HCHWA-DHereditary Cerebral Hemorrhage with Amyloidosis-Dutch Type (autosomal dominant disease)
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Through a collaboration with Amylon Therapeutics, the platform is also being developed for CNS indications including HCHWA-D. Recently, validation and expansion of the platform included positive results in clinical trials in cystic fibrosis and initiating a clinical trial in Leber's congenital amaurosis.