Report Detail

Pharmaceuticals & Healthcare Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline Review, H1 2018

  • RnM1785781
  • |
  • 23 March, 2018
  • |
  • Global
  • |
  • 67 Pages
  • |
  • Global Markets Direct
  • |
  • Pharmaceuticals & Healthcare

Table of Contents

    List of Tables

      List of Figures

        Introduction

          Global Markets Direct Report Coverage

            Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Overview

              Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Therapeutics Development

                Pipeline Overview

                  Pipeline by Companies

                    Pipeline by Universities/Institutes

                      Products under Development by Companies

                        Products under Development by Universities/Institutes

                          Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Therapeutics Assessment

                            Assessment by Target

                              Assessment by Mechanism of Action

                                Assessment by Route of Administration

                                  Assessment by Molecule Type

                                    Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Companies Involved in Therapeutics Development

                                      Alkeus Pharmaceuticals Inc

                                        Biovista Inc

                                          GenSight Biologics SA

                                            Ixchel Pharma LLC

                                              Khondrion BV

                                                Mitobridge Inc

                                                  Mitotech SA

                                                    Spark Therapeutics Inc

                                                      Stealth BioTherapeutics Inc

                                                        Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Drug Profiles

                                                          BVA-202 - Drug Profile

                                                            Product Description

                                                              Mechanism Of Action

                                                                R&D Progress

                                                                  BVA-203 - Drug Profile

                                                                    Product Description

                                                                      Mechanism Of Action

                                                                        R&D Progress

                                                                          Drug for Lebers Hereditary Optic Neuropathy - Drug Profile

                                                                            Product Description

                                                                              Mechanism Of Action

                                                                                R&D Progress

                                                                                  elamipretide - Drug Profile

                                                                                    Product Description

                                                                                      Mechanism Of Action

                                                                                        R&D Progress

                                                                                          Gene Therapy to Activate ND4 for Leber's Hereditary Optic Neuropathy - Drug Profile

                                                                                            Product Description

                                                                                              Mechanism Of Action

                                                                                                R&D Progress

                                                                                                  GS-010 - Drug Profile

                                                                                                    Product Description

                                                                                                      Mechanism Of Action

                                                                                                        R&D Progress

                                                                                                          GS-011 - Drug Profile

                                                                                                            Product Description

                                                                                                              Mechanism Of Action

                                                                                                                R&D Progress

                                                                                                                  IXC-201 - Drug Profile

                                                                                                                    Product Description

                                                                                                                      Mechanism Of Action

                                                                                                                        R&D Progress

                                                                                                                          IXC-203 - Drug Profile

                                                                                                                            Product Description

                                                                                                                              Mechanism Of Action

                                                                                                                                R&D Progress

                                                                                                                                  KH-176 - Drug Profile

                                                                                                                                    Product Description

                                                                                                                                      Mechanism Of Action

                                                                                                                                        R&D Progress

                                                                                                                                          MA-0211 - Drug Profile

                                                                                                                                            Product Description

                                                                                                                                              Mechanism Of Action

                                                                                                                                                R&D Progress

                                                                                                                                                  plastoquinone decyl triphenylphosphonium bromide - Drug Profile

                                                                                                                                                    Product Description

                                                                                                                                                      Mechanism Of Action

                                                                                                                                                        R&D Progress

                                                                                                                                                          SPK-LHON - Drug Profile

                                                                                                                                                            Product Description

                                                                                                                                                              Mechanism Of Action

                                                                                                                                                                R&D Progress

                                                                                                                                                                  Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Dormant Projects

                                                                                                                                                                    Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Product Development Milestones

                                                                                                                                                                      Featured News & Press Releases

                                                                                                                                                                        Feb 20, 2018: GenSight Biologics Announces Publication of Positive Data from Phase I/II Trial and Long-Term Follow-up of GS010 in Ophthalmology, the Journal of the American Academy of Ophthalmology

                                                                                                                                                                          Feb 02, 2018: Bascom Palmer Study Shows Gene Therapy Safe for Rare Eye Disease

                                                                                                                                                                            Jan 25, 2018: A potential drug for Leber’s hereditary optic neuropathy and Barth syndrome discovered at the IRCM

                                                                                                                                                                              Dec 18, 2017: Stealth BioTherapeutics Granted Fast Track Designation for Elamipretide for the Treatment of Leber's Hereditary Optic Neuropathy

                                                                                                                                                                                Dec 05, 2017: GenSight Biologics reports positive long-term visual acuity gains and safety results from Phase I/II Study of GS010 for the treatment of Leber Hereditary Optic Neuropathy (LHON)

                                                                                                                                                                                  Oct 16, 2017: GenSight Biologics to Present Data on GS010 at 2017 Congress of the European Society of Gene & Cell Therapy

                                                                                                                                                                                    Aug 01, 2017: GenSight Biologics Completes Enrollment of RESCUE Phase III Study of GS010 in the Treatment of Leber's Hereditary Optic Neuropathy

                                                                                                                                                                                      Jun 14, 2017: GenSight Biologics Reports Long-Term Positive Safety and Visual Acuity Results at Week 96 in Phase I/II Study of GS010 for the Treatment of Leber’s Hereditary Optic Neuropathy

                                                                                                                                                                                        May 08, 2017: GenSight Biologics to Present Data on GS010 at the Annual Meeting of ARVO

                                                                                                                                                                                          Feb 21, 2017: GenSight Biologics Completes Enrollment of REVERSE Phase III Study of GS010 in the treatment of Leber’s Hereditary Optic Neuropathy

                                                                                                                                                                                            Dec 20, 2016: GenSight Biologics Reports Sustained Visual Acuity Gain at 78 Weeks in Its Phase I/II Study with GS010 for the Treatment of Leber’s Hereditary Optic Neuropathy (LHON)

                                                                                                                                                                                              Sep 08, 2016: GenSight Biologics Receives Approval to Include Teenage Patients in RESCUE and REVERSE Phase III Trials with GS010 in Leber’s Hereditary Optic Neuropathy

                                                                                                                                                                                                Jun 08, 2016: GenSight Biologics Announces Additional Promising Results of Its Phase I/II Study with GS010 for the Treatment of Leber’s Hereditary Optic Neuropathy (LHON)

                                                                                                                                                                                                  Jun 01, 2016: Stealth BioTherapeutics Initiates Phase 2 Study of Elamipretide in Leber's Hereditary Optic Neuropathy

                                                                                                                                                                                                    Feb 29, 2016: GenSight Biologics Enrolls First Patient in Pivotal Phase III Program of GS010 in Leber’s Hereditary Optic Neuropathy

                                                                                                                                                                                                      Appendix

                                                                                                                                                                                                        Methodology

                                                                                                                                                                                                          Coverage

                                                                                                                                                                                                            Secondary Research

                                                                                                                                                                                                              Primary Research

                                                                                                                                                                                                                Expert Panel Validation

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                                                                                                                                                                                                                    Disclaimer

                                                                                                                                                                                                                    Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline Review, H1 2018

                                                                                                                                                                                                                    Summary

                                                                                                                                                                                                                    Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline Review, H1 2018, provides an overview of the Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) (Ophthalmology) pipeline landscape.

                                                                                                                                                                                                                    Leber’s hereditary optic neuropathy (LHON) is an inherited form of vision loss. This condition usually begins in a person's teens or twenties, rare cases may appear in early childhood or later in adulthood. Males are affected much more often than females. Symptoms include eye pain or discomfort, numbness, tingling and clouding of vision. Predisposing factors include age, tobacco smoke, alcohol, cancer treating drugs, stress related chemicals, some antibiotics and cyanogens.

                                                                                                                                                                                                                    Report Highlights

                                                                                                                                                                                                                    Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline Review, H1 2018, provides comprehensive information on the therapeutics under development for Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) (Ophthalmology), 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.

                                                                                                                                                                                                                    The Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) (Ophthalmology) pipeline guide also reviews of key players involved in therapeutic development for Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 1, 3, 2, 4, 1 and 1 respectively. Similarly, the Universities portfolio in Phase I stages comprises 1 molecules, respectively.

                                                                                                                                                                                                                    Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) (Ophthalmology) 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 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. 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.

                                                                                                                                                                                                                    Scope

                                                                                                                                                                                                                    - The pipeline guide provides a snapshot of the global therapeutic landscape of Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) (Ophthalmology).
                                                                                                                                                                                                                    - The pipeline guide reviews pipeline therapeutics for Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) (Ophthalmology) 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 Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) (Ophthalmology) therapeutics and enlists all their major and minor projects.
                                                                                                                                                                                                                    - The pipeline guide evaluates Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) (Ophthalmology) 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 Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) (Ophthalmology)

                                                                                                                                                                                                                    Reasons to buy

                                                                                                                                                                                                                    - 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 Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) (Ophthalmology).
                                                                                                                                                                                                                    - 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 Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) (Ophthalmology) 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.



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