Developing a safe therapy for the treatment of lipodystrophy.
Lipodystrophy encompasses a diverse range of disorders characterised by abnormal or degenerative conditions affecting adipose tissue. This uncommon and often undiagnosed ailment can manifest as partial/localised or generalised forms and is typically associated with metabolic issues like insulin resistance, diabetes, hyperglycemia, hyperlipidemia, and other serious health conditions. Lipodystrophy can either be present from birth or acquired later in life, and it frequently poses life-threatening risks due to complications affecting the liver, kidneys, and heart. All variations of lipodystrophy share common traits of severe insulin resistance and markedly low levels of leptin.
Presently, the only treatment for lipodystrophy involves replacing leptin using Metreleptin, administered via injections once or twice daily, but this treatment is costly, averaging $565,000 per patient per year, and may induce adverse effects. Zvelt Therapeutics is actively developing a safe, efficient, and economically viable therapy for lipodystrophy. This therapeutic approach involves utilising recombinant adeno-associated virus (rAAV) vectors, known for their prolonged transgenic expression and low immunogenicity. Zvelt intends to address these challenges by utilising an engineered AAV serotype called Rec2, which selectively targets adipose tissue. This strategy will be combined with a dual cassette platform designed to minimise transgene expression in the liver.