Research and gene therapy centre
Amicus Therapeutics built a global research and gene therapy centre of excellence in Philadelphia, Pennsylvania, US.
The facility was developed to serve as the headquarters of the company’s science organisation and to host the leadership team working on gene therapy.
The new facility was announced in February 2019 and completed in March 2020. It provides approximately 200 new jobs and allows the company to advance its pipeline of medicines for patients with rare metabolic diseases by boosting its gene therapy research capabilities.
The new research and gene therapy centre is located in a 14-storey 3675 Market Street building, spread across 345,000ft², in the uCity Square mixed-use community in Philadelphia.
The 6.5 million square feet uCity Square community houses offices, residential, retail, clinical, and office spaces, as well as laboratories.
Philadelphia, which is emerging as a biotechnology and gene therapy research hub, helps to advance the company’s gene therapy activities. The city’s close proximity to the company’s global headquarters in Cranbury, New Jersey, US, strengthens external collaborations to deliver high-quality medicines.
The site also offers easy reach to the University of Pennsylvania and Penn Medicine, which hosts some top gene therapy researchers, and hospitals in Philadelphia. Amicus also intends to extend its collaboration to other institutions, such as Drexel University.
Amicus’ new facility occupies 75,000ft² of office and laboratory space across the top three floors of the 3675 Market Street building, developed by Wexford Science & Technology in collaboration with the University City Science Center and Ventas.
The building currently houses a number of innovation centres, including CIC Philadelphia, [email protected] Philadelphia, Quorum, Venture Café Philadelphia, FirstHand, and other science centre commercialisation programmes.
A team of Amicus’ researchers is currently based in a temporary space in the building as a client of [email protected] Philadelphia, a co-working space with a wet laboratory and office facility. Researchers moved to the new facility after its completion.
The new facility is used for research and development (R&D) of biotechnology and gene therapy to treat rare metabolic diseases.
Amicus collaborated with the Perelman School of Medicine at the University of Pennsylvania on Gene Therapy Program in October 2018.
Partners carry out research and development work to innovate gene therapies for the treatment of Pompe disease, Fabry disease, CDKL5 deficiency and other rare metabolic disorders.
The collaboration develops AAV gene therapies using the University of Pennsylvania’s adeno associated virus (AAV) gene transfer technologies and the company’s protein engineering expertise.
Amicus acquired ten pre-clinical and clinical-stage gene therapy programmes developed at the Center for Gene Therapy at the Research Institute at Nationwide Children’s Hospital and the Ohio State University for the treatment of lysosomal storage disorders in children.
Amicus is a biopharmaceutical company focused on the discovery and development of high-quality medicines for the treatment of rare metabolic diseases.
The company developed certain technologies, including a pharmacological chaperone technology, enzyme-targeting technology, and proprietary Chaperone-Advanced Replacement Therapy (CHART®) technology to develop novel gene therapies for lysosomal storage disorders.
It has one FDA-approved drug in its portfolio, Galafold, indicated for the treatment of Fabry disease. The lead product candidate in the company’s pipeline is AT-GAA, which is being investigated to treat Pompe disease.
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