Leveraging a dual technology platform to develop gene therapies for a range of rare diseases

In this article, Dr Mary Spellman, Chief Medical Officer and Senior Vice President of R&D at Castle Creek Biosciences, discusses how a versatile dual platform of ex vivo and in vivo gene therapy technologies is being utilised for the development of novel gene therapies for a broad range of rare diseases in underserved patient populations.

Recent advancements in precision medicine have resulted in many promising cell and gene therapies being introduced and studied. Several treatments have been approved and others are advancing towards late-stage clinical development. Many of these therapies signal the prospect of effective treatment and even curative benefit, often in rare diseases that have limited or no treatment options. With each technologic advance and clinical success, drug developers gain a better understanding of how to leverage genetic engineering tools to optimise treatments for a range of genetic diseases.