Frataxin Mitochondrial – Pipeline Review, H2 2019

“Global Markets Direct’s, ‘Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1)- Pipeline Review, H2 2019’, provides in depth analysis on Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted pipeline therapeutics. The report provides comprehensive information complete with analysis by indications, stage of development, 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 Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics development and features dormant and discontinued projects. The report analyses the pipeline products from therapy areas Central Nervous System and Cardiovascular under development targeting Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1).

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.

*Updated report will be delivered in 48 hours of order confirmation.”

Scope


– The report provides a snapshot of the global therapeutic landscape for Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1)

– The report reviews Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) 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 Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) targeted therapeutics and enlists all their major and minor projects

– The report assesses Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) 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 Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) 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 Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1)

- 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 Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) 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

Companies mentioned

“Adverum Biotechnologies Inc

Biointaxis SL

Chondrial Therapeutics LLC

Design Therapeutics Inc

Fratagene Therapeutics Srl

Fulcrum Therapeutics Inc

Jupiter Orphan Therapeutics Inc

Panorama Research Inc

Pfizer Inc

PTC Therapeutics Inc

Voyager Therapeutics Inc”

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Frataxin Mitochondrial (Friedreich’s Ataxia Protein or FXN or EC

1.16.3.1) Overview

Frataxin Mitochondrial (Friedreich’s Ataxia Protein or FXN or EC

1.16.3.1) 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

Frataxin Mitochondrial (Friedreich’s Ataxia Protein or FXN or EC

1.16.3.1) Therapeutics Assessment

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Frataxin Mitochondrial (Friedreich’s Ataxia Protein or FXN or EC

1.16.3.1) Companies Involved in Therapeutics Development

Adverum Biotechnologies Inc

Pfizer Inc

Voyager Therapeutics Inc

Frataxin Mitochondrial (Friedreich’s Ataxia Protein or FXN or EC

1.16.3.1) Drug Profiles

ADVM-063 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AGIL-FA Drug Profile

Product Description

Mechanism Of Action

R&D Progress

CTI-1601 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Frataxin for Friedreich’s Ataxia Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Frataxin for Friedreich’s Ataxia Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Frataxin for Friedrich's Ataxia Drug Profile

Product Description

Mechanism Of Action

R&D Progress

JOT-101 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Activate Frataxin for Friedreich’s Ataxia Drug Profile

Product Description

Mechanism Of Action

R&D Progress

VYFXN-01 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Frataxin Mitochondrial (Friedreich’s Ataxia Protein or FXN or EC

1.16.3.1) Dormant Products

Frataxin Mitochondrial (Friedreich’s Ataxia Protein or FXN or EC

1.16.3.1) Product Development Milestones

Featured News & Press Releases

May 18, 2018: Voyager Therapeutics Announces Additional Data at the American Society of Gene and Cell Therapy 2018 Annual Meeting

Oct 31, 2017: Agilis Biotherapeutics Announces Orphan Product Designation Approval in Europe for the Treatment of Friedreich’s Ataxia

Aug 22, 2017: Jupiter Orphan Therapeutics Receives Orphan Drug Designation for its Trans-Resveratrol Product JOTROL for Treatment of Friedreich’s Ataxia

Aug 03, 2017: Chondrial Announces FDA Orphan Drug Designation for CTI-1601, a Novel Investigational Technology for the Treatment of Friedreich’s Ataxia

Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich’s Ataxia

Aug 05, 2015: Key Patent Granted for AAVLife’s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich’s Ataxia

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

“List of Tables

Number of Products under Development by Stage of Development, H2 2019

Number of Products under Development by Therapy Areas, H2 2019

Number of Products under Development by Indication, H2 2019

Number of Products under Development by Companies, H2 2019

Products under Development by Companies, H2 2019

Number of Products under Investigation by Universities/Institutes, H2 2019

Products under Investigation by Universities/Institutes, H2 2019

Number of Products by Stage and Mechanism of Actions, H2 2019

Number of Products by Stage and Route of Administration, H2 2019

Number of Products by Stage and Molecule Type, H2 2019

Pipeline by Adverum Biotechnologies Inc, H2 2019

Pipeline by Biointaxis SL, H2 2019

Pipeline by Chondrial Therapeutics LLC, H2 2019

Pipeline by Design Therapeutics Inc, H2 2019

Pipeline by Fratagene Therapeutics Srl, H2 2019

Pipeline by Fulcrum Therapeutics Inc, H2 2019

Pipeline by Jupiter Orphan Therapeutics Inc, H2 2019

Pipeline by Panorama Research Inc, H2 2019

Pipeline by Pfizer Inc, H2 2019

Pipeline by PTC Therapeutics Inc, H2 2019

Pipeline by Voyager Therapeutics Inc, H2 2019

Dormant Projects, H2 2019”

List of Figures

“List of Figures

Number of Products under Development by Stage of Development, H2 2019

Number of Products under Development by Therapy Areas, H2 2019

Number of Products under Development by Top 10 Indications, H2 2019

Number of Products by Mechanism of Actions, H2 2019

Number of Products by Stage and Mechanism of Actions, H2 2019

Number of Products by Routes of Administration, H2 2019

Number of Products by Stage and Routes of Administration, H2 2019

Number of Products by Molecule Types, H2 2019

Number of Products by Stage and Molecule Types, H2 2019”

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