FGF21 Gene Therapy for PKP2 Arrhythmogenic Cardiomyopathy
preclinicaldrug program · high · Thu Jan 01 2026 00:00:00 GMT+0000 (Coordinated Universal Time)
Assess whether AAV-mediated FGF21 delivery can prevent or mitigate catecholaminergic arrhythmias and cardiac dysfunction in PKP2-associated arrhythmogenic cardiomyopathy.
AAV8-FGF21 delivered by single tail-vein injection in adult cardiac-specific tamoxifen-activated PKP2 knockout mice, with echocardiography, electrocardiography, and cardiomyocyte calcium-transient assays.
Publication record reports AAV8-FGF21 efficacy in a PKP2 arrhythmogenic cardiomyopathy mouse model.
AAV-mediated FGF21 mitigated biventricular structural changes, decreased adrenergic arrhythmias, and rescued intracellular calcium imbalance in PKP2 haploinsufficiency models.
Chimeric AAV Library Analysis and Capsid Screening Platform
exploratoryplatform · medium · Wed Jan 01 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Analyze chimeric AAV libraries after random mutagenesis and identify AAV variants enriched for target tissues to support gene therapy and vaccine development.
Interactive computational tool for serotype composition and enrichment analysis of chimeric AAV variants; validated on synthetic datasets and applied to engineered chimeric AAV capsid libraries screened in human dermal fibroblasts, dendritic cells, canine muscle, and liver tissues.
2025 Gene Therapy publication introduced hafoe and reported validation and tissue-screening applications.
hafoe identified parental serotype compositions with 96.3% to 97.5% accuracy in validation datasets and identified enriched AAV variants across tested tissues.
FGF21/sTGFBR2 Combination Gene Therapy for Metabolic Dysfunction
preclinicalresearch program · high · Wed Jan 01 2025 00:00:00 GMT+0000 (Coordinated Universal Time)
Improve metabolic dysfunction, insulin resistance, hyperlipidemia, and age-related disease phenotypes by targeting FGF21 and TGF-beta signaling.
Combination gene therapy using FGF21 and soluble TGF-beta receptor 2, tested in obese and lipodystrophic mouse models with temperature-dependent comparisons.
2025 JCI Insight publication reports effects of FGF21/sTGFBR2 combination gene therapy in metabolic dysfunction models.
FGF21/sTGFBR2 improved insulin resistance and hyperlipidemia in obese mice more dramatically at warmer temperatures; in lipodystrophic mice, efficacy depended on environmental temperature and adipose tissue context.
AAV capsid discovery and engineering platform
exploratoryplatform · high · Thu Jun 20 2024 00:00:00 GMT+0000 (Coordinated Universal Time)
Identify, analyze, and engineer AAV capsids with tissue tropism useful for gene therapy and vaccine development.
Directed evolution and DNA shuffling of AAV capsid genes, computational analysis of chimeric AAV libraries with hafoe, and screening for tropism in human dermal fibroblasts, dendritic cells, canine muscle, and liver tissues.
Patent family published for adeno-associated virus capsids and 2025 publication describing hafoe and AAV library screening.
hafoe identified parental serotype composition with 96.3% to 97.5% accuracy in synthetic validation and identified enriched AAV variants in screened tissues.
Partial Reprogramming Gene Therapy
preclinicalresearch program · high · Mon Jan 01 2024 00:00:00 GMT+0000 (Coordinated Universal Time)
Extend lifespan, improve healthspan, and reverse age-related cellular or epigenetic changes through partial cellular reprogramming.
Systemic AAV delivery of an inducible OSK partial-reprogramming system in aged mice; human keratinocyte experiments expressing exogenous OSK.
2024 publication reports gene therapy-mediated partial reprogramming extended lifespan and reversed age-related changes in aged mice.
In 124-week-old male mice, systemically delivered inducible OSK AAV extended median remaining lifespan by 109% versus controls and improved frailty scores; human keratinocytes showed epigenetic markers of age reversal.
AAV8-FGF21 gene therapy for PKP2 arrhythmogenic cardiomyopathy
preclinicaldrug program · high
Treat PKP2-associated arrhythmogenic cardiomyopathy by mitigating cardiac dysfunction, adrenergic arrhythmias, and calcium imbalance.
AAV8-mediated FGF21 gene therapy delivered by single tail vein injection in adult cardiac-specific tamoxifen-activated PKP2 knockout mice.
Published 2026 preclinical study in Heart Rhythm.
AAV8-FGF21 mitigated biventricular structural changes, reduced adrenergic arrhythmias, and rescued intracellular calcium imbalance in PKP2 haploinsufficiency; acute one-hour in vitro FGF21 treatment did not affect calcium transients.
AAV capsid engineering and hafoe analysis platform
exploratoryplatform · high
Analyze chimeric AAV libraries after random mutagenesis and identify AAV variants with tissue tropism useful for gene therapy and vaccine development.
Interactive computational tool for exploratory analysis of chimeric AAV libraries, paired with engineered chimeric AAV capsid libraries screened in human dermal fibroblasts, dendritic cells, canine muscle, and liver tissues.
Published validation showing hafoe identified parental serotype compositions with 96.3% to 97.5% accuracy and characterized enriched AAV variants across target tissues.
The tool accurately decoded serotype composition and enrichment patterns in chimeric AAV variants and supported identification of tissue-enriched AAV variants.
AAV-FGF21 gene therapy for PKP2 arrhythmogenic cardiomyopathy
preclinicaldrug program · high
Treat or mitigate arrhythmogenic cardiomyopathy associated with PKP2 loss by addressing cardiac dysfunction, adrenergic arrhythmias, and calcium imbalance.
AAV8-mediated FGF21 gene therapy delivered by single systemic injection in adult PKP2 cardiac knockout mice.
Published preclinical mouse data showing mitigation of biventricular structural changes, reduced adrenergic arrhythmias, and rescued intracellular calcium imbalance.
AAV8-FGF21 reduced progression of cardiac structural changes, decreased adrenergic arrhythmias, and rescued intracellular calcium imbalance in PKP2 haploinsufficiency models.
Animal Health Program
undiscloseddrug program · medium
Develop animal-health gene therapies for age-related disease in companion animals, with animal development paths informing human clinical programs.
Targeted gene therapy for companion-animal indications.
Listed on Rejuvenate Bio's pipeline under Animal Health.
Canine MVD Trial 2020
undiscloseddrug program · low
Evaluate a Rejuvenate Bio program for canine mitral valve disease as part of the company's companion-animal longevity focus.
Clinical trial referenced on Rejuvenate Bio's site navigation as 'MVD Trial 2020'.
Company site materials reference a dedicated 'MVD Trial 2020' page under Trials.
Combination gene therapy for multiple age-related diseases
preclinicalresearch program · medium
Treat multiple age-related diseases with a single combination gene therapy approach.
Combination gene therapy using aging-related therapeutic targets.
Publication listed as 'A single combination gene therapy treats multiple age-related diseases'.
The supplied publication title reports treatment of multiple age-related diseases with a single combination gene therapy.
FGF21 and soluble TGFBR2 metabolic dysfunction gene therapy
preclinicaldrug program · high
Improve insulin resistance, hyperlipidemia, and metabolic dysfunction in obesity and lipodystrophy models.
Combination gene therapy targeting FGF21 and soluble TGF-beta receptor 2, tested in obese and lipodystrophic mice under different environmental temperatures.
Published preclinical study in JCI Insight in 2025.
FGF21/sTGFBR2 improved insulin resistance and hyperlipidemia in obese mice more strongly at warmer temperatures. In lipodystrophic mice, combination therapy required adipose tissue to improve insulin resistance at 30 C, while FGF21 alone improved insulin resistance at 22 C.
FGF21 and sTGFBR2 gene therapy for metabolic dysfunction in lipodystrophy
preclinicalresearch program · high
Improve insulin resistance, hyperlipidemia, and metabolic dysfunction in obesity and lipodystrophy models.
Combination gene therapy targeting FGF21 and TGF-beta signaling, tested in obese and lipodystrophic mice under different housing temperatures.
Published preclinical study evaluating FGF21/sTGFBR2 effects in obese and lipodystrophic mice.
Combination therapy improved insulin resistance and hyperlipidemia in obese mice, with stronger effects at warmer temperatures; effects in lipodystrophic mice depended on adipose tissue, temperature, and FGF21 contribution.
Gene Therapy for Desmoplakin-Associated Arrhythmogenic Cardiomyopathy
preclinicaldrug program · high
Develop a gene therapy targeting multiple pathological drivers of desmoplakin-associated arrhythmogenic cardiomyopathy.
Late-stage preclinical gene-therapy project supported by CIRM funding.
CIRM lists a $570,000 Late Stage Preclinical Projects award to Rejuvenate Bio investigator Noah Davidsohn for this program.
Human RJB-0402
undiscloseddrug program · medium
Develop a human gene-therapy program for aging or chronic age-related disease.
Targeted gene therapy; site analysis describes a liver-directed gene therapy approach emphasizing FGF21 biology.
Listed on Rejuvenate Bio's pipeline as Human-RJB-0402.
Klotho and soluble TGF-beta receptor 2 osteoarthritis gene therapy
preclinicaldrug program · medium
Reverse or treat osteoarthritis using gene therapy targets associated with aging biology.
Gene therapy using Klotho and soluble TGF-beta receptor 2.
Publication record listed for Klotho and sTGFbR2 reversing osteoarthritis.
The supplied publication title states that Klotho and sTGFbR2 reverse osteoarthritis, but no abstract-level details were provided.
Klotho and sTGFbR2 gene therapy for osteoarthritis
preclinicaldrug program · medium
Reverse or treat osteoarthritis using gene therapy targets associated with aging and inflammatory signaling.
Gene therapy using Klotho and soluble TGF-beta receptor 2 targets.
Publication listed as 'Klotho and sTGFbR2 reverse osteoarthritis'.
The supplied publication title reports reversal of osteoarthritis using Klotho and sTGFbR2.
Klotho and sTGFBR2 Gene Therapy for Osteoarthritis
preclinicalresearch program · medium
Reverse or treat osteoarthritis by targeting Klotho and TGF-beta signaling.
Gene-therapy research involving Klotho and soluble TGF-beta receptor 2.
Publication listed by Rejuvenate Bio and indexed as 'Klotho and sTGFbR2 reverse osteoarthritis.'
Mitral Valve Disease Program
undiscloseddrug program · medium
Treat mitral valve disease in companion animals as part of Rejuvenate Bio's animal-health pipeline.
Gene-therapy program; supplied site navigation references an MVD Trial 2020 page.
Listed on Rejuvenate Bio's pipeline as Mitral Valve Disease and site navigation includes Trials / MVD Trial 2020.
Novel gene therapy for desmoplakin-associated arrhythmogenic cardiomyopathy
preclinicaldrug program · high
Develop a gene therapy targeting multiple pathological drivers of desmoplakin-associated arrhythmogenic cardiomyopathy.
Late-stage preclinical gene therapy project funded through a CIRM award.
CIRM listed a $570,000 Late Stage Preclinical Projects award to Rejuvenate Bio for this program.
OSK partial reprogramming gene therapy
preclinicaldrug program · high
Extend lifespan, improve healthspan, and reverse age-related cellular or epigenetic changes using partial reprogramming.
Systemic adeno-associated virus delivery of an inducible OSK partial reprogramming system; related work includes OSK-mediated epigenetic restoration in vision models.
Published preclinical evidence in aged mice and human keratinocytes.
In aged male mice, systemically delivered inducible OSK AAV extended median remaining lifespan by 109% versus controls and improved frailty scores; human keratinocytes expressing OSK showed epigenetic markers of age reversal.
OSK partial reprogramming gene therapy for lifespan and age-related changes
preclinicalresearch program · high
Extend lifespan and reverse age-related changes using partial cellular reprogramming.
Systemically delivered adeno-associated viruses encoding an inducible OCT4, SOX2, and KLF4 partial reprogramming system in aged mice; OSK expression also tested in human keratinocytes.
Published study reporting lifespan extension and improved health parameters in aged mice.
In 124-week-old male mice, inducible OSK AAV treatment extended median remaining lifespan by 109% over controls and improved frailty scores; human keratinocytes showed epigenetic markers of age reversal.
