With the recent conclusion of the Biotechnology Innovation Organization’s BIO 2019 event, we thought we’d talk to some of the fascinating individuals who were present to showcase their innovative ideas or technologies. Irene Rombel, Senior Director, Head of Strategic Analysis – External Innovation, Discovery, Product Development & Supply, at Janssen Research & Development, spoke at BIO 2019 about gene therapy and the next generation of biotherapeutics. We spoke to her about her thoughts on the gene therapy field, and the future for companies in that space.

FLG: You Recently Spoke at BIO 2019 on Gene Therapy and Next-Generation Biotherapeutics. Could You Tell Us More About the Talk, and its Outcomes?

IR: The session was on the challenges and opportunities in the field of in vivo gene therapy, with an emphasis on how technology can be leveraged to develop the next generation of this important emerging class of therapeutics to bring it into mainstream medicine and help more patients.

FLG: Speaking of the Growth of Gene Therapy as a Field: Is that Explosion Starting to Level Out Now, or Will the Field Just Continue to Grow?

IR: The field isn’t levelling out at all – in fact we’re just at the beginning. We’re going from a more or less niche technology that’s difficult to manufacture in a scalable, cost effective manner. Currently gene therapy can only really address Mendelian diseases where the genetics are understood, and the populations are relatively small and well-defined. Going forward, the field will naturally start to expand to address larger disease indications that are typically polygenic in nature, where we’ll have to use different approaches to ameliorate the condition, if not cure it. In doing so, we’ll be bringing the benefits of gene therapy to many more patients.

To bring gene therapy into the mainstream will require investment to further develop the technology. Hence, we are seeing significant investment across the industry, from small academic spin-outs to large pharmaceutical companies. In addition to financing through VCs and public equities, we have seen a lot of licensing and M&A activity.

We’re still in the relatively early days of gene therapy as a medicine, but as I mentioned on the panel we’ve got to get our manufacturing efficiency and scale up, and costs down. To this end, we’re seeing some consolidation to achieve capacity on the gene therapy manufacturing front, as illustrated by the recent acquisitions of Brammer Bio and Paragon by Thermo Fisher and Catalent, respectively. While the issues of scalability, cost of goods and capacity seem a bit daunting for now, keep in mind that we saw the same thing with antibodies about 20 years ago – and look how far we’ve come!

FLG: What About the “Low-Hanging Fruit” in the Field? When Will We See that Starting to Drop?

IR: Low-hanging fruit in terms of Mendelian diseases are the most tractable, and so are the natural place to begin from a scientific and clinical perspective. Also, keep in mind that these include some of the most devastating diseases that cannot be treated with any other therapeutic modality today, so gene therapy may be the only hope for many of these patients for the foreseeable future. Given that gene therapy offers a potential cure for many of these rare diseases, the first therapy that is efficacious and safe may well capture most of the market for a given indication. However, this is still an emerging landscape, it remains to be seen how it plays out.

As I mentioned before, beyond rare genetic diseases there are also opportunities in larger disease indications, albeit they may be more challenging from a scientific, clinical and manufacturing perspective. Bottom line: the low-hanging fruit might go quickly, but there’s still a lot of fruit out there. One thing that came out of the BIO talk is that you shouldn’t try to use gene therapy as a hammer and see every disease as a nail – it’s a tremendously powerful modality that is uniquely able to address some severe unmet patient needs.

Different modalities should be used to treat different diseases. Ideally, you’d have a small molecule to treat everything, but that can’t happen for obvious biological reasons. Hence, for diseases untreatable by small molecules or antibodies, we need to use new innovative modalities such as gene therapy, and there are enough of those previously intractable diseases for a robust, long-term market.

FLG: A Big Problem in this Space is Affordability of Treatment. How Do You See that Changing in the Future?

IR: The issue of pricing and affordability of gene therapies gets a lot of attention mainly because of the large upfront cost, essentially creating a bit of a sticker shock. But these are life-time cures for absolutely devastating diseases, so we aren’t used to thinking about medicine in such terms. The nature of the medicine means that it is intrinsically costlier to produce than a traditional drug, but the cost of goods should go down over time as the manufacturing technologies and processes evolve.

The industry is well aware of this, and that’s something that big pharma can hopefully bring to the table, with expertise in manufacturing, scalability and efficiency. While the emerging pricing paradigm for gene therapies to treat rare diseases has been shown to be commensurate with the value that these innovative medicines bring to patients, this is likely not sustainable once we get to common diseases that affects very large numbers of patients.

This is why we need to develop “Gene Therapy 2.0.” Whether we get there by a big technological disruption, or through a series of meaningful incremental improvements, we’ll get there! Most important, we have a huge incentive – the patients themselves. As our company namesake, Dr. Paul Janssen once said, “Patients are waiting!”