Rocket Pharmaceuticals : Lonza Supporting Rocket Pharma with Clinical Manufacturing of LAD-I for the Treatment of Leukocyte Adhesion Deficiency-I

Basel - Lonza today announced that a clinical manufacturing agreement is in place with Rocket Pharmaceuticals, Inc., a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders.

The agreement covers development and manufacturing for LAD-I, a phase 1/2 candidate for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I).

The companies have been working together since 2018 and decided to make the collaboration public in parallel to the successful Preliminary Data from Phase 1/2 Trial announced by Rocket on December 9th, 2019.

The agreement covers the manufacturing of clinical product leveraging Lonza's lentiviral vector platform. Manufacturing takes place in both the Houston (TX), USA and Geleen, Netherlands Lonza cGMP manufacturing sites. The agreement also includes analytical assays and development services.

Autologous hematopoietic stem and progenitor cells (HSPCs) are transduced ex vivo with a lentiviral vector (LV) containing the ITGB2 gene encoding for the human CD18 receptor (2 integrin subunit). The therapy aims to treat Leukocyte Adhesion Deficiency-I (LAD-I). LAD-I is a rare immune disorder characterized by low or absent neutrophil CD18 expression, predisposing affected individuals to recurrent and fatal infections in childhood. Initial results from the first patient treated with RP-L201 demonstrated early evidence of safety and potential efficacy.

About Lonza

Lonza is a leading global supplier to the pharmaceutical, biotech and specialty ingredients markets. We work to promote a healthier lifestyle and prevent illness by supporting our customers to deliver innovative medicines that help treat or even cure a wide range of diseases. This is complemented by our broad range of microbial control solutions, which help to create and maintain a healthy environment.

Patients and consumers benefit from how we apply our scientific knowledge and advanced manufacturing technologies to the healthcare, hygiene and fast-moving consumer goods markets and to developing preservation and protection materials. Founded in 1897 in the Swiss Alps, Lonza today is a well-respected global company with more than 100 sites and offices and approximately 15,500 full-time employees worldwide (at the end of 2019). The company generated sales of CHF 5.9 billion in 2019 with a CORE EBITDA of CHF 1.6 billion.

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Additional Information and Disclaimer

Lonza Group Ltd has its headquarters in Basel, Switzerland, and is listed on the SIX Swiss Exchange. It has a secondary listing on the Singapore Exchange Securities Trading Limited ('SGX-ST'). Lonza Group Ltd is not subject to the SGX-ST's continuing listing requirements but remains subject to Rules 217 and 751 of the SGX-ST Listing Manual.

Certain matters discussed in this news release may constitute forward-looking statements. These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved. Investors are cautioned that all forward-looking statements involve risks and uncertainty and are qualified in their entirety. The actual results may differ materially in the future from the forward-looking statements included in this news release due to various factors. Furthermore, except as otherwise required by law, Lonza Group Ltd disclaims any intention or obligation to update the statements contained in this news release.

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