Science on Target: Q3 Research We Fund

$2.4 million Invested in Biomarker Innovation

ALS is a heterogeneous disease with distinct subpopulations of cases clustering together based on common underlying biology and with distinct differences in the clinical time course and manifestation of disease. Biomarkers are therefore critical for enabling precision medicine to deliver the right therapy to the right individual at the right time. While neurofilament assays represent an important breakthrough, aiding clinical evaluation of novel therapies, additional biomarkers are sorely needed.

Target ALS is poised to support consortia studying biomarkers of disease stratification and progression. As new therapeutics have begun moving the needle for people with ALS, biomarkers are needed to stratify ALS cases, detect response to therapeutics, and predict and track disease progression. We solicited letters of intent (LOIs)  from multidisciplinary groups focused on the discovery and development of novel fluid or tissue-based biomarkers of disease progression and stratification using novel, innovative, and complementary approaches, including applications of artificial intelligence (AI) and machine learning (ML). We are excited to announce the recipients of these awards for a total of $2.4 million dedicated to addressing this area of high unmet need. 

Pietro Fratta (University College London), Michael Ward (University of California, Berkeley), David Walt (Harvard), Sanjay Chandriani (Trace Neuroscience). “Circular RNA Biomarkers of TDP-43 Dysfunction in ALS”.   This team recently discovered a new class of molecules, called cryptic circular RNAs (c-circRNAs), that are produced specifically when TDP-43 function is lost. These molecules have been detected in ALS and frontotemporal dementia (FTD) brain tissue but not in healthy individuals. Importantly, circular RNAs are highly stable and may be measurable in blood, cerebrospinal fluid, and extracellular vesicles making them attractive candidates for minimally invasive biomarkers. This international consortium combines expertise in RNA biology, ultrasensitive molecular detection, and therapeutic development. Together, they will develop and compare multiple technologies to detect c-circRNAs to establish a new biomarker platform that enables patient stratification, monitors disease progression, and provides direct measures of therapeutic target engagement, accelerating the development of effective treatments for ALS.

Paul Lingor (Technical University of Munich), Johannes Dorst (University of Ulm), Michael Menden (University of Melbourne), and Alexander Brandt (Eli Lilly), “Proteomic signatures for ALS stratification and progression across presymptomatic and symptomatic disease stages”. Using blood and spinal-fluid samples already collected from people across the full course of ALS, including healthy individuals who carry ALS-causing genes but have no symptoms yet, they will measure thousands of proteins and other molecules. With advanced data analysis and artificial intelligence, they will search for distinct biological subgroups of patients, track how these signals change as the disease progresses, and test whether they shift in response to treatment. Their goal is to develop a reliable blood test that can sort patients into the right groups, show whether a therapy is working, and flag when the disease is about to begin in gene carriers. Such tools could make future clinical trials smaller, faster and fairer, and bring effective, personalised ALS treatments closer for everyone affected.

Alexander Thompson (University of Oxford), Johnathan Cooper-Knock (University of Sheffield), Eran Hornstein (Weizmann Institute of Science), Carrie Rubel (Biogen).  “ Redefining progression and treatment response in ALS” Their consortium aims to apply machine learning to  clinical and proteomic data from pALS who have enrolled in interventional studies, including the VALOR study, to identify and corroborate novel signatures of treatment response.  They will use existing datasets generated at Universities in Oxford, Sheffield and Tel Aviv to discover these signatures. Working with Biogen, they will then test how well they perform using samples from a successful clinical trial for the genetic form of ALS caused by alterations in the SOD1 gene, and from the ongoing UK EXPERTS-ALS clinical trial. Their goal is to develop reliable protein signatures for use in ALS clinical trials globally. These biomarkers could help much more quickly determine whether a treatment is working, reducing the size and duration of trials, accelerating the development of effective treatments for people living with ALS.

Grants & Funding Opportunities

Upcoming Industry-Led Consortium Funding Call 

Target ALS is deeply committed to catalyzing the development of new therapies grounded in a strong biological rationale based on human disease.  Since 2016, Target ALS has sponsored a funding call which aims to bring together investigators from pharma/biotech and academia to work collaboratively on a drug discovery project, with several projects delivering molecules tested in clinical trials today. Stay tuned for our November announcement sharing the details of the next Industry-Led Consortium funding call.

Related News

Science on Target: Q3 Publication Spotlight

October 7, 2026

Brooke Eby | 1988-2026

October 2, 2026

What are biomarkers in ALS research?

September 16, 2026

TDP-43: What It Is and Why It Matters in ALS

September 16, 2026