Breakthrough in Alzheimer’s research unveiled
Major strides in Alzheimer’s disease research, including FDA validation of organoid-based studies and the development of Tau-targeting therapies have been announced by Porosome Therapeutics.
The company introduces healthy porosomes into diseased neurons, through its “Reprogram, Restore, and Rescue” strategy, as the nanomachines restore secretory and metabolic function, hence, addressing the root causes of Alzheimer’s.
According to the CEO Guillermo Marmol, the Porosome Therapeutics’ platform targets directly the core biological dysfunctions of Alzheimer’s in order to restore both the neurotransmission and metabolic integrity.
“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,” he said.
Recent studies have also shown that Porosome Therapeutics’ approach is effective in reducing Tau protein levels, which is a key FDA-approved biomarker. The company said its therapy has reversed Alzheimer’s pathology in human brain organoids within two weeks of treatment.
Organoid models are three-dimensional cell cultures derived from stem cells that mimic or replicate human organ function, providing a more relevant platform for studying disease and drugs responses as well as development, unlike the traditional two-dimensional cell cultures.
According to the Founder and Chairman of Porosome Therapeutics, Dr Bhanu P Jena, “When using human brain organoids, we’re able to observe the molecular activity of the porosome at an entirely new scale.”
The company also identified three therapeutic classes for Alzheimer’s; small molecules and peptides to restore mitochondrial function, AI-designed peptides to neutralise beta amyloid, and biologics to reconstitute the porosome complex.
Alzheimer’s disease (AD), a neurodegenerative disease, accounts for about 60–70% of dementia cases, characterized by difficulty in remembering recent events.