Sections
Title | Starting Page | Number of Pages |
---|
Table of Contents | 2 | 5 |
List of Tables | 5 | 1 |
List of Figures | 6 | 1 |
Introduction | 7 | 1 |
Global Markets Direct Report Coverage | 7 | 1 |
Batten Disease Overview | 8 | 1 |
Therapeutics Development | 9 | 2 |
Pipeline Products for Batten Disease Overview | 9 | 1 |
Pipeline Products for Batten Disease Comparative Analysis | 10 | 1 |
Batten Disease Therapeutics under Development by Companies | 11 | 1 |
Batten Disease Therapeutics under Investigation by Universities/Institutes | 12 | 1 |
Batten Disease Pipeline Products Glance | 13 | 4 |
Late Stage Products | 13 | 1 |
Clinical Stage Products | 14 | 1 |
Early Stage Products | 15 | 1 |
Unknown Stage Products | 16 | 1 |
Batten Disease Products under Development by Companies | 17 | 1 |
Batten Disease Products under Investigation by Universities/Institutes | 18 | 1 |
Batten Disease Companies Involved in Therapeutics Development | 19 | 9 |
Abeona Therapeutics, Inc. | 19 | 1 |
BioMarin Pharmaceutical Inc. | 20 | 1 |
CereSpir Incorporated | 21 | 1 |
Evotec AG | 22 | 1 |
Ionis Pharmaceuticals, Inc. | 23 | 1 |
Mitochon Pharmaceuticals, Inc. | 24 | 1 |
RegenxBio Inc. | 25 | 1 |
Retrotope, Inc | 26 | 1 |
Spark Therapeutics, Inc. | 27 | 1 |
Batten Disease Therapeutics Assessment | 28 | 9 |
Assessment by Monotherapy Products | 28 | 1 |
Assessment by Target | 29 | 2 |
Assessment by Mechanism of Action | 31 | 2 |
Assessment by Route of Administration | 33 | 2 |
Assessment by Molecule Type | 35 | 2 |
Drug Profiles | 37 | 22 |
ABO-201 Drug Profile | 37 | 1 |
Antisense Oligonucleotides to Activate CLN3 for Batten Disease Drug Profile | 38 | 1 |
cerliponase alfa Drug Profile | 39 | 2 |
CSP-1103 Drug Profile | 41 | 2 |
DUOC-01 Drug Profile | 43 | 1 |
Gene Therapy to Activate CLN1 and CLN2 for Batten Disease Drug Profile | 44 | 1 |
Gene Therapy to Activate CLN6 for Batten Disease Drug Profile | 45 | 1 |
Gene Therapy to Activate Palmitoyl Protein Thioesterase 1 for Batten Disease Drug Profile | 46 | 1 |
Gene Therapy to Activate Tripeptidyl Peptidase 1 for Batten Disease Drug Profile | 47 | 2 |
INI-0602 Drug Profile | 49 | 1 |
MP-101 Drug Profile | 50 | 1 |
RT-002 Drug Profile | 51 | 1 |
Small Molecule for Batten Disease Drug Profile | 52 | 1 |
Small Molecules for Juvenile Batten Disease Drug Profile | 53 | 1 |
Small Molecules to Inhibit CDC42 for Batten Disease Drug Profile | 54 | 1 |
SNB-2401 Drug Profile | 55 | 1 |
SNB-4050 Drug Profile | 56 | 1 |
SPKTPP-1 Drug Profile | 57 | 1 |
XN-001 Drug Profile | 58 | 1 |
Batten Disease Dormant Projects | 59 | 1 |
Batten Disease Discontinued Products | 60 | 1 |
Batten Disease Product Development Milestones | 61 | 10 |
Featured News &Press Releases | 61 | 1 |
Jul 27, 2016: FDA Accepts BLA for BioMarins Cerliponase Alfa for CLN2 Disease, Form of Batten Disease | 61 | 1 |
May 03, 2016: BioMarin Announces EMA Grants Accelerated Assessment for Cerliponase Alfa, Experimental Treatment for a Form of Batten Disease | 61 | 1 |
Mar 02, 2016: Abeona Therapeutics Highlights New Preclinical Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) Data at WORLDSymposium 2016 | 62 | 1 |
Mar 02, 2016: BioMarin Announces Positive Data From Cerliponase Alfa Program for Treatment of CLN2 Disease, a Form of Batten Disease, at 12th Annual WORLDSymposium 2016 | 63 | 3 |
Nov 11, 2015: Spark Therapeutics Announces SPK-TPP1, Its First Program Targeting Neurodegenerative Disease | 66 | 1 |
Jan 12, 2015: BioMarin Provides Preliminary Data From Ongoing Phase 1/2 Pivotal Study of BMN 190 for Treatment of CLN2 Disorder, a Form of Batten Disease | 66 | 2 |
Sep 23, 2013: BioMarin Doses First Patient in Phase 1/2 Trial With BMN 190 for the Treatment of Neuronal Ceroid Lipofuscinosis Type 2, a Form of Batten Disease | 68 | 1 |
Apr 01, 2013: BioMarin Pharma Submits CTA For BMN-190 For Batten Disease | 68 | 1 |
Mar 15, 2012: Combination treatment in mice shows promise for fatal neurological disorder in kids | 69 | 2 |
Appendix | 71 | 2 |
Methodology | 71 | 1 |
Coverage | 71 | 1 |
Secondary Research | 71 | 1 |
Primary Research | 71 | 1 |
Expert Panel Validation | 71 | 1 |
Contact Us | 71 | 1 |
Disclaimer | 72 | 1 |