Accelerate drug development for neurological diseases SYNAXYS is French biotech specialized in the characterization of the response of the nervous system for nutraceutic, pharmaceutic and veterinary applications. We offer a complete range of R&D services to support drug development, from defining the dose effects in a human model, to identify biological targets and action mechanism, acute and chronic toxicity, or comparison to established treatments. Our field of application: - Drug positioning - Therapeutic evaluation in a diease model - Neuro-oncology - Data generation for IA modeling Neuro-engineering, Neuronal diseases modeling, Neuro-oncology, Autism, Alzheimer disease, and Glioblastoma

101 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

Bringing Precision Medicine to Neuroscience Drug Development Monument Tx is an independent UK based biotechnology company formed as a spin-out from Cambridge Cognition in 2021. Monument Tx develops products to treat serious diseases of the central nervous system. Our precision medicine approach utilises proprietary digital biomarkers to reduce heterogeneity and identify patients with homogenous underlying neurobiology, which are then matched with appropriately targeted compounds. Using biomarkers to more accurately identify patients with a disease sub-type most likely to respond to a particular treatment is already successfully used in the treatment of cancer. Now Monument Tx brings digital biomarkers to neuroscience …

26 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

Cognito Therapeutics is a pioneer in non-invasive neuromodulation to treat neurodegenerative diseases. Cognito Therapeutics is a pioneer in non-invasive neuromodulation to treat neurodegenerative diseases and improve human cognitive performance. The company has completed multiple clinical studies demonstrating its investigational therapeutic has the potential to safely slow or stop cognitive decline and loss of brain volume in Alzheimer’s disease. Cognito has received FDA Breakthrough Device Designation for the treatment of Alzheimer’s disease and expects to start pivotal studies in 2022. The company’s technology was based on pioneering optogenetics research by scientific co-founders Professors Li-Huei Tsai and Ed Boyden at MIT.

17 similar entities Type: Startup Activities: biotech deeptech healthtech Technologies: Synthetic Biology

we aim to develop new therapeutics for the treatment of brain disorders based on human cerebral organoids a:head’s drug discovery approach is based on human cerebral organoids, a revolutionary, paradigm-shifting new technology that allows the generation of mini brains from human stem cells in vitro. They capture the essential aspects of human embryonic brain development including brain patterning, formation of brain ventricles, differentiation into the various neuronal and supportive cell types and their correct three-dimensional arrangement. The technology has been pioneered by the company’s co-founders Madeline Lancaster and Juergen Knoblich and is exclusively licensed to a:head by the Institute of …

21 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

Therapeutics for the Disease of Aging Immune therapy platform targeting Dark Genome antigens that contribute to aging, cancer, diabetes, and neurodegenerative diseases Oncology, Aging, Dark Genome, ALS, Diabetes, and Longevity

198 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

Vivet Therapeutics is dedicated to developing innovative gene therapy treatments for orphan diseases. Vivet Therapeutics is focused on optimizing gene therapy through collaborating with the Fundacion para la Investigacion Medica Aplicada (CIMA, Universidad de Navarra) to develop new AAV vectors specifically targeting the liver and generating new technologies to optimize gene delivery and long term expression. Gene Therapy

91 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

We build high tech equipment supporting end-to-end development and manufacture of cell and gene therapies. We build high tech equipment supporting end-to-end development and manufacture of cell and gene therapies. biotech, medtech, cell therapy, gene therapy, CAR-T, MSC, iPS, ATMP, Tissue Engineering, cell based therapies, biotherapies, RMAT, and Manufacturing

428 similar entities Tags: FrenchTech2030 Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

Integra Therapeutics is a biotech company engineering the next generation of gene writing tools to cure genetic diseases At Integra Therapeutics we have combined the precision of CRISPR systems with the gene transfer efficiency of viral integrases and transposases. Harnessing what nature has evolved to introduce large pieces of DNA into the genome, together with the precision of sequence specific DNA binding proteins, has allowed for unprecedented efficiency in programmable gene delivery.

112 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

RNA Therapeutics We develop first-in-class RNA-based therapies, with lead programs in Head & Neck Squamous Cell Carcinoma (HNSCC) and Facioscalpulohumeral Muscular Dystrophy (FSHD). Our approach to drug design revolves around using genomic patient data to create highly tailored therapeutics – the right drug for the right patient. Our proprietary DREAmiRTM platform utilizes genomic and outcome data from thousands of patients to identify underlying genetic changes that cause their disease, and then creates a novel RNA therapeutic that can directly target and fix that genetic abnormality.

113 similar entities Type: Startup Activities: deeptech biotech healthtech Technologies: Synthetic Biology Data Analytics

Development of innovative neurological therapeutics based on pannexin channels Panntherapi develops innovative drugs in Neurology by acting on a specific target, the activated pannexin 1 channel, aiming a better efficacy and tolerance than current drugs. Panntherapi offers breakthrough innovation for the two main issues in development of current drugs in neurological diseases: - Innovative target addressing the root cause of the diseases - Representative non-clinical model using among others ex vivo human brain tissues Biotech, Neurology, Epilepsy, and Deeptech

65 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

Platelet-derived Regenerative Therapy for CNS Disorders InVenis Biotherapies is a biotech compagny, spin-off from academic laboratory U1172 and Taipei Medical University, developping innovative regenerative therapies based on blood platelet. Thanks to the high therapeutic potential of our product, we target the most severe diseases involving cell death, as the amyotrophic lateral sclerosis.

217 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

The CNS Regeneration Company Axoltis pharma is a biopharmaceutical company dedicated to develop a first-in-class disease-modifying drug to treat patients with neurodegenerative or traumatic neurological disorders with high unmet medical needs. Our innovative approach: combining Preventative, Neuroprotective, Regenerative & Remodeling properties in a single drug, as the key to cure complex neurological disorders. Our products are first-in-class multifunctional peptides with high potential in many CNS disorders. neurodegenerative diseases, neurodegeneration, spinal cord injury, peptide, neuroprotection, neural repair, regeneration, drug, biotechnology, pharma, CNS, neurology, ALS, Parkinson, and Multiple Sclerosis

62 similar entities Type: SMB Activities: biotech deeptech Technologies: Synthetic Biology

Neurotech & photomedicine REGEnLIFE is an innovative company developing cutting-edge therapy for neurodegenerative disorders. Our company supports a non-invasive medical approach based on physical sciences. Our mission is to develop innovative and responsible technologies to face up to medical challenges and public health needs. Our goal is to provide to healthcare professionals a technology able to alter the progression of neurodegenerative diseases, in order to improve the health and quality of life to promote a healthy aging process. Our technology consists in a novel medical device dedicated to people living with neurodegenerative diseases, specifically Alzheimer-type dementia. MedTech, NeuroTech, Neurophonic, Photonics, …

87 similar entities Type: Startup Activities: biotech deeptech healthtech manufacturing Technologies: Synthetic Biology

CNS/PNS - In vitro - In vivo - Drug Development Neuro-Sys is a research-intensive and innovative organisation specialising in pharmacology and in advanced in vitro and in vivo models of neurodegenerative diseases, mode of action research, and drug development in the CNS and PNS fields. We provide pharmacological profiles of lead compounds and explore their underlying mechanism of action and studies to determine the neuroprotective activity. We’re dedicated to providing high-level technical expertise in a collaborative relationship with our clients and accompany you along the drug development stages, from preclinical to clinical. Our expertise: › Alzheimer’s disease (AD) › Parkinson’s …

33 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

Coave therapeutics 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 …

86 similar entities Type: Startup Activities: deeptech biotech healthtech Technologies: Synthetic Biology

Delivering the next generation gene therapies AAVantgarde is a clinical-stage, international biotechnology company (headquartered in Italy) that has developed two proprietary adeno-associated viral (AAV) vector platforms to address the DNA cargo capacity limitations of AAV vectors Gene Therapy, Ophthalmology, and Rare diseases

338 similar entities Type: Startup Activities: healthtech biotech deeptech Technologies: Synthetic Biology

Targeting Glial Cells and Brain Metabolism to Treat Neurological Diseases GliaPharm is a Swiss-based biotechnology company that develops treatments for neurological and psychiatric disorders. GliaPharm's innovative therapeutic approach is to target glial cells, the 'support cells'​ of neurons, to sustain and enhance brain metabolism in pathological conditions. Our vision is to become a leading biotechnology company with a focus on glia-mediated pathways as agents for neuroprotection and maintenance of cognitive functions.

14 similar entities Type: Startup Activities: healthtech deeptech Technologies: Synthetic Biology

Precision treatment of immune disorders Polyneuron is a growing Basel-based biotechnology company active in the area of autoimmune disease treatments. The company was founded in 2014 as a spinout company from the University of Basel to develop novel treatments to combat immune disorders using its Antibody-CatchTM technology platform. The company currently employs about 10 people and has raised a significant amount in equity funding from top-tier VCs. We are a team of passionate and experienced scientists, entrepreneurs and investors willing to bring new therapies to patients.

129 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

AveXis is now Novartis Gene Therapies Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases. Utilizing cutting-edge technology, we are working to turn promising gene therapies into proven treatments, beginning with our transformative gene therapy for spinal muscular atrophy (SMA). Follow us on LinkedIn: https://www.linkedin.com/company/novartis-gene-therapies/ How to reach us: Novartis Gene Therapies 2275 Half Day Road, Suite 200 Bannockburn, IL 60015 Email: gtx.communications@novartis.com Office Phone: 847.572.8280 Toll-free Phone: 844.428.3947 Gene Therapy, Synthetic Biology, and Spinal Muscular Atrophy

68 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

Changing the paradigm in Friedreich's ataxia Biointaxis is an academic spin-off focused on the development of gene therapies for neurodegenerative disorders. Biointaxis lead product is a gene therapy product for Friedreich ataxia.

59 similar entities Type: Startup Activities: deeptech Technologies: Synthetic Biology

New treatments for central nervous system disorders Neurolixis Inc. is a biopharmaceutical company focused on the discovery and development of novel drugs for the treatment of human central nervous system diseases including movement disorders, autism spectrum disorders, depression and pain. Neuroscience, Drug development, Rett Syndrome, Parkinson's disease, Biased agonism, and Orphan Drug

142 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

Catalyzing the development of transformative genetic-based therapeutics from concept to clinic. Dedicated to discovery + translational research and led by Dr. James Wilson, the Gene Therapy Program (GTP) at the University of Pennsylvania focuses on the development of next-generation gene transfer vectors and their application in the treatment of a variety of acquired and inherited diseases. Dr. Wilson's commitment to patients with rare diseases stems from his days as a graduate student and medical student at the University of Michigan between 1977 and 1984. He was discouraged by the lack of available treatments for rare disease patients, but optimistic that …

12 similar entities Type: SMB Activities: biotech deeptech healthtech Technologies: Synthetic Biology

AstronauTx is tackling dementia through improving brain physiology AstronauTx is at the forefront of a novel approach to treating neurodegenerative diseases. We have a unique portfolio of small-molecule therapeutics to restore dysregulated physiology to both improve symptoms and modify disease.

101 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

Developing life-changing, innovative therapies for diseases with unmet needs Impel Pharmaceuticals is focused on developing and providing transformative therapies for people suffering from diseases with high unmet needs across various disease areas in addition to the central nervous system (CNS), through the pioneering of an optimized approach to drug delivery. We offer and develop treatments that pair the Company’s proprietary Precision Olfactory Delivery (POD®) technology with well-established therapeutics. Intranasal Drug Delivery, Migraine, Parkinson's Disease, CNS Therapeutics, and Autism

122 similar entities Type: Startup Activities: healthtech deeptech biotech Technologies: Synthetic Biology

TREVENTISTM Corporation is dedicated to treating and preventing protein misfolding diseases. We utilize a proprietary, patent-pending discovery engine – Common Conformational Morphology (CCM) – to identify druggable active sites in misfolded protein targets. CCM combines unique in silico models with deep expertise in model development (in vitro, ex vivo, in vivo) to enable rational drug design against misfolded protein targets. Our lead program focuses on anti-misfolding small molecules in tauopathy (3R, 4R, familial mutations and mixed) with relevance to Alzheimer’s disease. We have further CCM-driven efforts in oncology and other neurodegenerative diseases that show the wide utility of our technology …

Type: Startup Activities: healthtech deeptech biotech Technologies: Synthetic Biology

Unlocking New Targets to Transform the Treatment of Neurodegenerative Diseases Unlocking New Targets to Transform the Treatment of Neurodegenerative Diseases neurodegeneration, RNA therapeutics, physiological upregulation, post-transcriptional upregulation, mRNA, ASO, and SINEUP

279 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

Accelerating treatments for millions of patients Arctic Therapeutics is a clinical-stage biopharmaceutical company on a mission to treat and even prevent some of the world’s most pressing diseases by utilizing genetic sequencing to analyze and map the genetic causes of diseases at a granular level, applying our findings to develop effective treatments. Our company was founded by Dr. Hakon Hakonarson in 2015 as a spin-off from the US-based Center for Applied Genomics (CAG), which he launched in 2006 and established it as the largest pediatric biobank and genomic database in the world. We maintain close research relationships with CAG and …

165 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

Innovative disease modifying treatment for Parkinson's patients Synuca Therapeutics combines decades of groundbreaking research in P-type ATPases and neuroscience from Aarhus University to propose a novel disease-modifying treatment for patients suffering from Parkinson's Disease and associated disorders. By targeting the SERCA calcium transporter, our first-in-class small molecules salvage the root disease cause by repairing the neuronal calcium imbalances caused by pathological α-Synuclein brain species.

26 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

We turn blood stem cells into living drug factories At Immitra Bio, we are pioneering a next-generation gene therapy platform, harnessing Hematopoietic Stem Cells and their downstream cell lineages, such as red blood cells as living, in-vivo biotherapeutic protein factories. The CRISPR-Cas-based cell and gene therapy platform we are developing is versatile and promises to be easily reprogrammable.

221 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

A new hope for patients affected by Alzheimer’s disease CaSRevolution is a research company specialized in devising innovative therapies for neurodegeneration diseases We have discovered a new mechanism that explains the progression of Alzheimer’s Disease and identified a potential therapeutic approach biotech, neurodegeneration, startup, and alzheimer

115 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

Developing therapies for severe leukodystrophies Overview SynaptixBio is a rare disease biotech company that is pioneering ground-breaking treatments for severe leukodystrophies and other childhood neurodegenerative diseases. SynaptixBio’s mission is to deliver gene therapies for fatal leukodystrophies in children caused by loss of function or mutation in a single gene. Using a breadth of platforms including antisense oligonucleotide (ASO)-based technology, we can silence the expression of the toxic gene to reverse disease progression. This approach will create life saving treatments for severe genetic diseases that affect the central nervous system. Pharmaceutical, Biotech, Leukodystrophy, Rare Disease, H-ABC, Hypomyelination, Basal Ganglia, Cerebrllum, Neurology, …

59 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

We Fight Neurodegeneration FundaMental Pharma is engaged in pioneering research around a breakthrough molecular mechanism that allows to safely counteract glutamate neurotoxicity (also known as excitotoxicity) by specifically targeting the interface between NMDA receptors and TRPM4 channels with small molecule TwinF interface inhibitors (Yan et al., Science 2020). TwinF interface inhibitors constitute an entirely new class of drugs that safely ameliorate glutamate neurotoxicity, a common cause of neurodegeneration. Our initial focus is on amyotrophic lateral sclerosis (ALS). The applicability of our molecules extends to a range of neurogenerative disorders including Alzheimer’s disease (AD).

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Developing the Next Generation Gene Editing Technology Pencil Biosciences is developing a truly innovative gene modulation technology that can have an impact across a range of applications, including new therapeutic options for patients with rare diseases. Genome Engineering, Research and Development, and Gene Editing

299 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

Ascend is an advanced therapy development partner with a flexible platform featuring industry-leading analytics. Ascend is gene to GMP partner with deep expertise in advanced therapy development and commercialization. We offer unparalleled collaboration backed by expertise while working to develop your products from the bench through the clinic and beyond. Let’s build the future of advanced therapies together!

61 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

Better Technology. Better Delivery. Developing a new generation of disease-modifying therapies. Driven by our proprietary Enhanced Delivery Oligonucleotide (EDO) platform, we are creating a pipeline of disease-modifying therapeutics with the potential to safely and effectively target the root cause of serious genetic neuromuscular and neurological disorders. Biotechnology, Chemistry, Biology, Bioscience, Life sciences, and Therapeutics

436 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

Treating Neurodegenerative Disease by Restoring Autophagy At Samsara Therapeutics, we’re on a mission to unlock the full potential of autophagy activation in combating age-related neurodegenerative diseases. Our innovative approach aims to delay the progression of several debilitating conditions, offering new possibilities for patients and their families. biotechnology, biopharmaceuticals, rare disease, neurodegeneration, longevity, drug discovery, and drug development

159 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

Powering a drug discovery revolution to deliver cures for brain diseases that affect at least 1 in 10 people worldwide Brain diseases have no cures and affect 1 in 10 people worldwide and their families. BRAINCURES aims to tackle brain diseases with precision medicine based on the code for brain function. Our landmark decoding of the molecular framework behind brain health pinpointed SMART Target-Biology linkages that now empower novel translational avenues to conquer brain diseases through SMART Patient Stratification and SMART Treatments. The application of our proprietary platform technologies will lead to personalized treatments for the 350 million people worldwide …

Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

Outsmarting complex diseases with more intelligent medicines Our mission is to create a new generation of smarter medicines that outmaneuver complex diseases in ways previously inconceivable. To accomplish this mission, we are building a synthetic biology platform that could enable us to program next-generation cell and gene therapies with what we refer to as “gene circuits”. These gene circuits, which are created from novel and proprietary combinations of DNA sequences, are intended to reprogram cells with biological logic to sense inputs, compute decisions and respond to their cellular environments. We are designing gene circuits to improve the “intelligence” of cell …

82 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

Genewity is a pioneering biotech start-up focused on advanced thymus-driven therapies for immune reconstitution. Supported by the extensive business expertise of our management and advisors, we are at the forefront of developing and commercialising breakthrough therapies. With over 150 years of combined experience in immunology and gene therapy, our team is uniquely positioned to drive advancements in the field.

275 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

Follow Astellas Pharma for updates on our pipeline of genetic medicines for patients with rare diseases. The Astellas Gene Therapies page is no longer active or monitored. gene therapy and biotechnology

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Activating RNA, mastering disease Harnessing an innate mechanism of gene activation, MiNA Therapeutics'​ platform enables the development of new medicines that restore normal function to patients’ cells. We are applying our technology and clinical know-how to transform the therapy landscape of severe liver and other diseases. Gene activation, RNA therapeutics, Liver disease, and Drug discovery

213 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

Clinical-stage lentiviral gene therapy company, powered by plato®. Our vision: bring personalized gene therapy worldwide Our vision is to bring personalized gene therapy to the world. We target the root cause of genetic disease by introducing a functional copy of the affected gene into patients’ own hematopoietic stem cells (HSCs), with the goal of durably expressing the therapeutic protein throughout the body, including the central nervous system. Our first-in-class pipeline includes clinical programs for Gaucher disease and Hunter syndrome, as well as a preclinical program for Pompe disease. Our proprietary plato® gene therapy platform is scalable for planned global commercialization. …

45 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

Breaking through barriers in gene therapy and neurology Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, Friedreich’s ataxia, Parkinson’s disease, amyotrophic lateral sclerosis (ALS), and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACERTM AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of …

71 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

Precigen (Nasdaq: PGEN) is a biopharmaceutical company advancing the next generation of gene and cell therapies. Precigen: Advancing Medicine with PrecisionTM Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and …

63 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

Developing first in class therapies for the treatment of dementia. Cogentis Therapeutics is an early stage biotechnology company with a mission to develop and commercialize an innovative disease-modifying therapeutic for dementias such as frontotemporal dementia (FTD) and Alzheimer’s disease (AD) to improve patients’ quality of life and reduce the burden of disease on their families, providers, and the overall healthcare system. Our immediate goal is to develop a peptide-based compound, CT-526, for FTD. neurodegeneration, neuropeptide, frontotemporal dementia, Alzheimer's disease, and progressive supranuclear palsy

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Novel disease-modifying therapies for neurodegenerative diseases. From brains for brains. From us for you. We are a clinical-stage biotechnology company that strives to challenge established treatment options for neurodegenerative diseases in order to preserve the quality of life of patients. WE FOCUS ON UNMET MEDICAL NEEDS IN CHRONIC DISEASES Currently available treatments for many neurodegenerative diseases aim to manage symptoms, and do not stop disease progression. As a result, neurodegenerative diseases often lead to a reduced quality of life. Better treatments for these diseases are needed to give patients healthier, happier, and longer lives. We aim to develop therapeutic and …

4 similar entities Type: SMB Activities: biotech deeptech Technologies: Synthetic Biology

Pushing the boundaries of gene therapy to develop treatments for debilitating neurodegenerative diseases. Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinson’s disease, which affects millions of patients globally. Led by an experienced team of gene therapy development …

32 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

Transforming the lives of patients through gene therapy uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with severe genetic diseases. aav, Huntington's disease, gene therapy, ALS, epilepsy, and Fabry disease

160 similar entities Type: SMB Activities: biotech deeptech healthtech Technologies: Synthetic Biology

GeneCode is a private pharmaceutical development company focused on the development of disease-modifying therapeutics to combat neurodegeneration. The GDNF Mimetics platform leverages the disease-modifying properties of GDNF to small molecules with drug-like properties. R&D-wise, GeneCode has remarkably improved the properties of the identified hits, making them more active and effective, drug-like and better show their ability to combat neurodegeneration in Parkinson ́s disease (PD).

60 similar entities Type: SMB Activities: biotech deeptech Technologies: Synthetic Biology

Toward the next generation of gene therapy. Spur Therapeutics (formerly Freeline Therapeutics) is a clinical-stage biotechnology company focused on developing life-changing therapies for debilitating chronic conditions. By optimizing every component of our product candidates, we are improving genetic expression and targeted delivery to realize outsized clinical results. Building on the successes of our two potential first-in-class therapies for Gaucher disease and adrenomyeloneuropathy (AMN), we’re moving from rare diseases toward more widespread conditions, including forms of Parkinson’s and dementia, and even certain cardiovascular diseases. Expanding our impact, and pushing forward to new frontiers of genetic medicine. Toward life-changing therapies, and brighter …

88 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology