Sandhoff Disease Drug Research Report 2022: Comprehensive Insights About 5+ Companies and 5+ Pipeline Drugs Featuring IntraBio, Sanofi, Polaryx Therapeutics, & Azafaros –

Sandhoff Disease Drug Research Report 2022: Comprehensive Insights About 5+ Companies and 5+ Pipeline Drugs Featuring IntraBio, Sanofi, Polaryx Therapeutics, & Azafaros –

Sandhoff Disease Drug Research Report 2022: Comprehensive Insights About 5+ Companies and 5+ Pipeline Drugs Featuring IntraBio, Sanofi, Polaryx Therapeutics, & Azafaros –

DUBLIN–(BUSINESS WIRE)–The “Sandhoff disease – Pipeline Insight, 2022” drug pipelines has been added to’s offering.

The “Sandhoff disease – Pipeline Insight, 2022,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Sandhoff disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

The assessment part of the report embraces, in depth Sandhoff disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Sandhoff disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Sandhoff disease R&D. The therapies under development are focused on novel approaches to treat/improve Sandhoff disease.

Sandhoff disease Emerging Drugs Chapters

This segment of the Sandhoff disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Sandhoff disease Emerging Drugs

IB1001: IntraBio

IB1001, N-acetyl-L-leucine, is an orally administered modified amino acid. In vivo studies have identified N-acetyl-L-leucine to be the active isomer of N-acetyl-DL-leucine and can restore neuronal function and protect against/delay disease progression in multiple neurological circuits of the brain.

The mechanism of N-acetyl-L-leucine is known to be multi-modal, including altered glucose and antioxidant metabolism, reduced lysosomal storage, and the reduction of neuroinflammation in the cerebellum, leading to the attenuation of cell death. The company is conducting a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).

Venglustat: Sanofi

Venglustat is a novel, oral investigational therapy that has the potential to slow the progression of certain diseases by inhibiting abnormal glycosphingolipid (GSL) accumulation. GSLs are cellular building blocks whose abnormal accumulation is implicated in several rare diseases, responsible for both cell dysfunction and disease progression. Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of Sandhoff Disease.

Sandhoff disease: Therapeutic Assessment

This segment of the report provides insights about the different Sandhoff disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Sandhoff disease

There are approx. 5+ key companies which are developing the therapies for Sandhoff disease. The companies which have their Sandhoff disease drug candidates in the most advanced stage, i.e. phase III include, Sanofi.


The report covers around 5+ products under different phases of clinical development like

  • Late stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration

Products have been categorized under various ROAs such as

  • Oral
  • Parenteral
  • intravenous
  • Subcutaneous
  • Topical.
  • Molecule Type

Products have been categorized under various Molecule types such as

  • Monoclonal Antibody
  • Peptides
  • Polymer
  • Small molecule
  • Gene therapy
  • Product Type

Sandhoff disease Report Insights

  • Sandhoff disease Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

Sandhoff disease Report Assessment

  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs

Key Players

  • IntraBio
  • Sanofi
  • Polaryx Therapeutics
  • Azafaros

Key Products

  • IB1001
  • Venglustat
  • PLX 300
  • PLX-200
  • AZ 3102

For more information about this drug pipelines report visit


Laura Wood, Senior Press Manager

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