STAT3 Gain-of-Function Therapy R&D

I am moving forward with a program to create an effective therapeutic for STAT3 gain-of-function (GoF) disease and need an experienced scientist to accelerate the research and development phase. The immediate objective is to generate solid proof-of-concept data that can carry the project toward IND-enabling studies. Here’s the current landscape: • Target: STAT3 GoF mutations driving immune dysregulation and multi-system pathology. • Approach: We are still evaluating options—small-molecule inhibitors, antisense/siRNA, and genome-editing strategies are all on the table. Your first task will be to help pinpoint the most promising route through literature mining, in-silico modeling, and bench-level feasibility assays. Key responsibilities you would take on: • Design and execute molecular and cellular assays that quantify STAT3 activation (e.g., pSTAT3 Westerns, luciferase reporter systems, cytokine read-outs). • Screen candidate molecules or constructs, iterating on structure-activity relationships when applicable. • Interpret data rigorously and draft concise technical reports fit for regulatory review. • Recommend next experiments and outline a path toward in-vivo validation. Resources available: • Access to CRISPR/Cas9 and RNAi toolkits, flow cytometry, ELISA, and high-content imaging. • Collaboration with a core facility that can perform mouse PK and limited efficacy studies once lead assets are identified. Deliverables: 1. A detailed experimental plan for the first 6–8 weeks. 2. Weekly data summaries with raw files and analyzed results. 3. A go/no-go recommendation report that ranks lead candidates and maps required preclinical milestones. If you have a strong publication record in STAT signaling, immunology, or targeted drug discovery and are comfortable driving early-stage therapeutic programs, let’s talk about timelines and milestones so we can launch the lab work immediately.

Projekt-ID: 40063199