Leber Congenital Amaurosis (LCA) – Pipeline Review, H1 2018

Global Markets Direct’s, ‘Leber Congenital Amaurosis (LCA) – Pipeline Review, H1 2018’, provides an overview of the Leber Congenital Amaurosis (LCA) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Leber Congenital Amaurosis (LCA), 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 Congenital Amaurosis (LCA) 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 Congenital Amaurosis (LCA)

– The report reviews pipeline therapeutics for Leber Congenital Amaurosis (LCA) 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 Congenital Amaurosis (LCA) therapeutics and enlists all their major and minor projects

– The report assesses Leber Congenital Amaurosis (LCA) 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 Congenital Amaurosis (LCA)

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 Congenital Amaurosis (LCA)

- 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 Congenital Amaurosis (LCA) 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

Editas Medicine Inc

Novelion Therapeutics Inc

ProQR Therapeutics NV

Spark Therapeutics Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Leber Congenital Amaurosis (LCA) Overview

Leber Congenital Amaurosis (LCA) Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Leber Congenital Amaurosis (LCA) Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Leber Congenital Amaurosis (LCA) Companies Involved in Therapeutics Development

Editas Medicine Inc

Novelion Therapeutics Inc

ProQR Therapeutics NV

Spark Therapeutics Inc

Leber Congenital Amaurosis (LCA) Drug Profiles

A-001 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

EDIT-101 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate AIPL1 for Leber’s Congenital Amaurosis Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate AIPL1 for Ophthalmology Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Retinal Guanylyl Cyclase-1 for Leber Congenital Amaurosis-1 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate RPE65 for Leber Congenital Amaurosis Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate RPE65 for Leber Congenital Amaurosis Drug Profile

Product Description

Mechanism Of Action

R&D Progress

QR-110 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

voretigene neparvovec Drug Profile

Product Description

Mechanism Of Action

R&D Progress

zuretinol acetate Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Leber Congenital Amaurosis (LCA) Dormant Projects

Leber Congenital Amaurosis (LCA) Product Development Milestones

Featured News & Press Releases

Jan 03, 2018: Spark's Gene Therapy Treatment for Blindness LUXTURNA to Cost $850,000

Oct 19, 2017: Editas Medicine Demonstrates Dose-Dependent, In Vivo Editing with EDIT-101 in CEP290 Transgenic Mice

Sep 26, 2017: Editas Medicine Receives EMA's Orphan Medicinal Product Designation for EDIT-101 for the Treatment of LCA10

Sep 12, 2017: Editas Medicine Initiates Clinical Natural History Study to Evaluate Patients with Leber Congenital Amaurosis Type 10

Jul 31, 2017: Spark Therapeutics Submits Marketing Authorization Application to European Medicines Agency for Investigational LUXTURNA (voretigene neparvovec)

Jan 09, 2017: Editas Medicine Reports on Recent Progress and 2017 Goals at J.P. Morgan Healthcare Conference

Apr 27, 2016: Editas Medicine Announces Presentations at Upcoming Scientific Conferences

May 04, 2015: Penn Study Indicates that Gene Therapy Efficacy for LCA is Dynamic: Improvement is Followed by Decline in Vision

May 01, 2015: Spark Therapeutics to Have Multiple Presentations at 2015 ARVO Meeting

Dec 05, 2014: QLT Announces Results From Proof-of-Concept Trial With Oral Synthetic cis-Retinoid (QLT091001) in Adult Subjects With Impaired Dark Adaptation and/or Impaired Low Luminance Vision

Nov 06, 2014: Spark Therapeutics Receives FDA Breakthrough Therapy Designation for Its Lead Product Candidate, SPK-RPE65

Sep 12, 2014: QLT Announces Positive Final Results From Phase 1b Retreatment Trial With Oral Synthetic cis-Retinoid (QLT091001) in Subjects With LCA or RP Due to Mutations in RPE65 or LRAT

Jul 14, 2014: QLT Announces Publication in The Lancet of Phase 1b Data for QLT091001 in Leber Congenital Amaurosis Due to LRAT or RPE65 Mutations

May 08, 2014: Spark Therapeutics Scientific Advisor to Deliver Keynote at the Association for Research in Vision and Ophthalmology’s 2014 Annual Meeting

Feb 27, 2014: QLT Announces Positive Preliminary Results From Phase 1b Retreatment Trial of QLT091001 in Subjects With Leber Congenital Amaurosis and Retinitis Pigmentosa Due to Mutations in LRAT or RPE65

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 Congenital Amaurosis (LCA), H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Products under Development by Companies, H1 2018

Products under Development by Universities/Institutes, H1 2018

Number of Products by Stage and Target, H1 2018

Number of Products by Stage and Mechanism of Action, H1 2018

Number of Products by Stage and Route of Administration, H1 2018

Number of Products by Stage and Molecule Type, H1 2018

Leber Congenital Amaurosis (LCA) Pipeline by Editas Medicine Inc, H1 2018

Leber Congenital Amaurosis (LCA) Pipeline by Novelion Therapeutics Inc, H1 2018

Leber Congenital Amaurosis (LCA) Pipeline by ProQR Therapeutics NV, H1 2018

Leber Congenital Amaurosis (LCA) Pipeline by Spark Therapeutics Inc, H1 2018

Leber Congenital Amaurosis (LCA) Dormant Projects, H1 2018

List of Figures

List of Figures

Number of Products under Development for Leber Congenital Amaurosis (LCA), H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Number of Products by Targets, H1 2018

Number of Products by Stage and Targets, H1 2018

Number of Products by Mechanism of Actions, H1 2018

Number of Products by Stage and Mechanism of Actions, H1 2018

Number of Products by Routes of Administration, H1 2018

Number of Products by Stage and Routes of Administration, H1 2018

Number of Products by Molecule Types, H1 2018

Number of Products by Stage and Molecule Types, H1 2018

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