Retinitis Pigmentosa Pipeline Insight

DelveInsight’s, “Retinitis pigmentosa (RP)-Pipeline Insight, 2020,” report provides comprehensive insights about 25+ companies and 30+ 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. 

Geography Covered

  • Global coverage

Retinitis pigmentosa Understanding

Retinitis pigmentosa: Overview

Retinitis pigmentosa (RP) comprises a large group of inherited vision disorders that cause progressive degeneration of the retina, the light sensitive membrane that coats the inside of the eyes. Peripheral (or side) vision gradually decreases and eventually is lost in most cases. Central vision is usually preserved until late in these conditions.

Symptoms

RP usually begins as night or dim light visual impairment (that is, difficulty in seeing in dimly lit environments or at dusk, or adapting to, or recovering function in, dim light after being in bright light for any length of time). Symptoms are more often noticed between the age 10 and 40, but earlier and later onset forms of RP exist. Characteristically, symptoms develop gradually over time. The sudden onset of these same symptoms should point to a different cause, such as an autoimmune process. Older people with sudden onset of these symptoms are especially at risk for experiencing them as the result of having cancer (so called paraneoplastic retinopathy, which often co-occurs with an optic nerve involvement as well).

Diagnosis

RP is diagnosed by electroretinography (ERG) showing progressive loss in photoreceptor function, visual field testing, and retinal imaging [mainly by optical coherence tomography (OCT) and fundus auto-fluorescence (FAF) that show detailed microanatomical features that cannot be resolved by naked eye]. Molecular genetic testing for mutations in many of the genes associated with RP is available to confirm the diagnosis.  

Treatment

There is no single treatment for retinitis pigmentosa because there are over 100 genes that cause it. There is treatment for retinitis pigmentosa due to the RPE65 genetic defect. Long-term supplementation with these regimens of vitamin A palmitate appears to be safe, although older patients should be aware that there is some evidence (although not univocal) that vitamin A supplements may promote further bone density loss, worsen osteoporosis and, therefore, increase the risk of hip fractures.

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 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

  • AAV8-RPGR: Nightstar Therapeutics/Biogen

Nightstar Therapeutics (now Biogen Inc.) initiated the development of an AAV-based Adeno-Associated Viral Vector Encoding Retinitis Pigmentosa GTPase Regulator gene therapy (AAV8-RPGR) in phase II/III for X-linked Retinitis Pigmentosa (XLRP). After the acquisition by Biogen, AAV8-RPGR is now known as BIIB112.


  • Risuteganib: Allegro Ophthalmics

Risuteganib (ALG 1001) is a first-in-class oligopeptide therapy, being developed by Allegro Ophthalmics for the treatment of Retinitis Pigmentosa. Risuteganib regulates mitochondrial dysfunction and downregulates oxidative stress response to restore retinal homeostasis.


  • NPI 001: Nacuity Pharmaceuticals

NPI-001, a GMP-grade of N-acetylcysteine amide (NACA), is an experimental antioxidant drug for the treatment of RP. The drug is in phase II stage of development.

Further product details are provided in the report……..

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. 25+ 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 III include, Nightstar Therapeutics/Biogen.

 Phases

DelveInsight’s report covers around 30+ 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

  • Vaccines
  • Monoclonal Antibody
  • Peptides
  • Polymer
  • Small molecule
  • Gene therapy
  • Product Type

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 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.

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.
  • In January 2017, GenSight Biologics announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the Company’s product candidate GS030 for the treatment of retinitis pigmentosa.
  • In March 2020, MeiraGTx announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Advanced Therapy Medicinal Product (ATMP) designations to AAV-RPGR, MeiraGTx’s investigational gene therapy for the treatment of x-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.
  • In January 2019, MeiraGTx announced that it has entered into a broad strategic collaboration with Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to develop and commercialize gene therapies for the treatment of inherited retinal diseases (IRDs).
  • In January 2019, the Janssen Pharmaceutical Companies of Johnson & Johnson announced a worldwide collaboration and license agreement with MeiraGTx to develop, manufacture and commercialize its clinical stage inherited retinal disease portfolio, including leading product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3, and X-linked retinitis pigmentosa (XLRP).

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

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

  • Acucela Inc
  • Allergan Plc
  • Amarantus Bioscience Holdings Inc
  • Amgen Inc
  • Applied Genetic Technologies Corp
  • Asklepios BioPharmaceutical Inc
  • Astellas Pharma Inc
  • Caladrius Biosciences Inc
  • Dompe Farmaceutici SpA
  • GenSight Biologics SA
  • Grupo Ferrer Internacional SA
  • ID Pharma Co Ltd
  • Janssen Pharmaceutical
  • MeiraGTx
  • Anabasis Pharma
  • Allegro Ophthalmics
  • Nacuity Pharmaceuticals
  • ReNeuron
  • jCyte
  • Editas Medicine
  • SanBio
  • Nightstar Therapeutics/Biogen
  • OiDE OptoEye

Key Products

  • Cenegermin
  • LCA10 program
  • SB623:
  • Risuteganib
  • NPI 001
  • ReN 003
  • jCell
  • GtCCR4 gene therapy
  • AAV8-RPGR/BIIB112
  • EDIT 102
  • SB 623

 

Introduction

Executive 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 – DelveInsight’s 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

AAV8-RPGR/BIIB112: Nightstar Therapeutics/Biogen

• Product Description

• Research and Development

• Product Development Activities

Mid Stage Products (Phase II)

• Comparative Analysis

ReN 003: ReNeuron

• Product Description

• Research and Development

• Product Development Activities

jCell: jCyte

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I)

• Comparative Analysis

Risuteganib: Allegro Ophthalmics

• Product Description

• Research and Development

• Product Development Activities

NPI 001: Nacuity Pharmaceuticals

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Pre-clinical and Discovery Stage Products

• Comparative Analysis

LCA10 program: Editas Medicine

• Product Description

• Research and Development

• Product Development Activities

SB623: SanBio

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

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

Appendix

List 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 Products

List 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

• Acucela Inc

• Allergan Plc

• Amarantus Bioscience Holdings Inc

• Amgen Inc

• Applied Genetic Technologies Corp

• Asklepios BioPharmaceutical Inc

• Astellas Pharma Inc

• Caladrius Biosciences Inc

• Dompe Farmaceutici SpA

• GenSight Biologics SA

• Grupo Ferrer Internacional SA

• ID Pharma Co Ltd

• Janssen Pharmaceutical

• MeiraGTx

• Anabasis Pharma

• Allegro Ophthalmics

• Nacuity Pharmaceuticals

• ReNeuron

• jCyte

• Editas Medicine

• SanBio

• Nightstar Therapeutics/Biogen

• OiDE OptoEye

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  • Retinitis Pigmentosa drugs
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