Laboratory of Nanomedicine & Biomaterials

Nanotechnology has generated a significant impact in nearly every aspect of science. Research in the Farokhzad Laboratory seeks novel nanomaterials and nanotechnologies in order to develop advanced drug delivery systems with the promise to improve health care.

Targeted nanoparticle therapies for cancer and cardio disease.
Targeted nanoparticle therapies for cancer and cardiovascular diseaases.

Highly interdisciplinary and translational, our research is focused on multifunctional, nanoparticle-based drug delivery systems. We seek to improve nanoparticle synthesis and formulation and its therapeutic efficacy. Additionally, we develop robust engineering processes to accelerate translation of nanoparticle-based drugs into the drug development pipeline. At the same time, we emphasize a fundamental understanding of the interface between nanomaterials and the biological system, all in order to aid in nanoparticle drug development.

Developing Targeting Moieties for Nanoparticle Drug Delivery

Molecules that target biomarkers of a specific disease play an important role in nanoparticle drug delivery. These molecules, used in nanoparticle delivery systems, increase nanoparticle in vivo efficacy and significantly affect the biodistribution and pharmacokinetics of the uptake of such particles. We seek to develop advanced technologies, such as bacterial phage display and SELEX, in order to discover targeting moieties of high specificity and affinity, including small molecules, peptides, antibodies, and aptamers. Using these molecules, we can develop novel conjugation techniques and engineering processes to formulate particles that target a variety of diseases.

Systemic delivery of nano-burr.
Systemic delivery of ‘nano-burr’ to injured vasculature.
Therapies Using Biocompatible and Biodegradable Polymeric Nanoparticles

Using biocompatible and biodegradable polymers for targeted drug delivery not only improve drug safety, but also significantly shorten the timeline of drug development. We are particularly interested in using PLA- and PLGA-based drug delivery systems and strive to improve their therapeutic efficacy. We also develop novel chemistry to conjugate and encapsulate a variety of small molecule drugs. We explore methods that will further functionalize polymer backbones so that the polymer is suitable to carry both hydrophobic and hydrophilic therapeutic agents. In addition, we rationally design nanoparticle platforms that could facilitate the development and translation of entirely new classes of bioactive macromolecules such as siRNA.

PEG with observable shedding
PEG with observable shedding.
Understanding the Interactions of Nanomaterials and Biological Systems

We also emphasize a fundamental understanding of how nanomaterials interact with biological systems, such as cancer cells and cardiovasculature. The performance of nanoparticle drugs often depends on the sensitive interplay between the multiple functionalities of the nanoparticle and its response to the biological environment, which, in turn, can impact the transport, distribution, and drug release of the nanoparticles. The knowledge of how nanoparticle properties change with correspondence to the biological environment can aid in the design of nanoparticle drugs. Using this knowledge, we can then control the physical chemical properties, composition, and formulation process of the nanoparticles themselves.

Clinical Translation

Over the past 8 years, we have pioneered robust engineering and high throughput screening of multifunctional nanoparticles for medical applications. The technologies that we have developed with collaborators at Harvard Medical School, DFCI and MIT have formed the foundation for the launch of three biotechnology companies that are developing: (i) a new class of targeted nanoparticles for treatment of important human diseases such as cancer and cardiovascular disease; (ii) a new class of synthetic nanoparticle vaccines for prophylactic and therapeutic applications; and (iii) a new class of integrative combination nanomedicines for oncology, inflammation/pain, and infectious diseases. The targeted nanoparticles for the treatment of solid tumors, BIND-014, entered Phase I human clinical trials in January 2011, marking the first-in-human testing of a targeted polymeric nanoparticle for cancer chemotherapy (ClinicalTrials.gov Identifier: NCT01300533). Initial results as published in SCIENCE Translational Medicine demonstrated that in patients with advanced solid tumors, BIND-014 displays remarkable pharmacological properties and cases of effective tumor shrinkage. In November 2011, a nicotine nanoparticle vaccine, SEL-068, entered the clinical trials for smoking cessation and relapse prevention, and the clinical translation of SEL-068 marked the first-in-human testing of targeted polymeric nanoparticle vaccine (ClinicalTrials.gov Identifier: NCT01478893).

Research Funding

The Laboratory of Nanomedicine and Biomaterials research is currently supported by Prostate Cancer Foundation ($5,000,000 / 2007-2013), National Cancer Institute ($370,945 / 2010–2015), National Heart, Lung and Blood Institute ($1,438,035 / 2010-2015), National Institute of Biomedical Imaging and Bioengineering ($2,562,105 / 2012-2016), and Korean National Science Foundation (Global Research Laboratory-GRL) ($564,895 / 2012–2018). Dr. Farokhzad also serves as mentor for post-doctoral fellowships and other awards. The currently funded fellowships/awards are as follows. Dr. Jinjun Shi received an NIH K99/R00 career development award (2011–2016). Dr. Xiaoyang Xu received an NIH NRSA Fellowship (2012-2015). Dr. Giuseppe Palmisano received a two-year Innovation Award from the Danish Minister of Science, Technology and Innovation from the Danish Research Council (2012–2014); Lili Zhao, Visiting Scholar received a two-year scholarship through the China Scholarship Council (2012–2014); Xi Zhu, Visiting Scholar received a two-year scholarship through the China Scholarship Council (2012–2014); Renata Ferreira de Carvalho Leitão, Visiting Associate Professor received a one-year fellowship from the Brazilian National Council for Scientific and Technological Development (CNPq) (2013).

Additional Information

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