Translational Discoveries

Beginning in the late 1970s and 1980s, Timothy A. Springer, PhD, discovered and characterized adhesion receptors that allow immune cells to recognize one another, adhere to blood vessels, and migrate into tissues. His laboratory’s discoveries led directly to new therapeutic strategies and FDA-approved medicines for inflammatory and autoimmune diseases.

In 1993, Dr. Springer founded LeukoSite to translate discoveries in leukocyte adhesion and trafficking into new medicines. He subsequently co-founded biotechnology companies including Scholar Rock, Morphic Therapeutic, Tectonic Therapeutic, and Seismic Therapeutic, extending research in integrin biology, growth-factor regulation, structural biology, and immunology toward therapeutic development. He was also a founding investor in Moderna, whose scientific origins are described separately below.

In 2017, Dr. Springer and Andrew Kruse, PhD, established the nonprofit Institute for Protein Innovation to expand access to high-quality synthetic antibodies and accelerate research in protein science.

In 2003, the laboratory of Judy Lieberman, MD, PhD, provided the first demonstration that siRNA-mediated gene silencing could protect against disease in an animal model. By using small interfering RNA (siRNA) to silence a disease-related gene, the work provided an early therapeutic proof of principle for RNA interference and helped establish a path toward the development of siRNA-based medicines.

Derrick Rossi, PhD, a former PCMM investigator and principal faculty member of the Harvard Stem Cell Institute, co-founded Moderna in 2010. His laboratory developed an approach using modified messenger RNA to direct cells to produce desired proteins while avoiding the cellular antiviral response. This work helped establish the scientific foundation for a new class of mRNA-based therapeutics.

Dr. Rossi later co-founded other biotechnology companies, including Intellia Therapeutics, Magenta Therapeutics, and Stelexis Therapeutics.

MERFISH (Multiplexed Error-Robust Fluorescence in situ Hybridization) was first reported in 2015 by Xiaowei Zhuang, PhD, Jeffrey Moffitt, PhD, and colleagues at Harvard University. MERFISH enables highly multiplexed measurement of RNA abundance and spatial organization in intact cells and tissues.

In 2019, Dr. Moffitt co-founded Vizgen to commercialize MERFISH and make spatially resolved transcriptomic measurements broadly available to researchers

In 2020, Hao Wu, PhD, and Judy Lieberman, MD, PhD, pioneers in the study of the inflammasome pathway, were among the academic founders of Ventus Therapeutics. The company applies structural biology, immunology, and biophysics to the discovery of small-molecule medicines targeting innate immune pathways. Ventus is advancing clinical programs that include the cGAS inhibitor VENT-03 in a Phase 2a trial for lupus and the brain-penetrant NLRP3 inhibitor VENT-02 in a Phase 2a trial for Parkinson’s disease.

In 2023, the Wyss Institute for Biologically Inspired Engineering, Boston Children’s Hospital, and Northpond Labs entered into an agreement to support the “Lab-on-a-Molecule” project led by Wesley Wong, PhD, and colleagues. The project uses DNA nanoswitch technology developed in the Wong laboratory to create a high-throughput platform for identifying compounds that alter the function and interactions of disease-relevant proteins. The goal is to accelerate the discovery of new drug candidates and provide a path toward commercialization.