PRODUCT DESCRIPTION
MoNuDin is a novel gene-based therapeutic for motor neuron disease. The product is a LentiVector^® system carrying a neuroprotective gene.

INDICATION
Amyotropic Lateral Sclerosis (ALS), the major form of MND.

MARKET
In the USA an estimated 30,000 people are living with ALS, and 6,000 new cases are diagnosed annually (ALS association). Worldwide market is estimated to be US$200 million.

TECHNICAL DESIGN
LentiVector delivering the vascular endothelial growth factor (VEGF) gene.

DEVELOPMENT STATUS

• Preclinical efficacy has been demonstrated in an industry standard in vivo model of ALS
• Studies showed that both the onset and progression of disease were slowed and life expectancy was extended by 30%. They also suggest that MoNuDin is one of the most effective potential therapies in the field to date (Nature May 2004 – Vol:429, Issue: 6990, pp: 413 – 417)
• Process development for manufacturing scale-up has commenced
• Additional preclinical toxicology and efficacy studies are being conducted, designed to support a regulatory submission for the start of clinical trials

COMMERCIALISATION STRATEGY
Commercial partners sought to co-develop and market the product.

PARTNERS/FUNDING
ALS Association, USA
MND Association, UK

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