Interesting reading about the Alzeimers treatments.
Because ASOs do not cross the blood brain barrier The treatment is being injected directly into spinal canal. Could this be another situation where our tech might be successful as similar to the eye treatment we could limit off target effects by doing this. Imagine if we could attack alzeimers more effectively. That would certainly be a target group of many more than 4-8000 patients.
"Because ASOs do not cross the blood-brain barrier, both Ionis and Wave Life are currently delivering theirs intrathecally, in other words, by way of an injection into the spinal canal. The anti-tau ASO trial offers an open-label extension, at which point investigators see how much participants mind this invasive form of drug delivery. “Every person chose to continue to open-label; that tells us a lot about how tolerable this is,” Mummery said. Even so, she would like to transition to less-onerous delivery modes, for example using little pumps implanted under the skin.The goal of delivering ASO therapies in a way that enables them to cross the blood-brain barrier is an active area of study. “It’s a challenging problem that we have not solved yet,” Bennett told the families. But even though intrathecal injection is cumbersome, Miller insisted that people tolerate it well in ALS, Huntington’s, and other ASO indications. “Our goal is to develop drugs that have a major effect and are therefore worth delivering in this way,” he said."
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