Based in Philadelphia, Aro Biotherapeutics is a biotechnology company pioneering the development of tissue-targeted genetic medicines with a platform based on a proprietary protein technology called Centyrins. Earlier this month, Aro announced the closing of an $88 million Series A financing. The proceeds from the Series A will be used to advance the company’s lead therapeutic candidates into clinical development, with an initial focus in rare genetic and immune disorders. I caught up with Aro Biotherapeutics Susan Dillon, Ph.D., co-founder and CEO to learn more about mass production of Centyrins, their line of RNA-based therapies, and more.
Aro Biotherapeutics is known for its work with Centyrins. Why are they better suited for mass production, and how does this affect the patient?
Centyrins can be produced at high yields in bacterial strains (E coli) used for manufacturing small proteins, which is different than standard monoclonal antibody products which are typically produced in mammalian cells. Manufacturing in E coli tends to be less complex, faster and more cost effective than manufacturing in mammalian cells. While the manufacturing process itself won’t have a direct impact on patients, other attributes of Centyrins — in particular, their binding to specific cells, small size and stability in the intracellular environment — could have a meaningful impact on efficacy and safety for patients, along with the potential to be administered at lower doses with more convenient (sub-cutaneous) formulations vs other products.
Briefly explain Aro’s line of RNA-based therapies.
Aro is developing a new class of medicines called Centyrin-oligonucleotide conjugates where Centyrins can target the oligonucleotide to a specific cellular address. In doing so, we can overcome one of the greatest challenges in the RNA space, which is how to ensure that these promising therapies can achieve sufficient activity in extra hepatic disease tissues without causing excess toxicity in other tissues. In our early studies in animal models, we’ve seen very promising gene modulation data working with both antisense oligonucleotides and siRNA.
Aro is said to double its staff based on its most recent round. What positions will likely be added, and why?
We have ambitious goals for the future development of both our platform and our pipeline and to achieve these goals, we will need to increase the size of our scientific team. The vast majority of new hires will be scientists who can help us accelerate our drug discovery efforts. Over time, as we approach human clinical trials, we will also need to build out a team focused on clinical development of therapeutics.
It was stated that Aro plans to focus on drugs for rare genetic diseases before moving onto immune system diseases. Which rare diseases will Aro focus on and why?
We haven’t publicly disclosed the specific indications that we will pursue, but our initial efforts will be in the genetic muscle disease area in addition to rare autoimmune disorders. First and foremost, the diseases where Aro will focus are areas of high unmet medical need, where we believe we can have a transformational impact on patients. Beyond this guiding principle, we’ve also looked at other factors – biological rationale, clinical/regulatory path, competition and so on – in prioritizing our lead indications.
Who are Aro’s local/regional partners? Explain the symbiotic nature of the partnerships
As a relatively small scientific team, Aro’s success is dependent on establishing a network of partners in greater Philadelphia/NJ (as well as across the US and Europe) to both expand our capacity for managing multiple projects as well as to bring new capabilities on board. We collaborate with both large contract research organizations and with many smaller more specialized firms that have expertise in screening and in various disease models.