This Small Business Innovation Research (SBIR) Phase I project proposes to develop a new technology for monitoring aggregation in therapeutic protein solutions that will have wide-ranging impact in the pharmaceutical and biotechnology industries. Protein aggregation is a major problem across these sectors engaged in developing new protein pharmaceuticals. Protein aggregation can elicit immune responses against the protein itself, severely reduce bioavailability, and is a significant hurdle in the drug development pipeline. This new technology can simultaneously and quantitatively monitor the stability of many independent protein formulations for hours, days, months, or longer, all under the control of a single instrument and without any significant human intervention. Because the technology is based on laser light scattering it provides a rigorous means of monitoring the protein aggregation process, based on fundamental laws of physics, and not on empirical or inferential means. The implementation and use of this technology could revolutionize drug discovery, formulation, and quality control dimensions and accelerate development of new drugs while making existing ones safer and more efficiently produced. The technology can be used wherever proteins are under development for fighting diseases such as cancer, Alzheimer's, diabetes, and various forms of cardiovascular disease.
The broader impact/commercial potential of this project, if successful, will be the value to the pharmaceutical industry in that it can potentially accelerate not only research and discovery of new drugs, but also increase their production efficiency and safety, thereby expanding their availability to the public. The technology also has applicability in many other sectors; e.g. monitoring natural polymers, such as dissolution of gelatin used in food or pharmaceutical applications, flocculation of impurities by water purification polymers, entrapment of oil by surfactants and other agents used in combating oil spills, and more. The quantitative kinetics of molecular weight changes during all these processes will allow deeper understanding of the fundamental physical and chemical forces that drive them. Therefore, many commercial and scientific opportunities exist for the proposed technology platform, yielding the potential for significant impact across multiple industries. With demonstrated feasibility on applications in protein therapeutics and early adoption by several key users, the initial market for this technology and instrumentation could expand quickly, and sales could easily reach $10M within several years of delivering a commercial product. The overall market for the technology will increase in time as other application sectors are developed.
APMT’s project was centered on feasibility research of its Simultaneous Multiple Sample Light Scattering (SMSLS) technology which is expected to become an important research tool for pharmaceutical and biotechnology researchers in both industry and academia. SMSLS offers scientists a novel, high throughput way to analyze several key characteristics and identify problems found in the discovery and development of new protein therapeutics, especially as regards aggregation of proteins, a widespread problem in the field and a great hindrance towards achieving clinically effective biologic drugs. The project was very successful and achieved all original goals in the proposal, and also pushed the technology into new, unexpected directions, and identified additional market opportunities beyond biotechnology applications. For now, APMT will continue to maintain focus on biotechnology and pharmaceutical applications, but expects to approach characterization of nanoparticles, synthetic and natural. APMT produced abundant measurable progress throughout the project. Should APMT be awarded an SBIR Phase II grant, APMT has concrete plans to continue pushing the development of the SMSLS technology towards a commercial product. A high level bulleted list of results from the SBIR Phase I and IB project is found below. Feasibility of several new features were developed and validated for the SMSLS platform, including protein aggregation behavior with respect to stirring stress and liquid/air interfaces, and detection of the formation of micron sized particles in protein formulations 2 new provisional patent applications were filed (August 2013 and May 2014) 2 journal publications were made using results from the project 1 book chapter publication was produced using some results from the project 4 invited presentations at major biotechnology industry conferences and at a large biotechnology company collaborator were made A new 16-cell SMSLS prototype with accompanying software was developed and assembled, bringing the technology much closer to a beta product Extensive data were gathered, validating the technology on multiple different types of protein samples from industrial partners and academic collaborators
