Apertura Gene Therapy launched this week with a Series A financing of up to $67M from Deerfield Management Company. The start up plans to develop genetic medicines using platform technologies that address key limitations of genetic medicine delivery and expression. Deerfield Management has also committed additional operational support.
Apertura is founded on a pair of platform technologies developed in the labs of Ben Deverman, PhD, Senior Director of Vector Engineering and Institute Scientist at the Broad Institute of MIT and Harvard, and Michael Greenberg, PhD, the Nathan Marsh Pusey Professor of Neurobiology and Chair of the Department of Neurobiology at Harvard Medical School (HMS). The company’s platform leverages machine learning and high-throughput assays to engineer novel capsids, gene regulatory elements, such as promoters and enhancers, and payloads to simultaneously enhance multiple functions of gene therapies for greater translational potential.
“With these platform technologies from the Broad Institute and Harvard University, Deerfield saw an opportunity to bring together and support a unique and comprehensive platform that could address technical challenges that have prevented gene therapy from reaching its full potential,” said Dave Greenwald, PhD, Acting Chief Executive Officer of Apertura and Vice President, Business Development at Deerfield Management Company. “While next-generation approaches to gene therapy have largely focused on the innovation of delivery vectors, Apertura has the potential to innovate simultaneously across delivery, expression, and payloads.”
Ben Deverman, Scientific Founder of Apertura, said, “When developing a gene therapy, it has been common to use naturally occurring serotype AAV capsids. The technology we have developed uses proprietary assays and machine learning to design custom AAV capsids that have the chosen characteristics for treating specific diseases, and we believe this approach will result in new and effective gene therapies.”
Apertura has certain exclusive rights to AAV capsids developed in the Deverman Lab at the Broad Institute.
A separate sponsored research and licensing agreement with Harvard University, spearheaded by the Harvard Office of Technology Development, grants Apertura exclusive access to certain methods of identifying cell type-specific genetic regulatory elements (GREs), including access to the Paralleled Enhancer Single-Cell Assay (PESCA) platform, developed in the Greenberg Lab at Harvard University.
“A major challenge in developing effective gene therapies is having the payload of the therapy expressed at the correct level in target cells,” said Greenberg. “The technology we have developed at Harvard Medical School overcomes this hurdle by targeting transgene expression to specific cell types, fine-tuning expression levels in these cells, and, at the same time, avoiding expression of the transgene in non-target cell types.”
The start-up’s two technology platforms have capabilities to simultaneously engineer AAV capsids to exhibit enhanced cellular tropism, evasion of pre-existing immunity, while maintaining and potentially improving manufacturability. The company’s platform focuses on GREs and enhancers that drive cell type-specific expression, disease state-specific expression, and tunable expression levels. The company believes these capabilities together could enable Apertura to develop best-in-class gene therapies designed for specific indications.
“Our platform has the potential to unlock many new indications for gene therapy,” said Kristina Wang, Director of Corporate Development and Board Member of Apertura. “We aim to maximize our impact through dedicated internal programs and meaningful partnerships with other biopharma companies and academic groups.”