Global Tay-Sachs Disease Treatment Market - 2022-2029

Global Tay-Sachs Disease Treatment Market - 2022-2029

Market Overview

The global tay-sachs disease treatment market size was valued at US$ XX million in 2021 and is estimated to reach US$ XX million by 2029, growing at a CAGR of XX % during the forecast period (2022-2029).

Tay-Sachs disease is a rare genetic disorder passed down from one generation to the next. It's caused by a lack of an enzyme that aids in the digestion of fatty substances. These fatty substances, known as gangliosides, accumulate to toxic levels in the brain and spinal cord, impairing nerve cell function.

Market Dynamics

The factors influencing the global tay-sachs disease treatment market are the increasing research and development and increasing prevalence of the tay-sachs disease.

The increasing research and development is expected to drive the market growth in the forecast period

There is a need for significant research into therapeutic techniques that could be useful in treating Tay-Sachs disease because there is currently no effective treatment. At this time, enzyme replacement therapy (ERT), the Jacob sheep model, substrate reduction therapy, and increasing the activity of -hexosaminidase A is among the most promising research areas. Tay-Sachs disease and other lysosomal storage disorders have been considered for treatment with ERT. The goal of ERT is to create a replacement for the HEXA enzyme that can take the place of the diseased person's non-functional enzyme. However, this process is hampered by the enzyme's physical size, which appears to be too large to pass through the blood-brain barrier (BBB) as required.

To get around this problem, some research has looked into injecting the HEXA enzyme directly into the cerebrospinal fluid (CSF). However, even when the enzyme is administered directly to the central nervous system, current research has not been able to demonstrate successful uptake (CNS). Another treatment option for Tay-Sachs disease is to increase the activity of the HEXA enzyme, which would reduce the severity of the deficiency that characterizes the disease. Patients with infantile Tay-Sachs disease, on the other hand, usually lack the enzyme completely and would not benefit from an increase in the spectrum of activity. Treatments such as gene therapy, stem cell transplantation, and enzyme replacement therapy are being researched to find a cure or slow the progression of Tay-Sachs disease.

Limitations of tay-sachs disease treatment are expected to hamper the market growth

Tay-Sachs disease has no known cure, and no treatments have been proven to slow the disease's progression. Some treatments can help with symptom management and complication prevention. Treatment aims to alleviate some symptoms, manage infections, prevent complications, and improve the overall quality of life. Anticonvulsants for seizures in children and antipsychotic medications for psychiatric disorders in adults may be used to treat symptoms. Tricyclic antidepressants, in particular, are thought to be ineffective, and they may inhibit the small amount of enzyme activity that some people with the disease have.

COVID-19 Impact Analysis

The COVID-19 pandemic forced several countries to restructure their healthcare systems. There was a significant underdiagnosis of diseases during the COVID-19 pandemic. According to a WHO survey, noncommunicable disease (NCD) prevention and treatment services have been severely disrupted since the COVID-19 pandemic. The survey, conducted over three weeks in May and included 155 countries, confirmed global impact but low-income countries are the most affected. People with NCDs are at a higher risk of severe COVID-19-related illness and death, so this situation is extremely concerning. Hence, tay-sachs disease, a rare neurodegenerative disorder, is also expected to be impacted by covid-19.

Segment Analysis

Infantile segment is expected to dominate the market growth

The Infantile form of Tay-Sachs disease is the most common, beginning with reduced vision and an exaggerated startle response at around 6 months of age and progressing to a gradual loss of skills and seizures by age 2 and early death usually by the age of 5. Tay-Sachs disease in children is characterized by a complete or nearly complete lack of hexosaminidase. The activity of an enzyme. The disorder frequently worsens quickly, causing significant cognitive and physical impairment. At birth, infants may appear to be unaffected. Mild muscle weakness, twitching or jerking of muscles, and exaggerated startle response, such as a sudden or unexpected noise, are common early symptoms between 3 and 6 months. The startle response may be due to an increased sensitivity to sound (acoustic hypersensitivity).

The appearance of a macular cherry red spot is a common symptom of Tay-Sachs disease. The abnormal accumulation of pale undigested material in the macula contrasts with the thin foveal transparent background exposing the normal rich choroidal vasculature, resulting in this ophthalmological finding. Approximately 90% of people with infantile Tay-Sachs disease have this symptom. More serious complications, such as seizures, difficulty swallowing, vision loss, paralysis, and progressive hearing loss, may develop as affected infants grow older. Confusion, disorientation, or deterioration of intellectual abilities are additional cognitive deficits. Infants may eventually become unresponsive to their surroundings and environment. Life-threatening complications, such as aspiration pneumonia leading to respiratory failure, appear between three and five.

Geographical Analysis

North America region is expected to hold the largest market share in the global tay-sachs disease treatment market

The growing prevalence of tay-sachs disease and better healthcare facilities is expected to drive the market growth.

In the United States, one in every 27 Jews carries the Tay-Sachs disease gene. Tay-Sachs is also more common among non-Jewish French Canadians who live near the St. Lawrence River or Louisiana's Cajun community. Around one in every 250 people in the general population is a carrier. Moreover, on 9th November 2020, the FDA lifted its clinical hold on Axovant Gene Therapies' Investigational New Drug (IND) application to begin a registrational study of gene therapy to treat patients with Tay-Sachs disease and Sandhoff disease, according to the company. The Axovant therapy, AXO-AAV-GM2, is the first investigational gene therapy for Tay-Sachs and Sandhoff to receive IND clearance.

Competitive Landscape

The global tay-sachs disease treatment market is moderately competitive with mergers, acquisitions, and product launches. Some of the key players in the market are Recursion Pharmaceuticals, Inc., IntraBioInc, Sio Gene Therapies, Zacharon Pharmaceuticals Inc.

IntraBioInc

Overview: IntraBio is a biopharmaceutical company with a late-stage drug pipeline focused on genetic and neurodegenerative diseases. The company's management team and business consultants have extensive commercial and regulatory experience in drug product development, from small molecule manufacturing to clinical trials and regulatory approvals in the United States and Europe.

Product Portfolio: IB1001-202 (NCT03759665) is a multinational clinical trial testing IB1001 as a symptomatic and neuroprotective disease-modifying treatment for adults and children with GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease). Trial sites in the United States, the United Kingdom, and the European Union enrolled patients aged 6 and up.

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