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Other Disease Treatment

ACI-24

Active immunotherapy targeting Abeta in Down syndrome

The liposomal therapeutic vaccine ACI-24 is also being investigated in a phase 1b trial for the prevention of cognitive decline in people with Down syndrome. Individuals with Down syndrome have an extra copy of chromosome 21, the chromosome on which the gene for amyloid precursor protein resides, leading to Abeta accumulation. These individuals develop AD-like characteristics at a rate three to five times greater than that of the general population and at a much younger age. AD-like characteristics develop in more than 98% of people with DS over age 40 with up to 80% developing associated dementia over the age of 60. Preclinical studies demonstrated the reduction of Abeta level and compelling memory enhancement.

ACI-24 is in a phase 1 clinical study which is supported by substantial grants of the US NIH and the LuMind Research Down Syndrome Foundation. 

 

 

Morphomer Abeta

Small molecule for glaucoma prevention

Misfolded Abeta is reportedly involved in the retinal disease glaucoma, a major cause of irreversible blindness worldwide representing an important opportunity for our Morphomer Abeta program. Preclinical studies have shown a strong ability to protect the eyes exposed to ocular pressure and chronic ocular hypertension which are clinical features of glaucoma. 

Morphomer α-syn

Small molecule for Parkinson’s disease treatment

Misfolded and aggregated α-synuclein is a key protein involved in the pathology of Parkinson’s disease. Preclinical studies show significant reduction of Lewy body like structures combined with promising CNS drug features.

Morphomer Abeta (Abeta inhibitor)
Down syndrome*
Phase 1
Biologics
Glaucoma
Pre-clin.
Small molecules
Morphomer α-syn (α-synuclein inhibitor)
Parkinson's
Discovery
Small molecules

ACI-24

anti-Abeta vaccine

The liposomal therapeutic vaccine ACI-24 is also being investigated in a phase 1b trial for the prevention of cognitive decline in people with Down syndrome. Individuals with Down syndrome have an extra copy of chromosome 21, the chromosome on which the gene for amyloid precursor protein resides, leading to Abeta accumulation. These individuals develop AD-like characteristics at a rate three to five times greater than that of the general population and at a much younger age. AD-like characteristics develop in more than 98% of people with DS over age 40 with up to 80% developing associated dementia over the age of 60. Preclinical studies demonstrated the reduction of Abeta level and compelling memory enhancement.

ACI-24 is in a phase 1 clinical study which is supported by substantial grants of the US NIH and the LuMind Research Down Syndrome Foundation. 

 

 

Morphomer Abeta

Abeta inhibitor

Misfolded Abeta is reportedly involved in the retinal disease glaucoma, a major cause of irreversible blindness worldwide representing an important opportunity for our Morphomer Abeta program. Preclinical studies have shown a strong ability to protect the eyes exposed to ocular pressure and chronic ocular hypertension which are clinical features of glaucoma. 

Morphomer α-syn

α-synuclein inhibitor

Misfolded and aggregated α-synuclein is a key protein involved in the pathology of Parkinson’s disease. Preclinical studies show significant reduction of Lewy body like structures combined with promising CNS drug features.