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Our science
Transforming healthcare
Neurodegenerative diseases such as Alzheimer’s, ALS and Parkinson’s are more prevalent with age. They are exceptionally cruel conditions, with enormous consequences for both patients and their families. They also require many years of care, with a heavy burden on healthcare provision.
Each disease manifests differently but all are seen as life sentences. While medicine can offer some relief from symptoms, there are currently no effective treatments to halt or prevent disease progression.
But we believe autophagy – our cells’ natural self-cleaning process – holds the solution. A defining feature of these diseases is the appearance and accumulation of cytotoxic, misfolded proteins. Research suggests that impaired autophagy could be a cause of these misfolded proteins, and that restoring autophagy could be the key to effective therapeutic strategies.
Fresh hope for patients with rare diseases
At Samsara, we’re also working to uncover treatments for rare monogenic diseases – particularly peripheral nervous system diseases such as CMT. Many of these diseases are caused by a single mutation in a gene, which leads to an accumulation of misfolded, non-functional proteins. These proteins go on to damage the cell and often cause serious disease. They can also inhibit the cell’s autophagy processes, which leads to more damage.
Our research shows that boosting autophagy in these cells can remove the misfolded proteins or even restore their functionality. In fact, autophagy therapies could even be completely curative – a life-changing development for patients who currently have little hope of successful treatment.
A new paradigm for disease prevention
In the next few years, patients and healthcare systems will demand new ways to extend healthspan and prevent age-related diseases. Samsara’s team is committed to meeting this need through rigorous science and ground-breaking research technology.
Many age-related neurodegenerative conditions are thought to take hold years or even decades before symptoms appear. So as well as halting or preventing the progression of disease, we’re looking towards a future in which autophagy therapies have a role in preventative medicine. As part of our work, we’re studying new ways to identify early signs of disease, through genetic testing, imaging techniques and the evaluation of biomarkers. Breakthroughs could revolutionise the ways we prevent and treat illness.
With healthcare strategies moving towards preventing disease through increasingly personalised, integrated, AI-driven approaches, we believe our ground-breaking work in autophagy could help to transform the healthcare of tomorrow.
More science
LysoSeeker®
Our best-in-class technology platform, LysoSeeker®, dramatically accelerates our search for viable, effective autophagy-inducing agents. It’s capable of screening and testing the autophagy-boosting potential of thousands of molecules, to identify drug candidates, their mechanisms and the cell targets they interact with.
SAM001
The first drug candidate in its class to enter clinical trials, SAM001 boosts autophagy and reduces neuron damage in brain cells taken from people with Parkinson’s, and restores motor function in models of ALS. Our research indicates it even has the potential to reverse symptoms of disease.
Our pipeline
We’re rapidly advancing new drugs, with a number of promising therapies designed to treat neurodegenerative conditions and serious rare genetic diseases. Our focus is on diseases which are strongly linked to dysfunctional autophagy, and have limited or no known treatment options.
SAM0021
Our second autophagy-inducing candidate, a promising treatment for Parkinson’s Disease. Thanks to funding from the Michael J. Fox Foundation, we’ve accelerated our research with the aim of moving into clinical trials by the end of 2024.