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— Amyotrophic Lateral Sclerosis Pipeline Market Companies Involved in Therapeutics Development are 2-BBB Medicines BV, AB Science SA, Aestus Therapeutics Inc, Anavex Life Sciences Corp, Angion Biomedica Corp, Apodemus AB, Apogenix GmbH, Apotex Inc, ArmaGen Inc, BioCrea GmbH, Biogen Inc, Biohaven Pharmaceutical Holding Company Limited, BioHealthonomics Inc, BrainStorm Cell Therapeutics Inc, Calico LLC, Catabasis Pharmaceuticals Inc, Cellceutix Corp, Chronos Therapeutics Ltd, ContraVir Pharmaceuticals Inc, Corcept Therapeutics Inc, Curatis Pharma GmbH, Cytokinetics Inc, Daval International Ltd, Edison Pharmaceuticals Inc, Eisai Co Ltd, Ensemble Therapeutics Corp, Evotec AG, F. Hoffmann-La Roche Ltd, Flex Pharma, Inc., FPRT Bio Inc, Gemac, Genentech Inc, Genervon Biopharmaceuticals LLC, GeNeuro SA, GenKyoTex SA, Glialogix Inc, Grifols SA, Herantis Pharma Plc, ImStar Therapeutics Inc., InFlectis BioScience, Ionis Pharmaceuticals Inc, Italfarmaco SpA, Jeil Pharmaceutical Co Ltd, Kadimastem Ltd, Karyopharm Therapeutics Inc, KineMed Inc, Kringle Pharma Inc, Kyorin Pharmaceutical Co Ltd, Kyowa Hakko Kirin Co Ltd, Lascco SA, Lead Discovery Center GmbH, M's Science Corp, Maas Biolab, Mallinckrodt Plc, MedDay SA, MeiraGTx Limited, miCure Therapeutics Ltd., miRagen Therapeutics Inc, MitoDys Therapeutics Limited, Mitsubishi Tanabe Pharma Corp, Neuralstem Inc, Neuraltus Pharmaceuticals Inc, Neurimmune Holding AG, Neurotec Pharma SL, Neurotune AG, Orion Oyj, Orphazyme ApS, Pharnext SA, Plex Pharmaceuticals Inc, Prevacus Inc, Primary Peptides, Inc., ProMIS Neurosciences Inc, Q Therapeutics Inc, ReceptoPharm Inc, Regenesance BV, reMYND NV, Saneron CCEL Therapeutics Inc, SciFluor Life Sciences LLC, SK Biopharmaceuticals Co Ltd, Spherium Biomed SL, Symic Biomedical Inc, Teva Pharmaceutical Industries Ltd, Thera Neuropharma Inc, Treeway BV, Valeant Pharmaceuticals International Inc, ViroMed Co Ltd, VivaCell Biotechnology Espana SL, Voyager Therapeutics Inc, WAVE Life Sciences Ltd and Yooyoung Pharmaceutical Co Ltd. This research provides comprehensive information on the therapeutics under development for Amyotrophic Lateral Sclerosis (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Inquire more about this research at http://www.reportsnreports.com/contacts/inquirybeforebuy.aspx?name=774107 The Amyotrophic Lateral Sclerosis (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Amyotrophic Lateral Sclerosis and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Filing rejected/Withdrawn, Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical, Discovery and Unknown stages are 3, 1, 2, 23, 12, 2, 74, 25 and 1 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 42 and 9 molecules, respectively. Amyotrophic Lateral Sclerosis (Central Nervous System) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Directs proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data. Buy a copy of this research report at http://www.reportsnreports.com/purchase.aspx?name=774107 • The pipeline guide provides a snapshot of the global therapeutic landscape of Amyotrophic Lateral Sclerosis (Central Nervous System). • The pipeline guide reviews pipeline therapeutics for Amyotrophic Lateral Sclerosis (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources. • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages. • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities. • The pipeline guide reviews key companies involved in Amyotrophic Lateral Sclerosis (Central Nervous System) therapeutics and enlists all their major and minor projects. • The pipeline guide evaluates Amyotrophic Lateral Sclerosis (Central Nervous System) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. • The pipeline guide encapsulates all the dormant and discontinued pipeline projects. • The pipeline guide reviews latest news related to pipeline therapeutics for Amyotrophic Lateral Sclerosis (Central Nervous System) • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies. • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage. • Find and recognize significant and varied types of therapeutics under development for Amyotrophic Lateral Sclerosis (Central Nervous System). • Classify potential new clients or partners in the target demographic. • Develop tactical initiatives by understanding the focus areas of leading companies. • Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics. • Formulate corrective measures for pipeline projects by understanding Amyotrophic Lateral Sclerosis (Central Nervous System) pipeline depth and focus of Indication therapeutics. • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope. • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline. For more information, please visit http://www.reportsnreports.com/reports/774107-amyotrophic-lateral-sclerosis-pipeline-review-h2-2016.html


Grant
Agency: Cordis | Branch: H2020 | Program: RIA | Phase: PHC-14-2015 | Award Amount: 6.00M | Year: 2016

The goal of BATCure is to advance the development of new therapeutic options for a group of rare lysosomal diseases - neuronal ceroid lipofuscinoses (NCL) or Batten disease. There are > thousand affected across Europe, with a combined incidence of c.1:100 000. The NCLs are devastating and debilitating genetic disorders that mainly affect children, who suffer progressive dementia and motor decline, visual failure and epilepsy, leading to a long period of complete dependence on others, and eventually a premature death. Existing palliative treatment can reduce, but does not eliminate, the burden of seizures and the progressively worsening effects on the whole body due to decreasing CNS influence and control. There are no curative treatments in the clinic for any type of NCL. We will follow a novel integrated strategy to identify specific gene and small molecule treatments for three genetic types of Batten disease that include the most prevalent world-wide, juvenile CLN3 disease, and in southern and mediterranean Europe, CLN6 and CLN7 diseases. To develop new therapies for these 3 types of Batten disease, BATCure will: 1. Create new models, tools and technologies for developing and testing therapies 2. Further delineate disease biology and gene function to identify new therapeutic target pathways utilising yeast and pluripotent stem cell models 3. Identify biochemical therapeutic target pathways, facilitate effective evaluation of preclinical therapies and improve diagnostics 4. Extend a comprehensive natural history beyond the brain to include cardiology, the spinal cord, PNS, psychiatric and metabolic changes 5. Identify new and repurpose existing small molecule therapy 6. Triage new compound treatments in zebrafish, a high-throughput small vertebrate model 7. Deliver and monitor new treatments using mouse models 8. Provide a novel mechanism to involve patients and their families to inform and fully contribute to therapy development and prepare for clinical trials


The invention relates to the use of a chemical substance selected from the group consisting of N-2-hydroxy-3-(1piperidinyl)-propoxy]-1-pyridine-1-oxide-3-carboximidoyl chloride, the optically active enantiomers and the mixtures of enantiomers thereof and pharmaceutically acceptable salts of the racemic and optically active compounds in the preparation of a pharmaceutical composition for the treatment or prevention of neurodegenerative diseases.


Patent
Orphazyme | Date: 2014-07-14

The present invention concerns a method for modulating the enzymatic activity of an enzyme, wherein said enzyme interacts with BMP, said method comprising the step of administering or inducing Hsp70, or a functional fragment or variant thereof, in a form suitable for allowing interaction between BMP and Hsp70, or said functional fragment or variant thereof, and thereby modulating the enzymatic activity of an enzyme interacting with BMP.


Patent
Orphazyme | Date: 2013-11-06

The present invention concerns a method for modulating the enzymatic activity of an enzyme, wherein said enzyme interacts with BMP, said method comprising the step of administering or inducing Hsp70, or a functional fragment or variant thereof, in a form suitable for allowing interaction between BMP and Hsp70, or said functional fragment or variant thereof, and thereby modulating the enzymatic activity of an enzyme interacting with BMP.


Patent
Orphazyme | Date: 2016-06-15

The present invention concerns a method for modulating the enzymatic activity of an enzyme, wherein said enzyme interacts with BMP, said method comprising the step of administering or inducing Hsp70, or a functional fragment or variant thereof, in a form suitable for allowing interaction between BMP and Hsp70, or said functional fragment or variant thereof, and thereby modulating the enzymatic activity of an enzyme interacting with BMP.


Patent
Orphazyme | Date: 2011-11-22

The present invention relates to a bioactive agent capable of increasing the intracellular concentration and/or activity of Hsp70 for use in the treatment of a lysosomal storage disease which arise from a defect in an enzyme whose activity is not directly associated with the presence of lysosomal BMP as a co-factor; such as glycogen storage diseases, gangliosidoses, neuronal ceroid lipofuscinoses, cerebrotendinous cholesterosis, Wolmans disease, cholesteryl ester storage disease, disorders of glycosaminoglycan metabolism, mucopolysaccharidoses, disorders of glycoprotein metabolism, mucolipidoses, aspartylglucosaminuria, fucosidosis, mannosidoses, and sialidosis type II.


Patent
Orphazyme | Date: 2016-02-19

The present invention concerns a method for modulating the enzymatic activity of an enzyme, wherein said enzyme interacts with BMP, said method comprising the step of administering or inducing Hsp70, or a functional fragment or variant thereof, in a form suitable for allowing interaction between BMP and Hsp70, or said functional fragment or variant thereof, and thereby modulating the enzymatic activity of an enzyme interacting with BMP.


Patent
Orphazyme | Date: 2012-08-08

The present invention concerns a method for modulating the enzymatic activity of an enzyme, wherein said enzyme interacts with BMP, said method comprising the step of administering or inducing Hsp70, or a functional fragment or variant thereof, in a form suitable for allowing interaction between BMP and Hsp70, or said functional fragment or variant thereof, and thereby modulating the enzymatic activity of an enzyme interacting with BMP.


Patent
Orphazyme | Date: 2013-08-19

The present invention concerns a method for modulating the enzymatic activity of an enzyme, wherein said enzyme interacts with BMP, said method comprising the step of administering or inducing Hsp70, or a functional fragment or variant thereof, in a form suitable for allowing interaction between BMP and Hsp70, or said functional fragment or variant thereof, and thereby modulating the enzymatic activity of an enzyme interacting with BMP.

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