NEW YORK & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apertura Gene Therapy, a biotechnology company opening opportunities for treating debilitating diseases with limited options for patients, today announced that it has launched with a Series A financing of up to $67M from Deerfield Management Company 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 to further strengthen the companys ability to advance gene therapy discoveries.

Apertura is founded on a pair of platform technologies developed in the labs of Ben Deverman, Ph.D., Senior Director of Vector Engineering and Institute Scientist at the Broad Institute of MIT and Harvard, and Michael Greenberg, Ph.D., the Nathan Marsh Pusey Professor of Neurobiology and Chair of the Department of Neurobiology at Harvard Medical School (HMS). The companys 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, Ph.D., 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 powerful 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 Greenberg labs PESCA platform was advanced to commercial readiness through the strategic support of the Q-FASTR program at HMS and the Blavatnik Biomedical Accelerator at Harvard University.

The two technology platforms have unique capabilities to simultaneously engineer AAV capsids to exhibit enhanced cellular tropism, evasion of pre-existing immunity, while maintaining and potentially improving manufacturability. The companys GRE platform focuses on GREs and enhancers that drive cell type-specific expression, disease state-specific expression, and tunable expression levels. These capabilities together are expected to 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. Committed to advancing the field of gene therapy, Apertura seeks to collaborate broadly to accelerate impact to patients.

About Apertura Gene TherapyApertura is a biotechnology company opening opportunities for treating currently intractable diseases. We are uniquely positioned to develop genetic medicines by simultaneously engineering AAV capsids, genetic regulatory elements, and payloads to overcome limitations in cellular access, gene expression, pre-existing immunity, and manufacturability. Apertura is committed to growing the field of gene therapy and believes that together we maximize our impact by working with corporate and academic partners, patients, and foundations. Founded on technologies from the Broad Institute and Harvard University, and with support from Deerfield Management Company, the company is based at the Cure, Deerfields innovation campus in New York City. For more information, please visit our website at http://www.aperturagtx.com and follow us on LinkedIn and Twitter.

About Deerfield ManagementDeerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy. The Firm works across the healthcare ecosystem to connect people, capital, ideas and technology in bold, collaborative and inclusive ways. For more information, please visit https://deerfield.com/.

About Harvard Universitys Office of Technology DevelopmentHarvards Office of Technology Development (OTD) promotes the public good by fostering innovation and translating new inventions made at Harvard University into useful products that are available and beneficial to society. Our integrated approach to technology development comprises sponsored research and corporate alliances, intellectual property management, and technology commercialization through venture creation and licensing. More than 90 startups have launched to commercialize Harvard technologies in the past 5 years, collectively raising more than $4.5 billion in financing. To further bridge the academic-industry development gap, Harvard OTD manages the Blavatnik Biomedical Accelerator and the Physical Sciences & Engineering Accelerator. For more information, please visit https://otd.harvard.edu.

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Apertura Gene Therapy Launches with $67M Series A Financing from Deerfield and an Innovative Technology Platform to Develop Genetic Medicines -...