How Novartis R&D is advancing the next breakthrough innovations

“It always starts with patients. What are the biggest unmet patient needs, particularly in disease areas where we have already established ourselves and where we have a deep understanding of that patient community? 

Then we go to the next level, which is understanding the basics of the disease. We're looking at causal biology—what's really driving that disease, and how can we target those causal pathways to bring long-term benefits to patients? Our platform technologies provide us with a range of tools to address diverse biological challenges. I think that puts us in a great position going forward.”

- Fiona Marshall, Ph.D.

“The cell therapy platform is a place that we had pioneered. This is a rapidly evolving field and there are two main areas that are a focus. One is understanding T cell biology, immune biology, and how we can generate cell therapies, or CAR-Ts, that are even more effective. We could do this by making them more stem-like, making them more persistent, and seeing more activity even in more immunosuppressive settings like the solid tumor setting.

The second part of innovation in the cell therapy space focuses on the manufacturing front. This is a very rapidly evolving landscape, from improvements in the design of CAR-Ts themselves to in vivo CAR-T. We are exploring this in our T-charge platform. This platform enables us to develop more stem-like T cells—so better, fitter T cells—which we think could drive longer-term durability of response and more persistent T cells for patients. And then this platform also enables faster manufacturing time.”

- Shiva Malek, Ph.D.

“A critical pillar for us in prostate cancer, in addition to developing innovative isotopes, is to look for novel combinations with our radioligand therapy platform.

One of those opportunities is with an androgen receptor degrader which, when administered, has antitumor effect in its own right, but also is a nice potential combinatorial partner for our RLT. We also have an agent that is a DNA damage repair inhibitor, and we are advancing a small molecule with an epigenetic mechanism of action that may offer additional therapeutic benefits in prostate cancer.

Based on the breadth of this pipeline, spanning different modalities, our goal is to deliver meaningful clinical benefits and extensions in survival for patients with prostate cancer.”

- Mark Rutstein, M.D.

“As a neurologist, I became very familiar with the unmet need in these diseases. But I don't think you need to be a neurologist to appreciate what’s at stake. Nearly everyone has been touched directly or indirectly by neurologic diseases—Alzheimer's, Parkinson's, others—that have limited treatment options. They’re devastating to the patient; they’re devastating to the families.

The reason for optimism is the convergence of several factors, the first being biological understanding. The knowledge we have from genetics, from analyzing human tissue, has put us in a place where we understand better what are the key things that we need to alter in those diseases. Second, we now have therapeutic modalities that can hit those genetically defined targets that in many cases are behind the blood brain barrier or in neuromuscular tissue. And then the last is clinical developability, with improving biomarkers to measure interventions faster and earlier.”

- Bob Baloh, M.D., Ph.D.

“As a dermatologist, and I'm sure for Peter as well as a rheumatologist, when we started out in academia, our treatment choices were limited to broadly immunosuppressive therapies. Now very exciting things have happened. We have made broad steps forward to understanding disease pathology and to understanding the immune system in general. We now have targeted therapies for patients which allow us to treat the root cause of some of these diseases. We still have a lot to do, but we are optimistic that continued advances will enable us to develop more targeted and effective treatments for patients.”

- Angelika Jahreis, M.D., Ph.D.

“So many immune-mediated diseases have common or related pathways, and by targeting the underlying driver of many diseases through a shared mechanism, we can run clinical studies in multiple indications in parallel. Together with Angelika's group, we can thus streamline and accelerate the development process.
At the same time, some immune-mediated diseases are more complex and require different approaches, so we're also exploring multiple therapeutic approaches to personalized care for these patients.”

- Peter Gergely, M.D., Ph.D.

“About 10% of first heart attacks and first strokes are fatal, and survivors are often left with heart failure or permanent deficits. This speaks to the need for prevention, and we have some new ideas about how to go about that.

We're focused on addressing the causal risk drivers of atherosclerosis with semi-annual or annually delivered medicines designed to decrease risk and prevent disease. We recognize the challenges of preventing ASCVD at scale, but we're very excited about the potential of our long-acting medicines to establish a new paradigm, improve adherence, and contribute to a meaningful extension of healthy lifespan.”

- Shaun Coughlin, M.D., Ph.D.

“Here at Novartis, I'm excited because we're on a mission to ensure that no heart is lost too soon and to build a world where preventable heart disease and cardiovascular deaths are no longer part of our everyday reality. And actually, perhaps now the science is genuinely ready to deliver a step change for patients.

Ultimately, our job is to turn this strong biology that we talked about today and the powerful platforms that we shared into durable and meaningful outcomes for patients, and I think we do this through decisive development and an unwavering focus, because I really believe that behind every single one of the data points we've talked about is a person, their family, and their future. And if we get this right, we give people more of those moments that make life worth living.” 

- Ruchira Glaser, M.D., M.S.