Description:
047 Development of Microbiome-based Products for Infectious Diseases
Fast-Track proposals will be accepted.
Direct to Phase II proposals will be accepted.
Number of anticipated awards: 1-2
Budget (total costs):
Phase I: $225,000 for up to one year
Phase II: $1,500,000 for up to 3 years
Background:
The majority of microbiome research to date has been largely descriptive and has focused on the characterization of the microbiome composition. However, there has been a recent shift in the microbiome field to focus on the functional capacity of the microbes present. This shift in microbiome research has led to the development of potential microbiome-based products for use as therapeutic interventions, thus presenting a need for innovation in the characterization and preclinical development of this novel product class. Currently, several microbiome-based products consisting of live bacteria are being developed as therapeutic interventions for vaginal and enteric infectious diseases. These products can be very complex, oftentimes originating from human stool or other diverse microbial communities. As such, there are many challenges associated with the preclinical development of these complex microbiome-based products. New and innovative ways to conduct IND-enabling studies (e.g. characterization assays to support lot release) are now needed to further advance these products for future human clinical trials.
Project Goal:
The goal of this project is to enable small businesses that have an existing microbiome-based product (consisting of live microorganisms, such as bacteria) intended for the treatment or prevention of infectious diseases to further their product development by focusing on preclinical studies. In particular, small businesses are encouraged to focus on IND-enabling studies to support the characterization, manufacture and release using product-specific assays. Focus should be on characterizing the product in terms of identity, genetic stability, purity, potency, transference of genetic material, and mechanism(s) of action. New methods to set appropriate specifications are also needed. In addition, novel methods to manufacture complex microbial ecosystems and raw materials are encouraged. Finally, novel formulations such as spray drying, lyophilization, and packaging of microbiome-based products for long-term stability are encouraged. The following FDA document, entitled “Early Clinical Trials with Live Biotherapeutic Products: Chemistry, Manufacturing, and Control Information”, should serve as guidance to small businesses seeking to submit proposals on this topic.
Phase I activities can include but are not limited to:
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• Development of novel analytical methods to fully characterize individual and combinations of components (e.g. live microorganisms, such as bacteria) of microbiome-based products.
• Development or conduct of assays to support lot release testing, such as identity or potency testing.
• Development or conduct of assays to demonstrate product stability.
• Development of methods and analytical technologies to support chemistry, manufacturing and control information, such as formulation, encapsulation, and lyophilization.
Phase II activities can include but are not limited to:
• Development of methods and analytical technologies to support chemistry, manufacturing and control information, such as formulation encapsulation and lyophilization.
• Scale-up formulation activities that may help support future clinical trials.
• Conduct of appropriate safety (e.g. toxicology) studies of formulations intended for clinical evaluation in the appropriate systems.
• Conduct of long-term stability studies to ensure product shelf life.
• Validation of assays to support lot release testing.
This SBIR will not support:
The design and conduct of clinical trials (see http://www.niaid.nih.gov/researchfunding/glossary/pages/c.aspx#clintrial for the NIH definition of a clinical trial). For clinical trial support, please refer to the NIAID SBIR Phase II Clinical Trial Implementation Cooperative Agreement program announcement or the NIAID Investigator-Initiated Clinical Trial Resources webpage.
Studies focused on discovering new microbiome-based products.
A new indication for an existing microbiome-based product (e.g. probiotics).
