How Target ALS is fueling breakthroughs in research through data discovery

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Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s Disease, is a progressive and fatal neurodegenerative disease that impacts the brain and spinal cord, resulting in a loss of muscle control. There is currently no cure for ALS and only a limited number of therapeutics available on the market to slow the progression. While there is a mutated gene associated with development of this disease, about 90% of ALS cases occur without any known family history or genetic cause. 

Target ALS is a medical research foundation breaking down barriers to accelerate research for ALS through their Innovation Ecosystem model. This landmark initiative has fostered unprecedented collaborations between academia, pharma, and biotech, lowered barriers to access for critical research tools, and become a hub of communication and networking for the worldwide scientific community. 


Target ALS needed a software solution to support controlled, external access of well-characterized collections of postmortem tissue, longitudinal biofluids, and stem cell data. They wanted a comprehensive solution that could support access to dynamic, cloud-based data holdings, as well as enable multi-omics analysis by data consumers. Target ALS also needed an intuitive user interface to help increase the adoption of the solution by their network of global researchers.


Target ALS leveraged Omics AI to facilitate the connection, sharing, and analysis of genomics, histopathology, spatial transcriptomics, and other metadata through a custom-branded data platform. The solution uses Publisher to connect data and Explorer to share it to their research community. The platform was designed so that researchers can easily request access to the data collections through a data access request mechanism managed by Target ALS. Bioinformatics services were engaged to support metadata and bioinformatics data curation efforts, and to help guide the data connection process. Engineering services were used to develop custom platform features specific to the needs of the ALS research community.

As a result of this partnership, Target ALS was able to improve the accessibility of their comprehensive datasets, and increase the analytical power of their research community. This collaboration has made one the most high-quality human postmortem tissue datasets easily discoverable, accessible, and analyzable to thousands of ALS researchers across industry, academia, and the public sector. Through the use of Omics AI, researchers can now more easily perform multi-omic landscaping to:

  • Characterize heterogeneity in ALS
  • Study genotype-gene expression relationships
  • Study genotype-phenotype relationships
  • Study genomic-histopathological relationships
  • Identify novel causal mutations
  • Identify new therapeutic targets or biomarkers for sporadic and familial forms of ALS.


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Target ALS’ innovative model provides researchers with holistic support to accelerate their ideas toward the clinic. Access to comprehensive data through this Omics AI-powered platform will enable greater understanding of ALS and fully realize the potential impact of emerging technologies like artificial intelligence and machine learning on therapeutic development.
Manish Raisinghani
M.B.B.S., Ph.D., President and CEO of Target ALS

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