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

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by Charles Moore |

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Somerset, N.J. based Catalent Pharma Solutions, specialists in efficient delivery technology and development of medical and health care product solutions is collaborating with Valerion Therapeutics, LLC of Concord, Massachusetts – whose corporate focus is on bio-therapeutics for orphan genetic disease development. The alliance will join forces to develop Valerion’s lead product candidates — for delivery of muscle-protein fusions, and treatment of multiple indications respectively, in the muscular dystrophy, glycogen storage disease, myopathy, and enzyme-deficiency therapy fields.

Under terms of the cooperation alliance, Catalent will contribute cell line engineering, process development, and cGMP biomanufacturing associated with these Valerian-developed products, which are distinguished by a novel antibody-based targeting system, and capability for enhanced intracellular delivery of functional proteins, oligonucleotides and small molecules. The project will employ Catalent’s proprietary GPEx technology, which creates robust, stable, mammalian cell lines quickly and efficiently. Catalent claims that its methods are typically capable of getting drug development projects into the clinical market in one-third the time required with traditional approaches. GPEx technology advantages derive firm early feasibility studies that benefit clinical manufacturing and accelerate bringing commercial scale production online.

Should initial studies prove successful, the therapies will be produced at Catalent’s state-of-the-art facility in Madison, Wisconsin – a purpose-built biomanufacturing works for mammalian cell culture development and cGMP manufacturing.

“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,” says Valerion CEO Deborah Ramsdell. “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.”


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Catalent President of Advanced Delivery Technologies Barry Littlejohns observes: “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 order to meet customer demand, Catalent’s $26m Madison biomanufacturing facility which was completed in June 2013, had quadrupled the company’s biologics manufacturing capacity. Designed for flexible cGMP production ranging from 10L up to 1,000L, and non-GMP production up to 250L, the Madison site’s single-use technologies and unidirectional flow features provide maximized efficiency and safety.

Valerion Therapeutics, is an emerging biotech startup founded in 2007 as 4s3 Bioscience by Dustin Armstrong, Ph.D. and Timothy Harris. Valerion’s focus is on developing of bio-therapeutic medicines for orphan neuromuscular genetic diseases. The company develops product candidates employing a proprietary delivery technology platform designed to leverage tissue-targeting by way of a novel humanized antibody (VAL–1205) that is imbued with cell-penetrating properties utilizing tissue-localized membrane transporters (ENTs). Because these transporters are naturally enriched in critical tissues (such as skeletal muscles and the CNS), Valerion has been successful in constructing targeted fusion and chemical conjugation products (proteins, drugs & oligos) with functional intracellular activity; resulting in a novel way to treat a number of neuromuscular diseases that have limited or no current therapeutic options.

Catalent Pharma Solutions (the name is an amalgam of Catalyst + Talent) has served the industry for more than 80 years, and has developed proven expertise augmented by innovative technologies that help its clients get more customer products to market faster, as well as helping enhance product performance, and to ensure optimum, reliable manufacturing results and speedy, efficient clinical and commercial product supply. Catalent’s client base includes thousands of innovators, large and small, in over 100 markets, including 41 of the top 50 biotech companies and 48 of the top 50 pharmaceutical companies. The company employs approximately 8,000 people of which more than 1,000 are scientists, supports 40 percent of recent new drug approvals. Catalent’s intellectual property assets include oral, injectable, and respiratory dosage forms and advanced drug packaging, and it owns more than 1,200 patents and patent applications. It nearly 30 facilities across 5 continents make or package over 100 billion units annually. In fiscal 2014 the company generated revenues of more than $1.8 billion Catalent’s delivery technologies include softgel solutions, Liqui-Gels capsules, and Vegicaps capsules, Zydis fast dissolve, controlled release, and inhaled dose forms.

Catalent Pharma Solutions
Valerion Therapeutics, LLC

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Catalent Pharma Solutions