CDMO focus: Strategic gene therapy deals and $250m sell-off

Lonza strengthens gene therapy partnership with Prevail; Paragon and Brammer evolving form hyperstacks over to iCELLis units for Sarepta; Mallinckrodt sells off Biovectra. Welcome to Bioprocess Insider’s CDMO round-up.

First up in this contract development and manufacturing organization (CDMO) round-up is Swiss firm Lonza, which is upping its partnership with Prevail Therapeutics.

Lonza has worked on the process development of Prevail’s two lead gene therapy programs since 2018 and will now take the partnership to the next stage, using a baculovirus/Sf9 production system to manufacture Prevail’s pipeline of AAV-based gene therapy programs for patients with neurodegenerative diseases.

Work will take place at Lonza’s gene therapy site in Houston, Texas, the world’s largest dedicated cell-and-gene-therapy manufacturing facility – according to the firm – that opened in April 2018.

“Lonza and Prevail will work together closely on process development and scaling up production of PR001, our gene therapy for Parkinson’s disease with GBA1 mutations and neuronopathic Gaucher disease, to supply late-stage clinical trials and for commercial production,” Prevail CEO Asa Abeliovich said.

“In addition, we look forward to a collaborative relationship to support our PR006 gene therapy program for patients with frontotemporal dementia with GRN mutations, and for future gene therapy programs in our pipeline.”

Sarepta

Sticking with gene therapies, Sarepta Therapeutics has revealed its CDMO partners have been moving from hyperstacks to fixed-bed bioreactors for the production of its candidates looking to treat both Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies.

“We are evolving from hyperstacks over to iCELLis units both at Paragon and Brammer,” Sarepta CEO Douglas Ingram said earlier this month on a call discussing limb-girdle muscular dystrophy type 2e functional results. The firm has long-term partnerships with both Paragon Bioservices, now part of Catalent, and Brammer Bio, now part of Thermo Fisher.

“I will come back and provide additional updates early next year, but we are making very good progress across all of these programs and the most advanced of which from a manufacturing perspective, is of course, our micro-dystrophin program,” he added.

Last month we reported on Sarepta’s efforts on improving yields through the reworking of its analytical and process development efforts.

Mallinckrodt waved goodbye to microbial CDMO

And finally, news from Canada as pharma firm Mallinckrodt enters into an agreement to sell off its third-party microbial manufacturing subsidiary BioVectra. Mallinckrodt acquired BioVectra as part of its $5.6 billion acquisition of Questcor Pharmaceuticals in 2014, but the business was viewed as non-core.

“While we recognize the longer-term growth potential for BioVectra, we believe that the structure of this deal enables us to participate in the future success of the business, and therefore we see this sale as the best option for both Mallinckrodt and BioVectra moving forward,” Mark Trudeau, Mallinckrodt CEO said.

H.I.G. Capital, a private equity investment firm, will pay $250 million for the CDMO business, including 64,000 L of fermentation bioreactor capacity across four manufacturing plants in Atlantic Canada – three in Prince Edward Island and one in Nova Scotia.

“BioVectra demonstrates a tremendous ability to generate robust organic growth and utilizes a broad set of technical capabilities to deliver outstanding service and quality,” said Mike Gallagher, MD at H.I.G. Capital. “They are completing major capital expenditure programs to significantly expand capacity and the company is well positioned to capitalize on growing demand for their services.”