Forge Biologics, Inc. products

Lower cost for production; Improved safety profile – removed unnecessary adenoviral elements; Forge Biologics intellectual property; Available for pipeline use.

Royalty-free license for clients; Clonal HEK293 mammalian suspension cell line; Optimized for robust transient transfection; Animal component free; Scalable production.

Krabbe disease is a devastating neurodegenerative disease affecting about 1 -2.5 in 100,000 people in the U.S. and is inherited in an autosomal recessive manner. Krabbe disease is caused by loss-of-function mutations in the galactosylceramidase (GALC) gene, a lysosomal enzyme responsible for the breakdown of certain types of lipids such as psychosine. Without functional GALC, psychosine accumulates to toxic levels in cells. The psychosine toxicity is most severe in the protective cells surrounding the nerves in the brain and throughout the body (peripheral nervous system), eventually leading to the death of these cells.