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A Tale of Two Platforms – The Formation of Apertura
By: Dave Greenwald, PhD, Vice President, Business Development at Deerfield Management and Board of Managers of Apertura Gene Therapy
“It was the best of times, it was the worst of times, it was the age of wisdom, it was the age of foolishness, it was the epoch of belief, it was the epoch of incredulity, it was the season of light, it was the season of darkness, it was the spring of hope, it was the winter of despair.”
Charles Dickens’ opening lines of A Tale of Two Cities could be used to describe the current status of the gene therapy industry. On one hand, we have several approved gene therapy medicines that are providing life-saving or life-altering treatments to patients. On the other hand, there continue to be serious safety concerns and areas ripe for improvement in the field. I believe the two platforms at Apertura, Fit4Function for AAV capsid engineering which we licensed from Ben Deverman’s lab at The Broad Institute, and PESCA for genetic regulatory element discovery which we licensed from Mike Greenberg’s lab at Harvard Medical School, have the potential to address some of the major scientific and clinical concerns in the AAV gene therapy space.
Dave Greenwald, PhD
Both of these platforms, one from The Broad, and the other from Harvard Medical School, are insightful and cutting-edge technologies. It is our core belief at Apertura that the capsids and regulatory elements we are engineering should be utilized broadly to improve the medicines that will one day be able to address so many serious diseases.
The four critical aspects of Apertura’s platforms that will take us from where we are as a field today to the ‘season of light’ from Dickens are: (1) enhancing cellular tropism to enable organ- and cell-type specific delivery, (2) increasing manufacturability to increase quality of final drug product and lower cost of goods sold, (3) evading neutralizing antibodies, and (4) fine-tuning transgene expression to specific cell types and disease states. These are bold ambitions that the gene therapy field has acknowledged for some time; while they largely evaded previous efforts in the field, we believe the technologies at Apertura provide an opportunity to overcome these challenges.
After joining Deerfield Management Company over four years ago, I was introduced to Ben Deverman through Deerfield’s collaboration with the Broad Institute of MIT and Harvard. Ben is an expert in using machine learning to design AAV vectors for use in gene therapy, and we discussed his ideas about how to overcome the challenges of tolerability and delivery accuracy. From our conversation, it was clear to me that there was a mutual interest and vision for the future of gene therapy, and Ben and I began working on what would eventually become Apertura.
Around the same time, through another existing Deerfield collaboration with Harvard Medical School, Lab1636, I was also introduced to Kristina Wang, Sinisa Hrvatin and Eric Griffith, who were working in Mike Greenberg’s lab at Harvard Medical School to develop gene regulatory elements (GREs) to modify expression of gene therapies in target cell types. Both of these platforms, one from The Broad, and the other from Harvard Medical School, are insightful and cutting-edge technologies. It is our core belief at Apertura that the capsids and regulatory elements we are engineering should be utilized broadly to improve the medicines that will one day be able to address so many serious diseases. To that end, in addition to developing our own product candidates, we intend to partner with the broader gene therapy field – including academic labs, emerging biotech companies and large multi-national pharmaceutical companies.
Apertura’s formation was a combination of the right ingredients. Deerfield’s academic collaborations with the Broad and Harvard Medical School provided the opportunity to meet Ben, Kristina, Sinisa and Eric and initiate our collaboration. This founding team along with Andrew Steinsapir and I share a passion for developing next-generation gene therapies. Deerfield has been a longtime supporter of gene therapy and provided significant operational and financial support to manage initial day-to-day operations of the nascent company – including my initial role as Acting CEO.
Apertura has been a long time in the making and I’m excited to see what the company will achieve in expanding the promise of gene therapy. Collaborating with outstanding academic labs, we’re focused on developing improved genetic medicines that can treat a wide range of diseases and take us from a ‘season of darkness’ to a ‘season of light.’
Check out Apertura’s website for more about how we are leveraging our novel platform technologies to developing genetic medicines to treat debilitating diseases.