This Retinitis Pigmentosa - Pipeline Insight, 2021 report provides comprehensive insights about 45+ companies and 45+ pipeline drugs in Retinitis Pigmentosa 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.
Retinitis pigmentosa (RP) is an umbrella term for a group of hereditary progressive retinal disorders that cause progressive degeneration of the retina. The disease is inherited as autosomal recessive, autosomal dominant or X-linked recessive traits. Signs of retinitis pigmentosa usually appear during childhood or adolescence. Common symptoms include difficulty seeing at night and a loss of side (peripheral) vision. RP is diagnosed by electroretinography (ERG), visual field testing, and retinal imaging, fundus auto-fluorescence (FAF) and genetic testing. Currently, there is no therapy that stops the evolution of pigmentary retinopathies or restores the vision. Management of retinitis pigmentosa includes dietary supplements, ozone therapy, surgery etc. Gene therapy based treatments are under research focus for the treatment of Retinitis pigmentosa.
Retinitis Pigmentosa - Pipeline Insight, 2021 report by the publisher outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Retinitis Pigmentosa pipeline landscape is provided which includes the disease overview and Retinitis Pigmentosa treatment guidelines. The assessment part of the report embraces, in depth Retinitis Pigmentosa 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, Retinitis Pigmentosa collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
The companies and academics are working to assess challenges and seek opportunities that could influence Retinitis Pigmentosa R&D. The therapies under development are focused on novel approaches to treat/improve Retinitis Pigmentosa.
This segment of the Retinitis Pigmentosa report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, 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.
BIIB-112: Biogen
BIIB-112 (formerly called NSR-RPGR) consists of a standard AAV8 vector carrying a codon optimized RPGR gene. BIIB112 is in phase II clinical studies for X-linked retinitis pigmentosa treatment. In September 2018, NSR-RPGR was granted orphan drug designation for the treatment of inherited retinal dystrophies due to defects in the RPGR gene from the U.S. Food and Drug Administration.
QR 1123 (formerly ISIS RHO 2.5Rx): Ionis Pharmaceuticals
QR 1123 (formerly ISIS RHO 2.5Rx) is an antisense oligonucleotide, designed to specifically target the mutant P23H messenger ribonucleic acid (mRNA) in order to reduce the expression of the P23H protein selectively, while preserving expression of the wild type (WT) rhodopsin (RHO) protein. The therapy is currently in Phase II clinical evaluation for the treatment of Retinitis Pigmentosa. In November 2019, ProQR Therapeutics announced that it received Orphan Drug designation (ODD) from the Food and Drug Administration (FDA) for QR-1123. QR-1123 was in-licensed from Ionis Pharmaceuticals in 2018. QR-1123 has also received Fast Track designation from the FDA.
There are approx. 45+ key companies which are developing the therapies for Retinitis Pigmentosa. The companies which have their Retinitis Pigmentosa drug candidates in the most advanced stage, i.e. Phase II include, Biogen.
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Retinitis Pigmentosa therapeutic drugs key players involved in developing key drugs.
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Retinitis Pigmentosa drugs.
Geography Covered
- Global coverage
Retinitis Pigmentosa Understanding
Retinitis Pigmentosa: Overview
Retinitis pigmentosa (RP) is an umbrella term for a group of hereditary progressive retinal disorders that cause progressive degeneration of the retina. The disease is inherited as autosomal recessive, autosomal dominant or X-linked recessive traits. Signs of retinitis pigmentosa usually appear during childhood or adolescence. Common symptoms include difficulty seeing at night and a loss of side (peripheral) vision. RP is diagnosed by electroretinography (ERG), visual field testing, and retinal imaging, fundus auto-fluorescence (FAF) and genetic testing. Currently, there is no therapy that stops the evolution of pigmentary retinopathies or restores the vision. Management of retinitis pigmentosa includes dietary supplements, ozone therapy, surgery etc. Gene therapy based treatments are under research focus for the treatment of Retinitis pigmentosa.
Retinitis Pigmentosa - Pipeline Insight, 2021 report by the publisher outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Retinitis Pigmentosa pipeline landscape is provided which includes the disease overview and Retinitis Pigmentosa treatment guidelines. The assessment part of the report embraces, in depth Retinitis Pigmentosa 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, Retinitis Pigmentosa 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 Retinitis Pigmentosa R&D. The therapies under development are focused on novel approaches to treat/improve Retinitis Pigmentosa.
Retinitis Pigmentosa Emerging Drugs Chapters
This segment of the Retinitis Pigmentosa report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, 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.
Retinitis Pigmentosa Emerging Drugs
BIIB-112: Biogen
BIIB-112 (formerly called NSR-RPGR) consists of a standard AAV8 vector carrying a codon optimized RPGR gene. BIIB112 is in phase II clinical studies for X-linked retinitis pigmentosa treatment. In September 2018, NSR-RPGR was granted orphan drug designation for the treatment of inherited retinal dystrophies due to defects in the RPGR gene from the U.S. Food and Drug Administration.
QR 1123 (formerly ISIS RHO 2.5Rx): Ionis Pharmaceuticals
QR 1123 (formerly ISIS RHO 2.5Rx) is an antisense oligonucleotide, designed to specifically target the mutant P23H messenger ribonucleic acid (mRNA) in order to reduce the expression of the P23H protein selectively, while preserving expression of the wild type (WT) rhodopsin (RHO) protein. The therapy is currently in Phase II clinical evaluation for the treatment of Retinitis Pigmentosa. In November 2019, ProQR Therapeutics announced that it received Orphan Drug designation (ODD) from the Food and Drug Administration (FDA) for QR-1123. QR-1123 was in-licensed from Ionis Pharmaceuticals in 2018. QR-1123 has also received Fast Track designation from the FDA.
Retinitis Pigmentosa: Therapeutic Assessment
This segment of the report provides insights about the different Retinitis Pigmentosa drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Retinitis Pigmentosa
There are approx. 45+ key companies which are developing the therapies for Retinitis Pigmentosa. The companies which have their Retinitis Pigmentosa drug candidates in the most advanced stage, i.e. Phase II include, Biogen.
Phases
This report covers around 45+ 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
Retinitis Pigmentosa 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
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Retinitis Pigmentosa: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Retinitis Pigmentosa 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 Retinitis Pigmentosa drugs.
Retinitis Pigmentosa Report Insights
- Retinitis Pigmentosa Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Retinitis Pigmentosa Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Key Questions Answered
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Retinitis Pigmentosa drugs?
- How many Retinitis Pigmentosa drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Retinitis Pigmentosa?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Retinitis Pigmentosa 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 Retinitis Pigmentosa and their status?
- What are the key designations that have been granted to the emerging drugs?
Key Players
- Biogen
- Neurotech
- Ionis Pharmaceuticals
- Novartis Pharmaceuticals
- Nacuity Pharmaceuticals
- ReNeuron
- ID Pharma
- Allegro Ophthalmics
- SparingVision
- Editas Medicine
- OiDE OptoEye
Key Products
- BIIB-112
- NT 501
- QR 1123 (formerly ISIS RHO 2.5Rx)
- CPK 850
- N-acetylcysteine-amide
- ReN 003
- DVC1-0401
- Risuteganib
- SPVN 06
- SPVN 20
- LCA10 program
- GtCCR4 gene therapy
Table of Contents
IntroductionExecutive Summary
Retinitis Pigmentosa: 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
Retinitis Pigmentosa - Analytical Perspective
In-depth Commercial Assessment
- Retinitis Pigmentosa companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Retinitis Pigmentosa Collaboration Deals
- Company-Company Collaborations (Licensing / Partnering) Analysis
- Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
- Comparative Analysis
Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
Mid Stage Products (Phase II)
- Comparative Analysis
BIIB-112: Biogen
- Product Description
- Research and Development
- Product Development Activities
Early Stage Products (Phase I)
- Comparative Analysis
N-acetylcysteine-amide: Nacuity Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
Preclinical and Discovery Stage Products
- Comparative Analysis
SPVN 06: SparingVision
- Product Description
- Research and Development
- Product Development Activities
Inactive Products
- Comparative Analysis
Retinitis Pigmentosa Key Companies
Retinitis Pigmentosa Key Products
Retinitis Pigmentosa- Unmet Needs
Retinitis Pigmentosa- Market Drivers and Barriers
Retinitis Pigmentosa- Future Perspectives and Conclusion
Retinitis Pigmentosa Analyst Views
Retinitis Pigmentosa Key Companies
AppendixList of Tables
Table 1 Total Products for Retinitis Pigmentosa
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 ProductsList of Figures
Figure 1 Total Products for Retinitis Pigmentosa
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
Companies Mentioned
A selection of companies mentioned in this report includes:
- Biogen
- Neurotech
- Ionis Pharmaceuticals
- Novartis Pharmaceuticals
- Nacuity Pharmaceuticals
- ReNeuron
- ID Pharma
- Allegro Ophthalmics
- SparingVision
- Editas Medicine
- OiDE OptoEye
