Leber Congenital Amaurosis (LCA) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Leber Congenital Amaurosis (LCA) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Leber Congenital Amaurosis (LCA) – Drugs In Development, 2022, provides an overview of the Leber Congenital Amaurosis (LCA) (Genetic Disorders) pipeline landscape.

Leber congenital amaurosis is an eye disorder that primarily affects the retina, which is the specialized tissue at the back of the eye that detects light and color. This condition is the most common cause of inherited blindness in childhood. Symptoms include vision loss, roving eye movements, deep-set eyes, developmental delay, epilepsy, and motor skill impairment.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Leber Congenital Amaurosis (LCA) – Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Leber Congenital Amaurosis (LCA) (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Leber Congenital Amaurosis (LCA) (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Leber Congenital Amaurosis (LCA) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 3, 1, 12 and 1 respectively. Similarly, the Universities portfolio in Phase I, Preclinical and Discovery stages comprises 1, 2 and 1 molecules, respectively.

Leber Congenital Amaurosis (LCA) (Genetic Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Leber Congenital Amaurosis (LCA) (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Leber Congenital Amaurosis (LCA) (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Leber Congenital Amaurosis (LCA) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Leber Congenital Amaurosis (LCA) (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Leber Congenital Amaurosis (LCA) (Genetic Disorders)
Reasons to Buy
  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Leber Congenital Amaurosis (LCA) (Genetic Disorders).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Leber Congenital Amaurosis (LCA) (Genetic Disorders) pipeline depth and focus of Indication therapeutics.
  • 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.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.


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
Atsena Therapeutics Inc
Editas Medicine Inc
Generation Bio Co
Hubble Therapeutics Inc
Huida (Shanghai) Biotechnology Co Ltd
IVERIC bio Inc
LuxFiat Therapeutics LLC
MeiraGTx Holdings Plc
Ocugen Inc
Odylia Therapeutics Inc
Opus Genetics Inc
ProQR Therapeutics NV
Variant
Leber Congenital Amaurosis (LCA) – Drug Profiles
AAV-AIPL1 – Drug Profile
cevaretigene ritoparvovec – Drug Profile
EDIT-101 – Drug Profile
Gene Therapy to Activate CRX for Leber Congenital Amaurosis – Drug Profile
Gene Therapy to Activate Retinal Guanylyl Cyclase-1 for Leber Congenital Amaurosis-1 – Drug Profile
Gene Therapy to Activate RPE65 for Leber Congenital Amaurosis – Drug Profile
HG-004 – Drug Profile
HUB-101 – Drug Profile
LCA10 – Drug Profile
LX-101 – Drug Profile
miniCEP290: LCA10 – Drug Profile
OCU-400 – Drug Profile
OPGX-001 – Drug Profile
OPGx-002 – Drug Profile
OPGx-003 – Drug Profile
OT-004 – Drug Profile
QRX-136 – Drug Profile
SAR-439483 – Drug Profile
sepofarsen – Drug Profile
VAR-002 – Drug Profile
VAR-003 – Drug Profile
Leber Congenital Amaurosis (LCA) – Dormant Projects
Leber Congenital Amaurosis (LCA) – Product Development Milestones
Featured News & Press Releases
May 04, 2022: Opus Genetics announces promising new data highlighting potential of AAV-based gene therapy OPGx-001 for the treatment of rare inherited retinal diseases
May 04, 2022: Opus Genetics announces promising new data highlighting potential of AAV-based gene therapy OPGx-002 for the treatment of rare inherited retinal diseases
Nov 24, 2021: VARIANT receives European Orphan Drug Designation for its gene therapy product candidate, VAR002, for the treatment of both Leber Congenital Amaurosis and Cone-Rod dystrophy
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 Medicinal Product Designation for gene therapy product candidate, OCU400, for the treatment of both retinitis pigmentosa and Leber congenital amaurosis
Sep 18, 2019: Ocugen granted FDA orphan drug designation for OCU400 (NR2E3) gene therapy for the treatment of CEP290 mutation associated retinal disease
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Table 1: Number of Products under Development for Leber Congenital Amaurosis (LCA), 2022
Table 2: Number of Products under Development by Companies, 2022
Table 3: Number of Products under Development by Universities/Institutes, 2022
Table 4: Products under Development by Companies, 2022
Table 5: Products under Development by Companies, 2022 (Contd..1)
Table 6: Products under Development by Universities/Institutes, 2022
Table 7: Number of Products by Stage and Target, 2022
Table 8: Number of Products by Stage and Mechanism of Action, 2022
Table 9: Number of Products by Stage and Route of Administration, 2022
Table 10: Number of Products by Stage and Molecule Type, 2022
Table 11: Leber Congenital Amaurosis (LCA) – Pipeline by Atsena Therapeutics Inc, 2022
Table 12: Leber Congenital Amaurosis (LCA) – Pipeline by Editas Medicine Inc, 2022
Table 13: Leber Congenital Amaurosis (LCA) – Pipeline by Generation Bio Co, 2022
Table 14: Leber Congenital Amaurosis (LCA) – Pipeline by Hubble Therapeutics Inc, 2022
Table 15: Leber Congenital Amaurosis (LCA) – Pipeline by Huida (Shanghai) Biotechnology Co Ltd, 2022
Table 16: Leber Congenital Amaurosis (LCA) – Pipeline by IVERIC bio Inc, 2022
Table 17: Leber Congenital Amaurosis (LCA) – Pipeline by LuxFiat Therapeutics LLC, 2022
Table 18: Leber Congenital Amaurosis (LCA) – Pipeline by MeiraGTx Holdings Plc, 2022
Table 19: Leber Congenital Amaurosis (LCA) – Pipeline by Ocugen Inc, 2022
Table 20: Leber Congenital Amaurosis (LCA) – Pipeline by Odylia Therapeutics Inc, 2022
Table 21: Leber Congenital Amaurosis (LCA) – Pipeline by Opus Genetics Inc, 2022
Table 22: Leber Congenital Amaurosis (LCA) – Pipeline by ProQR Therapeutics NV, 2022
Table 23: Leber Congenital Amaurosis (LCA) – Pipeline by Variant, 2022
Table 24: Leber Congenital Amaurosis (LCA) – Dormant Projects, 2022
List of Figures
Figure 1: Number of Products under Development for Leber Congenital Amaurosis (LCA), 2022
Figure 2: Number of Products under Development by Companies, 2022
Figure 3: Number of Products under Development by Universities/Institutes, 2022
Figure 4: Number of Products by Top 10 Targets, 2022
Figure 5: Number of Products by Stage and Top 10 Targets, 2022
Figure 6: Number of Products by Top 10 Mechanism of Actions, 2022
Figure 7: Number of Products by Stage and Top 10 Mechanism of Actions, 2022
Figure 8: Number of Products by Routes of Administration, 2022
Figure 9: Number of Products by Stage and Routes of Administration, 2022
Figure 10: Number of Products by Molecule Types, 2022
Figure 11: Number of Products by Stage and Molecule Types, 2022

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