This protein, present in all cells, has been identified as the root cause of various neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and frontotemporal dementia.
ProMIS' data show that several of its antibody candidates selectively targeted toxic, misfolded intracellular aggregates of TDP-43 but did not bind to normal TDP-43 in the cell nucleus. The therapeutic candidates also selectively bound to the pathogenic, misfolded form of TDP-43 in postmortem brain tissue of patients with frontotemporal dementia.
"The momentum behind each of our programs is palpable," President and CEO Elliot Goldstein said in the release. "In the past 30 days, we've announced data advancing our development programs selectively targeting toxic misfolded proteins for treatment of Alzheimer's and Parkinson's disease and Multiple System Atrophy (MSA), ALS and FTD. Yesterday, Biogen's decision to submit its amyloid-beta targeting drug candidate, aducanumab, for FDA approval signaled a milestone shift in the promise of next-generation amyloid-beta targeting drugs. Our lead program, PMN310, offers sniper-like precision for the toxic, misfolded form of amyloid beta, a key feature that offers important potential advantages compared to aducanumab. We will continue to support the patient community by advancing our programs, in particular PMN310 for Alzheimer’s disease, leveraging our proprietary, drug discovery platform."[NLINSERT]
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