Biomedical & Human Health

Richard Sayre, Spartina Biotechnologies, NMC Research Scientist

Spartina Biotechnologies is developing a safe, fast-acting new antiviral therapeutic called SIREN™, not a vaccine, but a therapeutic that blocks the ability of viruses to replicate and activates the innate immune system response within the patient within hours.

SIREN technology directly activates the innate immune response while simultaneously blocking viral replication through the targeted and effective delivery of RNA interference (RNAi) molecules to virus infected cells. This technology addresses the major challenges that have impeded the application of RNA interference (RNAi) technology in the past including, optimization of RNAi molecular structures for targeted viral gene silencing, stable delivery of RNAi molecules to the patient in an effective dose, and specific targeting of RNAi molecules to virus infected cells.

See the Spartina Biotechnologies webpage for more information.

Thomas Terwilliger, LANL

X-ray crystallography and single particle cyro-EM at near atomic resolution, generate high quality atomic models at near atomic resolution, continue to extend the Phenix infrastructure to support both novice and expert researchers.

The major goals of this project are: (1) fully automated interpretation of density maps produced by cryo-EM and low-resolution crystallographic methods, (2) obtaining substantial improvements in algorithms for determination of very challenging structures by experimental phasing, and (3) adding local information from molecular dynamics simulations in structural analysis as a complementary source of information about structural variability.

The focus of this research has been the development of algorithms allowing automated interpretation of cryo-EM maps. Terwilliger has developed tools for map sharpening, map segmentation, and for fully automated interpretation and model-building from cryo-EM maps, and this research will continue the work of building and improving the models.

The purpose of this work is to provide tools for researchers and pharmaceutical companies to visualize the 3D structures of proteins and other macro-molecules that they are interested in.  For example, pharmaceutical companies use the Phenix software to visualize the binding of drugs that they are developing to proteins that are the targets of those drugs.

Bette Korber, NMC Affiliate Researcher, LANL Staff Scientist
Elena Giorgi, Postdoctoral Research Associate, Los Alamos National Laboratory

This HIV Phase I vaccine trial is a human trial to test the immune response and safety of an HIV vaccine insert designed by Bette Korber at LANL. The vaccine antigen design is an attempt to contend with HIV’s diversity. Korber has assisted with the statistical and analysis plan, designed testing reagents, and helped with the FDA approvals throughout the process. This project will evaluate the vaccine response in terms of the ability to cross react with different strains of HIV.

Bette Korber, NMC Affiliate Researcher, LANL Staff Scientist
James Theiler, Laboratory Fellow, Los Alamos National Laboratory

The goal of this program is to support the preclinical development of an HIV vaccine carried in an HCV vector. This program is a collaboration with the Picker laboratory at the University of Oregon.We have developed computation tools to design HIV inserts for the unusual vector.

Kshitij Wagh, NMC Affiliate Researcher, LANL Staff Scientist

This program is in collaboration with Dr. David Montefiori at Duke University. Our role is to assist in the analysis of broadly neutralizing antibodies and antibodies with other functionalities against HIV variants,design reagent panels for global vaccine trials, and to help develop and analyze antibody reagents for therapy and prevention.