Down Syndrome Clinical, Companies, Therapeutic Assessment, Therapies, Treatment Algorithm, Pipeline Analysis | ACI-24, Connecta Therapeutics

Down Syndrome Pipeline constitutes 5+ key companies continuously working towards developing 5+ Down Syndrome treatment therapies, analyzes DelveInsight.

Down Syndrome Overview:

Down Syndrome (DS) is a genetic condition resulting from the presence of an additional copy of chromosome 21, a phenomenon referred to as trisomy 21. This extra genetic material disrupts normal development, giving rise to recognizable physical traits such as a flattened facial appearance, upward-slanting eyes, and a single deep crease across the palm. People with Down Syndrome typically face developmental delays and intellectual challenges, although the extent of these difficulties can vary significantly among individuals. The condition is also frequently associated with health complications, including congenital heart defects, respiratory issues, and an increased vulnerability to infections.

Common signs and symptoms include characteristic facial features (e.g., flat facial profile, almond-shaped eyes), developmental delays, and varying levels of cognitive impairment. Additionally, affected individuals may suffer from medical problems such as heart abnormalities at birth, breathing difficulties, and immune system deficiencies that make them more prone to illness.

At the pathophysiological level, Down Syndrome stems from the extra chromosome 21, which causes an imbalance in gene expression. This overexpression affects a range of genetic and molecular pathways, influencing the development and function of multiple body systems. In the brain, it alters neuronal structure and connectivity, contributing to intellectual disabilities and delays in cognitive development. It also disrupts the functioning of the cardiovascular, immune, and endocrine systems, leading to conditions like heart defects, hypothyroidism, and immune dysfunctions. These widespread effects result from the intricate biological consequences of having an additional chromosome and account for the diverse symptoms and complications seen in individuals with Down Syndrome.

Request for a detailed insights report on Down Syndrome pipeline insights https://www.delveinsight.com/report-store/down-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

"Down Syndrome Pipeline Insight 2025" report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Down Syndrome Therapeutics Market.

Key Takeaways from the Down Syndrome Pipeline Report

DelveInsight's Down Syndrome pipeline report depicts a robust space with 5+ active players working to develop 5+ pipeline therapies for Down Syndrome treatment.
In April 2025, the first participant with Down Syndrome was dosed in the HERO trial, evaluating ION269, an investigational drug designed to reduce amyloid precursor protein (APP) and prevent plaque formation. This marks a pivotal shift toward early Alzheimer's prevention in individuals with Down Syndrome, who are genetically prone to developing the disease by around age 40.
In March 2025, Johns Hopkins reported a success story: an 11-year-old with Down Syndrome received this implant and experienced complete resolution of OSA and notable improvements in behavior and sleep quality.
Key Down Syndrome companies such as ACI-24, Connecta Therapeutics, and others are evaluating new drugs for Down Syndrome to improve the treatment landscape.
Promising Down Syndrome pipeline therapies in various stages of development include ACI-24, and others.

Down Syndrome Pipeline Analysis
The report provides insights into:
The report provides detailed insights into the key companies that are developing therapies in the Down Syndrome Market.
The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Down Syndrome treatment.
It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Down Syndrome market.

Download our free sample page report on Down Syndrome pipeline insights https://www.delveinsight.com/sample-request/down-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Down Syndrome Emerging Drugs

ACI-24 - AC Immune SA

ACI-24 is an anti-Aβ active immunotherapy developed by AC Immune SA, aimed at prompting the immune system to generate antibodies that specifically recognize and target misfolded amyloid-beta (Aβ) proteins. This approach seeks to prevent the build-up of toxic Aβ aggregates and support the removal of existing amyloid plaques. The vaccine, which is liposome-based, is designed to address neurological conditions linked to misfolded Aβ proteins. It incorporates the first 15 amino acids of the human Aβ peptide arranged in a stacked β-sheet configuration on the liposome surface, thereby stimulating the immune system to produce polyclonal antibodies against both soluble and insoluble aggregated Aβ forms, including oligomers-a particularly harmful species implicated in the development of Alzheimer's Disease and Down Syndrome. ACI-24 is currently undergoing Phase II clinical trials to evaluate its effectiveness in treating individuals with Down Syndrome.

Down Syndrome Companies

More than five notable companies are currently engaged in developing therapies for Down Syndrome. Among these, AC Immune SA stands out with its drug candidate in the most advanced stage of development-Phase II clinical trials.

DelveInsight's report covers around 75+ 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

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

Intravenous
Subcutaneous
Oral
Intramuscular

Down Syndrome Products have been categorized under various Molecule types such as

Monoclonal antibody
Small molecule
Peptide

Download Sample Pages to Get an in-depth Assessment of the Emerging Down Syndrome Therapies and Key Companies: Down Syndrome Clinical Trials and advancements https://www.delveinsight.com/report-store/down-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Down Syndrome Pipeline Therapeutic Assessment
• Down Syndrome Assessment by Product Type
• Down Syndrome By Stage
• Down Syndrome Assessment by Route of Administration
• Down Syndrome Assessment by Molecule Type

Download Down Syndrome Sample report to know in detail about the Down Syndrome treatment market @ Down Syndrome Therapeutic Assessment https://www.delveinsight.com/sample-request/down-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Table of Content

1. Report Introduction
2. Executive Summary
3. Down Syndrome Current Treatment Patterns
4. Down Syndrome - DelveInsight's Analytical Perspective
5. Therapeutic Assessment
6. Down Syndrome Late-Stage Products (Phase-III)
7. Down Syndrome Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Down Syndrome Discontinued Products
13. Down Syndrome Product Profiles
14. Down Syndrome Key Companies
15. Down Syndrome Key Products
16. Dormant and Discontinued Products
17. Down Syndrome Unmet Needs
18. Down Syndrome Future Perspectives
19. Down Syndrome Analyst Review
20. Appendix
21. Report Methodology

Request the Sample PDF to Get Detailed Insights About the Down Syndrome Pipeline Reports Offerings https://www.delveinsight.com/report-store/down-syndrome-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Contact Info:
Kritika Rehani
krehani@delveinsight.com
info@delveinsight.com
+14699457679

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

This release was published on openPR.