Project Overview

Inflammasomes are multiprotein signaling complexes that activate caspases to generate pro-inflammatory cytokines — but when dysregulated, they drive autoinflammatory disorders known as inflammasomopathies. This project investigates the mechanisms by which inflammasomes mediate inflammation in human cells, addressing a critical gap left by mouse models that do not fully recapitulate human biology. Key findings include discovery of a novel mechanism by which non-canonical inflammasomes activate caspase-4/5, which in turn activate a pore-forming protein contributing to downstream cytokine release; identification of a human-specific LPS-sensing receptor that activates caspase-1 in the absence of caspase-4/5; and identification of a putative cryptic pocket on caspase-1 that may be targetable by small molecule drugs from FDA-approved compound libraries.

Impact & Innovation

New human-specific mechanisms at the root of inflammasome disease.

 

By working in human cells rather than mouse models, this project uncovered novel non-canonical inflammasome pathways — including a human-specific LPS-sensing receptor and a cryptic caspase-1 pocket — that open entirely new avenues for therapeutic targeting in autoinflammatory disease.

  • Discovers human-specific inflammasome activation mechanisms absent in mice, establishing the critical importance of human cell models for mechanistic inflammasome research and therapeutic development
  • Identifies a putative cryptic pocket on caspase-1 as a novel small molecule drug target, with FDA-approved compound screening underway in collaboration with the Bowman lab at Penn
  • Advances the Consortium’s Shared Mechanisms Across Autoimmune Diseases pillar by elucidating conserved and human-specific inflammasome pathways with broad relevance across autoinflammatory disorders and sepsis
Research Approach

A framework designed for discovery

This project combines biochemical, structural, and cell-based approaches in human cells to uncover the mechanisms by which canonical and non-canonical inflammasomes activate caspases and generate pro-inflammatory cytokines, with the goal of identifying novel therapeutic targets for inflammasomopathies.


Peptide-based caspase inhibitor development and specificity profiling; mechanistic studies of caspase-4/5 activation of pore-forming proteins and downstream caspase-1 activation; characterization of a human-specific LPS-sensing receptor in the absence of caspase-4/5; computational identification of a cryptic pocket on caspase-1 in collaboration with the Bowman lab; and FDA-approved small molecule library screening targeting identified cryptic pockets.

Human cell-based inflammasome activation datasets; caspase inhibitor specificity profiling data; mechanistic datasets characterizing caspase-4/5 pore-forming protein activation and downstream cytokine release; structural and computational datasets identifying the caspase-1 cryptic pocket; and FDA-approved drug library screening data.

Elucidation of human-specific inflammasome activation mechanisms and identification of novel therapeutic targets — including a cryptic caspase-1 pocket and a human-specific LPS receptor — with translational goals including small molecule drug development for autoinflammatory disorders and sepsis. Findings underscore the necessity of human cell models for inflammasome research and have broad implications for therapeutic development across inflammasomopathies.

Investigators & Institutions

Powering the science

Principal Investigator

Cornelius Y. Taabazuing, PhD, Colton Consortium Member

Presidential Assistant Professor, Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania

Research Outputs

From insight to impact

Publications

Chemical tools based on the tetrapeptide sequence of IL-18 reveals shared specificities between inflammatory and apoptotic initiator caspases

bioRxiv [Preprint]
Bourne, CM; Raniszewski, NR; Kulkarni, M; Exconde, PM; Liu, S; Yost, W; Wrong, TJ; Patio, RC; Mahale, A; Kardhashi, M; Shosanya, T; Kambayashi, M; Discher, BM; Brodsky, IE; Burslem, GM; Taabazuing, CY February 2025
Biological & MechanisticCytokine SignalingExperimental Platforms & ModelsIn Vitro ModelsInnate ImmunityTherapeutic DevelopmentTranslational & ClinicalAutoinflammatory DiseasesOtherUniversity of Pennsylvania

Human non-canonical inflammasomes activate CASP3 to limit intracellular Salmonella replication in macrophages

PLOS Pathogens
Kulkarni, M; Bourne, CM; Mahale, AB; Exconde, PM; Murphy, C; Goodrow, HT; Cervantes, S; Kardhashi, M; Kambayashi, M; Yoo, W; Wrong, TJ; Patio, RC; Discher, BM; Taabazuing, CY April 2026
Biological & MechanisticCytokine SignalingExperimental Platforms & ModelsIn Vitro ModelsInnate ImmunityAutoinflammatory DiseasesOtherUniversity of Pennsylvania