Ceregene established a leadership position in the fields of gene therapy and neurotrophic factors for the treatment of neurodegenerative diseases. A total of over 100 patients have been safely dosed in two clinical programs: CERE-120 (AAV-NRTN) for Parkinson's disease and CERE-110 (AAV-NGF) for Alzheimer's disease. A randomized, controlled Phase 2 study of CERE-110 for Alzheimer's is continuing. It is fully enrolled and financially supported in large part by a grant from the NIH, with top line data expected by late 2014. In addition to the Parkinson's and Alzheimer's programs, Ceregene has conducted extensive preclinical work with CERE-120 for Huntington's disease, as well as another gene therapy/neurotrophic factor product (AAV-NT4) for blinding ocular diseases (such as Retinitis Pigmentosa, macular degeneration, diabetic retinopathy and glaucoma) and yet another (AAV-IGF1) for Lou Gehrig 's disease (ALS). Ceregene is currently looking at strategic alternatives to advance its AAV gene therapy platform.
About CERE-120 and its Application to Treating Parkinson's Disease
CERE-120 is composed of a harmless adeno-associated virus (AAV) vector carrying the gene for neurturin, a naturally occurring protein known to repair damaged and dying dopamine-secreting neurons, keeping them alive and restoring function. Neurturin is a member of the same protein family as glial cell line-derived neurotrophic factor (GDNF). The two molecules have similar pharmacological properties and both have been shown to benefit the midbrain dopamine neurons that degenera
|SOURCE Ceregene, Inc.|
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