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Published: Jun, 2017 | Pages:
70 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Dystrophin (DMD) - Pipeline Review, H1 2017 Summary According to the recently published report 'Dystrophin (DMD) - Pipeline Review, H1 2017'; Dystrophin (DMD) pipeline Target constitutes close to 26 molecules. Out of which approximately 23 molecules are developed by companies and remaining by the universities/institutes. Dystrophin (DMD) - Dystrophin is a cytoplasmic protein. It anchors the extracellular matrix to the cytoskeleton via F-actin. It acts as ligand for dystroglycan. It act as component of the dystrophin-associated glycoprotein complex which accumulates at the neuromuscular junction and at a variety of synapses in the peripheral and central nervous systems and has a structural function in stabilizing the sarcolemma. The report 'Dystrophin (DMD) - Pipeline Review, H1 2017' outlays comprehensive information on the Dystrophin (DMD) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type; that are being developed by Companies / Universities. It also reviews key players involved in Dystrophin (DMD) targeted therapeutics development with respective active and dormant or discontinued projects. Currently, The molecules developed by companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 2, 2, 1, 8 and 10 respectively. Similarly, the universities portfolio in Phase I and Preclinical stages comprises 1 and 2 molecules, respectively. Report covers products from therapy areas Genetic Disorders and Musculoskeletal Disorders which include indications Duchenne Muscular Dystrophy and Muscular Dystrophy. Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data. Scope - The report provides a snapshot of the global therapeutic landscape for Dystrophin (DMD) - The report reviews Dystrophin (DMD) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources - The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages - The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities - The report reviews key players involved in Dystrophin (DMD) targeted therapeutics and enlists all their major and minor projects - The report assesses Dystrophin (DMD) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type - The report summarizes all the dormant and discontinued pipeline projects - The report reviews latest news and deals related to Dystrophin (DMD) targeted therapeutics Reasons To Buy - Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage - Identify and understand the targeted therapy areas and indications for Dystrophin (DMD) - Identify the use of drugs for target identification and drug repurposing - Identify potential new clients or partners in the target demographic - Develop strategic initiatives by understanding the focus areas of leading companies - Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics - Devise corrective measures for pipeline projects by understanding Dystrophin (DMD) development landscape - 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
Table of Contents
List of Tables List of Figures Introduction Global Markets Direct Report Coverage Dystrophin (DMD) - Overview Dystrophin (DMD) - Therapeutics Development Products under Development by Stage of Development Products under Development by Therapy Area Products under Development by Indication Products under Development by Companies Products under Development by Universities/Institutes Dystrophin (DMD) - Therapeutics Assessment Assessment by Mechanism of Action Assessment by Route of Administration Assessment by Molecule Type Dystrophin (DMD) - Companies Involved in Therapeutics Development Asklepios BioPharmaceutical Inc BioMarin Pharmaceutical Inc Daiichi Sankyo Company Ltd Editas Medicine Inc Genethon SA Nippon Shinyaku Co Ltd Pfizer Inc Sarepta Therapeutics Inc WAVE Life Sciences Ltd Dystrophin (DMD) - Drug Profiles Antisense Oligonucleotide 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile Product Description Mechanism Of Action R&D Progress Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile Product Description Mechanism Of Action R&D Progress Antisense Oligonucleotide to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile Product Description Mechanism Of Action R&D Progress Biostrophin - Drug Profile Product Description Mechanism Of Action R&D Progress BMBD-001 - Drug Profile Product Description Mechanism Of Action R&D Progress DS-5141 - Drug Profile Product Description Mechanism Of Action R&D Progress Gene Therapy to Activate DMD for Duchenne Muscular Dystrophy - Drug Profile Product Description Mechanism Of Action R&D Progress Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile Product Description Mechanism Of Action R&D Progress Gene Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile Product Description Mechanism Of Action R&D Progress Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile Product Description Mechanism Of Action R&D Progress NS-065 - Drug Profile Product Description Mechanism Of Action R&D Progress Oligonucleotides 1 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile Product Description Mechanism Of Action R&D Progress Oligonucleotides 2 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile Product Description Mechanism Of Action R&D Progress Oligonucleotides 3 to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile Product Description Mechanism Of Action R&D Progress PRO-055 - Drug Profile Product Description Mechanism Of Action R&D Progress SGT-001 - Drug Profile Product Description Mechanism Of Action R&D Progress SRP-4008 - Drug Profile Product Description Mechanism Of Action R&D Progress SRP-4044 - Drug Profile Product Description Mechanism Of Action R&D Progress SRP-4045 - Drug Profile Product Description Mechanism Of Action R&D Progress SRP-4050 - Drug Profile Product Description Mechanism Of Action R&D Progress SRP-4052 - Drug Profile Product Description Mechanism Of Action R&D Progress SRP-4053 - Drug Profile Product Description Mechanism Of Action R&D Progress SRP-4055 - Drug Profile Product Description Mechanism Of Action R&D Progress SRP-5051 - Drug Profile Product Description Mechanism Of Action R&D Progress Stem Cell Therapy to Activate Dystrophin for Duchenne Muscular Dystrophy - Drug Profile Product Description Mechanism Of Action R&D Progress WVE-210201 - Drug Profile Product Description Mechanism Of Action R&D Progress Dystrophin (DMD) - Dormant Products Dystrophin (DMD) - Discontinued Products Dystrophin (DMD) - Product Development Milestones Featured News & Press Releases May 11, 2017: New Preclinical Data Support SGT-001 As A Novel Treatment Approach For Duchenne Muscular Dystrophy Apr 24, 2017: Daiichi Sankyo Announces Designation of Duchenne Muscular Dystrophy Treatment DS-5141 under "SAKIGAKE Designation System" Jan 06, 2017: WAVE Life Sciences Provides Update on Duchenne Muscular Dystrophy Program WVE-210201 Oct 24, 2016: Solid Biosciences Granted U.S. and E.U. Orphan Drug Designations for Lead Gene Therapy Candidate for Duchenne Muscular Dystrophy Sep 28, 2016: Sarepta Therapeutics Announces First Patient Dosed in Phase III Clinical Trial of SRP-4045 for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping Sep 28, 2016: Sarepta Therapeutics Announces First Patient Dosed in Phase III Clinical Trial of SRP-4053 for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping Sep 20, 2016: Sarepta Therapeutics Announces Favorable USPTO Decision in Exon 53 Composition of Matter Patent Interference Cases against BioMarin Pharmaceutical Feb 25, 2016: Daiichi Sankyo Announces Commencement of Clinical Trials in Japan for Duchenne Muscular Dystrophy Treatment Oct 22, 2015: Gene Therapy Treats All Muscles in the Body in Muscular Dystrophy Dogs Jan 14, 2015: Sarepta Therapeutics Announces First Patient Dosed in European Phase I/II Study of SRP-4053 in Duchenne Muscular Dystrophy Patients May 09, 2013: Nippon Shinyaku And NCPN To Initiate Clinical Study With Antisense Oligonucleotide For Treatment Of Duchenne Muscular Dystrophy Jan 15, 2013: A Quantum Leap in Gene Therapy of Duchenne Muscular Dystrophy Dec 12, 2012: Action Duchenne Announces €5.5m EU Funding For Research Into Exon 53 Mar 26, 2012: Action Duchenne Provides Update On Prosensa's PRO055 Drug Candidate Mar 25, 2010: AVI BioPharma To Present Update On AVI-5038 For Treatment Of Duchenne Muscular Dystrophy Appendix Methodology Coverage Secondary Research Primary Research Expert Panel Validation Contact Us Disclaimer
List of Tables
Number of Products under Development by Stage of Development, H1 2017 Number of Products under Development by Therapy Areas, H1 2017 Number of Products under Development by Indication, H1 2017 Number of Products under Development by Companies, H1 2017 Products under Development by Companies, H1 2017 Products under Development by Companies, H1 2017 (Contd..1), H1 2017 Number of Products under Investigation by Universities/Institutes, H1 2017 Products under Investigation by Universities/Institutes, H1 2017 Number of Products by Stage and Mechanism of Actions, H1 2017 Number of Products by Stage and Route of Administration, H1 2017 Number of Products by Stage and Molecule Type, H1 2017 Pipeline by Asklepios BioPharmaceutical Inc, H1 2017 Pipeline by BioMarin Pharmaceutical Inc, H1 2017 Pipeline by Daiichi Sankyo Company Ltd, H1 2017 Pipeline by Editas Medicine Inc, H1 2017 Pipeline by Genethon SA, H1 2017 Pipeline by Nippon Shinyaku Co Ltd, H1 2017 Pipeline by Pfizer Inc, H1 2017 Pipeline by Sarepta Therapeutics Inc, H1 2017 Pipeline by WAVE Life Sciences Ltd, H1 2017 Dormant Projects, H1 2017 Discontinued Products, H1 2017
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