RegenXBio Inc. filed an S-1 form with the Securities and Exchange Commission (SEC) regarding its initial public offering. There were no terms given in the filing but the offering is valued up to $100 million, although this number normally just asks as a placeholder. The company intends to file on the NASDAQ Global Market under the symbol RGNX.

The underwriters for the offering are Morgan Stanley, Merrill Lynch, Piper Jaffray, and Chardan Capital Markets.

This is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy. The proprietary AAV gene delivery platform consists of exclusive rights to over 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10 (NAV Vectors).

The company’s mission is to transform the lives of patients suffering from severe diseases with significant unmet medical needs by developing and commercializing gene therapy products administered directly into the body, based on its NAV Technology Platform. It seeks to accomplish its mission through a combination of internal development efforts and the efforts of third-party licensees.

The NAV Technology Platform is currently being applied in the development of 23 product candidates for a variety of diseases, including five internally developed product candidates and 18 partnered product candidates developed by the company’s NAV technology licensees.

The most advanced internally developed candidates include programs for the treatment of two severe, rare genetic diseases: homozygous familial hypercholesterolemia (HoFH) and Mucopolysaccharidosis Type I (MPS I). The company expects these programs to enter phase I/II clinical trials in the first half of 2016. It also has a program for wet age-related macular degeneration (wet AMD) that is in the preclinical stage which is expected to file an Investigational New Drug application (IND) in the second half of 2016. RegenXBio plans to build internal gene therapy franchises in the metabolic, neurodegenerative and retinal therapeutic areas, and develop multiple product candidates in these and other areas.

The company intends to use the proceeds from the offering to fund the research and development of its product candidates. The remainder of the proceeds will go towards working capital and general corporate purposes.

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