News Article | December 16, 2016
— Huntington Disease - Companies Involved in Therapeutics Development are Addex Therapeutics Ltd, AFFiRiS AG, Angita BV, Annexon Inc, Astellas Pharma Inc, Azevan Pharmaceuticals Inc, BioCrea GmbH, BrainStorm Cell Therapeutics Inc, Celon Pharma Sp z oo, Chong Kun Dang Pharmaceutical Corp, Diffusion Pharmaceuticals Inc, EncephRx Inc, Evotec AG, Galenea Corp, Genervon Biopharmaceuticals LLC, Horizon Pharma Plc, Immungenetics AG, Ionis Pharmaceuticals Inc, Ipsen SA, Kadmon Corp LLC, KineMed Inc, Krenitsky Pharmaceuticals Inc, Living Cell Technologies Ltd, Medesis Pharma SA, Mitochon Pharmaceuticals Inc, Neuralstem Inc, Neurimmune Holding AG, NeuroNascent Inc, New World Laboratories Inc, nLife Therapeutics SL, NsGene A/S, Omeros Corp, Oryzon Genomics SA, Pfizer Inc, PharmatrophiX Inc, ProQR Therapeutics NV, QR Pharma Inc, reMYND NV, Retrotope Inc, Rhenovia Pharma Ltd, Shire Plc, SOM Biotech SL, Spark Therapeutics Inc, Teva Pharmaceutical Industries Ltd, TyrNovo Ltd, Ultragenyx Pharmaceutical Inc, UniQure NV, Vaccinex Inc, Vertex Pharmaceuticals Inc, VistaGen Therapeutics Inc, Vitality Biopharma Inc, VivaCell Biotechnology Espana SL, Voyager Therapeutics Inc, Vybion Inc, WAVE Life Sciences Ltd and Wellstat Therapeutics Corp. Huntington's disease (HD) is an inherited disease that causes certain nerve cells in the brain to waste away. HD is a familial disease that is passed on from parent to child through a mutation in their genes. Early symptoms of HD include uncontrolled movements, clumsiness, and balance problems. Later, HD can take away the ability to walk, talk, and swallow. The Huntington Disease (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Huntington Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 10, 5, 46 and 17 respectively. Similarly, the Universities portfolio in Phase III, Preclinical and Discovery stages comprises 1, 19 and 4 molecules, respectively. Huntington Disease (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. Inquire more about this report at http://www.reportsnreports.com/contacts/inquirybeforebuy.aspx?name=786898 Note:Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data. • The pipeline guide provides a snapshot of the global therapeutic landscape of Huntington Disease (Central Nervous System). • The pipeline guide reviews pipeline therapeutics for Huntington Disease (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 Huntington Disease (Central Nervous System) therapeutics and enlists all their major and minor projects. • The pipeline guide evaluates Huntington Disease (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 Huntington Disease (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 Huntington Disease (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 its most promising pipeline therapeutics. • Formulate corrective measures for pipeline projects by understanding Huntington Disease (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/786898-huntington-disease-pipeline-review-h2-2016.html
Lehmann S.,Montpellier University |
Lehmann S.,French National Center for Scientific Research |
Relano-Gines A.,French National Center for Scientific Research |
Resina S.,Medesis Pharma SA |
And 15 more authors.
PLoS ONE | Year: 2014
One of the main challenges for neurodegenerative disorders that are principally incurable is the development of new therapeutic strategies, which raises important medical, scientific and societal issues. Creutzfeldt-Jakob diseases are rare neurodegenerative fatal disorders which today remain incurable. The objective of this study was to evaluate the efficacy of the down-regulation of the prion protein (PrP) expression using siRNA delivered by, a water-in-oil microemulsion, as a therapeutic candidate in a preclinical study. After 12 days rectal mucosa administration of Aonys/PrP-siRNA in mice, we observed a decrease of about 28% of the brain PrPC level. The effect of Aonys/PrP-siRNA was then evaluated on prion infected mice. Several mice presented a delay in the incubation and survival time compared to the control groups and a significant impact was observed on astrocyte reaction and neuronal survival in the PrP-siRNA treated groups. These results suggest that a new therapeutic scheme based an innovative delivery system of PrP-siRNA can be envisioned in prion disorders. © 2014 Lehmann et al.
PubMed | French National Center for Scientific Research, Montpellier University and Medesis Pharma SA
Type: Journal Article | Journal: PloS one | Year: 2014
One of the main challenges for neurodegenerative disorders that are principally incurable is the development of new therapeutic strategies, which raises important medical, scientific and societal issues. Creutzfeldt-Jakob diseases are rare neurodegenerative fatal disorders which today remain incurable. The objective of this study was to evaluate the efficacy of the down-regulation of the prion protein (PrP) expression using siRNA delivered by, a water-in-oil microemulsion, as a therapeutic candidate in a preclinical study. After 12 days rectal mucosa administration of Aonys/PrP-siRNA in mice, we observed a decrease of about 28% of the brain PrP(C) level. The effect of Aonys/PrP-siRNA was then evaluated on prion infected mice. Several mice presented a delay in the incubation and survival time compared to the control groups and a significant impact was observed on astrocyte reaction and neuronal survival in the PrP-siRNA treated groups. These results suggest that a new therapeutic scheme based an innovative delivery system of PrP-siRNA can be envisioned in prion disorders.
Mouri A.,Charles Gerhardt Institute |
Mouri A.,Medesis Pharma S.A |
Diat O.,CNRS Marcoule Institute for Separative Chemistry |
El Ghzaoui A.,Montpellier University |
And 5 more authors.
Journal of Colloid and Interface Science | Year: 2015
The phase behavior of the four-components Peceol®/lecithin/ethanol/water system has been studied in a part of the phase diagram poor in water and varying the lecithin/Peceol® ratio. Using several complementary techniques such as Karl Fischer titration, rheology, polarized microscopy and SAXS measurements several nanostructures of the complex systems were identified. W/O microemulsion (L2) as well as an inverted hexagonal (H2) liquid-crystal phase were studied. The analysis of the different phase transitions allows us to understand the effect of lecithin on the water solubilization efficiency of this clear gel and to show its pharmaceutical interest among lecithin organogels. © 2015 Elsevier Inc.
PubMed | Charles Gerhardt Institute, Medesis Pharma S.A and Montpellier University
Type: Journal Article | Journal: International journal of pharmaceutics | Year: 2016
Lithium biocompatible microemulsion based on Peceol(), lecithin, ethanol and water was studied in attempt to identify the optimal compositions in term of drug content, physicochemical properties and stability. Lithium solubilization in microemulsion was found to be compatible with a drug-surfactant binding model. Lithium ions were predominantly solubilized within lecithin head group altering significantly the interfacial properties of the system. Pseudo-ternary phase diagrams of drug free and drug loaded microemulsions were built at constant ethanol/lecithin weight ratio (40/60). Lithium loaded microemulsion has totally disappeared in the Peceol() rich part of phase diagram; critical fractions of lecithin and ethanol were required for the formation of stable microemulsion. The effect of lithium concentration on the properties and physical stability of microemulsions were studied using microscopy, Karl Fischer titrations, rheology analyses, conductivity measurements and centrifugation tests. The investigated microemulsions were found to be stable under accelerated storage conditions. The systems exhibited low viscosity and behaved as Newtonian fluid and no structural transition was shown.