STAT3 GOF Gene Therapy
Budget: ₹75,000 – ₹150,000 INR
I am initiating a research-stage program to create an in-vivo gene therapy that corrects the gain-of-function mutation in STAT3. The assignment begins at the concept stage and continues through pre-clinical validation. Because the intervention must be delivered directly to the patient, every step—from vector choice and payload design to dose-finding studies—needs to be tailored for systemic administration while keeping immunogenicity and off-target risks low.
Your brief will include:
• Selecting or engineering the optimal viral or non-viral vector for STAT3 targeting.
• Designing the expression cassette or genome-editing components that reverse or silence the pathogenic allele.
• Building a reproducible bench-to-animal workflow, including relevant assays (qPCR, Western blot, functional readouts) and in-vivo model selection.
• Delivering a written development plan with timelines, decision gates, and clear go/no-go criteria, plus all associated lab protocols and construct maps.
Acceptance criteria
1. A complete vector design package (sequence files, annotated maps, cloning strategy).
2. An experimental plan that can be executed in a GLP setting, detailing materials, methods, and expected milestones.
3. A risk-mitigation section covering immunogenicity, biodistribution, and regulatory considerations specific to STAT3 GOF.
If you have proven expertise in AAV, LNP, or other systemic delivery platforms and can demonstrate prior success in early-stage gene therapy R&D, I would value your collaboration on this program.
Your brief will include:
• Selecting or engineering the optimal viral or non-viral vector for STAT3 targeting.
• Designing the expression cassette or genome-editing components that reverse or silence the pathogenic allele.
• Building a reproducible bench-to-animal workflow, including relevant assays (qPCR, Western blot, functional readouts) and in-vivo model selection.
• Delivering a written development plan with timelines, decision gates, and clear go/no-go criteria, plus all associated lab protocols and construct maps.
Acceptance criteria
1. A complete vector design package (sequence files, annotated maps, cloning strategy).
2. An experimental plan that can be executed in a GLP setting, detailing materials, methods, and expected milestones.
3. A risk-mitigation section covering immunogenicity, biodistribution, and regulatory considerations specific to STAT3 GOF.
If you have proven expertise in AAV, LNP, or other systemic delivery platforms and can demonstrate prior success in early-stage gene therapy R&D, I would value your collaboration on this program.