Gene Therapy Saved Me – Lovelace Biomedical’s CSO recalls what gene therapy research has done not only for the company, but for himself as well


Gene Therapy Saved Me. I mean, not literally…but certainly from a figurative standpoint. In 2016 I took on the task or rebranding Lovelace Biomedical from the Lovelace Respiratory Research Institute. The company had a long history of performing Gene Therapy and other Regenerative Medicine programs. But nobody but us knew it! In fact, we were doing Gene Therapy before it was popular, way back in 2007 when we were established as a Center by the NIH. We were working with the Wilson lab and other pioneers in the industry, cutting our teeth on rare diseases such as Pompe, alpha-1-antitrypsin and other disorders. There was very little FDA guidance at the time. It was us, some innovators and the FDA working together to help design programs to advance promising new indications with little precedent.

I would often get asked the question, “can you guys dose by non-inhalation routes?” I always thought it was a bit ironic, as inhalation is among the most challenging routes and we were being asked if we could also do standard routes of administration in nonclinical studies that we were already performing every day.

Fast forward to 2016. We were faced with rebranding a company known mostly for excellence in inhalation toxicology, infectious diseases and medical countermeasure development.We needed a hook. Something to allow us to communicate that while we are everything people thought of us, we were so much more and we just need a message to communicate that to the world. In parallel, Gene Therapy started to rapidly explode in the biotechnology segment. Aha! Here we are an organization that had been specializing in an esoteric field (Gene Therapy) that all of a sudden started to explode with promise. Lovelace Biomedical can dose your gene therapy by inhalation!…And to the CNS, ear, heart, brain, and in a Doctor Seuss kind of way – wherever else you want to put it (I often tongue and cheek say that Gene Therapy is all about complicated dosing and then figuring out where it went).

So we started to build a story in this area, and it has been a fantastic area for us. We have invested further in our capabilities to augment what we have been working on since 2007, and established a core competency that is helping many companies advance their Gene Therapies from concept to clinic.

Last spring these opportunities came full circle. I was at the annual ‘Meeting on the Mesa’ specialty meeting in San Diego. I sat in awe thinking about the technological advances, and how fast they were coming. The idea that we are looking for transformative changes potentially in the way we administer medicine. After spending decades thinking about how to develop medicines to treat people, we were helping companies that are looking for cures! What a fantastic time to be in science, and an unbelievable opportunity to play a small role in the companies that are innovating in this space. In many ways, Gene Therapy has saved me.

-Dr. Jacob Mcdonald, CSO