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Open Aperture is Apertura’s new material transfer program, which aims to get human-relevant gene therapy capsids into the hands of qualified academic researchers quickly and efficiently. Dave Greenwald, Executive Chair of Apertura’s board and Managing Director at Deerfield Management, shares his perspective on why this program is needed — and what kinds of discoveries may lie ahead.

I did my PhD long enough ago that “gene therapy” was still something you had to explain at dinner parties, and CRISPR had not yet entered the scientific vocabulary. At the time, I was working in the lab of Rajendra Kumar-Singh at Tufts University School of Medicine, focused on gene therapy for ophthalmic diseases. My thesis work centered on retinitis pigmentosa, a monogenic loss-of-function disorder that causes blindness in thousands of people worldwide.

Even then, it was clear to me that gene therapy worked in principle. The limiting factor seemed to be whether a therapeutic payload could be delivered efficiently to the right cells. If you could get the payload where it needed to go, gene therapy stood an excellent chance of working. Delivery, not biology, was often the gating factor to efficacy.

Several years after finishing my program, I worked as a Director of Business Development at Johns Hopkins Technology Ventures. In this role I worked with many other academic technology transfer offices. It soon became clear to me that despite having a well-resourced technology transfer office, material transfer agreements (MTAs) were often slow, cumbersome, and in many cases never fully executed. While these agreements are essential for sharing scientific tools and assays across institutions, they frequently introduce months of delay. For researchers pushing new technologies forward, and for families waiting for meaningful progress, that lost time matters.

From Broad to biotech

In 2019, I met Ben Deverman of the Broad Institute. We discussed the work his lab was doing to engineer novel adeno-associated viruses (AAVs) with improved cellular tropism and manufacturability. It was immediately clear that our interests were aligned, and with financial and operational support from Deerfield Management, we founded a new company to democratize access to the best capsids for genetic medicine delivery.

Since launching Apertura, the company’s guiding principle has been to maximize the impact gene therapies can have for patients and their families. This principle was foundational from the outset, and it is one that every member of the Apertura team has shared. We believe the most effective way to advance the field is by building a broad, collaborative ecosystem that includes patient groups, academic laboratories, biotech companies, CMOs, CDMOs, and pharmaceutical companies. We also believe that scientific rigor and data transparency are incredibly important.

Closing the licensing gap

In academic research, mouse-specific capsids are often used to demonstrate proof of concept. As programs mature, researchers seek access to human-relevant capsids to support the next phase of development. However, licensing state-of-the-art capsid technology typically requires substantial upfront financial commitments that are simply not feasible for most academic labs. When this happens, promising programs can stall at exactly the moment when momentum matters most.

This is why we created Open Aperture, our material transfer program. Open Aperture is designed to provide qualified academic researchers with no-cost, non-commercial access to TfR1 CapX, our novel AAV capsid targeting human transferrin receptor (TfR1). TfR1 CapX is a second-generation capsid that builds on Ben’s research (published in Science), demonstrating significantly improved CNS delivery compared to our first-generation capsid, BI-hTFR1.

We built Open Aperture with practicality in mind. The program includes a standardized agreement, guidance on CMC and preclinical considerations, and introductions to experienced vendors, allowing research teams to focus on executing high-quality science rather than navigating unnecessary administrative overhead.

Opening the aperture

Our goal is straightforward: to reduce friction and compress timelines so that promising CNS gene therapy concepts can be evaluated quickly and rigorously.

We cannot do this alone. Developing better delivery technologies is not sufficient if access to them remains constrained. There are far more credible payload ideas than any single organization can pursue, and we believe that the fastest way to learn which will ultimately benefit patients is to enable rigorous work across the broader research community.

By collaborating with academic labs, we aim to advance a new generation of genetic medicines capable of treating a wide range of diseases. When our partners succeed, the field succeeds.

If you are an academic investigator working on CNS gene therapy and would like to learn more or apply to Open Aperture, start here. We look forward to expanding the field’s perception of which genetic diseases are treatable.