AD/PD 2026 | Overcoming the challenge of delivering gene therapy across the BBB in AD and PD

Disclosures

So major challenges in gene delivery, it’s really delivery systems which are not ideal so far. So each platform fits specific application, but bottom line there is no ideal platform for efficient gene delivery. And this field is evolving tremendously in recent years with novel serotypes based on adeno-associated vectors. So people try to develop serotypes which will cross the BBB, the blood-brain barrier, efficiently. However, to say, technology still has not evolved to the level that it can be applied in vivo or even in humans. So we bypassed this by using direct injection into the brain parenchyma and brain tissue, which has been approved for many, many applications. So people do take these viral particles and directly inject into the brain. And there are different ways to deliver and to tackle it in the brain directly, so this is really an efficient way to deliver and this is a way to sort of bypass this requirement for the blood-brain barrier, which people utilize so far as a gold standard delivery system. But in the future, when this delivery will be, the serotypes of adeno-associated vectors will allow and will support this delivery in a systemic way with novel serotypes already being developed by several groups and they are in preclinical stages now as well.

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