Porosome Therapeutics has 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.
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, catalysed 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 neutralise 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 neutralise 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.
Pictured is the presence of amyloid plaques in the brain (immunohistochemical staining).