Michael J. Fox Foundation

Accure Therapeutics is a private pharma R&D engine at clinical stage in the CNS field.

ndLyz Therapeutics, Inc. is developing disease-modifying therapeutics which restore endo-lysosomal function and abrogate neurodegeneration in Parkinson’s disease (PD) and other dementias.

C2N Diagnostics, LLC is a privately held protein diagnostic and therapeutic discovery company targeting progressive neurodegeneration. They formed in late 2007 by scientific co-founders Drs. David Holtzman and Randall Bateman of Washington University School of Medicine in St. Louis, and LifeTech Research, a Maryland-based technology research and commercialization firm (www.lifetechresearch.com). They reside at the Center for Emerging Technologies in St. Louis.

Bexion Pharmaceuticals, Inc. is a biotechnology company based in Covington, Kentucky, focused on developing biologic therapies for cancer. Established in 2006, the company is pioneering innovative treatments, notably BXQ-350, which employs a unique mechanism of action to target and eliminate tumor cells. This approach involves the identification of Phosphatidylserine patches found on tumor cells and neovasculature, leading to apoptosis through the activation of acid sphingomyelinase, resulting in ceramide generation and elevated caspase levels. Additionally, Bexion aims to address challenges like chemotherapy-induced peripheral neuropathy by programming cell death, reducing tumor proliferation, and stimulating the immune response.

NRG Therapeutics is a drug discovery company established in 2018 that specializes in developing therapeutic approaches aimed at restoring mitochondrial function to address neurodegenerative diseases, including Parkinson’s disease, Alzheimer’s disease, and motor neuron disease. The company focuses on creating novel medicines that preserve mitochondrial function and halt disease progression. With a team experienced in neuroscience drug discovery, NRG Therapeutics is initially concentrating on the discovery and development of brain-penetrant inhibitors of the mitochondrial permeability transition pore specifically for Parkinson’s disease. This strategic focus is intended to empower physicians with effective treatment options for patients suffering from chronic neurodegenerative disorders.

Selonterra is a biopharmaceutical company that works on Alzheimer's and Parkinson's disease therapies and diagnostics. The company is based in the San Francisco Bay Area that is backed by venture capital.

Herantis Pharma is a biotechnology company focused on addressing unmet clinical needs through the development of disease-modifying therapies for Parkinson's disease. The company's primary development program centers on HER-096, a peptidomimetic molecule engineered to preserve the biological activity of the neuroprotective CDNF protein. By targeting the underlying mechanisms of the disease, Herantis Pharma aims to provide innovative treatment options for patients suffering from Parkinson's disease.

MISSION Therapeutics is a biotechnology company specializing in developing drugs that target the cellular process of deubiquitylation for the treatment of cancer and other diseases. Their focus is on developing small molecule drugs that selectively inhibit specific deubiquitylating enzymes (DUBs), which play crucial roles in regulating protein degradation and cellular processes. These drugs aim to intervene in disease mechanisms where DUB dysregulation is implicated, offering potential therapeutic benefits.

Kannalife, a subsidiary of Neuropathix, Inc (OTCQB: NPTX), is a biopharmaceutical company focused on discovering, developing, and commercializing a product platform of novel molecules from our proprietary next-generation cannabinoid therapeutics as potent, non-opioid alternatives to treat patients with a variety of unmet medical needs.

Alchemab Therapeutics Ltd is a biotechnology company based in London, established in 2019 by Uri Laserson and Jane Osbourn. The company focuses on discovering and developing novel therapeutics that utilize naturally protective antibodies to combat hard-to-treat diseases, including certain cancers, neurodegenerative conditions, and infectious diseases. Alchemab employs an unbiased, function-first approach to identify drug targets, leveraging three complementary processes and technologies to create a robust pipeline of antibody therapeutics. By harnessing these naturally occurring antibodies, Alchemab aims to provide innovative treatment options for patients facing challenging medical conditions.

Lucy Therapeutics is a biotech company focused on developing innovative treatments for neurological diseases that originate from mitochondrial dysfunction. The company utilizes a unique approach to drug discovery by targeting early disease mechanisms, which holds potential for addressing a range of neurological disorders, including Parkinson's Disease and Alzheimer's Disease. In addition to its therapeutic efforts, Lucy Therapeutics is engaged in the development of proprietary small molecule therapies and diagnostic biomarkers aimed at enhancing the understanding and treatment of central nervous system conditions. By leveraging genetic technologies, the company aims to provide deeper insights into personalized medicine, thereby improving patient outcomes and enabling more precise healthcare interventions in the neurological field.

NeuroRestore is a research and innovation institution that develops and implements medical remedies to restore neurological functions.

IRLAB Therapeutics AB is a Swedish research and development company focused on creating innovative treatments for neurological disorders, particularly Parkinson’s disease. The company is advancing several drug candidates, including Mesdopetam, a dopamine D3 receptor antagonist, and IRL752, which targets postural dysfunction. Both compounds have successfully completed Phase IIa clinical trials. Additionally, IRLAB is exploring other research programs aimed at addressing neurodegenerative disorders and aging, such as IRL942, P001, and P003, which seeks alternatives to levodopa for Parkinson’s treatment. By focusing on the core dysfunctions associated with Parkinson’s disease and dementias, IRLAB aims to enhance the quality of life for patients suffering from these conditions. The company is headquartered in Gothenburg, Sweden.

Octave is a clinical platform developer focused on transforming the care landscape for multiple sclerosis and other neurodegenerative diseases. The company's platform generates, analyzes, and integrates data to provide a comprehensive approach aimed at reducing the overall cost of care. By optimizing healthcare medications and utilization, Octave enables pharmaceutical companies to enhance the entire lifecycle of drug discovery, clinical trials, and post-marketing processes through the use of real-world evidence. This innovative approach seeks to improve patient outcomes while streamlining healthcare practices.

Nine Square Therapeutics focuses on developing small-molecule therapeutics aimed at treating degenerative movement disorders such as Parkinson's disease and amyotrophic lateral sclerosis (ALS), as well as non-degenerative conditions like essential tremor and dyskinesias. Founded in 2020 and based in New York, the company employs a combination of computational chemistry, biophysics, and computational biology, utilizing machine-learning-enabled cell profiling platforms for target identification, chemical optimization, and patient stratification. This innovative approach allows for a more efficient and effective strategy in addressing the challenges posed by these complex medical conditions.

Cantabio Pharmaceuticals is a preclinical stage biotechnology company based in Palo Alto, California, dedicated to the research, development, and commercialization of innovative therapies for neurodegenerative diseases, particularly Parkinson's disease (PD) and Alzheimer's disease (AD). The company’s lead programs include CB101, a small molecule pharmacological chaperone targeting the DJ-1 protein for PD treatment; CB201, an engineered DJ-1 protein designed to penetrate the central nervous system for PD; CB301, a chaperone aimed at the Tau protein for AD; and CB401, which targets the Aβ protein for AD. Additionally, Cantabio has a collaboration with the Luxembourg Institute of Health to explore the therapeutic potential of the DJ-1 protein in immune-associated diseases. Founded in 2009, Cantabio is also involved in developing therapeutic proteins that can cross the blood-brain barrier to address protein function loss in various disease states.

Neuronity cures neurological disorders by promoting immunity of the central nervous system. Neuronity was founded in 2022 and was headquartered in Mansfield, United States.

Ventus Therapeutics is a biopharmaceutical company focused on discovering and developing novel small molecule medicines aimed at treating autoimmune diseases, inflammatory diseases, and cancer by targeting the innate immune system. The company employs a structural immunology platform that provides deep insights into the mechanisms and molecular structures involved in immune responses. This platform integrates proprietary protein engineering capabilities with advanced rational and structure-based drug design tools, allowing for precise targeting of the innate immune system. Ventus is actively building a pipeline of drug programs that address critical targets within this system, particularly proteins involved in inflammasome and nucleic acid-sensing signaling pathways, thereby enabling the treatment of both acute and chronic inflammatory and immune-related conditions.

Lys Therapeutics is a biotechnology company focused on developing therapies for patients with neurovascular and neurodegenerative disorders. The company is centered around its lead product, Glunozumab, a monoclonal antibody that operates through a unique mechanism of action. This drug induces off-target toxicity by hyperactivating vascular and neuronal NMDA receptors, which can lead to significant disruptions in the blood-brain and blood-spinal cord barriers, as well as neuronal excitotoxicity. These effects are particularly relevant in conditions such as stroke and multiple sclerosis, where the modulation of neuroinflammation is critical for treatment.

Olatec is a Phase 2 clinical stage biotech company at the forefront of an emerging class of innovative, oral inflammation therapeutics. The Company’s lead drug candidate, dapansutrile, is a proprietary specific NLRP3 inhibitor. The Company has advanced dapansutrile from discovery to its current later-stage clinical development in arthritis, diabetes, neuroinflammatory diseases and cancer. Dapansutrile is positioned to become a breakthrough treatment with early human data showing its potential to safely modulate the body’s immune system allowing for the restoration of immune balance, a necessary step to treat or prevent disease progression and prognosis in patients. Olatec’s preclinical and clinical studies have been published in top medical and scientific journals. On the strength of dapansutrile’s benign safety profile and robust clinical data, Olatec’s clinical trials are now being conducted in well-known international centers of excellence. With a mission to translate scientific potential into medical practice and become best-in-class in treating chronic inflammation that drives disease progression with a cost-effective and patient-friendly (orally dosed) therapeutic. The Company conducts operations in the United States and Europe and maintains offices in New York and the Netherlands.

Selonterra is a biopharmaceutical company that works on Alzheimer's and Parkinson's disease therapies and diagnostics. The company is based in the San Francisco Bay Area that is backed by venture capital.

NRG Therapeutics is a drug discovery company established in 2018 that specializes in developing therapeutic approaches aimed at restoring mitochondrial function to address neurodegenerative diseases, including Parkinson’s disease, Alzheimer’s disease, and motor neuron disease. The company focuses on creating novel medicines that preserve mitochondrial function and halt disease progression. With a team experienced in neuroscience drug discovery, NRG Therapeutics is initially concentrating on the discovery and development of brain-penetrant inhibitors of the mitochondrial permeability transition pore specifically for Parkinson’s disease. This strategic focus is intended to empower physicians with effective treatment options for patients suffering from chronic neurodegenerative disorders.

Muna Therapeutics is a biopharmaceutical company focused on discovering and developing therapies aimed at slowing or halting the progression of neurodegenerative diseases, such as Parkinson's, Alzheimer's, Frontotemporal Dementia, and Multiple Sclerosis. The company is dedicated to identifying new medicines that preserve cognitive function and enhance resilience against these debilitating conditions, thereby providing individuals affected by neurodegenerative diseases with access to effective treatment options. Through its innovative approach, Muna Therapeutics strives to improve the quality of life for those suffering from these challenging disorders.

Seelos Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics aimed at addressing unmet medical needs in central nervous system disorders and other rare conditions. The company's lead programs include SLS-002, an intranasal formulation of racemic ketamine designed to treat acute suicidal ideation and behavior in patients with major depressive disorder and post-traumatic stress disorder; SLS-005, a protein stabilizer for Sanfilippo syndrome; and SLS-006, a partial dopamine agonist for Parkinson's disease. Additionally, Seelos has several preclinical programs, such as SLS-007, an anti-alpha-synuclein peptidic inhibitor targeting Parkinson's; SLS-008, an antagonist for chronic inflammation in asthma; SLS-010, an oral treatment for narcolepsy; and SLS-012, an injectable therapy for post-operative pain management. Founded in 2016 and headquartered in New York, Seelos aims to improve patient outcomes in both psychiatric and movement disorders, including orphan diseases.

ZyVersa Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative drugs for patients suffering from inflammatory and renal diseases that have significant unmet medical needs. Based in Weston, Florida, the company has a promising pipeline that includes VAR 200, a cholesterol efflux mediator currently ready for phase 2a trials, aimed at treating focal segmental glomerulosclerosis, a rare kidney disease. Additionally, ZyVersa is developing a novel inflammasome inhibitor, designed for the treatment of various inflammatory diseases. The company utilizes advanced, proprietary technologies to create first-in-class therapies, underscoring its commitment to addressing the challenging medical needs in its target patient populations. ZyVersa Therapeutics was formerly known as Variant Pharmaceuticals, Inc., and rebranded in May 2019.

With a prolific platform of small molecule therapies, innate immune checkpoint inhibitors, cell based therapies, vaccines and oncolytic viruses, the central focus of EpicentRx Inc., a privately owned physician-led phase 3 clinical-stage immuno-oncology company located in San Diego, California, is on anticancer immunization and chemo/radioprotection of normal tissues. As a company of actively practicing oncologists/internists they at EpicentRx take cancer very very personally (in a literal and figurative sense) with the plight of the patient serving first and foremost as the motivation to customize or personalize their platform of immunotherapies both to individuals that are otherwise out of options and to groups of individuals with common and in many cases currently undruggable mutations.

Alzprotect is a biopharmaceutical company focused on developing drug candidates for the treatment of neurodegenerative diseases, particularly Alzheimer's disease and orphan tauopathies like Progressive Supranuclear Palsy. The company's research emphasizes the role of hyperphosphorylated Tau proteins and associated inflammation in neuronal degeneration. By advancing its drug candidates from discovery through clinical trials, Alzprotect aims to provide effective therapeutic options for conditions characterized by abnormal tau protein aggregation and related neurodegenerative processes.

Asceneuron develops effective therapeutics for orphan tauopathies and Alzheimer’s disease. By focusing on areas of high unmet medical need, Asceneuron aspires to become a leading biotech company specialized in small molecule drug discovery for neurodegenerative diseases. Tauopathies are currently untreatable neurodegenerative diseases that rapidly progress towards debilitating conditions. The appearance of deposits of the microtubule-associated tau protein as e.g. neurofibrillary tangles in neurons of the brain is a common feature of tauopathies that is shared with Alzheimer’s disease. Neuronal tau deposits are known to be a major contributor to neurodegeneration and mutations in the tau gene are causative of the tauopathy fronto-temporal dementia and parkinsonism linked to chromosome 17 (FTDP-17). Due to increasing life expectancy, Alzheimer’s disease is viewed as one of the largest healthcare problems of this century, imposing a major economic burden on societies in the Western and developing world. Current treatment options provide limited benefits supporting the urgent need for more efficacious and better tolerated medicines that address symptomatic relief as well as disease progression.

AcureX Therapeutics is a biotechnology company focused on addressing neurodegenerative diseases, with an initial emphasis on Parkinson's Disease. The company specializes in the enhancement of mitophagy, a cellular process that eliminates dysfunctional mitochondria, which is crucial in the development of Parkinson's and similar disorders. By targeting the mechanisms responsible for mitophagy impairment, AcureX Therapeutics aims to facilitate the identification of druggable targets that can lead to effective treatments for these debilitating conditions.

Lucy Therapeutics is a biotech company focused on developing innovative treatments for neurological diseases that originate from mitochondrial dysfunction. The company utilizes a unique approach to drug discovery by targeting early disease mechanisms, which holds potential for addressing a range of neurological disorders, including Parkinson's Disease and Alzheimer's Disease. In addition to its therapeutic efforts, Lucy Therapeutics is engaged in the development of proprietary small molecule therapies and diagnostic biomarkers aimed at enhancing the understanding and treatment of central nervous system conditions. By leveraging genetic technologies, the company aims to provide deeper insights into personalized medicine, thereby improving patient outcomes and enabling more precise healthcare interventions in the neurological field.

Arkuda Therapeutics is a biotechnology company based in Cambridge, Massachusetts, founded in 2018. The company focuses on developing innovative medicines for patients suffering from neurological diseases, particularly targeting neurodegenerative conditions. Arkuda leverages insights into progranulin and lysosomal biology to create therapies aimed at addressing progranulin deficiency and lysosomal dysfunction, specifically in genetically-defined frontotemporal dementia related to mutations in the GRN gene. Its lead compounds are designed to improve cellular health in the brain, with the goal of delaying disease progression and the onset of symptoms.

MISSION Therapeutics is a biotechnology company specializing in developing drugs that target the cellular process of deubiquitylation for the treatment of cancer and other diseases. Their focus is on developing small molecule drugs that selectively inhibit specific deubiquitylating enzymes (DUBs), which play crucial roles in regulating protein degradation and cellular processes. These drugs aim to intervene in disease mechanisms where DUB dysregulation is implicated, offering potential therapeutic benefits.

Sinopia Biosciences is focused on advancing drug discovery and disease research by utilizing a data-driven platform that emphasizes phenotypic screening. This approach has proven more effective than traditional target-based methods in identifying first-in-class medications, as it accounts for the complex nature of many diseases and their unclear etiology. The company's platform combines high-throughput screening, multi-omics data, artificial intelligence, and network analysis to uncover novel therapeutic targets and mechanisms of disease. By enabling researchers to investigate diseases and potential drug candidates in greater detail, Sinopia Biosciences aims to facilitate the development of innovative treatments.

NRG Therapeutics is a drug discovery company established in 2018 that specializes in developing therapeutic approaches aimed at restoring mitochondrial function to address neurodegenerative diseases, including Parkinson’s disease, Alzheimer’s disease, and motor neuron disease. The company focuses on creating novel medicines that preserve mitochondrial function and halt disease progression. With a team experienced in neuroscience drug discovery, NRG Therapeutics is initially concentrating on the discovery and development of brain-penetrant inhibitors of the mitochondrial permeability transition pore specifically for Parkinson’s disease. This strategic focus is intended to empower physicians with effective treatment options for patients suffering from chronic neurodegenerative disorders.

MapLight Therapeutics offers a platform designed to discover and develop therapeutics for brain disorders. It combines single-cell transcriptomics and optogenetics to identify novel drug targets and develop effective therapies for brain disorders.

Caraway Therapeutics, Inc. is a biotechnology company focused on developing innovative treatments for neurodegenerative diseases through the modulation of mitophagy and autophagy. By targeting genetically defined pathways, the company aims to create small molecules that restore cellular balance and address the underlying mechanisms of diseases such as Parkinson's disease and amyotrophic lateral sclerosis. Founded in 2018 and based in Cambridge, Massachusetts, Caraway Therapeutics seeks to improve the lives of patients affected by these debilitating conditions by leveraging its expertise in cellular degradation processes. The company was previously known as Rheostat Therapeutics, Inc. before rebranding in October 2019.

Lundbeck is a global pharmaceutical company highly committed to improving the quality of life of people living with psychiatric and neurological disorders. For this purpose, Lundbeck is engaged in the research, development, production, marketing, and sale of pharmaceuticals across the world. The company’s products are targeted at disease areas such as depression, schizophrenia, Parkinson’s disease, and Alzheimer’s disease. According to the World Health Organization, WHO, more than 700 million cases of psychiatric and neurological disorders are reported every year. These are serious and life-threatening diseases that affect the quality of life of the patients as well as of their relatives. As these diseases also involve major socio-economic costs, it is imperative for the general society that new and innovative pharmaceuticals are developed. Over the past 50 years, new pharmaceuticals have revolutionized the treatment options, but there remains a large unmet need for new and innovative therapeutics. Focus on R&D is the most important pillar in Lundbeck’s ambition to improve treatment for people living with psychiatric and neurological disorders. We are specialists in our area and have research facilities in Denmark and China, and more than 1,000 employees work in our R&D units. We cooperate closely with strategic partners all over the world, ensuring the best possible foundation for innovation and the development of new treatment solutions. Lundbeck employs approximately 5,300 people worldwide, 1,700 of whom are based in Denmark. We have employees in 55 countries, and our products are registered in more than 100 countries. We have production facilities in China, Denmark, France and Italy and research centers in Denmark and China.

Sinopia Biosciences is focused on advancing drug discovery and disease research by utilizing a data-driven platform that emphasizes phenotypic screening. This approach has proven more effective than traditional target-based methods in identifying first-in-class medications, as it accounts for the complex nature of many diseases and their unclear etiology. The company's platform combines high-throughput screening, multi-omics data, artificial intelligence, and network analysis to uncover novel therapeutic targets and mechanisms of disease. By enabling researchers to investigate diseases and potential drug candidates in greater detail, Sinopia Biosciences aims to facilitate the development of innovative treatments.

Nitrase Therapeutics is a biopharmaceutical platform company focused on developing therapies targeting a newly identified class of enzymes known as Nitrases. Initially concentrating on Parkinson’s disease, the company aims to create drugs that may slow or halt the progression of this and other related neurodegenerative conditions. By leveraging its expertise in nitration, Nitrase Therapeutics seeks to explore the therapeutic potential of biochemical modifications of proteins, particularly through the identification of nitro-substrates and their involvement in various diseases. This approach positions the company to potentially expand its platform to address a broader range of medical conditions in the future.

Casma Therapeutics, Inc. specializes in developing innovative therapeutic strategies centered on the natural cellular process of autophagy and lysosomal flux. By focusing on enhancing autophagy, the company aims to improve the clearance of unwanted proteins, organelles, and pathogens, thereby addressing the progression of various diseases. Its research targets several serious medical conditions, including lysosomal storage disorders, muscle disorders, inflammatory disorders, and neurodegeneration. Founded in 2017 and based in Cambridge, Massachusetts, Casma Therapeutics is dedicated to creating effective treatment options that meet significant unmet medical needs in the field of drug discovery and development.

Inflazome Ltd. is a biotechnology company based in Dublin, Ireland, founded in 2016. It specializes in developing orally available drugs aimed at addressing unmet clinical needs in inflammatory diseases by targeting the inflammasome, a component involved in the inflammatory process. The company’s innovative therapies are designed to block inflammasome signals, effectively tackling various inflammatory-driven conditions. These include orphan diseases, central nervous system disorders such as Alzheimer's and Parkinson's, systemic inflammatory diseases like cardiovascular and pulmonary conditions, and applications in ophthalmology and dermatology. By targeting the root causes of inflammation, Inflazome enables the development of precise treatments for a range of diseases, thereby enhancing therapeutic options for healthcare providers. As of September 2020, Inflazome operates as a subsidiary of Roche Holding AG.

Alkahest, Inc. is a clinical stage biopharmaceutical company based in San Carlos, California, founded in 2014. The company focuses on developing therapies derived from blood to address neurological diseases, particularly those associated with aging, such as Alzheimer’s disease and Parkinson’s disease. Alkahest's research has revealed that factors present in the blood plasma of younger mice can potentially reverse cognitive deficits in aging and Alzheimer’s disease models. Collaborating with Grifols, a global healthcare company known for its plasma therapies, Alkahest aims to enhance health and vitality through innovative treatments that target the effects of aging.

MISSION Therapeutics is a biotechnology company specializing in developing drugs that target the cellular process of deubiquitylation for the treatment of cancer and other diseases. Their focus is on developing small molecule drugs that selectively inhibit specific deubiquitylating enzymes (DUBs), which play crucial roles in regulating protein degradation and cellular processes. These drugs aim to intervene in disease mechanisms where DUB dysregulation is implicated, offering potential therapeutic benefits.

Inhibikase Therapeutics has developed the first small molecule platform capable of treating both bacterial and viral infectious diseases through a common mechanism of action in the host. The company's host targeting strategy creates multi-pathogen anti-infectives without stimulating resistance.

Addex Therapeutics Ltd is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, that specializes in the discovery, development, and commercialization of small-molecule drugs targeting central nervous system (CNS) disorders. The company primarily focuses on allosteric modulators, a novel class of orally available drugs that interact with G-protein coupled receptors, potentially offering advantages over traditional therapeutic approaches. Its lead product candidates include Dipraglurant for treating Parkinson's disease-related dyskinesia and dystonia, ADX71149 for epilepsy and other CNS disorders, and GABAB PAM for addiction. Addex has established a collaboration with Janssen Pharmaceuticals Inc. to develop mGluR2PAM compounds aimed at addressing various health issues. Founded in 2002, Addex Therapeutics continues to advance its innovative drug discovery platform to improve therapeutic outcomes for patients with neurological conditions.

Neuropore Therapies is a pharmaceutical company located in San Diego, CA committed to the discovery and development of disease-modifying treatments for neurodegenerative disorders, including Parkinson’s disease and Alzheimer’s disease. They are located on Road to the Cure in San Diego, CA.Their approach is based on the fundamental concept that the pathological accumulation of misfolded and aggregated neurotoxic proteins is the underlying basis of these disorders. Their therapeutic candidates directly target these proteins and restore cellular autophagy mechanisms to degrade and dispose of these neurotoxic proteins.

nLife Therapeutics S.L. is a biopharmaceutical company based in Granada, Spain, with an additional office in Barcelona. Founded in 2009, the company specializes in developing innovative treatments for central nervous system (CNS) and neuromuscular disorders through the use of cell-specific oligonucleotides. nLife Therapeutics is focused on creating a first-in-class disease-modifying therapy aimed at selectively silencing alpha-synuclein in monoaminergic neurons to address the toxic accumulation associated with Parkinson’s disease. Additionally, the company is working on therapies to treat Huntington's Disease by lowering levels of altered Htt in striatal and cortical neurons to hinder disease progression. Their technology platform enables the targeted delivery of conjugated nucleic acids to specific neurons via a single intra-nasal application, with ongoing efforts to advance clinical trials for these promising treatments.

Domain Therapeutics is a biopharmaceutical company located in Strasbourg, France, dedicated to the discovery and early development of small molecules targeting G-coupled Protein Receptors (GPCRs), one of the best classes of drug targets. Domain Therapeutics is using its innovative and proprietary technology platform DTect-Allâ„¢ to identify orthosteric and allosteric ligands of GPCRs, including the so called Silent Allosteric Modulators (SAMs) that represent a unique source of compounds that cannot be discovered by standard technologies. DTect-Allâ„¢ is able to address difficult GPCRs such as orphan and peptidic GPCRs.

Motus Therapeutics is a biotechnology company based in Boston, Massachusetts, focused on developing peptide therapeutics to address unmet medical needs in metabolic diseases, particularly gastrointestinal disorders. The company's lead product candidate, relamorelin, is a ghrelin agonist designed to treat diabetic gastroparesis, a complication associated with diabetes, as well as other gastrointestinal functional disorders. Motus Therapeutics aims to provide innovative solutions for patients suffering from these conditions.

ZelosDx specializes in the development of blood testing products aimed at advancing research in brain health and diseases. The company provides researchers with access to proprietary technology that can be licensed and adapted for blood testing kits and automated laboratory equipment. Additionally, it offers pharmaceutical collaborators biomarker technology to support drug discovery efforts. By delivering critical data for diagnosis, monitoring, and evaluating therapeutic responses, ZelosDx facilitates significant advancements in the understanding and treatment of brain-related conditions.

Addex Therapeutics Ltd is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, that specializes in the discovery, development, and commercialization of small-molecule drugs targeting central nervous system (CNS) disorders. The company primarily focuses on allosteric modulators, a novel class of orally available drugs that interact with G-protein coupled receptors, potentially offering advantages over traditional therapeutic approaches. Its lead product candidates include Dipraglurant for treating Parkinson's disease-related dyskinesia and dystonia, ADX71149 for epilepsy and other CNS disorders, and GABAB PAM for addiction. Addex has established a collaboration with Janssen Pharmaceuticals Inc. to develop mGluR2PAM compounds aimed at addressing various health issues. Founded in 2002, Addex Therapeutics continues to advance its innovative drug discovery platform to improve therapeutic outcomes for patients with neurological conditions.

Envoy Therapeutics focuses on drug discovery with the aim of developing new medications that demonstrate improved efficacy and reduced side effects compared to existing treatments. Utilizing its innovative bacTRAP® technology, the company identifies proteins produced by specific cell types in vivo, eliminating the need for cell isolation. This capability is particularly significant in brain tissues, where numerous cell types are closely intermingled. By allowing for the targeted modulation of specific cell types based on their unique protein expression, Envoy Therapeutics enhances the possibilities for therapeutic interventions, marking a significant advancement in the field of drug discovery.

Trevena, Inc. is a biopharmaceutical company based in Chesterbrook, Pennsylvania, established in 2007. The company is dedicated to the development and commercialization of innovative medicines aimed at treating central nervous system disorders. Its product candidates include Oliceridine injection, which is designed for managing moderate-to-severe acute pain and has completed Phase III clinical trials, and TRV250, a delta-opioid receptor agonist that has finished Phase I trials for acute migraine treatment. Other candidates in development include TRV734, targeting both acute and chronic pain, and TRV027, which is being evaluated for acute heart failure. Additionally, Trevena is collaborating with Imperial College London to study TRV027 in the context of COVID-19. The company employs a specialized approach using G protein biased ligands to enhance the efficacy and safety of its therapeutics, addressing the limitations of existing GPCR-targeted drugs.

Sapiens Steering Brain Stimulation GmbH is an emerging medical device company whose mission is to deliver superior solutions for deep brain stimulation (DBS) therapy. Sapiens' ambition is to provide a unique, high-resolution, and MRI- compatible DBS system that will improve patient comfort and therapeutic outcome, initially for patients with Parkinson's disease. Sapiens, with offices in Eindhoven, the Netherlands and Munich, Germany, is founded in 2011 as a spin-out of Royal Philips Electronics. Its steering brain stimulation (SBS) system and image-guided programming are based upon patented technologies.

Amicus Therapeutics is a biotechnology company dedicated to discovering, developing, and commercializing therapies for rare and orphan diseases. The company is known for its product Galafold, an orally administered small molecule pharmacological chaperone specifically designed for treating Fabry disease. Amicus is also advancing its clinical pipeline, including a Phase 3 study of AT-GAA for Pompe disease. The company focuses on creating novel therapies, particularly pharmacological chaperones, which are intended to stabilize and enhance the activity of misfolded enzymes in patients. This approach may improve treatment outcomes, especially when used in conjunction with enzyme replacement therapy. Amicus Therapeutics was founded in 2002 and is headquartered in Cranbury, New Jersey, collaborating with institutions like Nationwide Children's Hospital and the University of Pennsylvania to further its research and development efforts.

Ceregene is a privately held biotechnology company based in San Diego, specializing in the treatment of major neurodegenerative disorders through the delivery of nervous system growth factors. The company's clinical programs feature CERE-110, an AAV2-based vector expressing nerve growth factor, which is set to enter Phase 2 studies for Alzheimer's disease. Additionally, CERE-120 has recently completed a Phase 2 trial for Parkinson's disease and is under evaluation for future development. Ceregene is also advancing two other treatments, CERE-135 and CERE-140, which are in preclinical stages targeting amyotrophic lateral sclerosis (ALS) and ocular diseases, respectively.

Omeros Corporation is a Seattle-based biopharmaceutical company that focuses on discovering, developing, and commercializing small-molecule and protein therapeutics for a range of indications, including inflammation, complement-mediated diseases, and central nervous system disorders. The company is known for its commercial product, OMIDRIA, which is used during cataract surgery in the United States. Omeros has several clinical programs, with its lead candidate, Narsoplimab, in Phase III trials for conditions such as thrombotic microangiopathy and immunoglobulin A nephropathy, as well as Phase II trials for lupus nephritis. Other clinical programs include OMS405 for opioid and nicotine addiction and OMS527 for addiction and compulsive disorders. The company also has a robust pipeline of preclinical programs targeting various disorders, including MASP-3 for paroxysmal nocturnal hemoglobinuria and a GPCR platform aimed at treating a broad range of diseases. Omeros was incorporated in 1994 and remains dedicated to addressing significant unmet medical needs.