We’re Getting Closer to Patients

When we launched iECURE in September of last year, we were embarking on a bold journey to develop potentially curative in vivo gene editing approaches to treat genetically driven liver disorders. And today, we announced a $65 million financing that will ensure that patients suffering with Ornithine Transcarbamylase (OTC) Deficiency will, in the near future, have the potential to access GTP-506, our experimental gene editing treatment through our first clinical study.

 In the last 14 months, we’ve made significant progress. Most importantly, we’ve assembled an experienced team skilled at executing the crucial tasks needed to start clinical testing. Notably, George Diaz, M.D., Ph.D., our therapeutic area lead for urea cycle disorders, is one of the most experienced physicians treating individuals with urea cycle disorders, and he’s working closely with Brad Dickerson, our vice president of project management and patient advocacy, to establish close ties with patient communities as we prepare for clinical development.

 I also want to acknowledge the hard work that Matt Hall, our vice president of clinical operations, and Mark Semanick, our vice president, head of technical operations, have accomplished. Matt has been busy gearing up for our global first-in-human study for GTP-506, engaging with the KOL community and regulatory agencies to determine study design, site selection and building operational infrastructure critical to execute the studies. Mark, on the other hand, has been developing an integrated CMC and technical strategy that will ensure that we have a reliable supply of high-quality drug product for clinical studies and potential commercialization.

In addition to hiring a great team, we’ve achieved many of the milestones that will facilitate our OTC program such as securing both Orphan Drug Designation and Rare Pediatric Disease Designation from the US FDA. Concurrently, our partners at the University of Pennsylvania Gene Therapy Program (GTP) have been generating data that continue to support GTP-506, including long term stability of the edited gene in nonhuman primates.

 We are truly excited to welcome two new investors to iECURE: Novo Holdings A/S and LYFE Capital, which co-led the financing, and we are very grateful for the continued support of our existing investors, Versant Ventures and OrbiMed. With this financing, we’ve added Ray Camahort, Ph.D., Partner at Novo Ventures and Derek Yuan, Ph.D., Managing Director at LYFE Capital to our board. We also added Tal Zaks, M.D., Ph.D., Partner at OrbiMed as a new board member. Stephen Squinto, Ph.D. remains on our board as an independent member.

 I remain more hopeful than ever that our efforts will offer up a clinically meaningful therapeutic for the treatments of disorders such as OTC and Citrullinemia, both of which are inborn errors in the pathway to detoxify ammonia, as well as Phenylketonuria (PKU), another inborn error of metabolism. In addition, we are continually evaluating new opportunities to use a mutation-agnostic approach to treating inborn errors of metabolism.

I am looking forward to providing more good news about our progress.