Leber congenital amaurosis - Pipeline Insight, 2021
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DelveInsight’s, “Leber congenital amaurosis - Pipeline Insight, 2021,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Leber congenital amaurosis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
Leber congenital amaurosis: Overview
Leber congenital amaurosis (LCA) is a rare genetic eye disorder. Affected infants are often blind at birth. Other symptoms may include crossed eyes (strabismus); rapid, involuntary eye movements (nystagmus); unusual sensitivity to light (photophobia); clouding of the lenses of the eyes (cataracts); and/or a cone shape to the front of the eye (keratoconus). LCA is usually inherited as an autosomal recessive genetic condition. Children born with LCA have light-gathering cells (rods and cones) of the retina that do not function properly. Absence or reduction of the electrical activity of the retina is always observed and is necessary for the diagnosis of LCA. A decrease in visual responsiveness at birth is the first sign of the disease. Often the child will poke, press and rub the eyes to stimulate the retina to produce light (Franceschetti’s oculo-digital sign).This activity may cause the eyes to become sunken or deep set (enophthalmos). LCA is a monogenic disease and at least 27 genes are implicated. Changes (mutations) in these genes can account for about 80-90% of diagnosed cases of LCA. The genes responsible for the remaining 10-20% of diagnoses are not known. LCA is usually inherited as an autosomal recessive genetic condition.
'Leber congenital amaurosis - Pipeline Insight, 2021' report by DelveInsight 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
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
Further product details are provided in the report.
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:
Leber congenital amaurosis: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Leber congenital amaurosis therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Leber congenital amaurosis drugs.
Leber congenital amaurosis Report Insights
Current Treatment Scenario and Emerging Therapies:
DelveInsight’s, “Leber congenital amaurosis - Pipeline Insight, 2021,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Leber congenital amaurosis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
- Global coverage
Leber congenital amaurosis: Overview
Leber congenital amaurosis (LCA) is a rare genetic eye disorder. Affected infants are often blind at birth. Other symptoms may include crossed eyes (strabismus); rapid, involuntary eye movements (nystagmus); unusual sensitivity to light (photophobia); clouding of the lenses of the eyes (cataracts); and/or a cone shape to the front of the eye (keratoconus). LCA is usually inherited as an autosomal recessive genetic condition. Children born with LCA have light-gathering cells (rods and cones) of the retina that do not function properly. Absence or reduction of the electrical activity of the retina is always observed and is necessary for the diagnosis of LCA. A decrease in visual responsiveness at birth is the first sign of the disease. Often the child will poke, press and rub the eyes to stimulate the retina to produce light (Franceschetti’s oculo-digital sign).This activity may cause the eyes to become sunken or deep set (enophthalmos). LCA is a monogenic disease and at least 27 genes are implicated. Changes (mutations) in these genes can account for about 80-90% of diagnosed cases of LCA. The genes responsible for the remaining 10-20% of diagnoses are not known. LCA is usually inherited as an autosomal recessive genetic condition.
'Leber congenital amaurosis - Pipeline Insight, 2021' report by DelveInsight 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.
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
- EDIT-101: Editas Medicine, Inc.
Further product details are provided in the report.
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
- Phases
- 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
- Oral
- Parenteral
- intravitreal
- Subretinal
- Topical.
- Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Leber congenital amaurosis: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Leber congenital amaurosis therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Leber congenital amaurosis drugs.
Leber congenital amaurosis Report Insights
- Leber congenital amaurosis Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Leber congenital amaurosis drugs?
- How many Leber congenital amaurosis drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Leber congenital amaurosis?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Leber congenital amaurosis therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Leber congenital amaurosis and their status?
- What are the key designations that have been granted to the emerging drugs?
- ProQR Therapeutics
- Editas Medicine
- Coave Therapeutics
- MeiraGTx
- Novelion Therapeutics
- Applied Genetic Technologies Corporation
- Oxford BioMedica
- IVERIC bio
- Sepofarsen (QR-110)
- EDIT 101
- HORA RPE65
- SAR 439483
- Cevaretigene ritoparvovec
- QLT091001
- rAAV2-CB-hRPE65
- AAV AIPL1
- OXB 204
- Research programme: eye disorder gene therapies
Introduction
Executive Summary
Leber congenital amaurosis: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Leber congenital amaurosis – DelveInsight’s Analytical Perspective
Late Stage Products (Phase II/III)
Comparative Analysis
Sepofarsen (QR-110): ProQR Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Mid Stage Products (Phase II)
Comparative Analysis
Drug name: Company name
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Early Stage Products (Phase I/II)
Comparative Analysis
Cevaretigene ritoparvovec: MeiraGTx
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Preclinical and Discovery Stage Products
Comparative Analysis
OXB 204: Oxford BioMedica
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Inactive Products
Comparative Analysis
Leber congenital amaurosis Key Companies
Leber congenital amaurosis Key Products
Leber congenital amaurosis- Unmet Needs
Leber congenital amaurosis- Market Drivers and Barriers
Leber congenital amaurosis- Future Perspectives and Conclusion
Leber congenital amaurosis Analyst Views
Leber congenital amaurosis Key Companies
Appendix
Executive Summary
Leber congenital amaurosis: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Leber congenital amaurosis – DelveInsight’s Analytical Perspective
Late Stage Products (Phase II/III)
Comparative Analysis
Sepofarsen (QR-110): ProQR Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Mid Stage Products (Phase II)
Comparative Analysis
Drug name: Company name
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Early Stage Products (Phase I/II)
Comparative Analysis
Cevaretigene ritoparvovec: MeiraGTx
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Preclinical and Discovery Stage Products
Comparative Analysis
OXB 204: Oxford BioMedica
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report.
Inactive Products
Comparative Analysis
Leber congenital amaurosis Key Companies
Leber congenital amaurosis Key Products
Leber congenital amaurosis- Unmet Needs
Leber congenital amaurosis- Market Drivers and Barriers
Leber congenital amaurosis- Future Perspectives and Conclusion
Leber congenital amaurosis Analyst Views
Leber congenital amaurosis Key Companies
Appendix
LIST OF TABLES
Table 1 Total Products for Leber congenital amaurosis
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
Table 1 Total Products for Leber congenital amaurosis
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
LIST OF FIGURES
Figure 1 Total Products for Leber congenital amaurosis
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Figure 1 Total Products for Leber congenital amaurosis
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
