Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1) – Pipeline Review, H2 2016’, 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 on the Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC 1.16.3.1), targeted therapeutics, 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.

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.

Bio Blast Pharma Ltd.

ProQR Therapeutics N.V.

RaNA Therapeutics, Inc.

Voyager Therapeutics, Inc.

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) Overview 6

Therapeutics Development 7

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) - Products under Development by Stage of Development 7

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) - Products under Development by Therapy Area 8

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) - Products under Development by Indication 9

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) - Pipeline Products Glance 10

Late Stage Products 10

Early Stage Products 11

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) - Products under Development by Companies 12

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) - Products under Development by Universities/Institutes 14

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) - Therapeutics Assessment 16

Assessment by Monotherapy/Combination Products 16

Assessment by Mechanism of Action 17

Assessment by Route of Administration 19

Assessment by Molecule Type 21

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) - Companies Involved in Therapeutics Development 23

Adverum Biotechnologies, Inc. 23

Bio Blast Pharma Ltd. 24

ProQR Therapeutics N.V. 25

RaNA Therapeutics, Inc. 26

Voyager Therapeutics, Inc. 27

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) - Drug Profiles 28

AGIL-FA - Drug Profile 28

Product Description 28

Mechanism Of Action 28

R&D Progress 28

ANN-003 - Drug Profile 29

Product Description 29

Mechanism Of Action 29

R&D Progress 29

BB-FA - Drug Profile 31

Product Description 31

Mechanism Of Action 31

R&D Progress 31

Gene Therapy to Activate Frataxin for Friedreich’s Ataxia - Drug Profile 32

Product Description 32

Mechanism Of Action 32

R&D Progress 32

Gene Therapy to Activate Frataxin for Friedreich’s Ataxia - Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

Gene Therapy to Activate Frataxin for Friedrich's Ataxia - Drug Profile 34

Product Description 34

Mechanism Of Action 34

R&D Progress 34

JOT-101 - Drug Profile 35

Product Description 35

Mechanism Of Action 35

R&D Progress 35

Oligonucleotide to Activate Frataxin for Friedreich's Ataxia - Drug Profile 36

Product Description 36

Mechanism Of Action 36

R&D Progress 36

QRX-604 - Drug Profile 37

Product Description 37

Mechanism Of Action 37

R&D Progress 37

Recombinant Protein for Friedreich Ataxia - Drug Profile 38

Product Description 38

Mechanism Of Action 38

R&D Progress 38

TAT-FXN - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

VYFXN-01 - Drug Profile 40

Product Description 40

Mechanism Of Action 40

R&D Progress 40

Frataxin Mitochondrial (Friedreich Ataxia Protein or FXN or EC

1.16.3.1) - Featured News & Press Releases 41

Aug 02, 2016: Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich’s Ataxia 41

Aug 05, 2015: Key Patent Granted For AAVLife’s Gene-Therapy Program to Treat Cardiomyopathy in Friedreich’s Ataxia 41

Jun 18, 2015: BioBlast Pharma to Present Preclinical Proofs of Concept in Friedreich’s Ataxia at Two Leading Scientific Meetings in June 42

Mar 26, 2015: BioBlast Pharma Announces Positive Preclinical Proof-of-Concept Results of Its Novel Mitochondrial Protein Replacement Platform in Friedreich's Ataxia 43

Dec 17, 2014: BioBlast Receives Notice of Patent Issuance From USPTO for Its Novel Fusion Protein-Based Platform for the Treatment of Friedrich's Ataxia 43

Appendix 44

Methodology 44

Coverage 44

Secondary Research 44

Primary Research 44

Expert Panel Validation 44

Contact Us 44

Disclaimer 45

List of Tables

List of Tables

Number of Products under Development for, H2 2016 7

Number of Products under Development by Therapy Area, H2 2016 8

Number of Products under Development by Indication, H2 2016 9

Comparative Analysis by Late Stage Development, H2 2016 10

Comparative Analysis by Early Stage Products, H2 2016 11

Number of Products under Development by Companies, H2 2016 12

Products under Development by Companies, H2 2016 13

Number of Products under Investigation by Universities/Institutes, H2 2016 14

Products under Investigation by Universities/Institutes, H2 2016 15

Assessment by Monotherapy/Combination Products, H2 2016 16

Number of Products by Stage and Mechanism of Action, H2 2016 18

Number of Products by Stage and Route of Administration, H2 2016 20

Number of Products by Stage and Molecule Type, H2 2016 22

Pipeline by Adverum Biotechnologies, Inc., H2 2016 23

Pipeline by Bio Blast Pharma Ltd., H2 2016 24

Pipeline by ProQR Therapeutics N.V., H2 2016 25

Pipeline by RaNA Therapeutics, Inc., H2 2016 26

Pipeline by Voyager Therapeutics, Inc., H2 2016 27

List of Figures

List of Figures

Number of Products under Development for, H2 2016 7

Number of Products under Development by Therapy Area, H2 2016 8

Number of Products under Development by Indication, H2 2016 9

Comparative Analysis by Early Stage Products, H2 2016 11

Assessment by Monotherapy/Combination Products, H2 2016 16

Number of Products by Mechanism of Actions, H2 2016 17

Number of Products by Stage and Mechanism of Actions, H2 2016 17

Number of Products by Routes of Administration, H2 2016 19

Number of Products by Stage and Routes of Administration, H2 2016 19

Number of Products by Molecule Types, H2 2016 21

Number of Products by Stage and Molecule Type, H2 2016 21

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