Advanced technology platforms

Novartis is investing in new technologies and platforms that offer more targeted approaches to fighting – and, in some cases, potentially curing – serious diseases. We have the depth and scale to discover, develop and commercialize therapies using these advanced platforms.

Radioligand therapy delivers precision-targeted radiation to cancer cells widely disseminated in the body, with the goal of limiting damage to surrounding tissue. It has the potential to address a wide range of cancers and become a major pillar of cancer care.

Novartis has built a strong foundation in this area with Lutathera, our first approved radioligand therapy, and with177Lu-PSMA-617, our investigational therapy for prostate cancer (see “Oncology” on previous page). In 2021, we acquired fibroblast associated protein (FAP) targeted assets from iTheranostics. We launched a collaboration with Artios to explore novel combinations of radioligand therapy and DNA damage pathway inhibitors and with PeptiDream to work on macrocyclic and constrained peptides. We also established a partnership with Molecular Partners to work on DARPin protein-based binding domains. We have more than 15 research and clinical programs underway to identify and accelerate the next generation of radioligand therapies for cancer.

In gene therapy, one of our technologies uses benign viruses called adeno-associated viruses (AAVs) to deliver genes to cells inside the body. The goal is to repair the cells with a one-time treatment. Our first approved AAV-based therapy was Zolgensma for SMA, and we are now exploring experimental forms of gene therapy for other diseases, from brain disease to blood disorders. Altogether we have more than 20 research programs in gene therapy.

The Novartis early-stage pipeline features a growing arsenal of optogenetic gene therapies, based on genetically engineered light-sensitive proteins that can rewire cells in the eye, allowing them to act as replacement photoreceptors. In 2020 and 2021, we obtained two key optogenetic technologies through the acquisitions of Vedere Bio and Arctos Medical. The resulting therapies could potentially treat blinding diseases such as inherited retinal dystrophies and age-related macular degeneration, which affect millions of patients worldwide.

With cell therapy, a patient has key cell types extracted and genetically modified in a clinical lab before being injected back into the body. An example of this is CAR-T therapy, a treatment generated from a patient’s own T-cells. Novartis was the first pharmaceutical company to significantly invest in pioneering CAR-T research and initiate global CAR-T trials. Our flagship CAR-T therapy, Kymriah, was the first cell therapy approved in the US for certain kinds of leukemia and lymphoma, and is now available to patients in 30 countries. We aim to broaden the impact of CAR-T technology to help patients with a variety of difficult-to-treat hematological cancers and solid tumors.

Novartis has a resilient global CAR-T manufacturing footprint, spanning seven facilities across four continents. In 2021, we announced the development of a novel manufacturing platform – created at NIBR – called T-Charge, which is expected to improve the therapeutic potency of the T-cell product while halving the time needed to take cells from the patient, manufacture the product, and return it to a healthcare professional for administration.