Amicus opens gene therapy ‘research engine’ in Philadelphia

By Ben Hargreaves

- Last updated on GMT

(Image: Getty/FlySnow)
(Image: Getty/FlySnow)

Related tags Amicus Therapeutics Gene therapy Pennsylvania

Amicus announces the opening of a gene therapy center that has been established in partnership with the University of Pennsylvania.

The 75,000-square-foot research space, known as the Global Research and Gene Therapy Center of Excellence, is part of Amicus Therapeutics’ ongoing gene therapy partnership with the University of Pennsylvania (Penn).

The latest update to the partnership sees the opening of the US facility, which will contain 200 researchers and drug developers focused exclusively on the development of gene therapies, with the newly opened area containing offices and ‘state-of-the-art’ laboratories.

A previous part of the agreement sees Amicus hold the worldwide rights for the ‘majority’ of treatments discovered to treat lysosomal storage disorders, as well as a further 12 rare disease areas, including Rett Syndrome, Angelman Syndrome and selected other muscular dystrophies.

According to John Crowley, CEO of Amicus, the Philadelphia area is a “magnet for talent in gene therapy”​ and, on a Q4 investor call, he referred to the partnership with Penn as a “tremendous research engine for the future growth of [the company].”

Beyond this latest lab opening, Crowley provided an update on the progress on other areas within the company’s portfolio of gene therapies. In particular, he noted that CLN6, Amicus’ drug candidate for Batten disease, which is currently at the Phase I/II stage, would post results of 13 patients in the second half of the year.

Beyond this, the company needs to ready “lots of other activities,”​ which includes a number of activities around manufacturing.

The company is partnered with Thermo Fisher Scientific on this work, where Crowley confirmed that tech transfer and scale up activities are nearly complete.

In terms of building out its own capacity to manufacture such products, Daphne Quimi, CFO for the company, confirmed that the company plans “to take a phased approach to build out [its] facilities”​ and internal manufacturing capabilities.

A spokesperson for Amicus did not respond to a request for further information on the most recent update.

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