Catalent has started making viral vectors to support trials of PBGM01, a gene therapy for the rare lysosomal storage disease GM1 being developed by Passage Bio. 

The US contract development and manufacturing organization (CDMO) set up a dedicated manufacturing suite at for Passage at its facility in Harmans, Maryland in August.

Work since then has focused on bringing the suite online and validating it for the production of the AAVhu68 vector. The vector is used to deliver a modified DNA molecule encoding a functioning version of the enzyme b-gal that is defective in GM1.

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A Passage spokesman told us “Clinical supply for the GM1 Phase I/II trial is already in place through Passage Bio’s ongoing partnership with our CDMO Catalent.

He added, “We have also begun manufacturing operations to support adeno-associated virus (AAV) production for all of our lead product candidates through the completion of a dedicated CGMP manufacturing suite at Catalent Cell & Gene Therapy’s facility in Maryland. It will support clinical trials for lead programs through early commercialization.

Passage signed a supply and manufacturing deal with Catalent in 2019. Under the deal the CDMO agreed to use Pall’s iCELLis single-use fixed-bed bioreactor to make vectors.

Flexibility

The bioreactor technology provides a scalable alternative for the cultivation of adherent cells and – according to the firms – is capable of meeting demand for both clinical and commercial-scale volumes.

Catalent also will be involved in delivering candidates to clinical trial sites through its packaging, labelling and distribution service.

The collaboration with Catalent combined with an existing agreement with the University of Pennsylvania’s Gene Therapy Program will help Passage tailor suppliers to market needs according to company chief technical officer Alex Fotopoulos.

“Investing in dedicated CGMP manufacturing infrastructure at Catalent, augmented by our access to vector supplies, technology, and expertise at Penn, provides us the flexibility and capacity to advance multiple programs in parallel and to rapidly deliver supplies to support clinical trials worldwide.

“Our ability to create an agile, global supply chain as we advance our lead product candidates positions us for clinical and commercial success, enhancing our competitiveness in the gene therapy arena.”