Over the past decade, I’ve had the privilege of being on the leading edge of the evolution of gene therapy as it entered the forefront of innovative science in biotech. When I first joined Spark Therapeutics in 2013, gene therapy was re-emerging after a decade-plus in academic hibernation as a therapeutic modality. The field was once again warming up to the tremendous potential to not only treat the symptoms of a disease, but to effectively cure patients and enable them to live disease-free. A stronger understanding of the human genome, increased access to capital and improved technology led to a de-risking of the field and a resurgence of gene therapy players.
Throughout my tenure at Spark, I was incredibly fortunate to witness firsthand the power of gene therapy and the profound impact it can have on patients and their caregivers. Leading the teams who brought the first approved gene therapy for a genetic disease across the finish line in the U.S. and Europe taught me a great deal about what it takes to advance a genetic medicine through clinical development, regulatory approval, and commercialization. Seeing the life-altering impact that these therapies have on patients – helping them live more fulfilling, disease-free lives – is a truly rewarding experience, and one that I hope to accomplish at Apertura.
Since those early days at Spark, the field of gene therapy has exploded with over 1700 active studies registered on www.clinicaltrials.gov. The emergence of several safety- and efficacy-related challenges also surfaced in that time period, which severely limited the field from realizing the full potential of gene therapy. At Apertura, we are focused on tackling these challenges by harnessing the power of the platform technologies originating from Ben Deverman’s lab at The Broad Institute and Michael Greenberg’s lab at Harvard. We believe these platforms offer unique approaches to engineering the gene therapy vector to avoid many of the safety signals in the field today and widen the field’s opportunity to many genetic diseases. This effort will require advances on many fronts including engineering capsids that express more tissue-specific tropism and evade pre-existing neutralizing antibodies, and developing novel gene regulatory elements to drive gene expression in a cell-type specific and inducible manner.
I aspire to lead Apertura in pioneering the next wave of genetic medicines. It is a priority for us to ensure that the novel discoveries from these platforms make their way into the gene therapy ecosystem with an eye toward maximizing patient impact. Not only do we aim to build an internal pipeline of programs and therapeutics that harness the power of these platforms, but we also will partner with large pharmaceutical and biotechnology companies and smaller organizations such as non-profits, academics, and patient groups to enable and support growth of the field at large.
As we continue to establish Apertura and bring the future of gene therapy into focus, I hope to foster an environment and company culture that effectively collaborates, values high science, and holds the patient as a guiding north star. Gene therapy holds enormous promise to treat a broad range of genetic diseases and I believe Apertura can deliver on this promise.
Together with our dedicated team of scientists at Apertura, I am excited to embark on this journey of realizing the full potential of gene therapy and advancing medicines that have the capacity to fundamentally change the progression of debilitating diseases. Visit Apertura’s website to learn more about our team, mission, and technology.
Visit Apertura’s website to learn more about our team, mission, and technology.