Harvard's Office of Technology Development and LabCentral invite you to the latest event in the Guppy Tank series, where Harvard life-science innovators will pitch their super-early-stage concepts to a panel of entrepreneurs and investors for constructive, in-depth feedback.

Please join us on Thursday, March 7, from 4:30 to 6:30 p.m. at Harvard's Northwest Building to hear the presentation, learn from experts on startup formation, and participate in giving audience feedback. The event is accompanied by a reception with food and drinks. All are welcome!

The Wagers Lab developed a new delivery platform – DAEUS – enabling predictable, tunable gene transfer across multiple organ systems in vivo. The research team will discuss their findings and the ability for DAEUS to provide therapeutically meaningful multi-organ gene replacement.

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The Panel:

Steve Tregay, PhD, Managing General Partner at Mission BioCapital

Rebecca Silberman, PhD, Investment Director at RA Capital

Fiona Mack, PhD, Vice President and Head, Cell and Gene Therapy at Bayer Co.Lab Cambridge

Ricardo Dolmetsch, PhD, President at Tempero Bio

About the Project:

To overcome present-day difficulties in the clinical implementation of adenoassociated virus (AAV) vector-mediated gene therapies for neuromuscular, CNS and ophthalmologic genetic disease, we developed a new delivery platform – DAEUS – enabling predictable, tunable gene transfer across multiple organ systems in vivo. We tested DAEUS in preclinical mouse models, including the progeroid disease Wolfram Syndrome II, and find that it provides therapeutically meaningful multi-organ gene replacement. Current gene transfer approaches have generally eschewed treatment of such multi-system diseases because the conventional AAVs on which they rely exhibit tissue-biased tropisms, producing in vivo patterns of transduction that are often mismatched to the clinical need and creating significant safety concerns when undesired tissue targets are favored over the therapeutic target of interest. In contrast, DAEUS’s uniquely modular design enables optimization of the ratio of its components to “program” user-defined levels of transgene expression across different cell types, thereby achieving robust, tunable and predictable gene transfer across major organ systems. This feature allows DAEUS to more closely match endogenous expression profiles, or to deliver higher transgene levels to certain organs and cell types to enhance therapeutic efficacy, positioning DAEUS as a potentially generalizable gene therapeutic platform. Moving forward, we aim to optimize the DAEUS platform and expand its applicability to additional organs and tissues. Once optimized, our platform could enable the treatment of several rare genetic diseases with multi-organ involvement, such as Ehlers-Danlos syndromes, Wilson’s disease, and PTENopathies (such as Cowden syndrome), and may offer a novel approach to reversing the systemic downregulation of key genes and metabolic pathways that appear to underlie certain age-acquired disorders.