Apertura Launches Leveraging Harvard, MIT Gene Therapy Platforms: Updated

Apertura Platform Developers_Apertura

From Left: Apertura Developers Ben Deverman and Michael Greenberg/Courtesy of Apertura

With $67 million in Series A financing, newly-launched Apertura Gene Therapy hit the ground with a goal to develop genetic medicines using platform technologies that address key limitations of gene delivery and expression.

Based in New York, Apertura was founded on full-stack platform technologies developed by the Broad Institute of MIT and Harvard and researchers from Harvard Medical School. The platform is designed to engineer novel capsids, gene regulatory elements and payloads that are able to address key limitations of gene delivery and expression. Developed in the labs of Ben Deverman from the Broad Institute and Harvard’s Michael Greenberg, the platform harnesses machine learning capabilities to develop next-generation gene therapies that are expected to offer greater translational potential.

Greenberg said one of the key challenges with current gene therapies is ensuring the therapeutic payload is expressed at the correct level in target cells. The technology that is the basis of Apertura’s approach overcomes that 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,” Greenberg said in a statement.

“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,” Deverman said of the Broad Institute, which is the scientific founder of Apertura.

Dave Greenwald, the acting CEO of Apertura and vice president of business development at Deerfield, said the gene therapy platform provides Apertura with the potential to innovate next-generation therapies across delivery vectors, as well as in expression and payload capabilities. The technology platform provides Apertura with the opportunity to “address technical challenges that have prevented gene therapy from reaching its full potential.”

Dave Greenwald_courtesy of AperturaIn an interview with BioSpace, Greenwald said Deerfield became familiar with Deverman’s work following a 2017 collaboration with the Broad Institute. Deerfield and Broad forged the research partnership to solve “complex, early-stage therapeutic challenges related to serious unmet medical needs.”

By harnessing the platform capabilities, Greenwald said it’s “all about making better, safer drugs for serious diseases.” The company is not disclosing potential targets at this time but he shared that Apertura's technology provides it with a broad opportunity to target rare diseases.

“There are more than 7,000 rare diseases and most of those are caused by a genetic mutation of some kind. There are lots of opportunities out there,” he said.

Greenwald was also quick to point out that a small company like Apertura cannot target every rare disease on its own. He said the company will be open to partnerships with biopharma companies large and small, as well as potential academic institutions.

Kristina Wang_courtesy AperturaKristina Wang, director of corporate development and a board member at Apertura, noted that a significant number of the known rare diseases are out of the reach of existing gene therapy technologies. Apertura’s next-generation technologies should create new target opportunities, she said.

Although no specific targets have yet to be announced, Greenwald said in the coming weeks Apertura will begin to make some announcements regarding preclinical data. He said that the company will announce some findings as it prepares to participate in upcoming scientific conferences.

The Apertura platforms are capable of simultaneously engineering AAV capsids to exhibit enhanced cellular tropism and the evasion of pre-existing immunity. In addition to the technology platforms that form the basis of its gene therapy program, Apertura also secured methods of identifying cell-type-specific genetic regulatory elements (GREs) from Harvard and also gained access to the Harvard-developed Paralleled Enhancer Single-Cell Assay (PESCA) platform.

The GRE platform is designed to focus on genetic regulatory elements and enhancers that drive cell type-specific expression, disease state-specific expression, and tunable expression levels, the company said. These capabilities are expected to enable Apertura to develop best-in-class gene therapies.

“Our platform has the potential to unlock many new indications for gene therapy,” Wang said in a statement. “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.”

The Series A was backed by Deerfield Management Company, which also committed additional operational support to the startup in order to bolster the company’s ability to advance gene therapy discoveries.

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