Building on the success of gene therapies in treating rare diseases, the field is now rapidly evolving into a cornerstone of longevity science. By targeting the biological mechanisms of aging at the genetic level, researchers and biotechnology companies are pioneering novel therapies designed to extend healthspan - the years of life lived in good health, free from chronic disease and age-related decline.
Partial Cellular Reprogramming: Rejuvenating Without Resetting
One of the most promising approaches in this domain is partial cellular reprogramming. This method involves the use of Yamanaka factors: a set of four transcription factors capable of reverting adult cells to a more youthful, stem-like state. Unlike full reprogramming, which risks loss of cellular identity and tumorigenesis, partial reprogramming aims to rejuvenate aging cells while maintaining their function. Startups such as YouthBio Therapeutics are developing gene therapies using viral vectors to deliver the reprogramming factors to tissues such as the brain to treat age-related neurodegenerative diseases.
Gene Expression Modulation for Age-Related Conditions
Other gene therapy strategies focus on enhancing expression of genes associated with longevity. For instance, TERT encodes a component of the telomerase enzyme and plays a critical role in maintaining telomere length – a key marker of cellular aging. Telomere Therapeutics is advancing a preclinical AAV-based therapy delivering TERT and is focussed on treating idiopathic pulmonary fibrosis. Another example is Rejuvenate Bio, which employs AAV-based delivery of combinations of FGF21, αKlotho, and TGFβR2. Their therapies have shown promising results in treating a wide spectrum of age-related conditions, including heart failure, kidney disease, diabetes, and obesity, in both rodent and large animal models.
Innovation in Discovery and Delivery Platforms
Progress in the field also relies on innovation in discovery platforms to identify new or better gene targets, and in gene delivery technologies. Shift Bioscience, for example, is developing a discovery platform to identify transcription factor combinations that safely rejuvenate cells without activating oncogenic pathways. Meanwhile, Oisín Biotechnologies has created a Proteo-Lipid Vehicle (PLV) delivery platform, designed to offer tissue- and cell-specific gene delivery. Oisín is currently applying this platform in preclinical studies for conditions such as muscle wasting and fat accumulation.
Protecting Innovation Through Strategic IP
As gene therapy technologies advance, in the longevity space and beyond, robust IP strategies are critical to protect and commercialise innovations. Patent protection can be secured for novel gene constructs, vectors, methods of treatment and manufacturing processes. Patent protection can also be secured more broadly around new therapeutic approaches targeting the molecular drivers of aging e.g. around modulation of a novel target gene or pathway. Such broad patents can create valuable licensing and partnership opportunities. Similarly, patents protecting delivery and manufacturing platforms represent particularly valuable assets as the field grapples with commercialisation challenges; with broad claims offering licensing potential across a range of age-related indications.
As partnerships and funding in the longevity sector accelerate, a well-considered and strategically aligned IP portfolio will be key for companies to solidify interest and secure investment.
