Global Leber Congenital Amaurosis Pipeline Insight Report 2021: Comprehensive Insights About 10+ Companies and 10+ Pipeline Drugs –

Global Leber Congenital Amaurosis Pipeline Insight Report 2021: Comprehensive Insights About 10+ Companies and 10+ Pipeline Drugs –

Global Leber Congenital Amaurosis Pipeline Insight Report 2021: Comprehensive Insights About 10+ Companies and 10+ Pipeline Drugs –

DUBLIN–(BUSINESS WIRE)–The “Leber Congenital Amaurosis – Pipeline Insight, 2021” drug pipelines has been added to’s offering.

This Leber congenital amaurosis – Pipeline Insight, 2021 report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Leber congenital amaurosis pipeline landscape.

“Leber congenital amaurosis – Pipeline Insight, 2021” report by the publisher outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Leber congenital amaurosis pipeline landscape is provided which includes the disease overview and Leber congenital amaurosis treatment guidelines.

The assessment part of the report embraces, in depth Leber congenital amaurosis commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Leber congenital amaurosis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Leber congenital amaurosis R&D. The therapies under development are focused on novel approaches to treat/improve Leber congenital amaurosis.

Leber congenital amaurosis Emerging Drugs Chapters

This segment of the Leber congenital amaurosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Leber congenital amaurosis Emerging Drugs

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.

The p.Cys998X mutation causes a mistake in the CEP290 RNA and therefore a process called splicing is not done properly. The cell can therefore not use the RNA to produce a working CEP290 protein that is essential for vision. Currently, it is in Phase II/III stage of development to treat Leber congenital amaurosis.

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. This approach may restore normal protein expression, photoreceptor function, and ultimately, vision.

Leber congenital amaurosis: Therapeutic Assessment

This segment of the report provides insights about the different Leber congenital amaurosis drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Leber congenital amaurosis

There are approx. 10+ key companies which are developing the therapies for Leber congenital amaurosis. The companies which have their Leber congenital amaurosis drug candidates in the most advanced stage, i.e. phase II/III include, ProQR Therapeutics.


This report covers around 10+ products under different phases of clinical development like

  • Late stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & 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
  • Product Type

Leber congenital amaurosis Report Insights

  • Leber congenital amaurosis Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

Leber congenital amaurosis Report Assessment

  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs

Key Players

  • ProQR Therapeutics
  • Editas Medicine
  • Coave Therapeutics
  • MeiraGTx
  • Novelion Therapeutics
  • Applied Genetic Technologies Corporation
  • Oxford BioMedica
  • IVERIC bio

Key Products

  • Sepofarsen (QR-110)
  • EDIT 101
  • HORA RPE65
  • SAR 439483
  • Cevaretigene ritoparvovec
  • QLT091001
  • rAAV2-CB-hRPE65
  • OXB 204

For more information about this drug pipelines report visit


Laura Wood, Senior Press Manager

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