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Sachi Bio lead shows Effect in Multiple Sclerosis Model: Findings published in ACS Chemical Neuroscience

Multiple sclerosis (MS) is a serious autoimmune disease that affects millions of people worldwide, with women being four times more likely than men to develop it. The disease often strikes in the prime of life, when people are highly productive, and it causes significant damage to the brain and spinal cord. This damage leads to severe inflammation, the breakdown of the protective covering of nerve fibers (called myelin), and eventually, nerve damage. As a result, people with MS often experience symptoms like difficulty moving and, over time, may lose mobility entirely. While existing treatments that suppress the immune system can help manage symptoms and delay the progression of the disease, they don’t work for everyone, and many patients struggle to achieve lasting remission. Sachi Bio recently published findings in ACS Chemical Neuroscience about Nanoligomer leads NI112 and NI111 that show promise in Multiple Sclerosis models.

Recent research has explored new ways to target the specific parts of the immune system responsible for inflammation in MS without affecting the rest of the immune system. One promising approach is the use of Nanoligomers—tiny molecules that can block specific inflammatory pathways. In this study, scientists from Sachi Bio screened several potential Nanoligomer combinations that target molecules like NF-κB, TNFR1, and IL-6, all of which are involved in the inflammatory response in MS. They found that two combinations, called NI111 and NI112, performed better than existing treatments in reducing inflammation in lab-grown human brain models without any negative effects on brain function.


To test these treatments further, researchers at Colorado State University used a mouse model of MS called experimental autoimmune encephalomyelitis (EAE), which mimics the inflammation and nerve damage seen in human MS. They found that mice treated with NI112, which targets both NF-κB and NLRP3, showed minimal signs of disease. These mice maintained their mobility, had very little inflammation in their brain and spinal cord, and showed no significant nerve damage, similar to healthy control mice that did not have the disease. Another combination, NI111, also reduced inflammation but was not as effective as NI112, and the treated mice experienced more severe symptoms compared to those treated with NI112.


The researchers also tested an oral version of NI112 to see if it could be administered more easily. The oral treatment showed promise, suggesting it could be a practical option for future therapies.


Overall, these findings highlight the potential of using inflammasome-targeting Nanoligomers like NI112 to treat MS and possibly other neurodegenerative diseases. This targeted approach could offer a more effective way to reduce inflammation and prevent nerve damage, providing new hope for patients with MS who struggle with current treatments. Further development and testing of these Nanoligomers could lead to breakthroughs in managing this debilitating disease.


Read more about this study:


Sadhana Sharma, Sydney Risen, Vincenzo S. Gilberto, Sean Boland, Anushree Chatterjee, Julie A. Moreno, and, Prashant Nagpal* (2024).  ACS Chemical Neuroscience  15, 7, 1596–1608.

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