Expanding the drug discovery pipeline to increase effective therapies for ALS.
Most ALS cases (90%) are “sporadic,” meaning they have no clear cause. Despite years of research, most treatments target the effects of the disease, not what starts it. That may be why many have failed or had limited impact. A new wave of therapies is aiming to act earlier in the disease process. Target ALS is helping lead this shift by funding cutting-edge research through its Industry-Led Collaborative Consortia, with many programs now moving into clinical trials.
Read about each project below:
Targeting the Root: Advancing ALS Therapies and Biomarkers Through Precision Science
QurAlis and partners are pioneering a new wave of ALS research,combining targeted therapies like QRL-101 with innovative biomarker discovery to address the disease at its core.
Denali Therapeutics: Learning from the DNL343 Trial in ALS
Denali Therapeutics shared top-line results from their DNL343 trial (Regimen G) in the Healey ALS Platform Trial at the Target ALS 2025 Annual Meeting, which took place in Boston in early May.
Splice Correction of UNC13A: A New Frontier in ALS Treatment
A multi-institutional team from Trace Therapeutics, Stanford, University of Michigan, and FMP Berlin is developing a promising and exciting new therapy that corrects RNA splicing errors in the UNC13A gene; a mistake seen in most ALS cases and linked to faster disease progression.
Unlocking RNA Granules: A Promising Therapeutic Pathway in ALS
A groundbreaking collaboration between Denali Therapeutics and researchers at UC San Diego (UCSD) is advancing a bold new strategy to treat ALS by targeting a previously overlooked player: the RNA granule protein OTUD4.
T Cells and ALS: Clues from the Immune System
Could the immune system hold the key to unlocking new treatments for ALS? Two innovative research projects are challenging long-held assumptions by exploring how T cells, the immune system’s frontline responders, behave in ALS.
Beyond “Junk” DNA: Targeting Transposable Elements in ALS
A collaborative consortium led by NeuroDex that includes scientists from Twilight Neuro, BrainEver Pharma, and NINDS is analyzing NDEs to detect HERV-K and LINE1 RNA, both implicated in ALS pathology. Early findings show that these elements are not only elevated in a subset of ALS patients, but also strongly correlate with TDP-43 protein mislocalization, the central hallmark of ALS. This makes them promising biomarkers; potential tools to identify who is most at risk, and who might benefit from therapies that suppress transposable element activity.
Autophagy and Metabolism: Two Sides of ALS Cellular Breakdown
At the intersection of energy failure and impaired waste clearance, two Target ALS Springboard Fellows, Dr. Yang Liu (Johns Hopkins University) and Dr. Juliet Goldsmith (Thomas Jefferson University), are uncovering how disruptions in metabolism and autophagy; the cell’s “cleanup and recycling” system, interact to drive neurodegeneration in ALS.
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