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Leber Congenital Amaurosis Pipeline Analysis (2023) Covering Clinical Trials, Emerging Therapies, FDA, EMA, and PMDA Approvals, Competitive Landscape | ProQR, Editas, Atsena, IVERIC bio, MeiraGTx

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Leber Congenital Amaurosis Pipeline Analysis (2023) Covering Clinical Trials, Emerging Therapies, FDA, EMA, and PMDA Approvals, Competitive Landscape | ProQR, Editas, Atsena, IVERIC bio, MeiraGTx

September 15
08:08 2023
Leber Congenital Amaurosis Pipeline Analysis (2023) Covering Clinical Trials, Emerging Therapies, FDA, EMA, and PMDA Approvals, Competitive Landscape | ProQR, Editas, Atsena, IVERIC bio, MeiraGTx
Delveinsight Business Research LLP
As per DelveInsight’s assessment, globally, about 10+ key pharma and biotech companies are working on 10+ pipeline drugs in the Leber Congenital Amaurosis therapeutics landscape based on different Routes of Administration (ROA), Mechanism of Action (MOA), and molecule types. Several of the therapies are in the advanced stages of clinical development and are expected to launch in the coming years.

Leber Congenital Amaurosis Pipeline Insight, 2023” report by DelveInsight outlines a comprehensive assessment of the present clinical/non-clinical development activities and growth prospects across the Leber Congenital Amaurosis Market. 

The Leber Congenital Amaurosis Pipeline report embraces in-depth commercial, regulatory, and Leber Congenital Amaurosis clinical trial assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the emerging Leber Congenital Amaurosis drugs, including the mechanism of action, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers, acquisition, funding, designations, and other product-related details.

Leber Congenital Amaurosis Pipeline Analysis

The report provides insights into: 

  • The report provides detailed insights into the emerging therapies for Leber Congenital Amaurosis treatment and the aggregate therapies developed by major pharma companies.

  • It accesses the different Leber Congenital Amaurosis therapiessegmented into early-stage, mid-stage, and late-stage of clinical development.

  • It outlines the major Leber Congenital Amaurosis companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

  • The report evaluates the Leber Congenital Amaurosis drugs that are under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. 

  • It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in the Leber Congenital Amaurosis therapeutic market.

Leber Congenital Amaurosis Therapeutics Landscape

Treatment for LCA is symptomatic and supportive. Genetic counseling is recommended for families of affected children. Gene therapy aims to halt retinal degeneration by replacing the mutated gene with a normal healthy copy; this enables the affected cells to regain some function and produce functioning proteins. Gene therapy is currently available for LCA caused by two mutations in a gene called RPE65, which accounts for about 6% of LCA cases.

In 2017, the gene therapy Luxturna (voretigene neparvovec-rzyl) was approved by the US Food and Drug Administration (FDA) to treat children and adults with two mutations in the RPE65 gene, which includes a type of LCA called LCA2. Luxturna is manufactured by Spark Therapeutics.

There are approx. 10+ key companies developing the therapies for Leber Congenital Amaurosis. Currently, ProQR Therapeutics is leading the therapeutics market with its Leber Congenital Amaurosis drug candidates in the most advanced stage of clinical development.

Leber Congenital Amaurosis Companies Actively Working in the Therapeutic Market Include:

  • ProQR Therapeutics

  • Editas Medicine

  • Atsena Therapeutics Inc.

  • Applied Genetic Technologies Corporation

  • IVERIC bio

  • MeiraGTx

And Many Others

Emerging and Marketed Leber Congenital Amaurosis Drugs Covered in the Report Include:

  • Sepofarsen (QR-110): ProQR Therapeutics

Sepofarsen (QR-110) is an RNA therapy that aims to restore vision in Leber Congenital Amaurosis 10 (LCA10) due to the most common p.Cys998X mutation in the CEP290 gene. Sepofarsen aims to restore vision loss in people with Leber Congenital Amaurosis due to a specific mutation in the CEP290 gene. This p.Cys998X mutation, also known as c.2991+1655A>G, is the most common mutation causing LCA10. 

  • EDIT-101: Editas Medicine, Inc.

The company’s approach to Leber Congenital Amaurosis 10 (LCA10) is to target a disease-causing mutation in the CEP290 gene that causes degeneration in ocular photoreceptor cells—cells critical for experiencing normal vision. They have developed an experimental CRISPR medicine called EDIT-101 designed to remove the CEP290 mutation.

Request the Sample PDF to Get a Better Understanding of the Emerging Drugs and Leber Congenital Amaurosis Companies Working in the Market @


Analysis of Emerging Leber Congenital Amaurosis Therapies by Phases

The report covers the emerging products under different phases of clinical development like –

  • Late-stage products (Phase III)

  • Mid-stage products (Phase II)

  • Early-stage product (Phase I) 

  • Pre-clinical and Discovery stage candidates

  • Discontinued and inactive candidates

Route of Administration

Leber Congenital Amaurosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

  • Oral

  • Parenteral

  • Intravitreal

  • Subretinal

  • Topical.

Molecule Type

Products have been categorized under various Molecule types such as

  • Monoclonal Antibody

  • Peptides

  • Polymer

  • Small molecule

  • Gene therapy

Learn How the Leber Congenital Amaurosis Treatment Outlook will Evolve with the Ongoing Clinical and commercial Activities in the Therapeutic Market @


Table of Content (TOC)

1. Report Introduction

2. Executive Summary

3. Leber Congenital Amaurosis Treatment Patterns

4. Leber Congenital Amaurosis – DelveInsight’s Analytical Perspective

5. Therapeutic Assessment

6. Leber Congenital Amaurosis Late Stage Products (Phase-III)

7. Leber Congenital Amaurosis Mid-Stage Products (Phase-II)

8. Leber Congenital Amaurosis Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Leber Congenital Amaurosis Discontinued Products

13. Leber Congenital Amaurosis Product Profiles

14. Major Leber Congenital Amaurosis Companies in the Market

15. Key Products in the Leber Congenital Amaurosis Therapeutics Segment

16. Dormant and Discontinued Products

17. Leber Congenital Amaurosis Unmet Needs

18. Leber Congenital Amaurosis Future Perspectives

19. Leber Congenital Amaurosis Analyst Review  

20. Appendix

21. Report Methodology

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

Download Sample PDF to Explore the Key Offerings of the Report:



About DelveInsight

DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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