Church Lab Combinatorial Aging Gene Therapy — Gene Therapy & Genetic Interventions

George Church's multiplex longevity gene therapy approach targeting multiple aging pathways with a single treatment.

Overview

George Church's lab at Harvard has pioneered combinatorial gene therapy approaches for aging, systematically testing combinations of longevity-associated genes delivered via AAV vectors. In a landmark 2019 preprint, they demonstrated that a combination of three genes (FGF21, sTGFBR2, and aKlotho) delivered via AAV could simultaneously reverse obesity, type 2 diabetes, heart failure, and kidney failure in aged mice. The combinatorial approach is based on the hypothesis that aging is a multi-factorial process requiring multi-target intervention. This work led to the founding of Rejuvenate Bio.

Indications

  • Multi-organ aging reversal
  • Age-related metabolic syndrome
  • Heart failure in aging
  • Kidney function decline
  • Obesity and insulin resistance

Mechanism of Action

Three AAV8 vectors transduce hepatocytes, which become biofactories secreting FGF21, sTGFBR2, and soluble Klotho into circulation

Dosing

CompoundDoseFrequencyNotes
AAV triple combination (FGF21 + sTGFBR2 + aKlotho)3 x 10^11 vg per vectorSingle injectionMurine dose; human-equivalent dose not established

Evidence Grade

GRADE C

Safety & Contraindications

  • Multi-gene delivery complexity increases unpredictable interactions
  • AAV dose requirements scale with number of transgenes
  • Individual gene overexpression may have tissue-specific adverse effects
  • FGF21 overexpression can cause bone loss in preclinical models
  • TGF-beta pathway manipulation affects wound healing and immune function
  • Human translation requires extensive safety characterization