Catalent Pharma Solutions has entered into a collaboration with Valerion Therapeutics to develop two of Valerion’s lead product candidates to treat orphan genetic diseases. The collaboration will focus on the delivery of muscle-protein fusions, and treatment of multiple indications, in the fields of muscular dystrophies, glycogen storage diseases, myopathies and enzyme-deficiency disorders.
 
Catalent will provide all cell line engineering, process development and cGMP biomanufacturing for the products utilizing an antibody-based targeting system capable of enhanced intracellular delivery of functional proteins, oligonucleotides and small molecules. The project will employ Catalent’s GPEx technology, which creates high-expression, stable mammalian cell lines.
 
If initial studies are successful, the therapies will be produced at Catalent’s Madison, WI site, a purpose built facility for mammalian cell culture development and cGMP manufacturing utilizing up to 1000L single use bioreactors (SUBs).
 
“We selected Catalent as our development and manufacturing partner for this exciting project as the company has both the expertise and proprietary technologies required to bring these candidates to the clinic in the shortest possible timeframe,” said Deborah Ramsdell, chief executive officer of Valerion. “Catalent’s cGMP facility capabilities are a great match for Valerion’s current supply requirements and we will look to partner with them on future development and manufacturing needs as well.”
 
Barry Littlejohns, president of Advanced Delivery Technologies at Catalent, added, “We are pleased to be working with Valerion to address the need for biologic based therapeutics for orphan genetic diseases. Valerion’s proprietary tissue targeting delivery platform has demonstrated huge potential in terms of bringing new treatments to market.”