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Down Syndrome - Pipeline Insight, 2020

  • ID: 4330922
  • Drug Pipelines
  • October 2020
  • Region: Global
  • 70 pages
  • DelveInsight

FEATURED COMPANIES

  • AC Immune
  • Anvyl
  • Biogen
  • Eisai Co Ltd.
  • Elixirgen
  • Neuronascent

This “Down Syndrome - Pipeline Insight, 2020,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Down Syndrome pipeline landscape. It covers the pipeline drug profiles, including clinical and non-clinical 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

Down Syndrome Understanding

Down Syndrome: Overview

Down syndrome is a chromosome disorder associated with intellectual disability, a characteristic facial appearance, with a small nose and an upward slant to the eyes, and low muscle tone in infancy. The degree of intellectual disability varies from mild to moderate. People with Down syndrome may also be born with various health concerns such as heart defects or digestive abnormalities, as well as short stature and a single deep crease across the center of the palm. They also have an increased risk to develop gastroesophageal reflux, celiac disease, hypothyroidism, hearing and vision problems, leukemia, and Alzheimer's disease.

Symptoms

People with Down syndrome may develop the following medical problems:

  • Congenital hypothyroidism
  • Hearing loss
  • Congenital heart defects
  • Seizures
  • Vision disorders
  • Decreased muscle tone (hypotonia)

Diagnosis

Down syndrome is an incurable condition but there are many children with this condition who grow up to lead fulfilling lives. Down syndrome can be identified before birth using antenatal screening and further tests to confirm the likelihood of a child being born with the condition. To detect the likelihood of Down syndrome, the number and appearance of chromosomes present in the nucleus are assessed using a process called karyotyping.

Treatment

The U.S. Food and Drug Administration has not approved any drugs specifically to treat dementia associated with Down syndrome. In the United Kingdom, cholinesterase inhibitors, a class of drugs approved in the United States and many other countries to treat Alzheimer's, are approved to treat dementia in people with Down syndrome. Early intervention services, quality educational programs, a stimulating home environment, good health care, and positive support from family and friends can help people with Down syndrome develop to their full potential. The overall goal of treatment is to boost cognition by improving learning, memory, and speech. Other treatments depend on the specific health problems or complications present in each affected person.

Down Syndrome Emerging Drugs Chapters

This segment of the Down Syndrome 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.

Down Syndrome Emerging Drugs

ACI-24: AC Immune SA

ACI-24 is a liposomal therapeutic anti-Abeta vaccine candidate, which generates antibodies specific to disease-causing conformations. The vaccine is designed to stimulate a patient's immune system to produce antibodies that specifically target the oligomeric and fibrillary Abeta proteins to prevent plaque accumulation and to enhance plaque clearance. Preclinical data demonstrated a significant activity in plaque reduction and memory restoration as well as a favorable safety profile characterized by a lack of local inflammation and a mode of action independent of T-cells. The vaccine is being studied in Phase 2 clinical trial in patients with mild to moderate AD and in a Phase 1b study in young adult DS subjects, and has been proven to be safe with preliminary trends of efficacy.

Down Syndrome: Therapeutic Assessment

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

Major Players in Down Syndrome

There are approx. 10+ key companies which are developing the therapies for Down Syndrome. The companies which have their Down Syndrome drug candidates in the mid to advanced stage, i.e. phase III and Phase II include, Transition Therapeutics Ireland, Eisai, AC Immune etc.

Phases

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

  • Mid-stage products (Phase II and Phase I/II)
  • Early-stage products (Phase I/II and Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration

Down Syndrome 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
  • Intravenous
  • Intramuscular
  • Molecule Type

Products have been categorized under various Molecule types such as

  • Small molecules
  • Gene therapies
  • Vaccines
  • Monoclonal antibodies
  • Product Type

The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Down Syndrome: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Down syndrome 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 Down Syndrome drugs.

Report Highlights

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

Down Syndrome Report Insights

  • Down Syndrome Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

Down Syndrome 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 Down Syndrome drugs?
  • How many Down Syndrome drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Down Syndrome?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to Down syndrome 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 Down syndrome and their status?
  • What are the key designations that have been granted to emerging drugs?

Key Questions

  • Anvyl
  • Elixirgen
  • Neuronascent
  • AC Immune
  • Eisai Co Ltd.
  • Biogen
  • Transition Therapeutics Ireland

Key Questions

  • Research programme: CNS disorder therapeutics
  • ZSCAN4 gene therapy
  • AC Immune
  • BAN 2401
  • Scyllo-inositol


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Note: Product cover images may vary from those shown

FEATURED COMPANIES

  • AC Immune
  • Anvyl
  • Biogen
  • Eisai Co Ltd.
  • Elixirgen
  • Neuronascent
Introduction

Executive Summary

Down Syndrome: 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
Down Syndrome - Analytical Perspective

In-depth Commercial Assessment
  • Down Syndrome companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Down Syndrome Collaboration Deals
  • Company-Company Collaborations (Licensing / Partnering) Analysis
  • Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
  • Comparative Analysis
BAN 2401: Eisai Co Ltd.
  • Product Description
  • Research and Development
  • Product Development Activities
Mid Stage Products (Phase II)
  • Comparative Analysis
Scyllo-inositol: Transition Therapeutics Ireland
  • Product Description
  • Research and Development
  • Product Development Activities
Pre-clinical and Discovery Stage Products
  • Comparative Analysis
  • Product Description
  • Research and Development
  • Product Development Activities
Inactive Products
  • Comparative Analysis
Down Syndrome Key Companies

Down Syndrome Key Products

Down Syndrome- Unmet Needs

Down Syndrome- Market Drivers and Barriers

Down Syndrome- Future Perspectives and Conclusion

Down Syndrome Analyst Views

Down Syndrome Key Companies

Appendix

List of Tables
Table 1 Total Products for Down Syndrome
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 Down Syndrome
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
Note: Product cover images may vary from those shown
  • Anvyl
  • Elixirgen
  • Neuronascent
  • AC Immune
  • Eisai Co Ltd.
  • Biogen
  • Transition Therapeutics Ireland
Note: Product cover images may vary from those shown
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