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

Published: Aug, 2016 | Pages: 45 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

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

Summary

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.

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

 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

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