According to DelveInsight’s analysis, the Down Syndrome pipeline features over five key companies actively developing more than five therapeutic candidates aimed at treating Down Syndrome.
Down Syndrome Overview:
Down Syndrome (DS) is a genetic disorder caused by the presence of an extra copy of chromosome 21, a condition known as trisomy 21. This additional genetic material disrupts typical developmental processes, leading to distinctive physical characteristics such as a flattened facial profile, upward-slanting eyes, and a single deep crease on the palm. Individuals with Down Syndrome often experience intellectual disabilities and developmental delays, though the severity varies widely. The condition is also linked to several health issues, including congenital heart defects, respiratory complications, and increased susceptibility to infections.
Common signs and symptoms include characteristic facial features, delayed physical and cognitive development, and varying levels of intellectual impairment. Medical complications may include heart abnormalities, breathing and digestive issues, and immune system weaknesses, which heighten vulnerability to illness.
At the biological level, Down Syndrome arises from the overexpression of genes on the extra chromosome 21, leading to imbalances in multiple genetic and molecular pathways. These disruptions affect the development and function of various organ systems. In the brain, they alter neuronal structure, connectivity, and signaling, contributing to learning and memory difficulties. The extra chromosome also impacts the cardiovascular, immune, and endocrine systems, leading to conditions such as congenital heart disease, hypothyroidism, and immune dysfunction. Collectively, these genetic and molecular disturbances explain the broad spectrum of symptoms and health challenges observed in individuals with Down Syndrome.
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"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
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DelveInsight’s Down Syndrome pipeline report highlights a growing and dynamic landscape, with over five active companies developing five or more investigational therapies aimed at improving treatment for Down Syndrome.
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In April 2025, the first participant with Down Syndrome was dosed in the HERO trial investigating ION269, an experimental therapy designed to lower amyloid precursor protein (APP) levels and prevent plaque buildup—an important step toward early Alzheimer’s prevention in individuals genetically predisposed to the condition by around age 40.
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In March 2025, Johns Hopkins announced a remarkable case in which an 11-year-old patient with Down Syndrome experienced a complete resolution of obstructive sleep apnea (OSA) and significant improvements in behavior and sleep quality following the implantation of a novel device.
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Key players in this space include ACI-24, Connecta Therapeutics, and other emerging biopharmaceutical companies actively advancing therapeutic innovation. Among the most promising pipeline candidates currently in development are ACI-24 and several additional investigational therapies targeting various aspects of Down Syndrome pathology.
Down Syndrome Pipeline Analysis
The report provides insights into:
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The report provides detailed insights into the key companies that are developing therapies in the Down Syndrome Market.
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The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Down Syndrome treatment.
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It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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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.
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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Down Syndrome market.
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Down Syndrome Emerging Drugs
ACI-24 – AC Immune SA
ACI-24 is an anti-amyloid beta (Aβ) active immunotherapy developed by AC Immune SA, designed to stimulate the body’s immune system to produce antibodies that selectively recognize and eliminate misfolded Aβ proteins. This therapeutic strategy aims to prevent the accumulation of toxic Aβ aggregates and enhance the clearance of existing amyloid plaques. The liposome-based vaccine incorporates the first 15 amino acids of the human Aβ peptide arranged in a stacked β-sheet conformation on the liposome surface, effectively inducing the generation of polyclonal antibodies targeting both soluble and insoluble Aβ aggregates, including oligomers—the most neurotoxic form associated with Alzheimer’s disease and Down Syndrome.
Currently, ACI-24 is in Phase II clinical trials, evaluating its safety and efficacy in individuals with Down Syndrome.
Down Syndrome Companies
Over five key biopharmaceutical companies are actively developing therapies for Down Syndrome. Among them, AC Immune SA is leading the field, with its candidate ACI-24 being the most advanced, currently in Phase II clinical development.
DelveInsight’s report covers around 75+ products under different phases of clinical development like
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Late stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I) along with the details of
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Pre-clinical and Discovery stage candidates
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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
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Intravenous
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Subcutaneous
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Oral
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Intramuscular
Down Syndrome Products have been categorized under various Molecule types such as
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Monoclonal antibody
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Small molecule
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Peptide
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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
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
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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.
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