New York City – Target ALS is pleased to announce that it has awarded target discovery and development funding to six research consortia that are working toward the development of ground-breaking therapeutic approaches to treating ALS.

In 2016 – 2017, Target ALS provided multi-year grants to a number of unique research consortia to encourage ALS research ideation and active involvement from both the pharma/biotech industry as well as the most talented new investigators in the field. In a historic first, either a pharma/biotech researcher or a new investigator led each team. These teams were awarded funding for a range of novel projects from early stage target discovery to the development of new therapeutic approaches.

A wave of creative thinking and collaboration followed. Indeed, many projects have led to the development of new, ground-breaking therapeutic approaches while others are advancing our understanding of pathophysiology of ALS.  In response to this growing momentum, the Target ALS Independent Review Committee (IRC) granted six of the most promising collaborative projects an additional year of funding.

The Awardees

  • Targeting Stress Granule Dynamics for Familial and Sporadic ALS: Joe Lewcock (Denali Therapeutics, Project Leader), Gene Yeo (University of California, San Diego), Steve Finkbeiner (Gladstone Institutes).
  • Developing human-derived antibodies to target dipeptide-repeat protein toxicity in C9ORF72 disease: Fabio Montrasio (Neurimmune, Project Leader) Laura Ranum (University of Florida), Magdalini Polymenidou (University of Zurich), Clotilde Lagier-Tourenne (Massachusetts General Hospital).
  • Nuclear Export Inhibitor KPT-350 for C9orf72 and Sporadic ALS: Alex McCampbell (Biogen, Co-Project Leader), Jeff Rothstein (Johns Hopkins University, Co-Project Leader), Thomas Lloyd (Johns Hopkins University), Laura Ranum (University of Florida).
  • Exploiting yeast to discover small-molecule drugs for ALS caused by aberrant TDP-43, FUS, and c9orf72 dipeptide repeat protein homeostasis: Dean Brown & Nick Brandon (AstraZeneca, Co-Project Leaders), Jim Shorter (University of Pennsylvania), Steve Finkbeiner (Gladstone Institutes), Aaron Gitler (Stanford University).
  • Identification of RAN Translation Regulators as Therapeutic Targets in C9ORF72-Related ALS: Shuying Sun (Johns Hopkins University, Project Leader), Michael Bassik (Stanford University), Aaron Gitler (Stanford University), Fen-Biao Gao (University of Massachusetts), Joel Richter (University of Massachusetts).
  • Spatially resolved transcriptome wide analysis of gene expression in the spinal cord in ALS: Hemali Phatnani (New York Genome Center), Joakim Lundeberg (KTH Royal Institute), Richard Bonneau (New York University).

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