Sandhoff Disease Therapeutics Market

Sandhoff Disease Therapeutics Market

Global Sandhoff Disease Therapeutics Market reached US$ YY million in 2023 and is expected to reach US$ YY million by 2031, growing at a CAGR of YY% during the forecast period 2024-2031.

Sandhoff disease is a rare, inherited disease that progressively destroys nerve cells in the brain and spinal cord. It occurs when fatty materials called lipids accumulate in brain cells (and in other parts of the body), forcing them to malfunction and die.

Sandhoff disease comprises a phenotypic continuum encompassing acute infantile, subacute juvenile, and late-onset disease. Although classification into these phenotypes is somewhat arbitrary, it is helpful in understanding the variation observed in the timing of disease onset, presenting manifestations, rate of progression, and life span.

Market Dynamics: Drivers & Restraints

Rise in the prevalence of sandhoff disease

The sandhoff disease therapeutics market is expanding due to the rise in prevalence of the condition, driven by the use of gene therapy, enzyme replacement therapy, and small molecule drugs. These treatments have shown promising results in preclinical trials, and if successful in later trials and regulatory approval, they could significantly impact the market.

For instance, in February 2021, Sio Gene Therapies stated the first patient to be dosed with AXO- AAV-GM2, which is a significant milestone for the company as well as for the field of gene therapy. The goal of the Phase 1/2 trial is to evaluate the safety and efficacy of AXO-AAV-GM2, which has the potential to restore lysosomal enzyme activity and potentially change the course of the disease.

Furthermore, the global sandhoff disease therapeutics is also driven by various other factors like regulatory support and expedited approvals, globalization of research and clinical trials and others helping the market to grow during the forecast period.

High cost of treatment

The market for advanced sandhoff disease therapeutics is expected to be significantly impacted by factors like limited availability and high costs, which may hinder growth due to hospitalization, intensive care, or specialized diagnostic tests. Furthermore, significant side effects and other factors also hamper the market growth.

Segment Analysis

The global sandhoff disease therapeutics market is segmented based on type, therapy type, end-user and region.

The gene therapy from the therapy type segment accounted for approximately 42.1% of the sandhoff disease therapeutics market share

The gene therapy from the therapy type segment accounted for approximately 42.1%. Gene therapy is being researched to potentially treat both Tay-Sachs and Sandhoff disease. Gene therapy aims to be a one-time treatment that could slow or stop disease progression by delivering working HEXA and HEXB genes into the cells using a viral vector. Scientists know that viruses are good at getting into cells, so they have learned how to safely use this ability as a carrier to deliver working genes. But don’t worry, the viral genes are removed, so only therapeutic (intended) genes are delivered.

For instance, in November 2020, Axovant Gene Therapies Ltd, a clinical-stage company developing innovative gene therapies, stated that the U.S. Food and Drug Administration (FDA) had lifted its clinical hold and cleared the Investigational New Drug (IND) Application to initiate a registrational study of AXO-AAV-GM2 gene therapy to treat patients with Tay-Sachs disease and sandhoff disease.

Geographical Analysis

North America is estimated to hold about 38.4% of the total market share throughout the forecast period

North America is estimated to hold about 38.4% of the total market share throughout the forecast period owing to the factors like rising prevalence of the condition in the region along with the high healthcare expenditure and a large geriatric population, the presence of a large number of pharmaceutical companies, which are investing heavily in the development of new, targeted treatments for the condition, and high level of healthcare expenditure leading to a large patient population are some of the factors responsible for the growth of the market in the region.

For instance, in April 2021, the USA reported on the first baby in a gene therapy clinical trial for Sandhoff, and Tay-Sachs diseases. The study, based on technology developed at and licensed from UMass Medical School, is an open-label, two-stage, clinical trial designed to evaluate safety and dose-escalation, and the safety and efficacy of surgical delivery of AXO-AAV-GM2 directly to the brain and spinal fluid of pediatric participants with both infantile and juvenile GM2 gangliosidosis.

Covid 19 Impact Analysis

COVID-19 had a substantial impact on the sandhoff disease therapeutics market where there was a significant reduction in the number of tests requested for the investigation of inborn errors of metabolism (IEM) during the COVID-19 pandemic, which could lead to underdiagnosis of IEM, including GM2 gangliosidosis (Sandhoff diseases). Also, individuals with sandhoff disease faced many challenges there was a disruption in receiving diagnosis and medical attention etc.

Market Segmentation

By Type
• Acute infantile Sandhoff disease
• Subacute juvenile Sandhoff disease
• Late-onset Sandhoff disease

By Therapy Type
• Gene Therapy
• Enzyme Replacement Therapy
• Stem Cell Therapy

By End User
• Hospitals
• Homecare
• Specialty Clinics
• Others

By Region
• North America
U.S.
Canada
Mexico
• Europe
Germany
UK
France
Italy
Spain
Rest of Europe
• South America
Brazil
Argentina
Rest of South America
• Asia-Pacific
China
India
Japan
Australia
Rest of Asia-Pacific
• Middle East and Africa

Competitive Landscape

The major global players in the market include Bioasis Technologies Inc., Nuo Therapeutics Inc., Sio Gene Therapies, Inc., Intrabio Ireland Limited, Sanofi, Roivant Sciences Ltd., Azafaros, Polaryx Therapeutics, Merck & Co., Inc., Intellia Therapeutics among others.

Key Developments

 In June 2023, Azafaros B.V. stated that the first patient had been enrolled on its Phase 2 RAINBOW study (NCT05758922). The clinical trial is being conducted in Brazil and the US and will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics across two doses of its lead asset, AZ-3102, in patients with GM2 gangliosidosis and Niemann-Pick disease type C (NP-C). In 2022, the compound received Fast Track Designation for GM1 and GM2 gangliosidoses as well as NP-C and Orphan Drug Designations (ODD) for GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NP-C from the FDA.

Why Purchase the Report?
• To visualize the global sandhoff disease therapeutics market segmentation based on type, therapy type, end user and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analysing trends and co-development.
• Excel data sheet with numerous data points of sandhoff disease therapeutics market level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global sandhoff disease therapeutics market report would provide approximately 64 tables, 61 figures and 186 pages.

Target Audience 2023
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies