Achromatopsia - Pipeline Review, H1 2016 Summary Global Markets Direct's, 'Achromatopsia - Pipeline Review, H1 2016', provides an overview of the Achromatopsia pipeline landscape. The report provides comprehensive information on the therapeutics under development for Achromatopsia, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Achromatopsia and features dormant and discontinued projects. Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct's 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. The report 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. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data. Scope - The report provides a snapshot of the global therapeutic landscape of Achromatopsia - The report reviews pipeline therapeutics for Achromatopsia 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 Achromatopsia therapeutics and enlists all their major and minor projects - The report assesses Achromatopsia therapeutics based on drug target, 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 related to pipeline therapeutics for Achromatopsia 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 important and diverse types of therapeutics under development for Achromatopsia - 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 Achromatopsia 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 - Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline
Table of Contents
Table of Contents 2 List of Tables 4 List of Figures 5 Introduction 6 Global Markets Direct Report Coverage 6 Achromatopsia Overview 7 Therapeutics Development 8 Pipeline Products for Achromatopsia - Overview 8 Achromatopsia - Therapeutics under Development by Companies 9 Achromatopsia - Pipeline Products Glance 10 Clinical Stage Products 10 Early Stage Products 11 Achromatopsia - Products under Development by Companies 12 Achromatopsia - Companies Involved in Therapeutics Development 13 Amgen Inc. 13 Applied Genetic Technologies Corporation 14 Achromatopsia - Therapeutics Assessment 15 Assessment by Monotherapy Products 15 Assessment by Route of Administration 16 Assessment by Molecule Type 17 Drug Profiles 19 Gene Therapy for Achromatopsia - Drug Profile 19 Product Description 19 Mechanism of Action 19 R&D Progress 19 Gene Therapy for X-linked retinoschisis and Achromatopsia - Drug Profile 21 Product Description 21 Mechanism of Action 21 R&D Progress 21 Gene Therapy to Activate CNGA3 for Achromatopsia - Drug Profile 22 Product Description 22 Mechanism of Action 22 R&D Progress 22 Gene Therapy to Activate CNGB3 for Achromatopsia - Drug Profile 23 Product Description 23 Mechanism of Action 23 R&D Progress 23 Gene Therapy to Activate Cone Photoreceptor cGMP-Gated Channel for Achromatopsia - Drug Profile 24 Product Description 24 Mechanism of Action 24 R&D Progress 24 NT-501 - Drug Profile 25 Product Description 25 Mechanism of Action 25 R&D Progress 25 Achromatopsia - Recent Pipeline Updates 28 Achromatopsia - Product Development Milestones 32 Featured News & Press Releases 32 Apr 20, 2016: AGTC Announces Publication of Safety and Biodistribution Data Supporting Future Clinical Studies of Novel Gene Therapy for Treating Achromatopsia 32 Jan 07, 2016: AGTC Announces Preclinical Data Evaluating Cone-Specific Promoters for Use in Gene Therapy of Achromatopsia and Other Retinal Diseases 33 Nov 23, 2015: AGTC Announces U.S. FDA Orphan Drug Designation for Gene Therapy to Treat Achromatopsia 34 Nov 02, 2015: AGTC Files Investigational New Drug Application for the Treatment of Achromatopsia Caused by Mutations in the CNGB3 Gene 34 Oct 22, 2015: AGTC Announces Orphan Drug Designation in the European Union for Gene Therapy to Treat Achromatopsia 35 Aug 27, 2015: AGTC Announces Achievement of XLRS Patient Enrollment Milestone in Biogen Collaboration 35 May 18, 2015: AGTC Announces New Data Supporting Novel Gene-Based Therapies for Rare Inherited Retinal Diseases 36 May 04, 2015: Novel Gene-Based Therapy for Achromatopsia Demonstrates Functional Rescue of Cone Cells 37 Jul 23, 2013: The University of Florida and AGTC Secure $8.4M Grant to Study Rare Eye Disease 38 Appendix 39 Methodology 39 Coverage 39 Secondary Research 39 Primary Research 39 Expert Panel Validation 39 Contact Us 39 Disclaimer 40
List of Tables
Number of Products under Development for Achromatopsia, H1 2016 8 Number of Products under Development by Companies, H1 2016 9 Comparative Analysis by Clinical Stage Development, H1 2016 10 Comparative Analysis by Early Stage Development, H1 2016 11 Products under Development by Companies, H1 2016 12 Achromatopsia - Pipeline by Amgen Inc., H1 2016 13 Achromatopsia - Pipeline by Applied Genetic Technologies Corporation, H1 2016 14 Assessment by Monotherapy Products, H1 2016 15 Number of Products by Stage and Route of Administration, H1 2016 16 Number of Products by Stage and Molecule Type, H1 2016 18 Achromatopsia Therapeutics - Recent Pipeline Updates, H1 2016 28
List of Figures
Number of Products under Development for Achromatopsia, H1 2016 8 Number of Products under Development by Companies, H1 2016 9 Comparative Analysis by Early Stage Products, H1 2016 11 Assessment by Monotherapy Products, H1 2016 15 Number of Products by Molecule Types, H1 2016 17 Number of Products by Stage and Molecule Types, H1 2016 17
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