With the company expected to graduate from the BioEscalator in 2025 – the incubator’s model is to nurture start-ups for up to three years before they expand elsewhere – we talked to co-founder Dr Nenad Svrzikapa recently about how Orfonyx Bio started and its ambitions to ‘own’ the protein upregulation area. Discussions also touched on how the BioEscalator has helped its growth and we were pleased to hear that there’s ‘no better place’ for life sciences start-ups in Oxford!
The right time for Orfonyx Bio
The inception of Orfonyx Bio was catalyzed by four factors coming together to create the ideal environment, Nenad said. The first of these is the massive increase in the understanding of the regulatory elements in genes, coupled secondly with the variety of mechanisms with which we can intervene at the nucleic acid level as developed over the past three decades. This includes antisense oligonucleotides, exon skipping and gene editing – all showing the tractability of nucleic acids for therapeutic benefit.
Third, Nenad and co-founder Associate Professor Thomas Roberts have deep experience of the oligonucleotide space. Finally, once it became clear that their APEX platform (see below) had clinical potential, forming a company was a clear next step. Perhaps this isn’t such a surprise, given that the company was conceived in the laboratory of co-founder Professor Matthew Wood, serial entrepreneur and Chair of the BioEscalator Management Board. In addition, Nenad’s experience at the US companies Alnylam Pharmaceuticals (a ‘trailblazer’ in RNA interference) and Wave Life Sciences (a pioneer in advancing stereopure genetic medicines which he helped establish) surely played a role too.
The potential of Orfonyx Bio's APEX technology to have clinical impact. Source: Orfonyx Bio
Technology progress and leveraging connections at the University of Oxford
Having made progress at the BioEscalator with the APEX platform, which generates oligonucleotide modulators (APEX-ASO in the figure above) of novel genetic sequences which upregulate protein expression, Orfonyx Bio is finding ‘very exciting targets’ to exploit. Close connections with the University of Oxford have brought benefits via a sponsored research collaboration and relationships with the Department of Chemistry and the Institute of Developmental and Regenerative Medicine. One of the objectives of the BioEscalator is to enhance and support the University’s cutting-edge research through interactions such as these.
More BioEscalators needed
Nenad is very positive about the start-up experience at the BioEscalator (‘more BioEscalators are needed’!), highlighting the access to research facilities and technical support it provides. This access is a ‘big deal’ for companies as it allows them to focus funds on the science rather than additional equipment and reduces the amount of space they need. Orfonyx Bio also appreciates being on the Old Road Campus and the academic research proximity it brings, as well as the BioEscalator team’s ability to link them to the Oxford innovation ecosystem and alumni of the incubator. A recent meeting in which a BioEscalator graduate company talked about planning a corporate move was timely – and eye-opening – for Orfonyx Bio as it learned about how to transition to other environments and the care it takes to get it right.
Building the technology and the team
The next few months are busy for Orfonyx Bio as it closes its next financing round, moves to new premises and continues to exploit the APEX platform. Proceeds from the fundraising will be used to advance R&D towards nomination of its first drug candidate and into IND-enabling studies. The Orfonyx team will be strengthened and built out too as the company matures, complementing the skills of Executive Chair Dr Jonathan Jones (ex-Aviado Bio, CRISPR Therapeutics) and CSO Dr Stuart Milstein (ex-Alnylam Pharmaceuticals, Korro Bio andSenda), two highly experienced leaders in the genetics medicines space.
We’ll be watching the company’s progress with interest as it continues to grow!