Meet AveXis - 2019 Facility of the Year Award Honorable Mention Category Winner

Projects selected for recognition with an Honorable Mention Award are typically projects that utilize new technology or are located in a challenging region of the world. These projects do not win for a specific category but are clearly successful projects that overcame significant challenges in planning, execution, and delivery and deserve recognition for their achievements.

Founded in 2013, Chicago-based AveXis is a gene therapy company focused on developing and commercializing novel treatments for patients and families affected by rare and life-threatening neurological genetic diseases. The goal of the company’s cutting-edge science is to stop and prevent further disease progression by replacing a lost or defective gene with a fully functioning copy of the gene using a one-time delivery method. In order to manufacture its proprietary investigational gene replacement therapy, AVXS-101, AveXis built a 49,000-square-foot state-of-the-art manufacturing facility in suburban Chicago.

AVXS-101, approved by the FDA in the summer of 2019, is their novel treatment for spinal muscular atrophy (SMA), a severe, progressive neuromuscular disease caused by a defective or missing SMN1 gene, the primary gene responsible for making the survival motor neuron (SMN) protein. SMA affects approximately one in 10,000 live births and is the leading genetic cause of infant mortality. SMA Type 1 is the most severe form of the disease. Without the SMN protein, infants rapidly lose motor neurons after birth, impacting all muscle functions, including breathing, swallowing and basic movement. If left untreated, most babies with SMA Type 1 die or require permanent breathing support by their second birthdays. AVXS-101 is delivered as a single one-time dose and addresses the underlying genetic cause of SMA. It works by replacing the missing or defective SMN1 gene with a working copy, which starts making SMN protein immediately and continuously, thereby promoting survival and proper functioning of motor neurons.

Manufacturing a gene replacement therapy is a highly complex process that differs substantially from manufacturing a small molecule. AveXis is one of the first companies in the world to have built a novel facility and created a manufacturing process for a gene replacement therapy that can be easily replicated. Without any existing infrastructure or process, AveXis, with the collaboration and support of a dedicated, integrated team of employees and partners, was able to convert a warehouse to a fully-operational, state-of-the art GMP facility – manufacturing through primary packaging – in only 15 months, while simultaneously creating all of the functions needed to operate the company.

By creating a new paradigm to manufacture a gene replacement therapy at commercial scale in a first-of-its-kind facility that uses cutting-edge, modular, portable, single-use technology, AveXis has developed a potentially transformational therapy that could change the treatment of SMA and bring hope to the community affected by this devastating genetic disease.

ISPE congratulates the AveXis team for their achievements and their FOYA award winning entry for an Honorable Mention. Learn more about the 2019 FOYA Honorable Mention winners.

Has your company recently designed, built or renovated a state-of-the-art pharmaceutical or biotechnology facility that is best in its class? ISPE is now accepting entries into the 2020 ISPE Facility of the Year Awards (FOYA) Program, and your facility may be eligible to apply. Learn more about the Facility of the Year Awards submission process and your company could be the next Facility of the Year Awards Category winner.

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Facility of the Year Awards Banquet

Join ISPE and prominent industry leaders as we recognize the 2019 Facility of the Year Awards (FOYA) Category Winners for their innovation and creativity in pharmaceutical and biotechnology facility design, construction, and operation, at the FOYA Banquet on the evening of Sunday, 27 October 2019 in Las Vegas, NV during the 2019 ISPE Annual Meeting and Expo.