GM2 Gangliosidoses – Market outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2020 To 2030

GM2 gangliosidoses are three related genetic disorders due to a deficiency of the beta-hexosaminidase enzyme, which catalyses the biodegradation of fatty acid derivatives known as gangliosides in lysosomes. Tay-Sachs disease is the most common and involves a defect in the HEXA gene for beta-hexosaminidase A (in particular, the alpha subunit).

·       Thelansis captures epidemiology on incidence, prevalence, diagnosed, and treated prevalence for all forms of GM2 such as Tay-Sachs disease, Sandhoff disease, and AB variant.

·       The prevalence of Tay-Sachs disease is The prevalence of infection is 1 case per 320 000 live births. The AB variant is scarce; only a few cases have been reported worldwide. Based on a few sources, the incidence of Sandhoff disease is approximately 1 out of every 180,000 live births worldwide.

·       A Phase II drug candidate leads pipeline sensation for GM-2 gangliosidosis; IB-1001 from IntraBio Inc. Apart from this Phase II therapy, the other clinical drug candidate is a viral gene therapy; AXO-AAV-GM2, which is under Phase I stage of development by Axovant Gene Therapies Ltd. A treatment aimed at inhibiting gangliosides synthesis (Miglustat) is also being investigated for the slowly progressive forms by institutional investigators.

·       In pre-clinical stages, therapies like CAM-GM201 (Beta-hexosaminidase) from Sanofi, a Glucosylceramide Synthase from J&J, and a gene therapy program from Gene Therapy Research Institution Co., Ltd. are under evaluation.

 

The competitive landscape of GM2 Gangliosidoses includes country-specific approved and pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are tracked and supplemented with analyst commentary.

KOLs insights of GM2 Gangliosidoses across 8 MM market from the centre of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

GM2 Gangliosidoses Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard) which Data Inputs with sourcing, Market Event, and Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Read more: GM2 Gangliosidoses – Market outlook, Epidemiology, Market Forecast, and Competitive Landscape Report – 2020 To 2030

S. No    Asset               Company                                 Stage

1          TSHA-101         Taysha Gene Therapies Phase 2

2          venglustat GZ402671    Genzyme          Phase 3

3          Zavesca (Miglustat)       Actelion Phase 3

4          AXO-AAV-GM2 Sio Gene Therapies       Phase 1

 

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