Partnering to Expand the Impact of Novel Genetic Medicines
By: Kristina Wang, Director of Corporate Development
During my graduate studies at Harvard, I took the unconventional path for someone with a business background and picked up pipettes to work in a lab. I leapt at an opportunity to work on a next-generation gene regulation technology because I believed it would bring the transformative benefit of gene therapies to a broader set of diseases. I am delighted to spin out the gene regulatory element (GRE) platform I worked on in the Greenberg lab at Harvard Medical School and build Apertura Gene Therapy as its first employee alongside world-class scientists in the Deverman lab at the Broad Institute and our investors at Deerfield Management. While there were many challenges on this journey, my conviction of the power of gene therapy remains steadfast.
Apertura is industrializing and scaling the technology platforms originated from the Deverman and Greenberg labs to develop novel vectors that overcome the delivery and expression limitations of current gene therapy technologies. Through the combination of capsid and gene regulatory element engineering platforms, we aim to deliver genetic payloads to the right cells and tune payload expression to the right levels. We envision shifting the field from the existing paradigm of using natural serotype capsids and strong ubiquitous promoters for all diseases to using novel capsids and regulatory elements engineered for the target product profile for a genetic disease.
A core tenet of our philosophy at Apertura is to enable the delivery of as many differentiated gene therapies to patients as possible. In that vein, we aim to augment our platform impact through both internal development and partnerships to address diseases where there are few or no meaningful treatment options for patients. As we believe the future of gene therapy requires strong collaboration among all stakeholders, Apertura strives to be the partner-of-choice for novel capsids and regulatory elements for gene therapies. We seek to work closely with drug developers of all sizes to rapidly develop best-in-class capsids and regulatory elements for gene therapy programs, and we are fully committed to move the field forward. When our partners are successful, we will be successful.
When considering partnering with Apertura, a question we often receive is, “how is your technology different?” Our capsid and GRE engineering platforms deploy a massively high throughput screening strategy in generating quantitative and reproducible data that enable machine learning (ML)-guided predictions of function. This enables us to deeply sample the sequence space to optimize vectors for multiple functions (e.g., tissue tropism, translation across species, and manufacturability) rapidly and systematically. We apply this ML-guided approach in engineering novel capsid tropisms through both function-focused and mechanism-focused assays to increase our confidence in translation to humans. In parallel, we are expanding the repertoire of gene regulatory elements to finely tune payload expression by looking beyond promoters to naturally evolved and synthetic enhancers, repressors, microRNAs, and other regulatory elements.
The time is right to innovate at the intersection of protein engineering, gene regulation, machine learning, and next-generation sequencing. Having assembled a phenomenal team at our state-of-the-art lab in the Cure. building in New York City, we’re ready to accelerate our impact to patients. We welcome partnerships to address the challenges in gene delivery and expression. Let’s work together to develop transformative gene therapies to treat devastating disorders.
Contact us if you are interested in collaborating with Apertura to bring transformative gene therapies to patients.