(MENAFN- GlobeNewsWire - Nasdaq) Burlingame, CA, Dec. 03, 2025 (GLOBE NEWSWIRE) -- The Global Tay-Sachs Disease Market is estimated to be valued at USD 2.64 Bn in 2025 and is expected to reach USD 4.08 Bn by 2032, exhibiting a compound annual growth rate (CAGR) of 6.4% from 2025 to 2032. This growth is propelled by continuous advancements in diagnostic technologies, rising awareness of genetic disorders, and strengthened government initiatives supporting rare disease research and treatment. Additionally, a growing pipeline of targeted therapies is further accelerating market expansion throughout the forecast period. Request Sample Report:
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Global Tay-Sachs Disease Market Key Takeaways
Based on disease type, infantile Tay-Sachs disease segment is set to dominate the industry, accounting for 65.3% of the market share in 2025.
Gene therapy is expected to remain a highly sought-after treatment for Tay-Sachs disease, holding a market share of 55.3% by 2025
In terms of route of administration, oral segment is slated to account for more than half of the global Tay-Sachs disease market share in 2025.
North America, with an estimated share of 37.3% in 2025, is projected to retain its market dominance.
Asia Pacific is poised to emerge as the fastest-growing Tay-Sachs disease market, capturing a global share of 23.4% in 2025.
Increasing Awareness and Early Diagnosis Rates Fueling Market Growth
Coherent Market Insights' latest Tay-Sachs disease market analysis offers insights into major factors driving growth. Rising awareness about the disease and improvements in early diagnosis rates are among the primary factors contributing to market expansion.
Growing awareness among clinicians, patients, and advocacy groups of Tay Sachs and other lysosomal storage / GM2 gangliosidosis disorders is prompting more screening and diagnosis. This, in turn, is expected to boost growth of the Tay-Sachs Disease market during the forecast period.
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High Treatment Costs Limiting Tay-Sachs Disease Market Growth
The global Tay-Sachs disease market outlook remains positive, owing to increasing awareness and diagnostics for rare genetic disorders. However, absence of widely approved curative treatments and high cost of emerging experimental therapies might limit market growth to some extent during the forthcoming period.
Rare‐disease therapies, including genetic therapies and enzyme replacement, are quite expensive. This limits their accessibility across low- and middle-income regions, thereby slowing down Tay-Sachs disease market growth.
Therapeutic Innovations Creating Lucrative Growth Avenues
Therapeutic innovations are opening promising avenues in Tay‐Sachs disease. For example, gene therapy (including gene editing) and substrate reduction therapy are under investigation, and enzyme replacement approaches have been attempted. Although no approved disease‐modifying therapy yet exists, these efforts are expected to create growth opportunities for biotech companies focused on rare neurodegenerative disorders.
Emerging Tay-Sachs Disease Market Trends
Leading companies and research institutions are intensifying efforts to explore effective treatments for rare genetic disorders like Tay-Sachs. For example, Scientists at the National Institutes of Health (NIH) recently used gene‐editing in human cell models and mice to correct the defective gene in late‐onset Tay‐Sachs disease. These advances will likely support market expansion during the forecast period.
Governments and health authorities are increasingly implementing initiatives and programs to address the burden of rare diseases. For instance, in May 2025, WHO's 78th World Health Assembly adopted a resolution recognizing rare diseases as a global health priority. The resolution asks WHO to develop a 10‐year global action plan to improve diagnosis, access to treatment, and integrated care for people living with rare diseases.
Surge in genetic screening and carrier testing programs is expected to drive growth in the Tay-Sachs disease market during the assessment period. Expanded newborn, preconception, and family screenings enable earlier diagnosis and identification of more patients. Advances in cost-effective next-generation sequencing (NGS) technologies are further driving market expansion by allowing faster and more comprehensive detection of Tay-Sachs mutations.
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Analyst's View
“The global Tay-Sachs disease market is expanding steadily, owing to increasing awareness and early diagnosis rates, therapeutic innovation, especially gene- and enzyme-based treatments, expanding screening programs, and growing support from governments and rare disease advocacy organizations,” said a senior analyst at CMI.
Current Events and Their Impact on the Tay-Sachs Disease Market
| Event | Description and Impact |
| New Drug Research | Description: Researchers at McMaster University discovered that an existing FDA‐approved drug called 4‐phenylbutyric acid (4‐PBA) showed improved motor function and longer lifespan in mouse models of both Tay‐Sachs disease and Sandhoff disease. Impact: This could speed up treatment development and attract investment in therapies. |
| Gene Therapy Trials | Description: A Phase I/II clinical trial by UMass Chan Medical School delivered a dual‐vector gene therapy for GM2‐gangliosidosis (which includes Tay‐Sachs) and saw increased enzyme activity, safety in patients, fewer/severity of seizures, and longer oral feeding. Impact: This increases hope for effective treatments and boosts R&D interest in the market. |
| Rare Disease Initiatives | Description: Many nations are launching new rare‐disease diagnosis and care programmes. Impact: This could create growth opportunities for the Tay‐Sachs disease market during the forecast period. |
Competitor Insights
Key companies in the Tay-Sachs disease market report:
IntraBio Inc. Sanofi Forge Biologics Taysha Gene Therapies Recursion Pharmaceuticals, Inc. Sio Gene Therapies JCR Pharmaceuticals Co., Ltd.
Key Developments
In August 2025, JCR Pharmaceuticals and MEDIPAL Holdings signed a global licensing and Japan co-development agreement for JR‐479, a new therapy for GM2 gangliosidosis. Under the deal, JCR will lead development and commercialisation in Japan, while MEDIPAL holds rights and will develop and commercialise the drug outside Japan.
In June 2023, Forge Biologics and New Hope Research Foundation announced cGMP manufacturing partnership to advance gene therapy for patients with GM2 gangliosidosis, including Tay-Sachs Disease. The collaboration will support process development and production of the AAV-based gene therapy to accelerate its clinical development.
Market Segmentation
Disease Type Insights (Revenue, USD Bn, 2020 - 2032)
Infantile Tay-Sachs Disease Juvenile Tay-Sachs Disease Late-Onset Tay-Sachs Disease
Treatment Type Insights (Revenue, USD Bn, 2020 - 2032)
Gene Therapy N-Acetyl-L-Leucine Venglustat (Substrate Reduction Therapy) Other Pipeline Drugs/Therapy
Route of Administration Insights (Revenue, USD Bn, 2020 - 2032)
End User Insights (Revenue, USD Bn, 2020 - 2032)
Hospitals and Specialty Neurology Clinics Diagnostic Laboratories and Genetic Centers Research and Academic Institutes Homecare Settings Patient Support Organizations
Distribution Channel Insights (Revenue, USD Bn, 2020 - 2032)
Hospital Pharmacies Retail Pharmacies Online Pharmacies
Regional Insights (Revenue, USD Bn, 2020 - 2032)
North America Latin America Brazil Argentina Mexico Rest of Latin America
Europe Germany U.K. Spain France Italy Russia Rest of Europe
Asia Pacific China India Japan Australia South Korea ASEAN Rest of Asia Pacific
Middle East GCC Countries Israel Rest of Middle East
Africa South Africa North Africa Central Africa
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Gene Therapy for Rare Disease Market Outlook for 2025-2032
Gene Therapy Market Size, Share, Trends & Opportunities for 2025-2032
Rare Disease Drugs Market Analysis & Forecast for 2025-2032
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