their vision is to restore health and improve the quality of life for millions of patients worldwide suffering from neurodegenerative diseases and CNS injuries.


13. Lysogene

  • Website: lysogene.com
  • Headquarters: Neuilly Sur Seine, Ile-De-France, France
  • Founded: 2009
  • Headcount: 11-50
  • Latest funding type: Post Ipo Debt
  • LinkedIn

Lysogene is a French company developing gene therapies for children with CNS diseases. They aim to improve the lives of these children by targeting the causes of disease using their advanced gene therapy technology.


14. Spark Therapeutics, Inc.

  • Website: sparktx.com
  • Headquarters: Philadelphia, Pennsylvania, United States
  • Founded: 2013
  • Headcount: 501-1000
  • Latest funding type: Acquired
  • LinkedIn

Spark is a gene therapy company that strives to challenge the inevitability of genetic disease. They aim to discover, develop, and deliver potential treatments in ways unimaginable, combining their proprietary gene therapy platform with excellence in research and manufacturing.


15. NeurATRIS

  • Website: neuratris.com
  • Headquarters: Fontenay-Aux-Roses, Idf, France
  • Founded: 2012
  • Headcount: 1001-5000
  • LinkedIn

NeurATRIS is a research and service organization that supports innovative therapies for neurodegenerative diseases such as Alzheimer's, Parkinson's, and Huntington's. They also offer therapeutic solutions for multiple sclerosis and neurodevelopmental disorders.


16. Lacerta Therapeutics

  • Website: lacertatx.com
  • Headquarters: Alachua, Florida, United States
  • Founded: 2017
  • Headcount: 51-200
  • LinkedIn

Lacerta Therapeutics is a biotech company specializing in the development and production of AAV-based gene therapies. They aim to provide novel therapeutic options by leveraging their expertise in the nervous system, proprietary AAV capsid technologies, and scalable manufacturing platforms.


17. Capsida Biotherapeutics

  • Website: capsida.com
  • Headquarters: Newbury Park, California, United States
  • Founded: 2019
  • Headcount: 51-200
  • Latest funding type: Corporate Round
  • LinkedIn

Capsida Biotherapeutics is a gene therapy platform company focused on creating targeted, non-invasive gene therapies for patients with debilitating and life-threatening disorders. They aim to improve patient lives by blending innovation with execution.


18. bit.bio

  • Website: bit.bio
  • Headquarters: Cambridge, Cambridgeshire, United Kingdom
  • Founded: 2016
  • Headcount: 51-200
  • Latest funding type: Series B
  • LinkedIn

bit.bio is a cell coding company that specializes in precision reprogrammed human cells for research and drug discovery. They have developed a platform that can create any human cell type and manufacture it at scale with precision and consistency.


19. Sio Gene Therapies

  • Website: siogtx.com
  • Headquarters: New York, New York, United States
  • Founded: 2014
  • Headcount: 51-200
  • Latest funding type: Post Ipo Equity
  • LinkedIn

Sio Gene Therapies is a company that combines cutting-edge science with bold imagination to develop one-time therapies for patients with unmet medical needs. Its aim is to deliver lifelong benefits through gene therapies that transform the treatment of serious neurodegenerative diseases.


20. KIF1A.ORG

  • Website: kif1a.org
  • Headquarters: Alexandria, Kentucky, United States
  • Founded: 2016
  • Headcount: 1-10
  • LinkedIn

KIF1A.org is a nonprofit 501(c)(3) organization working to improve the lives of individuals and families affected by KIF1A Associated Neurological Disorder (KAND) and accelerating research to find treatments and cures. KIF1A Associated Neurological Disorder (KAND) is an ultra-rare neurodegenerative disease caused by variants in the KIF1A gene. Mutations in KIF1A cause a constellation of medical complexities, including cognitive impairment, cerebellar atrophy, ataxia, spastic paraplegia, optic nerve atrophy and epilepsy. KIF1A.ORG works with members of the rare disease community to enable research and discovery of treatments for this generation of people living with KIF1A Associated Neurological Disorder.


21. Coave Therapeutics

  • Website: coavetx.com
  • Headquarters: Paris, Ile-De-France, France
  • Founded: 2014
  • Headcount: 1-10
  • Latest funding type: Series B
  • LinkedIn

Coave Therapeutics is a clinical stage biotechnology company focused on developing life changing gene therapies in rare Ocular and CNS (Central Nervous System) diseases. We are pushing the boundaries of gene therapy through application of our AAV-Ligand Conjugates platform (ALIGATER) that enables targeted delivery, enhanced gene transduction and tissue distribution to improve the effectiveness of advanced gene therapies for rare diseases. Our strategy is to advance a pipeline of novel therapies targeting rare ocular and CNS diseases where targeted gene therapy has the potential to be most effective, where there is a clear unmet need and where success will provide rapid validation of our ALIGATER platform.


22. Tetraneuron

  • Website: tetraneuron.com
  • Headquarters: Madrid, Spain, Spain
  • Founded: 2012
  • Headcount: 1-10
  • Latest funding type: Undisclosed
  • LinkedIn

TETRANEURON is a drug discovery company, focused on the development of a disruptive Gene Therapy for Alzheimer’s disease (AD) and an Early Biomarker for this neurodegenerative condition.


23. Brain Neurotherapy Bio

  • Website: brainneubio.com
  • Headquarters: Raleigh, North Carolina, United States
  • Founded: 2018
  • Headcount: 11-50
  • Latest funding type: Seed

Brain Neurotherapy Bio is a company specializing in gene therapies for patients with neurodegenerative diseases, including Parkinson’s disease and Multiple System Atrophy.


24. Imago Pxi

Imago Pharmaceuticals is a biopharmaceutical company dedicated to the discovery and development of novel medicines to treat neurodegenerative diseases including Alzheimer's Disease, Parkinson's Disease, Idiopathic Pulmonary Fibrosis and Spinal Muscular Atrophy.


25. Ceregene

  • Website: ceregene.com
  • Headquarters: San Diego, California, United States
  • Founded: 2000
  • Headcount: 11-50
  • Latest funding type: Acquired
  • LinkedIn

Ceregene is a San Diego-based biotechnology company that focuses on developing neurotrophic growth factor treatments for major neurodegenerative disorders using a proprietary gene delivery technology.


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