Lonza Expands Partnership with Rocket Pharmaceuticals for Childhood Disorder Treatment

January 30, 2020

Clinical manufacturing of the therapy will take place at Lonza's Houston, TX, and Netherlands cGMP manufacturing sites.

Lonza announced on Jan. 28, 2020 that it is expanding its clinical manufacturing agreement with New York City-based Rocket Pharmaceuticals for the development and manufacture of RP-L201, a Phase I/II candidate for the treatment of leukocyte adhesion deficiency-I (LAD-I), a disorder that causes recurring and fatal infections during childhood.

Although the companies have been in collaboration since 2018, the partnership only became public in December 2019 after Rocket Pharma published positive preliminary data from a Phase I/II trial of the drug, according to a Lonza press release. Clinical manufacturing of the therapy will take place at Lonza's Houston, TX and Netherlands cGMP manufacturing sites. The agreement also involves analytical assays and development services.

“We are firm believers and supporters of the science developed by Rocket Pharma. Our close collaboration with this team started over two years ago and has enabled the delivery of life-saving treatments to young patients left with no other options,” said Alberto Santagostino, senior vice president, head of Cell and Gene Technologies, Lonza Pharma & Biotech, in the press release. “With the presentation of the preliminary data from the first of these patients, Rocket Pharma is now demonstrating the potential of its novel platform technology to treat rare and devastating diseases successfully with gene therapies. We are fully committed to continuing to enable Rocket Pharma to deliver these treatments to many more patients in the [United States] and in Europe, as they progress towards commercialization."

Source: Lonza