Amicus, Paragon to Collaborate on Gene Therapies for Batten, Other Diseases

Amicus Therapeutics entered a collaboration with Paragon Gene Therapy, a unit of Catalent Biologics, for the production and development of potential gene therapies to tackle lysosomal storage disorders (LSDs), including Batten disease.

They will concentrate on advancing gene therapy programs that are at a preclinical stage — those  that have not yet moved to clinical testing in patients. Particular focus will be given to the program for Pompe disease, a lysosomal disorder.

Another important part of Amicus’ pipeline focuses on gene therapies for different types of Batten disease. Potential therapies for CLN8 disease are at preclinical stage; those for CLN1 disease, also known as infantile Batten disease, are under discovery.

Gene therapies for CLN3 disease and CLN6 disease are more advanced, with two Phase 1/2 clinical trials underway — one is currently enrolling children ages 3–10 with CLN3 (NCT03770572); the other is testing a gene therapy in children and adults with CLN6-associated Batten disease (NCT02725580).

Under the new collaboration, Paragon will run a platform for the production of adeno-associated viral (AAV) particles — commonly used carriers for gene therapy delivery. The AAV platform will be used in Amicus’ preclinical testing, with plans to leverage it for clinical supply and commercial product as well.

Paragon has two facilities in Baltimore dedicated to processing development through commercial manufacturing of the most promising AAV platforms. Since 2016, the company has completed more than 100 clinical AAV batches across 40 programs. Amicus is collaborating with the University of Pennsylvania throughout this process.

“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,” John F. Crowley, chairman and CEO of Amicus, said in a press release.

Crowley added, “Advancing a robust manufacturing process and supply is critical to our success. 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.”

Pete Buzy, president of Paragon, said: “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 holds long-term agreements with industrial suppliers of plasmids — circular DNA molecules widely used in genetic engineering and in the transfer of genes to cells — to support advancement of all current gene therapy programs.

The company recently established a Global Research and Gene Therapy Center of Excellence in Philadelphia to evolve its science, research, and gene therapy capabilities.