Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Leber Congenital Amaurosis (LCA) - Overview
Leber Congenital Amaurosis (LCA) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Leber Congenital Amaurosis (LCA) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Leber Congenital Amaurosis (LCA) - Companies Involved in Therapeutics Development
Atse Therapeutics Inc
Editas Medicine Inc
Generation Bio Co
IVERIC bio Inc
MeiraGTx Holdings Plc
Ocugen Inc
Odylia Therapeutics Inc
Oxford BioMedica Plc
ProQR Therapeutics NV
Variant
Leber Congenital Amaurosis (LCA) - Drug Profiles
AAV-AIPL1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
cevaretigene ritoparvovec - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EDIT-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Leber’s Congenital Amaurosis, Stargardt Disease And Wet Age-related Macular Degeneration - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate CEP290 for Leber Congenital Amaurosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate CRX for Leber Congenital Amaurosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Retil Guanylyl Cyclase-1 for Leber Congenital Amaurosis-1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate RPE65 for Leber Congenital Amaurosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate RPE65 for Leber Congenital Amaurosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate RPGRIP1 for Leber Congenital Amaurosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LX-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OCU-400 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OXB-204 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
QRX-136 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SAR-439483 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
sepofarsen - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VAR-002 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VAR-003 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Leber Congenital Amaurosis (LCA) - Dormant Projects
Leber Congenital Amaurosis (LCA) - Product Development Milestones
Featured News & Press Releases
Jun 23, 2021: Editas Medicine announces enrollment of the first pediatric cohort in the BRILLIANCE clinical trial of EDIT-101 for the treatment of LCA10 following IDMC endorsement
Mar 30, 2021: Variant confirms breakthrough in treatment of hereditary CRX-dependent retinopathies with innovative gene therapy product VAR002
Feb 23, 2021: European Commission grants Ocugen Orphan Medicil Product Desigtion for gene therapy product candidate, OCU400, for the treatment of both retinitis pigmentosa and Leber congenital amaurosis
Aug 06, 2020: Editas Medicine regains full global rights to ocular medicines
Mar 04, 2020: Allergan and Editas Medicine announce dosing of first patient in landmark phase 1/2 clinical trial of CRISPR medicine AGN-151587 (EDIT-101) for the treatment of LCA10
Jan 13, 2020: Editas Medicine reports presentation on EDIT-101 at J.P. Morgan Healthcare Conference
Sep 18, 2019: Ocugen granted FDA orphan drug desigtion for OCU400 (NR2E3) gene therapy for the treatment of CEP290 mutation associated retil disease
Jul 26, 2019: Allergan and Editas to assess in-vivo CRISPR editing to treat LCA10
Apr 29, 2019: Allergan and Editas Medicine announce initial data from tural history study to evaluate patients with leber congenital amaurosis 10 (LCA10)
Jan 21, 2019: Editas medicine announces publication in ture medicine of data supporting the development of EDIT-101 to treat Leber Congenital Amaurosis 10 (LCA10)
Jan 07, 2019: Editas Medicine reports on recent progress at J.P. Morgan Healthcare Conference
Nov 30, 2018: Editas Medicine Announces FDA Acceptance of IND Application for EDIT-101
Aug 27, 2018: Editas Medicine announces transition of chief medical officer
Aug 16, 2018: Editas Medicine Announces the Completion of the Recombint D Advisory Committee (RAC) Registration Process
May 18, 2018: Editas Medicine Reports Data Demonstrating Subretil Injection of EDIT-101 Well-tolerated in Non-human Primates
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures

List of Figures
Number of Products under Development for Leber Congenital Amaurosis (LCA), 2021
Number of Products under Development by Companies, 2021
Number of Products under Development by Universities/Institutes, 2021
Number of Products by Targets, 2021
Number of Products by Stage and Targets, 2021
Number of Products by Mechanism of Actions, 2021
Number of Products by Stage and Mechanism of Actions, 2021
Number of Products by Routes of Administration, 2021
Number of Products by Stage and Routes of Administration, 2021
Number of Products by Molecule Types, 2021
Number of Products by Stage and Molecule Types, 2021

List of Tables

List of Tables
Number of Products under Development for Leber Congenital Amaurosis (LCA), 2021
Number of Products under Development by Companies, 2021
Number of Products under Development by Universities/Institutes, 2021
Products under Development by Companies, 2021
Products under Development by Universities/Institutes, 2021
Number of Products by Stage and Target, 2021
Number of Products by Stage and Mechanism of Action, 2021
Number of Products by Stage and Route of Administration, 2021
Number of Products by Stage and Molecule Type, 2021
Leber Congenital Amaurosis (LCA) - Pipeline by Atsena Therapeutics Inc, 2021
Leber Congenital Amaurosis (LCA) - Pipeline by Editas Medicine Inc, 2021
Leber Congenital Amaurosis (LCA) - Pipeline by Generation Bio Co, 2021
Leber Congenital Amaurosis (LCA) - Pipeline by IVERIC bio Inc, 2021
Leber Congenital Amaurosis (LCA) - Pipeline by MeiraGTx Holdings Plc, 2021
Leber Congenital Amaurosis (LCA) - Pipeline by Ocugen Inc, 2021
Leber Congenital Amaurosis (LCA) - Pipeline by Odylia Therapeutics Inc, 2021
Leber Congenital Amaurosis (LCA) - Pipeline by Oxford BioMedica Plc, 2021
Leber Congenital Amaurosis (LCA) - Pipeline by ProQR Therapeutics NV, 2021
Leber Congenital Amaurosis (LCA) - Pipeline by Variant, 2021
Leber Congenital Amaurosis (LCA) - Dormant Projects, 2021