Amicus Partners with Catalent on Lysosomal Disease Gene Therapy Programs

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Metabolic-disease biotech Amicus Therapeutics announced a manufacturing agreement with Catalent’s Paragon Gene Therapy unit for gene therapy manufacturing.

Amicus Therapeutics, Inc. announced on July 2, 2019 that the company has entered into a strategic manufacturing agreement with Paragon Gene Therapy, a unit of Catalent Biologics, for clinical manufacturing capabilities and capacity for multiple active preclinical lysosomal disorder programs currently in development in collaboration with the University of Pennsylvania (Penn). 

Penn has transferred technology to Paragon in the past and will collaborate with Amicus throughout the process, Amicus reported in a press statement announcing the agreement.

Under the collaboration, current research and development production technologies and capabilities related to multiple active preclinical lysosomal disorder programs will be transferred to and developed at Paragon.

The establishment of an adeno-associated virus (AAV) manufacturing platform is intended to advance and establish manufacturing for Amicus preclinical gene therapy programs using Paragon’s manufacturing capabilities for initial clinical supply and commercial product.

The collaboration also will include initial technology transfer and process development to focus on the Amicus Pompe AAV gene therapy program.


“As we continue to articulate our near- and long-term Amicus gene therapy manufacturing strategy, our collaboration with Paragon Bioservices is a significant next step in securing clinical scale-up and supply for our Pompe gene therapy, as well as our other active preclinical programs. Advancing a robust manufacturing process and supply is critical to our success,” said John F. Crowley, chairman and chief executive officer of Amicus in the press statement.  “Leveraging our internal expertise and process development in combination with Paragon’s expertise and platform capabilities, we hope to expedite the process of moving our preclinical gene therapy programs into the clinic as quickly as possible.”

“Amicus has a strong gene therapy pipeline focused on addressing many life-threatening conditions where there are urgent needs for treatments and cures,” said Pete Buzy, president of Paragon Gene Therapy. “Paragon has a proven track record to develop and successfully manufacture complex biotherapeutics using commercially-scalable processes. This agreement with Amicus highlights our world-class manufacturing team and our track record as a center of excellence for gene therapy, trusted by top biopharmaceutical companies.”

Amicus also reported that the company has also established a plasmid supply network and supply agreements to support current gene therapy pipeline programs.


Source: Amicus Therapeutics