Challenges and opportunities: lessons learned in developing potential gene therapy cures

While the full potential of gene therapies continues to be realised, their successes in rare diseases promote them as a promising treatment modality. Dr Gaurav Shah, Chief Executive Officer of Rocket Pharmaceuticals discusses the future of gene therapies as well as the company’s progress in in vivo and ex vivo programmes.

For the first time in human history, science is enabling not just effective treatments but potential cures at the genetic level, which is the deepest essence of who we are as physical living beings. As a multi-platform gene therapy company advancing a pipeline of four best-in-class programmes, we recognise the potential impact of our work in advancing treatments for rare diseases. Yet, despite gene therapy’s potential, there are several obstacles the field must collectively overcome.

Rocket Pharmaceuticals is not immune to these challenges and we are eager to share our learnings with the broader scientific and patient community. Whether related to safety, efficient scaling and manufacturing or target identification, we can support each other to advance the development of potentially curative treatments and ultimately elevate the lives of patients and families worldwide living with rare diseases.