Science on Target: Q2 Grants & Funding Opportunities Update
July 9, 2026 Amy Easton
Smarter Grants, Greater Impact: Target ALS Moves to SmartSimple
Target ALS has recently transitioned from Blackbaud to SmartSimple as its grants management platform, marking a significant step forward in how we support and engage with our research community. SmartSimple’s robust, purpose-built grants management capabilities offer a more streamlined and intuitive experience for both applicants and administrators, enabling greater transparency throughout the grant lifecycle, more flexible reporting, and improved tracking of research milestones and outcomes. This upgrade reflects our ongoing commitment to operational excellence and to providing our funded researchers with the tools and support they need to focus on what matters most — accelerating the discovery of treatments for ALS. If you’ve applied for or have received a Target ALS grant in the past, all of your application and reporting information can be found in SmartSimple.
Basic Biology Consortia Awards Announced in May
Target ALS is excited to award new grants totaling $2.75M to 6 consortia to evaluate the basic biology and underlying disease mechanisms of ALS. This set of proposals is notable for their incorporation of innovative methods including AI/ML algorithms to further elucidate ALS disease pathobiology.
Dr. Tayana Shelkovnikova (Sheffield) leads a consortium that includes Drs. Antonio Calabrese (Leeds), Gareth Wright (Essex), and Vera Wiersma (Amsterdam University Medical College), and will investigate mechanisms underlying TDP-43 oligomerization. Shelkovnikova et al. will utilize state-of-the-art mass photometry and spectrometry methods, innovative proximity ligation assays and novel photo-crosslinkers, and confocal nanoscanning to characterize TDP-43 oligomerization state and the interplay between RNA and DPR binding. Finally, Shelkovnikova et al. will target TDP-43 mis-oligomerization using novel therapeutic candidates.
Dr. Pietro Fratta (University College London) and consortium members, Drs. Sarosh Irani (Mayo Clinic), Leonard Petrucelli (University of Miami), and Jenny Jiang (University of Pennsylvania), will work to characterize how TDP-43 pathology drives peripheral immune response and migration into the central nervous system. Dr. Fratta and his consortium members will use a novel tetramer-associated T-cell receptor sequencing assay to evaluate cryptic peptide expression in different cell types in parallel with single-cell RNA sequencing, targeted mass spectrometry, and interactive bleaching to extend multiplexity technology.
Dr. Panos Roussos (Mount Sinai), alongside Drs. Steve Finkbeiner (University of California, San Francisco), Guo-Cheng Yuan (Mount Sinai), and Zhenyu Yue (Mount Sinai), will assess how microglial autophagy and proteostasis underlie TDP-43 pathology. Dr. Roussos and his colleagues will integrate spatial transcriptomics, single-nuclei RNA-sequencing, quantitative neuropathology, and induced microglia and motor neurons to identify shared and distinct microglial states driving neuronal TDP-43 dysfunction.
Dr. Jiou Wang (Johns Hopkins University) will collaborate with Drs. Norman Haughey (Tulane) and Ling Hao (Maryland) to explore ALS-associated lipid metabolic vulnerabilities. The consortium will leverage untargeted lipidomics using high-resolution LC-HRMS/MS, deep global, proximity labeling, and dynamic SILAC-based turnover proteomics, and superresolution imaging techniques.
Dr. Jessica Mandrioli (Università degli Studi di Modena e Reggio Emilia) will partner with Drs. Laura Brunelli (Mario Negri Institute), Amedeo Amedei (Florence), and Luca Marchetti (Trento) to develop and implement an AI-driven multi-omics platform to identify therapeutic targets by integrating lipidomics, immune profiling, and inflammatory transcriptomic signatures.
Finally, Dr. Jeffrey Rothstein (Johns Hopkins University) will team with Drs. Julia Kaye (University of California, San Francisco) and Andre Hoelz (Caltech), and Juro Gottweis (Google) to study and model how rare nucleoporin coding variants initiate TDP-43 mislocalization and sporadic ALS. The consortium will integrate genetic, multi-omic, and imaging data gathered from robotic longitudinal microscopy, using Google artificial intelligence (DeepMind) to predict nucleoporin protein structure and drug targets.
New In Vivo Target Validation Grants to be Announced
Target ALS provides support for in vivo proof-of-concept for promising therapeutics in mouse models of ALS. We have partnered with the contract research organization Biospective to enable evaluation of novel therapeutics for target engagement and disease-modifying potential in ALS. Test articles (e.g., small molecules, biologics, gene therapy, or antisense oligonucleotides) are provided by the investigator. Results from these studies have the potential to catapult ALS drug candidates from preclinical to clinical pipelines, accelerating development of novel therapeutics. In this funding cycle, we received 47 proposals, and awards will be announced in mid-July.
C9orf72 RE KI mouse model characterization underway at Biospective
Preparations are underway for the upcoming launch of Target ALS’s In Vivo Target Validation funding calls, with phenotype characterization of Regeneron’s C9orf72-545 repeat expansion mice currently in progress at Biospective, a contract research organization based in Montreal. This critical groundwork will ensure that validated, well-characterized animal models are ready to support rigorous in vivo pharmacology studies when the funding calls open later in 2027.
Biomarker Consortia Funding Call Closed
ALS is a heterogeneous disease with distinct subpopulations of patients 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 patient 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 ALS patients, biomarkers are needed to stratify patient populations, understand response to therapeutics, and quantify 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). Recently, we extended Invitations to submit full proposals to top applicants. Final decisions will be announced mid-September.
Check our website, social media, or email notifications for information on new funding calls to be announced in late Q4.
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