Dr. Mordes is an Assistant Professor and board-certified neuropathologist at UCSF. His work connects C9 dipeptide repeat toxicity to changes in gene expression through SRSF7 and links TBK1 (another ALS-associated protein) to the C9ORF72 protein complex. His lab is building a “wiring diagram” of C9 disease, mapping the molecular nodes that are disrupted and how they converge on TDP-43 dysfunction. With collaborators in Boston, he has recently demonstrated a shared molecular pathology between ALS and advanced forms of chronic traumatic encephalopathy (CTE) in individuals with a history of playing contact sports. Together, these insights define how cellular systems fail in neurodegenerative disease and help turn “we know the gene” into actionable therapeutic targets.

Dr. Mancuso is a group leader and Deputy Director at the VIB–Center for Molecular Neurology, Belgium. He obtained his PhD in Barcelona in 2014 and since then his primary focus has been to investigate the underlying molecular mechanisms that drive Alzheimer’s disease (AD) and Frontotemporal degeneration (FTD), with special focus on inflammatory networks and particularly the contribution of microglia. In his lab, research is performed using a wide range of techniques and technologies, including classical mouse models, iPSC and cutting-edge humanised mouse systems to determine the immune component of these disorders and determine how genetics alter microglial function and contribute to the initiation and perpetuation of brain disease. Dr. Mancuso has been awarded with several young investigator awards (including the Grand Prix by the French Alzheimer’s foundation, ARUK), for his seminal contributions to our understanding of microglial pathogenic mechanisms. He has also received several prestigious grants, including an ERC Starting Grant to investigate neuro-glia interactions in AD.

Dr. Donnelly, Ph.D., is an Associate Professor of Neurology at the Peter O’Donnell Jr. Brain Institute at UT Southwestern and Neuromuscular Section Chief. He earned his Ph.D. from the University of Delaware, where he studied axonal RNA transport and local translation in nerve regeneration with Dr. Jeff Twiss, and completed postdoctoral training with Dr. Jeff Rothstein at Johns Hopkins School of Medicine, investigating cellular dysfunction in C9orf72-associated ALS and FTD. Prior to joining UT Southwestern in 2026, he was the founding Scientific Director of the LiveLikeLou Center for ALS Research at the University of Pittsburgh School of Medicine.
 
The Donnelly lab focuses on the molecular mechanisms underlying neurodegenerative disease, with an emphasis on how dysfunction of RNA-binding proteins, including TDP-43 and FUS, drives pathology and neuronal vulnerability in C9orf72 and sporadic ALS/FTLD. His laboratory investigates how RNA regulates protein function and phase behavior, and how defects in nuclear and axonal transport and pathogenic protein interactions drive ALS/FTLD. The goal of this work is to identify strategies to restore normal protein function in disease. The Donnelly lab integrates patient-derived neuronal models with RNA-based therapeutic approaches to target disease mechanisms. Dr. Donnelly’s prior research identified key disease pathways, including nucleocytoplasmic transport defects, and advanced therapeutic strategies targeting toxic C9orf72 gene products with antisense oligonucleotide (ASO) therapies. More recently, his lab developed short RNA oligonucleotides (bait/chaperone RNAs) approaches to reverse pathological protein aggregation and restore physiological function in ALS/FTLD.

Dr. Boeynaems is an Assistant Professor in Molecular and Human Genetics at Baylor College of Medicine, an Investigator at the Duncan Neurological Research Institute at Texas Children’s Hospital, and a Faculty Member of the Therapeutic Innovation Center, the Dan L Duncan Comprehensive Cancer Center, and the Center for Alzheimer’s and Neurodegenerative Diseases. He is a CPRIT Scholar in Cancer Research and an NIH Director’s New Innovator. Dr. Boeynaems has over a decade of expertise in the study of tandem repeats and protein aggregation in human disease and has contributed important insights into the pathophysiology of C9orf72 ALS/FTD.

Dr. McEachin is an assistant professor in Human Genetics and Cell Biology at Emory University. His laboratory is focused on understanding the molecular mechanisms underlying neurodegenerative disease, particularly repeat expansion disorders such as C9orf72-ALS/FTD. His research integrates multiomic analyses with diverse experimental models to uncover how genetic and molecular alterations contribute to disease pathogenesis and to identify new therapeutic targets for neurodegeneration.

Dr. Jafar-Nejad, MD, is a scientific leader in RNA therapeutics and currently serves as Executive Director at Ionis Pharmaceuticals, Inc., a pioneering biotechnology company in RNA-targeted drug discovery and development. He brings more than two decades of experience in neuroscience and RNA biology, including over a decade focused on oligonucleotide therapeutics, and has contributed to a substantial body of scientific publications in the field.

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Dr. Jafar-Nejad earned his medical degree from Tehran University of Medical Sciences and completed postdoctoral training in Dr. Huda Zoghbi’s laboratory at Baylor College of Medicine, where he investigated mechanisms of neurodegeneration. At Ionis, he has led research programs in neurology and rare diseases, advancing RNA-based therapies into clinical trials for ALS, Angelman syndrome, genetic epileptic encephalopathies, and related disorders. In recognition of this work, he and his colleagues received the 2023 Lalji & Family ALS Endowed Award for Innovative Healing from the Healey & AMG Center at Massachusetts General Hospital.

Dr. Clelland is building what every C9 carrier hopes for: a one-time fix at the genomic level. Her CRISPR work has shown the most complete reversal of C9 pathology demonstrated to date in patient-derived neurons. She is now partnering with Denali Therapeutics to solve the hardest remaining problem: getting that edit into the brain via a simple IV infusion. A project she is not yet public about is her work screening and optimizing gene therapy for the CNS using brain-dead trauma patients. This use of humans to improve gene therapy in a compartment nobody has successfully targeted is nearly unparalleled in utility for C9 and other neurological diseases. The cost for a first-in-human dose is ~$10M. This is one of the projects that should be proposed for funding by CureC9. Dr. Clelland chose C9orf72 because her patients have FTD. The most common cause of familial FTD is the C9 repeat expansion, so to cure FTD she needs to cure C9. Beyond the science, she brings a focus on translation that keeps every conversation grounded in what actually matters: getting a therapy to patients.

Dr. Gregory is developing an early warning system for neurodegenerative disease, focused on detecting pathology long before clinical symptoms appear. Her work with RNA aptamers identifies TDP-43 pathology with unprecedented sensitivity, and her skin biopsy research has demonstrated detectable pathology up to 26.5 years before diagnosis, opening the possibility of transforming presymptomatic monitoring from a source of uncertainty into a strategy for timely intervention. Her research is driven by a central question: not just what is happening in disease progression, but when it can first be detected and acted upon.
Beyond her own laboratory, Dr. Gregory serves as a co-investigator on a research program using synchrotron X-ray fluorescence imaging at the European Synchrotron Radiation Facility to investigate the C9orf72 paradox. Her team leads the pathomolecular profiling and low-field MRI platform aimed at translating nanoscale metal–aggregate signatures into a scalable diagnostic tool. She also builds and leads large presymptomatic research cohorts designed to enable early-stage disease discovery and intervention trials. As a practising pathologist and clinical lead for a major tissue bank, she integrates patient-derived biospecimens, clinical phenotyping, and molecular pathology to create resources that support collaborative discovery across the ALS research community.