Intracellular Therapies and Oligonucleotide Applications
Karina Thorn, Ph.D.,
Corporate Vice President, RNA and Gene Therapies,
Global Research Technologies
Genetic medicine is moving beyond the rare disease space according to Novo Nordisk, which says DNA and RNA therapies will soon be available for a broader range of illnesses.
Rare diseases have always been a major focus for developers of “genetic medicines” – drugs that enter the cell and treat or cure diseases at the DNA or RNA level. From uniCure’s pioneering Glybera through to more recent products like Spark’s Luxturna and Novartis’ Kymriah, most approved gene therapies target diseases affecting just a handful of patients.
But things are changing according to Dr. Karina Thorn, Ph.D., Corporate Vice President, RNA and Gene Therapies, Global Research Technologies, Novo Nordisk, who told delegates at TIDES Europe use of genetic medicines is expanding.
“We no longer see this as only a rare disease space…We see more and more examples of these types of genetic medicines being applied outside of the rare disease space” she said, citing oncology, cardiometabolics and diabetes as examples of areas were activity is increasing.
And there are opportunities for those with the right genetic medicine technologies according to Thorn. “We are looking into expanding the technologies and our ambition is really to help transform gene therapy from [a treatment for] the few today to the many tomorrow.”
Intracellular Opportunities
To that end, NovoNordisk is reappraising its intracellular gene therapy enabling technologies to identify those with potential to be used to treat common diseases.
Thorn cited Novo Nordisk’s GalXC technology – an RNAi drug platform the firm acquired with Dicerna Pharmaceutical in 2021 – as an example
“It has many good features when we think about this technology as a drug. It has subcutaneous administration, it is readily soluble in water. We have the long duration of action, maybe not the PK as it will disappear from the blood circulation pretty fast, but it has long duration of action in the target tissue.”
“And what is also good with this is that it is a very fast platform, so that when we start looking into various types of genetic targets of origin, we will run these cassettes of one or multiple genes for a certain disease signal and can quickly find some hits to validate.”
She cited the genes LXRa and MARC1 as examples, explaining two RNAi drugs targeting these genes have entered clinical trials.
“We could probably spend the whole presentation of each one of these targets if we wanted to dive into the therapeutic potential and the overall discovery of these RNAi molecules.
“But the reason that I brought them here today was more to give examples of how we are approaching getting new targets into what we think is the right fit for our drug modality platform.”
“So these are examples of how we are looking into applying the technology platforms that Novo and others have [more widely].”
COVID Experience
Thorn also cited learnings from the COVID-19 RNA vaccine space as a factor driving the expanded use of genetic medicine, particularly in terms of how drugs based on such technologies could be commercialised.
“Some thought that [COVID mRNA vaccines] could not be produced at scale, but the pandemic demonstrated if there's a will, there's a way. Now we really start to see the application of what this technology can do at scale,” she said.
