New FDA validation on organoid studies, first-of-its-kind Tau protein developments, and an advanced AI-designed neutralizing infrastructure have restructured the future of Alzheimer’s research and therapeutic modalities.
BOSTON–(BUSINESS WIRE)–Porosome Therapeutics, Inc. (Porosome Therapeutics) today announced key advancements in its first-in-class, disease-modifying neurological platform, which will transform Alzheimer’s Disease (AD) research and treatment by offering a new approach that goes beyond symptom management. Following the recent FDA validation of organoid-based studies, the company has demonstrated a relatively rapid reduction of the Alzheimer’s pathology in AD human brain organoids treated with its principal therapy; shown a significant reduction of the Tau protein, acknowledged by the Food and Drug Administration (FDA) as an AD biomarker, in other studies; and established new classes of AD therapeutics.
Implementing the company’s signature “Reprogram, Restore, and Rescue” strategy, researchers introduce healthy porosomes – neuronal secretory nanomachines – into diseased neurons to target the root causes of AD. By restoring the neuron’s secretory and metabolic systems, Porosome Therapeutics’ first-of-its-kind approach addresses the cellular function of AD rather than traditional symptom management.
“Unlike therapies that merely dissolve beta amyloid plaques, our platform directly targets the core biological dysfunctions of Alzheimer’s, restoring both neurotransmission and metabolic integrity,” said Guillermo Marmol, CEO, Porosome Therapeutics. “Given Alzheimer’s is a serious and life-threatening disease with no effective treatment options, and based on our ability to deliver quantifiable, biomarker-driven improvements, including Tau reduction and disease reversal in human brain organoids, we are actively exploring the FDA’s accelerated approval pathway.”
In recent studies, the company’s approach to porosome restoration has been shown to significantly reduce Tau protein levels – a key FDA-approved biomarker of Alzheimer’s. The FDA’s recent approval of the Tau test is a landmark development in the research for AD therapies, catalyzed by the groundbreaking science of Porosome Therapeutics.
Furthermore, the company’s therapeutic approach has been validated using FDA-recommended human brain organoid models, demonstrating fast clinical results with a significant reversal of Alzheimer’s pathology shown within two weeks. Organoid models are three-dimensional cell cultures derived from stem cells that mimic the structure and function of human organs.
“When using human brain organoids, we’re able to observe the molecular activity of the porosome at an entirely new scale,” said Bhanu P. Jena, PhD, Founder and Chairman, Porosome Therapeutics, and a distinguished cell biologist known for his discovery of the porosome nanomachine, the secretory portal of the cell. “The ability to create an immense impact in just two weeks is a promising step forward and marks an important milestone as we advance the future of Alzheimer’s research and care.”
In a complementary development, the company is leveraging artificial intelligence (AI) to design proprietary decoy peptides that target and neutralize the toxic beta amyloid peptide (1-42), which is known for disrupting protein-protein interactions within the neuronal porosome complex and impairing neurotransmitter release. These specially designed AI-decoys bind more strongly to the beta amyloid (1-42), diverting the peptide from interfering with essential porosome functions.
With these advances, Porosome Therapeutics has identified three distinct therapeutic classes for AD:
- Small Molecules and Peptides – Cross the blood-brain barrier to restore mitochondrial function.
- Biologics – Reconstitute the porosome complex to reverse neuronal secretory dysfunction.
- AI-Designed Peptides – Decoy peptides designed by AI to neutralize beta amyloid peptides (1-42) and protect neurotransmission.
Porosome Therapeutics has expanded its portfolio of products to reflect these breakthroughs and the company’s commitment to advancing novel treatment modalities beyond the current standard of care.
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.
Contacts
Laramie Hunter
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(916) 658-0144
