New Chemistries for Un-drugged Targets through A Specialized Platform for Innovative Research Exploration (ASPIRE) Collaborative Research Program (UG3/UH3 Clinical Trials Not Allowed) | RFA TR 21 001

Frequently Asked Questions (FAQs)

What is the ASPIRE Collaborative Research Program?

The New Chemistries for Un-drugged Targets through A Specialized Platform for Innovative Research Exploration (ASPIRE) Collaborative Research Program is an NIH funding opportunity led by the National Center for Advancing Translational Sciences (NCATS). It is designed to speed up early drug discovery for biological targets that currently have no known drugs that can modulate their function.

What problem is this program trying to solve?

The program targets a long-standing bottleneck in biomedical research: many disease-relevant proteins and pathways remain "undrugged" because conventional chemistry approaches, screening libraries, and workflows often fail to produce useful chemical starting points. ASPIRE aims to expand what drug discovery can reach by developing new chemistry capabilities and practical platform modules.

What does "un-drugged targets" mean in this opportunity?

In this context, "un-drugged targets" refers to biological targets for which there are currently no known drugs capable of modulating their function. These targets may be considered hard-to-drug or never-before-drugged using existing approaches.

Which NIH organization is sponsoring this funding opportunity?

This opportunity is from the National Institutes of Health (NIH) and is sponsored by the National Center for Advancing Translational Sciences (NCATS).

What is the main approach ASPIRE uses to accelerate early drug discovery?

ASPIRE centers on an integrated NCATS platform that combines automated synthetic chemistry with computational and experimental tools. The platform supports iterative cycles of designing, making, and evaluating compounds, with the goal of producing validated chemical starting points for downstream drug development.

What is the NCATS ASPIRE platform?

The NCATS ASPIRE platform is described as an integrated set of physical and virtual modules that combine automated synthetic chemistry with modern computational and experimental capabilities. It is intended to act as a shared engine that helps move projects from concept to validated chemical starting points.

How are artificial intelligence (AI) and machine learning (ML) used in ASPIRE?

On the computational side, the ASPIRE platform leverages AI and ML to help guide molecule design and decision-making. These methods are part of the platform's design tools that inform iterative chemistry efforts.

What experimental capabilities are emphasized in the platform?

On the experimental side, the platform integrates engineering, informatics, and biological testing to support repeated design-make-test cycles. The intent is to connect chemistry generation with biological evaluation in an iterative and scalable way.

Is this opportunity meant to fund stand-alone projects?

No. The program emphasizes true collaboration rather than independent, stand-alone projects. Extramural teams are expected to work closely with NCATS intramural scientists and leverage the shared ASPIRE platform infrastructure.

Who are the collaborators in these projects?

The collaboration model specifically brings together NCATS intramural scientists (researchers working inside NIH) with outside (extramural) investigators to jointly create and apply new chemistry capabilities through the ASPIRE platform.

What types of projects is the funding announcement aiming to support?

The announcement specifically aims to support collaborations that develop additional physical modules to expand what the ASPIRE platform can do. Applicants are expected to propose tangible, buildable enhancements that strengthen end-to-end platform capabilities.

What does "module-building" mean for an application?

Module-building refers to proposing concrete additions or improvements to the ASPIRE platform, such as new automated synthesis approaches, novel hardware or workflows to improve throughput or reliability, improved integration between chemistry and biological testing, or other modular additions that make the platform more powerful and generalizable across targets.

What is meant by "platform-driven innovation" in this opportunity?

Platform-driven innovation means the focus is not only on discovering a single interesting molecule. It also includes improving the underlying tools, machinery, and processes so future discoveries across many targets can become easier, faster, and more reproducible.

What kinds of deliverables or outcomes are anticipated?

Anticipated outcomes include identifying, designing, synthesizing, and validating new chemical entities that can serve as credible starting points (often described as "hits" or "leads") for downstream drug development against novel targets. The program also emphasizes expanding chemical space by generating structurally diverse molecules and new chemotypes suitable for screening against challenging targets.

What are "hits" and "leads" in the context of this program?

"Hits" or "leads" are described as credible chemical starting points for drug development. In this opportunity, the expectation is that successful projects will generate validated chemical matter that can seed later-stage efforts beyond the scope of this program.

What does it mean to "expand chemical space available for screening"?

It means generating structurally diverse molecules and new chemotypes that broaden the types of compounds available for testing. This is intended to increase the odds of finding active compounds for targets where traditional libraries and conventional medicinal chemistry strategies have not succeeded.

Are clinical trials allowed under this funding opportunity?

No. The funding mechanism explicitly states "Clinical Trials Not Allowed." The work is intended to remain in a preclinical-to-translational space focused on methods, prototypes, platform modules, and validated chemical matter rather than clinical testing.

What funding instrument is used for this opportunity?

This opportunity uses a cooperative agreement funding instrument. This reflects that NIH, through NCATS intramural scientists, expects substantial involvement in the conduct of the project rather than serving only as a funder.

What does "cooperative agreement" imply for how the project will be run?

Based on the description provided, it implies that NCATS intramural scientists will have substantial involvement during the project and that the collaboration is intended to be active and integrated with NCATS platform resources.

What is the Assistance Listing (CFDA) number for this program?

The Assistance Listing (CFDA) number provided is 93.350.

What is the FOA identifier for this opportunity?

The Funding Opportunity Announcement (FOA) is identified as RFA-TR-21-001.

What is the activity category for this opportunity?

The activity category is health.

When was this opportunity created and when was the original application closing date?

The opportunity was created on December 10, 2020. The original application closing date listed is July 8, 2021.

Is the award ceiling or the expected number of awards provided?

No. The provided source data did not specify an award ceiling or the expected number of awards.

Who is eligible to apply?

Eligibility is broad. Eligible applicants include many types of organizations such as state, county, city, township, and special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; federally recognized Native American tribal governments; other Native American tribal organizations; public housing authorities/Indian housing authorities; nonprofits with and without 501(c)(3) status; for-profit organizations (other than small businesses) and small businesses; and additional categories listed in the announcement.

Are minority-serving institutions and tribally controlled institutions eligible?

Yes. The announcement highlights eligibility for institutions such as Alaska Native and Native Hawaiian Serving Institutions, AANAPISI institutions, Hispanic-serving institutions, Historically Black Colleges and Universities (HBCUs), Tribally Controlled Colleges and Universities (TCCUs), and other categories.

Are U.S. territories or non-U.S. (foreign) organizations eligible?

Yes. The eligibility list includes U.S. territories or possessions and also includes non-U.S. entities (foreign organizations).

Are faith-based or community-based organizations eligible?

Yes. The eligibility list explicitly includes faith-based or community-based organizations.

Are federal agencies eligible to apply?

Yes. The announcement includes eligible federal agencies among the eligible applicant types.

What makes this program different from typical drug discovery grants?

Key distinguishing features described include: a strong emphasis on collaboration between NCATS intramural scientists and extramural investigators; reliance on the NCATS ASPIRE platform as shared infrastructure; and a focus on building scalable, practical platform modules that can be broadly reused, rather than supporting isolated efforts focused only on a single target or molecule.

What stage of research is this program targeting?

The program is positioned to support early drug discovery and translational, preclinical work. It aims to generate validated chemical starting points and platform improvements, not clinical trials.

What kinds of enhancements might strengthen the platform's end-to-end capabilities?

Examples mentioned include new automated synthesis approaches, new hardware or workflows to improve throughput or reliability, improved integration between chemistry and biological testing, and other modular additions that increase the platform's power and generalizability.

What is the overall goal of the ASPIRE collaborative model?

The overall goal is to use a shared platform that combines automation, AI/ML, and biological testing to enable faster, more reproducible early discovery for targets that have been difficult or impossible to address with existing drug discovery methods, while also improving the underlying infrastructure for future projects.