Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline Review, H2 2018

Global Markets Direct’s, ‘Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) - Pipeline Review, H2 2018’, provides an overview of the Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy), 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 Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 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 Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)

- The report reviews pipeline therapeutics for Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 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 Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) therapeutics and enlists all their major and minor projects

- The report assesses Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy) 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 Leber’s Hereditary Optic Neuropathy (LHON) (Leber optic atrophy)

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

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

Companies mentioned

Alkeus Pharmaceuticals Inc

Bactevo Ltd

Biovista Inc

Fortify Therapeutics Inc

GenSight Biologics SA

Ixchel Pharma LLC

Khondrion BV

Mitobridge Inc

Mitotech SA

Santhera Pharmaceuticals Holding ...

Alkeus Pharmaceuticals Inc

Bactevo Ltd

Biovista Inc

Fortify Therapeutics Inc

GenSight Biologics SA

Ixchel Pharma LLC

Khondrion BV

Mitobridge Inc

Mitotech SA

Santhera Pharmaceuticals Holding AG

Spark Therapeutics Inc

Stealth BioTherapeutics Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Overview

Leber ...

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Overview

Leber’s Hereditary Optic Neuropathy (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 (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 (Leber Optic Atrophy) – Companies Involved in Therapeutics Development

Alkeus Pharmaceuticals Inc

Bactevo Ltd

Biovista Inc

Fortify Therapeutics Inc

GenSight Biologics SA

Ixchel Pharma LLC

Khondrion BV

Mitobridge Inc

Mitotech SA

Santhera Pharmaceuticals Holding AG

Spark Therapeutics Inc

Stealth BioTherapeutics Inc

Leber’s Hereditary Optic Neuropathy (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-011 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

idebenone – 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

lenadogene nolparvovec – 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

Small Molecules for Leber’s Hereditary Optic Neuropathy – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules for Mitochondrial and Central Nervous System Disorders – 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 (Leber Optic Atrophy) – Dormant Projects

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Product Development Milestones

Featured News & Press Releases

Jun 20, 2018: GenSight Biologics : Key Opinion Leaders Highlight GS010 Efficacy and Patient Benefits in Discussion of Findings from REVERSE Phase III Clinical Trial

Jun 12, 2018: Santhera Submits NDA in South Korea for Raxone for the Treatment of LHON

Jun 12, 2018: GenSight Biologics Reports Positive Additional Data from REVERSE Phase III Clinical Trial of GS010 for Treatment of Leber Hereditary Optic Neuropathy (LHON)

Apr 30, 2018: Stealth BioTherapeutics Granted Orphan Drug Designation of Elamipretide for Treatment of Leber's Hereditary Optic Neuropathy

Apr 26, 2018: GenSight Biologics to Present Data on GS010 at the Annual Meeting of Association for Research in Vision and Ophthalmology

Apr 03, 2018: GenSight Biologics announces topline results from REVERSE Phase III clinical trial of GS010 in patients with Leber Hereditary Optic Neuropathy (LHON)

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 23, 2017: First International Consensus Statement on the Clinical and Therapeutic Management of LHON published in the Journal of Neuro-Ophthalmology

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

Aug 31, 2017: Santhera Announces Approval of Raxone for LHON in Israel

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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy), H2 2018

Number of Products under Development by ...

List of Tables

Number of Products under Development for Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy), H2 2018

Number of Products under Development by Companies, H2 2018

Number of Products under Development by Universities/Institutes, H2 2018

Products under Development by Companies, H2 2018

Products under Development by Universities/Institutes, H2 2018

Number of Products by Stage and Target, H2 2018

Number of Products by Stage and Mechanism of Action, H2 2018

Number of Products by Stage and Route of Administration, H2 2018

Number of Products by Stage and Molecule Type, H2 2018

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Pipeline by Alkeus Pharmaceuticals Inc, H2 2018

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Pipeline by Bactevo Ltd, H2 2018

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Pipeline by Biovista Inc, H2 2018

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Pipeline by Fortify Therapeutics Inc, H2 2018

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Pipeline by GenSight Biologics SA, H2 2018

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Pipeline by Ixchel Pharma LLC, H2 2018

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Pipeline by Khondrion BV, H2 2018

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Pipeline by Mitobridge Inc, H2 2018

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Pipeline by Mitotech SA, H2 2018

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Pipeline by Santhera Pharmaceuticals Holding AG, H2 2018

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Pipeline by Spark Therapeutics Inc, H2 2018

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Pipeline by Stealth BioTherapeutics Inc, H2 2018

Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy) – Dormant Projects, H2 2018

List of Figures

List of Figures

Number of Products under Development for Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy), H2 2018

Number of Products under Development by ...

List of Figures

Number of Products under Development for Leber’s Hereditary Optic Neuropathy (Leber Optic Atrophy), H2 2018

Number of Products under Development by Companies, H2 2018

Number of Products by Targets, H2 2018

Number of Products by Stage and Targets, H2 2018

Number of Products by Mechanism of Actions, H2 2018

Number of Products by Stage and Mechanism of Actions, H2 2018

Number of Products by Routes of Administration, H2 2018

Number of Products by Stage and Routes of Administration, H2 2018

Number of Products by Molecule Types, H2 2018

Number of Products by Stage and Molecule Types, H2 2018

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