Catalent contracted to manufacture Valerion's orphan drug candidates

03-Dec-2014 - Last updated on 03-Dec-2014 at 12:18 GMT

Catalent Contract

Valerion Therapeutics has inked an agreement for the manufacture of two orphan drug candidates using Catalent’s GPEx mammalian cell line platform.

The deal will see the contract development and manufacturing orgnaisation (CDMO) aid the development of Valerion’s two lead candidates focused on patients with rare muscle diseases by providing cell line engineering, process development and cGMP biomanufacturing as they approach clinical trials.

“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 Valerion CEO Deborah Ramsdell.

Financial details were not provided but Catalent will use its GPEx technology to create mammalian cell lines. The platform uses retroviral vector-based technology the firm says ensures the stable transduction of targeted cells, leading to a level of efficiency which eliminates the requirement for selectable markers.

The therapies – if initial studies are successful - will be produced at Catalent’s biomanufacturing facility in Madison, Wisconsin, an ex-GE Healthcare facility which opened last year. The site makes extensive use of disposable technologies with single-use bioreactors ranging from 10L to 1,000L.

“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,” Ramsdell added.

VAL-0620 and VAL-0411

Valerion’s two lead candidates are VAL-0620, to treat patients with myotubular myopathy, a deficiency of the enzyme MTM1 which causes severe muscle disease, and VAL-0411, being investigated to for adult muscular dystrophy.

Currently in the preclinical stage of development, both candidates were generated using Valerion’s drug delivery platform that uses not previously deliverable substrates (proteins, enzymes, oligonucleotides, etc.) to target tissues.

“Valerion’s proprietary tissue targeting delivery platform has demonstrated huge potential in terms of bringing new treatments to market,” said Barry Littlejohns, President of Advanced Delivery Technologies at Catalent.