Catalent and Valerion Therapeutics Collaborate to Bring New Treatments for Orphan Genetic Disorders to Market

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Catalent Pharma Solutions, the leading global provider of advanced delivery technologies and development solutions for drugs, biologics and consumer health products, today announced that it is to collaborate with Valerion Therapeutics, LLC, an emerging science-driven company, focused on the development of bio-therapeutics for orphan genetic diseases, to develop Valerion’s two lead product candidates. 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.

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

Catalent Pharma Solutions, the leading global provider of advanced delivery technologies and development solutions for drugs, biologics and consumer health products, today announced that it is to collaborate with Valerion Therapeutics, LLC, an emerging science-driven company, focused on the development of bio-therapeutics for orphan genetic diseases, to develop Valerion’s two lead product candidates. 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.

Under the agreement, Catalent will provide all cell line engineering, process development and cGMP biomanufacturing activities associated with the two lead products, which utilize a novel antibody-based targeting system, capable of enhanced intracellular delivery of functional proteins, oligonucleotides and small molecules. The project will utilize Catalent’s proprietary GPEx® technology, which creates high-expression, extremely stable mammalian cell lines with speed and efficiency, typically capable of getting drug development projects to clinic in one-third the time of traditional approaches. The advantages of applying GPEx technology span from early feasibility studies, to clinical manufacturing, to commercial scale production.

Should initial studies prove successful, the therapies will be produced at Catalent’s state-of-the-art 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,” commented Deborah Ramsdell, CEO of Valerion. She added. “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.”

Constructed in response to customer demand, and completed in June 2013, Catalent’s $26m Madison biomanufacturing facility quadrupled the company’s biologics manufacturing capacity. Designed for flexible cGMP production from 10L up to 1,000L, and non-GMP production up to 250L, the site features single-use technologies and unidirectional flow to maximize efficiency and safety.

About Valerion Therapeutics

Valerion Therapeutics is an emerging science-driven company focused on the development of bio-therapeutics for orphan neuromuscular genetic diseases. Valerion generates unique product candidates utilizing a proprietary delivery technology platform that takes advantage of tissue targeting via a novel humanized antibody (VAL-1205) with cell-penetrating properties utilizing a tissue-localized membrane transporter family (ENTs). Because the transporter is naturally enriched in critical tissues (ex. skeletal muscle and CNS), Valerion is able to construct targeted fusion and chemical conjugation products (proteins, drugs & oligos) with functional intracellular activity; thereby, providing a novel way to treat a number of neuromuscular diseases with limited or no current therapeutic options.

About Catalent

Catalent Pharma Solutions is the leading global provider of advanced delivery technologies and development solutions for drugs, biologics and consumer health products. With over 80 years serving the industry, Catalent has proven expertise in bringing more customer products to market faster, enhancing product performance and ensuring reliable clinical and commercial product supply. Catalent employs approximately 8,000 people, including over 1,000 scientists, at nearly 30 facilities across 5 continents and in fiscal 2014 generated more than $1.8 billion in annual revenue. Catalent is headquartered in Somerset, N.J. For more information, visit http://www.catalent.com

More products. Better treatments. Reliably supplied.™

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