The neurodegenerative disorder gene therapy industry focuses on innovative treatments targeting diseases like Alzheimer’s, Parkinson’s, and ALS through genetic interventions. Companies in this sector typically leverage advanced biotechnology to develop therapies aimed at modifying disease progression rather than merely managing symptoms. Recently, the sector has gained momentum, supported by increased investment and technological advancements in gene delivery systems. The merging of genetics and neurology presents exciting potential for personalized treatments, aiming to address significant unmet medical needs. There’s a growing emphasis on research collaboration and strategic partnerships, propelling industry growth forward.

This list includes a range of companies from global biotechnology firms to innovative startups, differentiating in headquarters from the USA to Europe and Asia. Most companies were founded within the last decade, emphasizing a youthful dynamism in addressing neurodegenerative disorders. Employee sizes vary, with small teams leveraging specialized expertise, while larger companies harness resources for extensive clinical trials. They focus on therapies using gene manipulation techniques, with products targeting Alzheimer’s, Parkinson’s, and related disorders. From venture capital-backed startups to corporate subsidiaries, these companies represent a concentrated effort to tackle challenging health issues.

Read on to discover the top neurodegenerative disorder gene therapy companies.

Top 25 Neurodegenerative Disorder Gene Therapy Companies

1. Prevail Therapeutics

• Website: prevailtherapeutics.com
• Ownership type: Corporate
• Headquarters: New York, New York, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: $880.0M, December 2020
• Founded year: 2017
• Headcount: 51-200
• LinkedIn: prevail-therapeutics

Prevail Therapeutics, founded in 2017 and based in New York City, is a biotechnology firm dedicated to developing innovative gene therapies for neurodegenerative diseases. The company targets conditions such as Parkinson's disease, Gaucher disease, and frontotemporal dementia, aiming to provide treatments that modify the disease process rather than just alleviate symptoms. Prevail's research is grounded in recent advancements in human genetics, focusing on lysosomal dysfunction as a key factor in these disorders. Their pipeline includes several promising programs, notably PR001 and PR006, which are currently in Phase 1/2 clinical trials. Prevail Therapeutics was acquired by Eli Lilly and Company in 2021, becoming a wholly owned subsidiary, which further enhances its capabilities and resources in the biotechnology sector. The company has successfully raised $880 million in funding, underscoring its potential impact in the field of gene therapy for neurodegenerative diseases.

2. AviadoBio

• Website: aviadobio.com
• Ownership type: Venture Capital
• Headquarters: London, England, United Kingdom (UK)
• Employee distribution: United Kingdom (UK) 100%
• Latest funding: $20.0M, October 2024
• Founded year: 2019
• Headcount: 11-50
• LinkedIn: aviadobio

AviadoBio is a biotechnology firm based in London, UK, founded in 2019. The company is dedicated to developing innovative gene therapies aimed at treating neurodegenerative diseases, specifically frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). AviadoBio leverages proprietary gene therapy platforms and advanced delivery technologies to create potentially life-changing treatments for patients suffering from these debilitating conditions. Their research is rooted in pioneering studies from King’s College London and the UK Dementia Research Institute, highlighting their strong academic foundation. Recently, AviadoBio secured $20 million in funding, which underscores investor confidence in their mission and capabilities. The company is currently conducting clinical trials, including a Phase 1/2 study for AVB-101, a gene therapy designed to address genetic mutations associated with FTD. This active involvement in clinical research and their focus on unmet medical needs in neurodegenerative disorders solidify their position in the biotechnology industry.

3. AskBio Inc.

• Website: askbio.com
• Ownership type: Corporate
• Headquarters: Durham, North Carolina, United States (USA)
• Employee distribution: United States (USA) 75%, United Kingdom (UK) 14%, France 6%, Other 5%
• Latest funding: $2.0B, October 2020
• Founded year: 2001
• Headcount: 501-1000
• LinkedIn: askbio

AskBio Inc., also known as Asklepios BioPharmaceutical, Inc., is a biotechnology firm based in Durham, North Carolina. Founded in 2001, the company focuses on gene therapy, utilizing advanced adeno-associated virus (AAV) technology to develop treatments for genetic diseases. AskBio collaborates with researchers and healthcare providers to create therapies that address significant unmet medical needs. The company has a robust pipeline that includes programs targeting neuromuscular and central nervous system disorders, such as Huntington's disease and Parkinson's disease. In 2020, AskBio secured substantial funding, amounting to $2 billion, to support its research and development efforts. This financial backing underscores the company's commitment to advancing gene therapy solutions for patients.

4. Tetraneuron

• Website: tetraneuron.com
• Ownership type: Private
• Headquarters: Madrid, Madrid, Spain
• Employee distribution: Spain 100%
• Latest funding: $5.3M, December 2022
• Founded year: 2012
• Headcount: 1-10
• LinkedIn: tetraneuron-sl

Tetraneuron is a private biotechnology firm based in Madrid, Spain, founded in 2012. The company is dedicated to developing innovative gene therapies aimed at treating neurodegenerative diseases, with a particular emphasis on Alzheimer's disease. Tetraneuron emerged as a spin-off from the Cajal Institute, which is affiliated with the Spanish National Research Council (CSIC). Their scientific team boasts over 30 years of experience in neuroscience, and they are committed to addressing the challenges posed by neurodegenerative conditions through multifactorial mechanisms of action. Tetraneuron's pipeline includes promising therapies that have undergone various stages of validation, and they have recently engaged in strategic alliances to enhance their research capabilities. In December 2022, the company secured funding amounting to approximately €5.3 million, which will support their ongoing projects and initiatives in the field of gene therapy for neurodegenerative disorders.

5. Capsida Biotherapeutics

• Website: capsida.com
• Ownership type: Venture Capital
• Headquarters: Thousand Oaks, California, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: February 2023
• Founded year: 2019
• Headcount: 51-200
• LinkedIn: capsida-biotherapeutics

Capsida Biotherapeutics, founded in 2019 and based in Thousand Oaks, California, is a biotechnology firm dedicated to advancing gene therapy. The company specializes in creating targeted, non-invasive therapies for conditions like genetic epilepsy and Parkinson's disease. Utilizing cutting-edge capsid engineering, Capsida aims to improve the delivery of gene therapies to the central nervous system while minimizing exposure to non-targeted organs. Their pipeline includes wholly owned programs that are designed to achieve significant neuronal transduction, with a focus on addressing unmet medical needs in neurodegenerative disorders. Capsida has established partnerships with notable companies such as AbbVie and Lilly, enhancing their capabilities in clinical development and manufacturing. The company is committed to rapidly advancing their therapies to patients, reflecting their focus on both rare and common diseases across all age groups.

6. BlueRock Therapeutics

• Website: bluerocktx.com
• Ownership type: Venture Capital
• Headquarters: Cambridge, Massachusetts, United States (USA)
• Employee distribution: Canada 51%, United States (USA) 48%
• Latest funding: $1.0B, August 2019
• Founded year: 2016
• Headcount: 201-500
• LinkedIn: bluerocktx

BlueRock Therapeutics LP, based in Cambridge, Massachusetts, is a biotechnology company that specializes in developing innovative cell therapies aimed at treating degenerative diseases. Founded in 2016, the company focuses on conditions such as Parkinson's disease and inherited retinal diseases. BlueRock's mission is to restore lost functions through advanced regenerative medicine, utilizing pluripotent stem cells to create specific cell types that can replace those damaged or lost due to disease. Their lead program, bemdaneprocel, is a cell therapy designed to transplant dopamine-producing neurons into the brains of Parkinson's patients, addressing a significant unmet need in the treatment of this condition. The company operates as a wholly-owned subsidiary of Bayer AG, which supports its efforts in cell and gene therapy. BlueRock has successfully raised substantial funding, totaling $1 billion, to further its research and development initiatives, underscoring its potential in the biotechnology sector.

7. Alcyone Therapeutics Inc.

• Website: alcyonetx.com
• Ownership type: Private
• Headquarters: Lowell, Massachusetts, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: April 2025
• Founded year: 2010
• Headcount: 11-50
• LinkedIn: alcyonelifesciences

Alcyone Therapeutics Inc., founded in 2010 and based in Lowell, Massachusetts, is a biotechnology firm dedicated to pioneering gene-based therapies for neurological disorders. The company is particularly focused on developing innovative solutions for complex conditions such as Rett syndrome. Alcyone's work revolves around precision intra-CSF (cerebrospinal fluid) drug delivery systems and X-reactivation therapies, which aim to enhance the effectiveness of treatments for patients with severe neurological challenges. Their proprietary platforms, including the Falcon™ delivery system and the miREX X-reactivation therapy, showcase their commitment to addressing the unmet needs in the field of neurodegenerative disorders. Alcyone collaborates with leading research institutions to advance their therapeutic technologies, ensuring that their approaches are grounded in rigorous scientific research. The company is actively engaged in clinical studies to evaluate their therapies, demonstrating their ongoing relevance in the biotechnology sector.

8. Encoded Therapeutics Inc.

• Website: encoded.com
• Ownership type: Private Equity
• Headquarters: South San Francisco, California, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Series D, $135.0M, July 2020
• Founded year: 2014
• Headcount: 51-200
• LinkedIn: encodedtherapeutics

Encoded Therapeutics Inc., founded in 2014 and based in South San Francisco, California, is a biotechnology firm dedicated to the development of gene therapies for central nervous system disorders. The company operates with a clear mission to create transformative treatment options that address the underlying genetic causes of these conditions. Encoded Therapeutics has established a robust pipeline, including their lead program ETX101, which is aimed at treating Dravet Syndrome. This condition is characterized by severe seizures and can lead to neurodegenerative outcomes. The company has received significant funding, with a Series D round raising $135 million in July 2020, which supports their research, clinical studies, and manufacturing capabilities. Encoded Therapeutics is committed to advancing gene therapy as a viable treatment option for both common and rare CNS disorders, leveraging their proprietary technology platform to enhance the specificity and efficacy of their therapies.

9. Exegenesis Bio

• Website: exegenesisbio.com
• Ownership type: Private Equity
• Headquarters: Philadelphia, Pennsylvania, United States (USA)
• Employee distribution: China 65%, United States (USA) 35%
• Latest funding: Series B, February 2021
• Founded year: 2019
• Headcount: 51-200
• LinkedIn: exegenesis

Exegenesis Bio is a biotechnology firm based in Philadelphia, Pennsylvania, founded in 2019. The company specializes in gene and cell therapies, with a mission to develop innovative genetic medicines for various diseases, including neurodegenerative disorders. Their pipeline features therapies targeting Spinal Muscular Atrophy and Parkinson's Disease, showcasing their commitment to addressing significant health challenges. Exegenesis Bio has established a strong research and development foundation, supported by advanced manufacturing capabilities. The company has raised over $120 million in funding, which underscores its potential for growth and innovation in the gene therapy sector. With a global presence that includes offices in the United States, China, and Singapore, Exegenesis Bio aims to enhance access to cutting-edge therapies for patients worldwide.

10. Astellas Gene Therapies

• Website: astellasgenetherapies.com
• Ownership type: Private
• Headquarters: San Francisco, California, United States (USA)
• Employee distribution: United States (USA) 100%
• Founded year: 2012
• Headcount: 201-500
• LinkedIn: audentes-therapeutics

Astellas Gene Therapies, based in San Francisco, California, is a biotechnology firm dedicated to creating innovative gene therapies aimed at treating rare genetic diseases. Founded in 2012, the company operates with a workforce of approximately 409 employees. Their mission is to transform the lives of patients suffering from conditions such as X-Linked Myotubular Myopathy and Pompe disease. Astellas Gene Therapies is recognized for its robust pipeline of gene therapies, particularly focusing on rare diseases affecting the eye, central nervous system, and neuromuscular system. They utilize an advanced adeno-associated virus (AAV) platform for their gene therapy drug discovery process, which is complemented by their internal manufacturing capabilities. The company emphasizes strong relationships with patients and families to enhance their understanding of the diseases they target, thereby optimizing their research and clinical development efforts. Astellas has also been involved in significant collaborations, such as a recent agreement for a gene therapy targeting Frontotemporal Dementia, further solidifying their commitment to addressing neurodegenerative disorders.

11. Coave Therapeutics

• Website: coavetx.com
• Ownership type: Venture Capital
• Headquarters: Paris, Île-De-France, France
• Employee distribution: France 100%
• Latest funding: Series A, $32.9M, January 2025
• Founded year: 2014
• Headcount: 1-10
• LinkedIn: horama

Coave Therapeutics is a biotechnology firm based in Paris, France, founded in 2014. The company specializes in developing innovative gene therapies aimed at treating rare ocular and central nervous system (CNS) diseases. Utilizing their proprietary ALIGATER platform, Coave Therapeutics enhances the delivery and effectiveness of genetic medicines. Their pipeline includes promising candidates for neurodegenerative diseases such as Multiple System Atrophy and ALS, showcasing their commitment to addressing unmet medical needs in this area. The company has received significant funding, including a recent Series A round totaling approximately €33 million, which underscores their potential for growth and development in the gene therapy sector. Coave Therapeutics collaborates with various research institutions to advance their therapeutic solutions, positioning themselves as a notable player in the gene therapy landscape.

12. Aspen Neuroscience, Inc.

• Website: aspenneuroscience.com
• Ownership type: Venture Capital
• Headquarters: San Diego, California, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Other (Grant), $8.0M, May 2024
• Founded year: 2018
• Headcount: 51-200
• LinkedIn: aspen-neuroscience

Aspen Neuroscience, Inc., based in San Diego, California, is a biotechnology firm founded in 2018. The company specializes in personalized cell therapies aimed at treating Parkinson's disease, a condition that affects millions globally. Aspen leverages advanced stem cell biology and genomics to develop autologous neuron replacement therapies, which are designed to address significant unmet medical needs in neurodegenerative diseases. Their flagship product, ANPD001, is currently under investigation in a Phase 1/2a clinical trial for sporadic Parkinson's disease. The company has received $8 million in funding through a grant in May 2024, indicating ongoing support for their innovative research and development efforts. Aspen's mission is to provide effective, patient-specific treatments that could potentially modify the course of Parkinson's disease, making them a notable player in the field of gene therapy for neurodegenerative disorders.

13. remynd

• Website: remynd.com
• Ownership type: Venture Capital
• Headquarters: Leuven, Flanders, Belgium
• Employee distribution: Belgium 100%
• Latest funding: Series B, $14.6M, January 2021
• Founded year: 2002
• Headcount: 11-50
• LinkedIn: remynd-nv

reMYND is a biotechnology firm based in Leuven, Belgium, founded in 2002. The company specializes in developing treatments for Alzheimer's and Huntington's diseases, utilizing a proprietary drug discovery platform that enables the identification of novel mechanisms of action and first-in-class small molecules. Their lead program, ReS19-T, is currently in clinical trials, demonstrating promising results in restoring synaptic function and managing calcium homeostasis in Alzheimer's patients. Additionally, reMYND's Huntington program, ReS18-H, aims to neutralize the neurotoxic effects of the mutated huntingtin protein, showing potential for significant therapeutic impact. The company has received substantial funding, including a Series B round that raised approximately €24 million, which supports their ongoing research and development efforts. Furthermore, reMYND operates a Contract Research Organization (CRO) that collaborates with major pharmaceutical companies, providing critical insights into the pharmacokinetics and efficacy of experimental treatments for neurodegenerative diseases.

14. Atalanta Therapeutics

• Website: atalantatx.com
• Ownership type: Venture Capital
• Headquarters: Boston, Massachusetts, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Series B, $97.0M, January 2025
• Founded year: 2018
• Headcount: 51-200
• LinkedIn: atalanta-therapeutics

Atalanta Therapeutics, founded in 2018 and based in Boston, Massachusetts, is a biotechnology firm dedicated to pioneering treatments for neurodegenerative diseases. The company leverages innovative RNA interference technology to develop therapies aimed at halting diseases like Huntington's at their source. With a team of 51-200 employees, Atalanta is focused on creating effective interventions for patients and families affected by these conditions. The company has recently secured $97 million in Series B funding, underscoring its potential and the interest of investors in its mission to address urgent healthcare needs in the neurodegenerative space.

15. Sania Therapeutics

• Website: saniarx.com
• Ownership type: Venture Capital
• Headquarters: London, England, United Kingdom (UK)
• Employee distribution: United Kingdom (UK) 100%
• Latest funding: Seed, $5.0M, March 2022
• Founded year: 2021
• Headcount: 1-10
• LinkedIn: sania-therapeutics

Sania Therapeutics is a biotechnology company based in London, UK, founded in 2021. The company is dedicated to creating precision therapies aimed at treating nervous system disorders. Their mission is to transform medicine by addressing the root causes of prevalent and underserved diseases. With a team of experts in neuroscience and drug development, Sania Therapeutics is developing innovative treatments that leverage targeted delivery systems and controllable therapeutics. They have received seed funding amounting to approximately $5 million in March 2022, which supports their research and development efforts. The company is currently preparing for clinical trials in 2026 for their first indication, focusing on spasticity, and they are exploring additional indications in areas of high unmet need.

16. Latus Bio

• Website: latusbio.com
• Ownership type: Venture Capital
• Headquarters: Philadelphia, Pennsylvania, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Series A, $54.0M, May 2024
• Founded year: 2022
• Headcount: 11-50
• LinkedIn: latus-biosciences

Latus Bio, founded in 2022 and based in Philadelphia, Pennsylvania, is a biotechnology firm dedicated to advancing gene therapy solutions for central nervous system disorders. The company is particularly focused on conditions such as Huntington's disease and CLN2, aiming to enhance patient care through innovative treatment methods. Latus Bio employs cutting-edge technology to develop gene therapy candidates that utilize precision delivery systems, addressing challenges associated with traditional gene therapy approaches. Their recent Series A funding of $54 million, secured in May 2024, underscores their potential for growth and innovation in the neurodegenerative disorder space. With a team of experienced scientists and clinicians, Latus Bio is committed to transforming the treatment landscape for patients suffering from debilitating CNS diseases.

17. Neuraly Inc.

• Website: neuralymed.com
• Ownership type: Venture Capital
• Headquarters: Gaithersburg, Maryland, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Series A, $36.0M, July 2018
• Founded year: 2016
• Headcount: 51-200
• LinkedIn: neuraly-inc

Neuraly Inc. is a clinical-stage biotechnology company based in Gaithersburg, Maryland, founded in 2016. The company is dedicated to developing innovative treatments for neurodegenerative diseases, particularly Parkinson's and Alzheimer's. Neuraly aims to translate scientific discoveries into new drugs that can significantly improve the lives of patients suffering from these debilitating conditions. Their lead product, NLY01, is a long-acting GLP-1 receptor agonist that targets glial cell activation, which plays a critical role in neuroinflammation and neuronal cell death. Neuraly has received substantial funding, with a reported $36 million in Series A financing in 2018, which supports their ongoing clinical trials and research efforts. The company is committed to advancing its pipeline of therapies that could potentially modify the course of neurodegenerative diseases, addressing a significant unmet medical need.

18. BIORCHESTRA Co., Ltd.

• Website: biorchestra.com
• Ownership type: Private Equity
• Headquarters: Cambridge, Massachusetts, United States (USA)
• Employee distribution: South Korea 100%
• Latest funding: Series C, $45.7M, February 2022
• Founded year: 2016
• Headcount: 51-200
• LinkedIn: biorchestra-co-ltd

BIORCHESTRA Co., Ltd. is a biotechnology firm based in Cambridge, Massachusetts, with a significant operational presence in Daejeon, South Korea. Founded in 2016, the company specializes in RNA-based therapeutics aimed at treating neurodegenerative diseases, including Alzheimer's disease and amyotrophic lateral sclerosis (ALS). BIORCHESTRA has developed a proprietary drug delivery system known as BDDS™ (BIORCHESTRA Drug Delivery System), which facilitates the effective delivery of RNA therapies across the blood-brain barrier. This technology is crucial for ensuring that therapeutic agents reach their intended targets in the central nervous system. The company is currently advancing its lead compound, BMD-001, which has demonstrated potential in preclinical studies to reverse cognitive deficits in relevant animal models. In February 2022, BIORCHESTRA successfully raised $45.7 million in Series C funding, underscoring its commitment to expanding its research and development efforts. The company collaborates with pharmaceutical partners and healthcare providers to bring innovative therapeutic solutions to market, addressing critical needs in the treatment of neurodegenerative disorders.

19. Belief BioMed

• Website: beliefbiomed.com
• Ownership type: Private
• Headquarters: Shanghai, Shanghai, China
• Employee distribution: China 100%
• Founded year: 2018
• Headcount: 201-500
• LinkedIn: belief-biomed

Belief BioMed Inc. is a biotechnology firm based in Shanghai, China, established in 2018. The company specializes in gene therapy products, utilizing advanced adeno-associated virus (AAV) vectors to develop innovative treatments for severe genetic and chronic diseases. Their research and development efforts are robust, with a pipeline that includes therapies for conditions such as hemophilia, Parkinson's disease, and neuromuscular diseases. Belief BioMed has made significant strides in establishing manufacturing capabilities in China, ensuring they can produce clinical-grade gene therapy products. They have received notable recognition, including Orphan Drug Designation from the U.S. FDA for their hemophilia B treatment, BBM-H901, which is the first gene therapy under development for hemophilia in China. The company is committed to making effective therapies accessible to patients and healthcare providers.

20. QurAlis

• Website: quralis.com
• Ownership type: Venture Capital
• Headquarters: Cambridge, Massachusetts, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Series B, $88.0M, March 2023
• Founded year: 2016
• Headcount: 51-200
• LinkedIn: quralis1

QurAlis is a biotechnology firm based in Cambridge, Massachusetts, founded in 2016. The company is dedicated to developing precision medicines for neurodegenerative diseases, particularly focusing on ALS and FTD. QurAlis leverages advanced scientific research and proprietary platforms, such as their FlexASO™ and QR43™ platforms, to create therapeutic candidates that address significant unmet medical needs. Their innovative approach is underscored by a commitment to understanding the underlying mechanisms of diseases like ALS, which is linked to TDP-43 pathology. The company has made notable strides in the industry, including the first human dosing of their lead candidate, QRL-201, and has received significant funding, raising $88 million in their Series B round in March 2023. This funding supports their ongoing research and development efforts, positioning QurAlis as a key player in the neurodegenerative disorder gene therapy space.

21. AcuraStem

• Website: acurastem.com
• Ownership type: Private
• Headquarters: Pasadena, California, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Other (Grant), $4.0M, September 2024
• Founded year: 2016
• Headcount: 11-50
• LinkedIn: acurastem-inc.

AcuraStem is a biotechnology firm based in Pasadena, California, founded in 2016. The company specializes in developing innovative treatments for neurodegenerative diseases, particularly amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Utilizing their proprietary iNeuroRx® technology platform, AcuraStem aims to create effective, patient-based therapeutics that address the complexities of these conditions. Their approach emphasizes the importance of understanding human disease mechanisms, moving beyond traditional animal models. Recently, AcuraStem secured $4 million in grant funding from the California Institute for Regenerative Medicine to advance their targeted therapeutic program for the UNC13A gene, which plays a significant role in ALS and FTD. The company is committed to accelerating the development of their promising treatments and actively engages in research and development to fulfill their mission.

22. Skyhawk Therapeutics

• Website: skyhawktx.com
• Ownership type: Venture Capital
• Headquarters: Waltham, Massachusetts, United States (USA)
• Employee distribution: Switzerland 50%, United States (USA) 50%
• Latest funding: $133.0M, September 2021
• Founded year: 2016
• Headcount: 51-200
• LinkedIn: skyhawk-therapeutics

Skyhawk Therapeutics, founded in 2016 and based in Waltham, Massachusetts, is a biotechnology firm dedicated to developing small molecule therapeutics that modify RNA expression. The company is particularly focused on addressing challenging diseases, including neurodegenerative disorders like Huntington's Disease. Their lead candidate, SKY-0515, is currently in clinical trials and has demonstrated a significant reduction in HTT mRNA levels, indicating its potential effectiveness in treating Huntington's Disease. Skyhawk operates with a robust pipeline that also includes programs targeting Spinocerebellar Ataxia Type 3 and Frontotemporal Dementia, both of which currently lack approved therapeutic options. The company has secured substantial funding, with a recent round totaling $133 million, which supports their research and development efforts. Collaborations with major pharmaceutical companies such as Biogen and Merck further enhance their capabilities and reach within the industry.

23. Neuron23

• Website: neuron23.com
• Ownership type: Venture Capital
• Headquarters: South San Francisco, California, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Series C, $100.0M, March 2022
• Founded year: 2018
• Headcount: 51-200
• LinkedIn: neuron23-inc

Neuron23 is a biotechnology company based in South San Francisco, California, founded in 2018. The company is dedicated to developing precision medicines aimed at genetically defined neurological and immunological diseases. Neuron23 utilizes cutting-edge data science and human genetics to create targeted therapies, particularly focusing on conditions like Parkinson's disease. They are currently advancing their clinical-stage program, which includes a first-in-human trial of NEU-723, a potent LRRK2 inhibitor designed to modify the progression of Parkinson's disease. This program is notable as it represents one of the few small molecules targeting LRRK2 in clinical development. Neuron23 has also established collaborations, such as with QIAGEN, to develop companion diagnostics that will help identify patients likely to benefit from their therapies. The company has successfully raised $100 million in funding as of March 2022, which underscores their potential and commitment to addressing significant unmet medical needs in neurodegenerative diseases.

24. TrueBinding

• Website: truebinding.com
• Ownership type: Venture Capital
• Headquarters: Foster City, California, United States (USA)
• Latest funding: $57.7M, July 2023
• Founded year: 2016
• Headcount: 51-200
• LinkedIn: immutics

TrueBinding Inc., founded in 2016 and based in Foster City, California, is a clinical-stage biotherapeutic company dedicated to creating novel monoclonal antibody therapeutics. The company specializes in addressing neurodegenerative diseases such as Alzheimer's and Parkinson's, as well as other medical conditions with significant unmet needs. TrueBinding's innovative approach leverages its proprietary platform technology to develop targeted treatments that aim to improve patient outcomes. The company is actively conducting clinical trials, including an Expanded Access Program for its investigational drug TB006, which shows promise in treating Alzheimer's disease. TrueBinding has recently secured funding of approximately $57.7 million, indicating investor confidence in its potential to advance therapies in the biopharmaceutical sector. Additionally, TrueBinding offers contract development services, enhancing its role in the biopharmaceutical industry by supporting other companies in the efficient production of monoclonal antibodies.

25. Ionis Pharmaceuticals, Inc.

• Website: ionis.com
• Ownership type: Private
• Headquarters: Carlsbad, California, United States (USA)
• Employee distribution: United States (USA) 99%, Other 1%
• Founded year: 1989
• Headcount: 501-1000
• LinkedIn: ionis-pharmaceuticals

Ionis Pharmaceuticals, Inc., founded in 1989 and based in Carlsbad, California, is a biotechnology firm that focuses on developing genetic medicines and RNA-targeted therapeutics. The company has a robust pipeline aimed at treating a variety of diseases, with a significant emphasis on neurological conditions. Ionis collaborates closely with patients to enhance their understanding of disease management and improve therapeutic outcomes. Their innovative approach has led to the development of several approved therapies, including SPINRAZA for spinal muscular atrophy and Qalsody for amyotrophic lateral sclerosis. Ionis is recognized for its pioneering work in antisense oligonucleotide technology, which has positioned it at the forefront of genetic medicine. The company continues to push the boundaries of science to deliver meaningful treatments for patients suffering from debilitating diseases.

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