Sections
Title | Starting Page | Number of Pages |
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Table of Contents | 2 | 4 |
List of Tables | 4 | 1 |
List of Figures | 5 | 1 |
Introduction | 6 | 1 |
Global Markets Direct Report Coverage | 6 | 1 |
Leber Congenital Amaurosis (LCA) Overview | 7 | 1 |
Therapeutics Development | 8 | 2 |
Pipeline Products for Leber Congenital Amaurosis (LCA) Overview | 8 | 1 |
Pipeline Products for Leber Congenital Amaurosis (LCA) Comparative Analysis | 9 | 1 |
Leber Congenital Amaurosis (LCA) Therapeutics under Development by Companies | 10 | 1 |
Leber Congenital Amaurosis (LCA) Therapeutics under Investigation by Universities/Institutes | 11 | 1 |
Leber Congenital Amaurosis (LCA) Pipeline Products Glance | 12 | 3 |
Late Stage Products | 12 | 1 |
Clinical Stage Products | 13 | 1 |
Early Stage Products | 14 | 1 |
Leber Congenital Amaurosis (LCA) Products under Development by Companies | 15 | 1 |
Leber Congenital Amaurosis (LCA) Products under Investigation by Universities/Institutes | 16 | 1 |
Leber Congenital Amaurosis (LCA) Companies Involved in Therapeutics Development | 17 | 6 |
AmpliPhi Biosciences Corporation | 17 | 1 |
Applied Genetic Technologies Corporation | 18 | 1 |
Editas Medicine, Inc. | 19 | 1 |
ProQR Therapeutics N.V. | 20 | 1 |
QLT Inc. | 21 | 1 |
Spark Therapeutics, Inc. | 22 | 1 |
Leber Congenital Amaurosis (LCA) Therapeutics Assessment | 23 | 9 |
Assessment by Monotherapy Products | 23 | 1 |
Assessment by Target | 24 | 2 |
Assessment by Mechanism of Action | 26 | 2 |
Assessment by Route of Administration | 28 | 2 |
Assessment by Molecule Type | 30 | 2 |
Drug Profiles | 32 | 17 |
A-001 Drug Profile | 32 | 2 |
Gene Therapy for Leber Congenital Amaurosis 10 Drug Profile | 34 | 1 |
Gene Therapy to Activate Retinal Guanylyl Cyclase-1 for Leber Congenital Amaurosis-1 Drug Profile | 35 | 1 |
Gene Therapy to Activate Retinal Pigment Epithelium 65 for Leber Congenital Amaurosis Drug Profile | 36 | 2 |
HORARPE-65 Drug Profile | 38 | 1 |
QR-110 Drug Profile | 39 | 1 |
voretigene neparvovec Drug Profile | 40 | 4 |
zuretinol acetate Drug Profile | 44 | 5 |
Leber Congenital Amaurosis (LCA) Dormant Projects | 49 | 1 |
Leber Congenital Amaurosis (LCA) Product Development Milestones | 50 | 10 |
Featured News &Press Releases | 50 | 1 |
Jun 01, 2016: ProQR s Drug Candidate QR-110 for Leber s Congenital Amaurosis Type 10 Receives EMA and FDA Orphan Drug Designation | 50 | 1 |
Apr 25, 2016: ProQR Announces an Investor and Analyst Event and Pre-Clinical Data Presentations for QR-110 for LCA10 at the 2016 ARVO Annual Meeting | 50 | 1 |
May 04, 2015: Penn Study Indicates that Gene Therapy Efficacy for LCA is Dynamic: Improvement is Followed by Decline in Vision | 50 | 2 |
May 01, 2015: Spark Therapeutics to Have Multiple Presentations at 2015 ARVO Meeting | 52 | 1 |
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 | 52 | 2 |
Nov 06, 2014: Spark Therapeutics Receives FDA Breakthrough Therapy Designation for Its Lead Product Candidate, SPK-RPE65 | 54 | 1 |
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 | 55 | 1 |
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 | 56 | 1 |
May 08, 2014: Spark Therapeutics Scientific Advisor to Deliver Keynote at the Association for Research in Vision and Ophthalmology s 2014 Annual Meeting | 57 | 1 |
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 | 57 | 1 |
Jan 14, 2014: Spark Therapeutics Achieves Recruitment Goal in Phase 3 Gene Therapy Clinical Study for Inherited Blindness | 58 | 2 |
Appendix | 60 | 2 |
Methodology | 60 | 1 |
Coverage | 60 | 1 |
Secondary Research | 60 | 1 |
Primary Research | 60 | 1 |
Expert Panel Validation | 60 | 1 |
Contact Us | 60 | 1 |
Disclaimer | 61 | 1 |