Faculty

The Penelope Booth Rockwell Professor in Biomedical Research, professor of microbiology & physiological systems, director of the Horae Gene Therapy Center and co-director of the Li Weibo Institute for Rare Diseases Research

Dr. Gao is researching rAAV-based recombinant vaccines for COVID-19. The goal is to develop vectors that are capable of continually expressing soluble inhibitors of SARS-CoV-2. This would prevent the virus that causes COVID-19 from entering and infecting cells in the airway and lungs.

An international leader in gene therapy research, Gao has played a key role in developing adeno-associated virus (AAV) gene therapies for a range of rare genetic disorders such as amyotrophic lateral sclerosis (ALS), Rett syndrome and Canavan disease.

Gao Lab

Professor of RNA therapeutics

The Khvorova lab is investigating a cocktail of siRNA and antisense RNA oligonucleotides that are specific for SARS-CoV-2, which in concept could be delivered to the macrophages and epithelial cells in the lungs and target the mRNA of the virus to significantly reduce replication of the virus in those cells.

Dr. Khvorova is a leading expert in the biochemistry of RNA therapeutics. Scientists in the Khvorova lab identify the chemical and biological properties that drive small RNA tissue distribution, retention, cellular uptake and biological availability. Their goal is to deliver oligonucleotide and RNA-based therapies to tissues other than the liver through chemical engineering. By screening a wide range of chemically engineered and naturally occurring bioactive conjugates, they have already identified novel chemical modalities that support delivery of robust amounts of RNAs to the heart, kidneys, muscle, placenta, vasculature and brain—tissues previously not targeted by RNAi.

Khvorova Lab

Professor of medicine and executive vice chancellor of MassBiologics

MassBiologics has previously demonstrated that human monoclonal antibodies are able to prevent infection with a related coronavirus, SARS CoV-1, and can be used for treatment of SARS-CoV-1 infections. Dr. Klempner has identified two human monoclonal antibodies that neutralize SARS-CoV-2 , including one that blocks binding of SARS-Cov-2 to its ACE2 receptor. Current efforts are directed at producing sufficient quantities of these antibodies for testing in an animal model of COVID-19.

Another research effort at MassBiologics involves IgA antibodies, which are stabilized by the mucous that lines the respiratory tract. Klempner and colleagues are developing IgA antibodies for delivery directly to the respiratory tract for both prevention and treatment of COVID-19.

Another approach being studied at MBL focuses on single-domain antibodies, also known as nanobodies—antibody fragments consisting of a single monomeric variable antibody domain that access unique sites on coronaviruses. Current efforts are directed at testing candidate nanobodies for cross neutralization and for in vivo testing against several coronavirus isolates.

Diagnostics

In collaboration with investigators at the New York Public Health laboratories at Wadsworth New York, MassBiologics has designed, engineered and manufactured three antigens associated with virus naturalizing antibodies.

The David J. Freelander Professor in AIDS Research and professor of molecular medicine

The Luban lab is moving rapidly into the study and analysis of the virologic mechanism of COVID-19 infection, seeking to understand what makes its mechanism unique and why it might be susceptible to antiviral drugs like chloroquine. Dr. Luban’s 2016 paper on the singular infectivity of Ebola virus in Cell presaged the need for a more precise understanding of infectivity of novel viruses like SARS-CoV-2.

Luban Lab

The Gladys Smith Martin Chair in Oncology, professor of biochemistry & molecular pharmacology and director of the Institute for Drug Resistance

Dr. Schiffer is collaborating with Paul Thompson, PhD, to identify and design antiviral drugs for SARS-CoV-2, using high-throughput drug screening and innovative structure-based drug design.

Schiffer studies how viruses become resistant to drugs and uses a variety of research tools to determine how changes in the biochemistry of a drug can make resistance less likely.

Schiffer Lab

Professor of biochemistry & molecular pharmacology and director of the Chemical Biology Interface Program

Dr. Thompson is collaborating with Celia Schiffer, PhD, to identify and design antiviral drugs for SARS-CoV-2, using high-throughput drug screening and innovative structure-based drug design. One area of particular promise is the identification and development of protease inhibitors specific to the virus. Thompson’s lab has vast experience in small-molecule screening, where researchers test thousands of potential therapeutics against infectious agents like viruses. Thompson’s identification of a family of enzymes critical to the development of autoimmune diseases helped advance the understanding of the biochemistry and biological effects of these enzymes.

Thompson Lab

Associate professor of RNA therapeutics

Scientists in the Watts lab are developing viable mechanisms for delivering small interfering RNAs (siRNA) to the lungs, which can significantly reduce the expression of the SARS-CoV-2 viral genes.

The Watts lab is developing new approaches to several classes of oligonucleotides with the goal of harnessing the chemistry and biology of these molecules to help make oligonucleotides an effective therapeutic for disease. Oligonucleotides are short DNA or RNA molecules that have a wide range of applications. By synthesizing oligonucleotides that can bind to the messenger RNA, it may be possible to effectively turn off genes that cause illness and disease.

Watts Lab

Associate professor of microbiology & physiological systems

Dr. Xie, collaborating with Guangping Gao, PhD, is developing rAAV-based recombinant vaccines for COVID-19. Xie researches AAV vector technology and AAV-mediated miRNA therapeutics.