AI modeling, Alzheimer’s tissue analyses, and preclinical porosome studies have identified a fundamental cause of Alzheimer’s disease, paving the way for innovative treatment pathways aimed at reversing early-stage disease pathology
BOSTON–(BUSINESS WIRE)–Porosome Therapeutics, Inc. (Porosome Therapeutics) today announced its latest development in identifying the secretory dysfunction responsible for Alzheimer’s Disease along with advancements in therapeutic neuronal nanomachines. These innovations may transform the future of Alzheimer’s research and treatment approaches. The company’s new findings suggest that combining porosome restoration with small molecule and peptide therapy may not only slow the progression of Alzheimer’s disease (AD) but also potentially reverse early-stage pathology, moving beyond the traditional focus on amyloid accumulation.
“For the first time in over a century since Alzheimer’s disease was identified, we have achieved a molecular-level understanding of how this condition impairs neurosecretion and disrupts communication within the brain, ultimately resulting in dementia,” said Bhanu P. Jena, PhD, founder of Porosome Therapeutics and a distinguished cell biologist known for his discovery of the porosome nanomachine, the secretory portal of the cell. “By utilizing AlphaFold, an artificial intelligence-driven tool, we have elucidated how beta-amyloid (Aβ) disrupts essential protein interactions within the neuronal porosome complex, consequently hindering neurotransmitter release in individuals affected by Alzheimer’s. Immunocytochemistry studies of human brain tissue confirmed the AlphaFold results. This significant advancement positions us to develop innovative therapies that tackle the fundamental secretory dysfunction causes of the disease.”
Continuing research into the secretory dysfunction dimension of Alzheimer’s, conducted by Porosome Therapeutics’ subsidiary, NeuroTher LLC, has advanced a new class of treatments designed to restore neuronal function by addressing the secretory and metabolic defects contributing to AD.
Preclinical studies led by Xin Qi, PhD, an expert in mitochondrial pathology and peptide therapies for neurodegenerative diseases, have validated a novel therapeutic strategy targeting both secretory dysfunction and mitochondrial defect at the cellular level.
AD is characterized by the loss of neurons due to secretory and metabolic defects, resulting in mitochondrial dysfunction and neurotransmitter depletion. Through AlphaFold analysis, NeuroTher has discovered how the beta amyloid peptide alters proteins within the porosome. This suggests that incorporating functional porosomes into neurons affected by AD could potentially restore neurotransmitter release, communication, and plasticity—critical components for perception, thought, and memory.
“This research has underscored the crucial role of proteins associated with mitochondrial dynamics in cellular functions and their beneficial link to porosomes,” stated Dr. Qi. “We are currently investigating the impact of porosomes on neurodegenerative diseases and how this discovery could reshape treatment methodologies and research approaches, offering a beacon of hope for patients and families around the globe.”
Recent preclinical studies utilizing this technique showed positive results in restoring function, presenting a compelling new direction for AD treatment. Unlike conventional therapies focusing on symptom management, our approach targets the cellular level to restore mitochondrial and neuronal secretory function. Its emphasis on repairing the critical secretory and metabolic machinery sets it apart from other available or under-development treatments. Porosome Therapeutics has submitted the findings of its study to a leading, peer-reviewed scientific journal.
“I am strongly convinced by our findings regarding the future of Alzheimer’s research,” stated Juan Ruiz, PhD, MBA, former Deputy Director for Science in the Office of Translational Sciences at the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research and a member of the Scientific Advisory Board of Porosome Therapeutics. “Restoring neuronal function at the cellular level has the potential to revolutionize our approach to treating Alzheimer’s disease, instilling renewed hope in millions of patients and their families worldwide. It is inspiring to witness the advancements in porosome replacement therapy research and the promise it holds for individuals affected by conditions previously deemed untreatable, including Alzheimer’s disease, cystic fibrosis, and various other significant health challenges.” The Company’s treatment for cystic fibrosis was granted Orphan Drug Designation from the FDA in 2025.
“This discovery marks a significant advancement in the quest for effective Alzheimer’s treatments,” said Guillermo Marmol, CEO of Porosome Therapeutics. “Through transformative and innovative research, in collaboration with leading scientists from academic and research institutions, Porosome Therapeutics is committed to the accelerated development of groundbreaking therapies and partnerships with pioneering organizations and regulatory agencies to swiftly deliver safe, life-changing medicines for Alzheimer’s and beyond.”
About Porosome Therapeutics, Inc.
Porosome Therapeutics, Inc. is a biopharmaceutical company based in Boston that leverages the groundbreaking discovery of the porosome—the cell’s essential secretory machinery. The company employs innovative technologies to identify and develop proprietary therapeutics aimed at currently undruggable porosome proteins linked to secretory and hydration disorders such as cystic fibrosis, diabetes, and cancer. Porosome Therapeutics is the first company to employ a technology platform dedicated to creating novel therapies targeting the porosome, recognized as the universal secretory machinery in cells. This platform is built on decades of research and is supported by over 200 published studies, proving effective against various serious diseases involving secretory defects. The platform focuses on highly specific nanobody-mediated therapies, aiming to significantly reduce drug side effects. Porosome Therapeutics’ work on Alzheimer’s Disease is carried out through a subsidiary, NeuroTher LLC.
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