Down Syndrome Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Global Markets Direct’s latest Pharmaceutical and Healthcare disease pipeline guide Down Syndrome – Drugs In Development, 2022, provides an overview of the Down Syndrome (Genetic Disorders) pipeline landscape.

Down syndrome is a genetic disorder that causes lifelong mental retardation, developmental delays and other problems. Down syndrome varies in severity, so developmental problems range from moderate to serious. Children with Down syndrome have a distinct facial appearance. Though not all children with Down syndrome have the same features, some of the more common features are flattened facial features, small head, short neck, protruding tongue, upward slanting eyes, unusual for the child’s ethnic group and unusually shaped ears. Children with Down syndrome may also have poor muscle tone, broad, short hands with a single crease in the palm, relatively short fingers and excessive flexibility.

Report Highlights

Global Markets Direct’s Pharmaceutical and Healthcare latest pipeline guide Down Syndrome – Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Down Syndrome (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

Down Syndrome Drugs in Development_GMDHC22036IDB

The Down Syndrome (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Down Syndrome and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase I, Preclinical, Discovery and Unknown stages are 1, 5, 10, 3 and 1 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 3 and 2 molecules, respectively.

Down Syndrome (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

– The pipeline guide provides a snapshot of the global therapeutic landscape of Down Syndrome (Genetic Disorders).

– The pipeline guide reviews pipeline therapeutics for Down Syndrome (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.

– The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.

– The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.

– The pipeline guide reviews key companies involved in Down Syndrome (Genetic Disorders) therapeutics and enlists all their major and minor projects.

– The pipeline guide evaluates Down Syndrome (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.

– The pipeline guide encapsulates all the dormant and discontinued pipeline projects.

– The pipeline guide reviews latest news related to pipeline therapeutics for Down Syndrome (Genetic Disorders)

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.

- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.

- Find and recognize significant and varied types of therapeutics under development for Down Syndrome (Genetic Disorders).

- Classify potential new clients or partners in the target demographic.

- Develop tactical initiatives by understanding the focus areas of leading companies.

- Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.

- Formulate corrective measures for pipeline projects by understanding Down Syndrome (Genetic Disorders) pipeline depth and focus of Indication therapeutics.

- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.

- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Companies mentioned

AC Immune SA

Aelis Farma SAS

Alzheon Inc

Annovis Bio Inc

Avanti Biosciences Inc

Balance Therapeutics Inc

Eisai Co Ltd

Elixirgen Therapeutics Inc

F. Hoffmann-La Roche Ltd

Felicitex Therapeutics Inc

KinoPharma Inc

Laminar Pharmaceuticals SA

Les Laboratoires Servier SAS

ManRos Therapeutics

NeuroCircuit Therapeutics Inc

NeuroNascent Inc

Perha Pharmaceuticals SAS

Prothena Corp Plc

SanBio Co Ltd

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Down Syndrome - Overview

Down Syndrome - Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Down Syndrome - Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Down Syndrome - Companies Involved in Therapeutics Development

AC Immune SA

Aelis Farma SAS

Alzheon Inc

Annovis Bio Inc

Avanti Biosciences Inc

Balance Therapeutics Inc

Eisai Co Ltd

Elixirgen Therapeutics Inc

F. Hoffmann-La Roche Ltd

Felicitex Therapeutics Inc

KinoPharma Inc

Laminar Pharmaceuticals SA

Les Laboratoires Servier SAS

ManRos Therapeutics

NeuroCircuit Therapeutics Inc

NeuroNascent Inc

Perha Pharmaceuticals SAS

Prothena Corp Plc

SanBio Co Ltd

Down Syndrome - Drug Profiles

ABI-02 - Drug Profile

Product Description

Mechanism Of Action

ACI-24 - Drug Profile

Product Description

Mechanism Of Action

History of Events

AEF-0217 - Drug Profile

Product Description

Mechanism Of Action

History of Events

ANVS-405 - Drug Profile

Product Description

Mechanism Of Action

ARN-23746 - Drug Profile

Product Description

Mechanism Of Action

buntanetap - Drug Profile

Product Description

Mechanism Of Action

History of Events

D-217 - Drug Profile

Product Description

Mechanism Of Action

DYR-219 - Drug Profile

Product Description

Mechanism Of Action

FX-8546 - Drug Profile

Product Description

Mechanism Of Action

Gene Therapy to Activate ZSCAN4 for Chromosomal Disorders - Drug Profile

Product Description

Mechanism Of Action

History of Events

KPO-1143 - Drug Profile

Product Description

Mechanism Of Action

History of Events

LAM-226 - Drug Profile

Product Description

Mechanism Of Action

LCTB-21 - Drug Profile

Product Description

Mechanism Of Action

lecanemab - Drug Profile

Product Description

Mechanism Of Action

History of Events

minocycline hydrochloride - Drug Profile

Product Description

Mechanism Of Action

NNI-351 - Drug Profile

Product Description

Mechanism Of Action

pegaspargase - Drug Profile

Product Description

Mechanism Of Action

History of Events

pentylenetetrazol - Drug Profile

Product Description

Mechanism Of Action

History of Events

RO-4938581 - Drug Profile

Product Description

Mechanism Of Action

Small Molecule for Alzheimer's Disease and Down Syndrome - Drug Profile

Product Description

Mechanism Of Action

Small Molecule to Inhibit DYRK1A for Alzheimer’s Disease and Down Syndrome - Drug Profile

Product Description

Mechanism Of Action

Small Molecule to Inhibit DYRK1A for Down Syndrome - Drug Profile

Product Description

Mechanism Of Action

History of Events

Small Molecules for Down Syndrome - Drug Profile

Product Description

Mechanism Of Action

Small Molecules to Inhibit OLIG2 for Down Syndrome - Drug Profile

Product Description

Mechanism Of Action

valiltramiprosate - Drug Profile

Product Description

Mechanism Of Action

History of Events

Down Syndrome - Dormant Projects

Down Syndrome - Discontinued Products

Down Syndrome - Product Development Milestones

Featured News & Press Releases

Jan 05, 2022: Annovis Bio announces official unique name buntanetap for lead candidate ANVS401

May 13, 2021: Annovis Bio files for FDA Orphan Drug Designation for ANVS401 for the treatment of Alzheimer's disease in persons with Down syndrome

Apr 29, 2021: Annovis Bio to study potential of ANVS401 to normalize brain development in Down syndrome

Mar 16, 2021: AC Immune announces new clinical results in Down syndrome and plans for future development of anti-Amyloid-Beta vaccine

Mar 15, 2021: BioArctic and Eisai presented latest data regarding lecanemab at AD/PD 2021

Mar 11, 2021: Annovis Bio signs GMP manufacturing agreement to support planned late-stage studies

Mar 05, 2021: Latest lecanemab data to be presented at the AD/PD congress

Feb 02, 2021: Annovis bio's lead candidate ANVS401 improves cognitive and functional outcomes in stroke mice study

Dec 15, 2020: Annovis Bio showcases its unique approach to Alzheimer’s at the New York Academy of Sciences

Sep 29, 2020: Annovis Bio demonstrates improved axonal transport in nerve cells and brain of down syndrome mice, an animal model of Alzheimer’s disease

Jul 23, 2020: AC Immune to present advances in two of the company’s world leading programs at the upcoming Alzheimer’s Association International Conference

Jul 10, 2020: Discovery of a novel drug candidate to develop effective treatments for brain disorders

Jul 11, 2019: AC Immune hosts KOL event and reports initial interim clinical data for ACI-24 vaccine to treat Alzheimer’s Disease-like symptoms in subjects with Down Syndrome

Aug 20, 2018: AC Immune announces important clinical milestones for ACI-24 vaccine in alzheimers disease and down syndrome

Sep 12, 2017: AC Immune Completes Recruitment For Low-Dose Cohort In World's First Clinical Trial For Anti-Abeta Vaccine Targeting Alzheimer's Disease-Like Characteristics In Individuals With Down Syndrome

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Down Syndrome, 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Universities/Institutes, 2022

Products under Development by Companies, 2022

Products under Development by Universities/Institutes, 2022

Number of Products by Stage and Target, 2022

Number of Products by Stage and Mechanism of Action, 2022

Number of Products by Stage and Route of Administration, 2022

Number of Products by Stage and Molecule Type, 2022

Down Syndrome – Pipeline by AC Immune SA, 2022

Down Syndrome – Pipeline by Aelis Farma SAS, 2022

Down Syndrome – Pipeline by Alzheon Inc, 2022

Down Syndrome – Pipeline by Annovis Bio Inc, 2022

Down Syndrome – Pipeline by Avanti Biosciences Inc, 2022

Down Syndrome – Pipeline by Balance Therapeutics Inc, 2022

Down Syndrome – Pipeline by Eisai Co Ltd, 2022

Down Syndrome – Pipeline by Elixirgen Therapeutics Inc, 2022

Down Syndrome – Pipeline by F. Hoffmann-La Roche Ltd, 2022

Down Syndrome – Pipeline by Felicitex Therapeutics Inc, 2022

Down Syndrome – Pipeline by KinoPharma Inc, 2022

Down Syndrome – Pipeline by Laminar Pharmaceuticals SA, 2022

Down Syndrome – Pipeline by Les Laboratoires Servier SAS, 2022

Down Syndrome – Pipeline by ManRos Therapeutics, 2022

Down Syndrome – Pipeline by NeuroCircuit Therapeutics Inc, 2022

Down Syndrome – Pipeline by NeuroNascent Inc, 2022

Down Syndrome – Pipeline by Perha Pharmaceuticals SAS, 2022

Down Syndrome – Pipeline by Prothena Corp Plc, 2022

Down Syndrome – Pipeline by SanBio Co Ltd, 2022

Down Syndrome – Dormant Projects, 2022

Down Syndrome – Discontinued Products, 2022

List of Figures

List of Figures

Number of Products under Development for Down Syndrome, 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Universities/Institutes, 2022

Number of Products by Targets, 2022

Number of Products by Stage and Targets, 2022

Number of Products by Mechanism of Actions, 2022

Number of Products by Stage and Mechanism of Actions, 2022

Number of Products by Routes of Administration, 2022

Number of Products by Stage and Routes of Administration, 2022

Number of Products by Molecule Types, 2022

Number of Products by Stage and Top 10 Molecule Types, 2022

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