Tay-Sachs Disease Market Outlook 2025–2032: Strong CAGR of 6.4% with USD 4.08 Billion Valuation

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: https://www.coherentmarketinsights.com/insight/request-sample/8609

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.

Immediate Delivery Available | Buy This Premium Research Report: https://www.coherentmarketinsights.com/insight/buy-now/8609

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.

Request For Customization: https://www.coherentmarketinsights.com/insight/request-customization/8609

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)

Oral
Parenteral
Others

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
- U.S.
- Canada
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

Our Trusted Partners:
Worldwide Market Reports, Coherent MI, Stratagem Market Insights
Get Recent News:
https://www.coherentmarketinsights.com/news

About Us:

Coherent Market Insights leads into data and analytics, audience measurement, consumer behaviors, and market trend analysis. From shorter dispatch to in-depth insights, CMI has exceled in offering research, analytics, and consumer-focused shifts for nearly a decade. With cutting-edge syndicated tools and custom-made research services, we empower businesses to move in the direction of growth. We are multifunctional in our work scope and have 450+ seasoned consultants, analysts, and researchers across 26+ industries spread out in 32+ countries.

Contact Us:

Mr. Shah
Coherent Market Insights
533 Airport Boulevard,
Suite 400, Burlingame,
CA 94010, United States
US: + 12524771362
UK: +442039578553
AUS: +61-8-7924-7805
India: +91-848-285-0837
Email: sales@coherentmarketinsights.com
Website: https://www.coherentmarketinsights.com
For Latest Update Follow Us:
LinkedIn | Facebook | Twitter

Legal Disclaimer:

EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.