We are witnessing the coming of age of Healthspan and Lifespan globally with the population pyramid changes, and as a result, the aging related diseases continue to grow exponentially. The field of Cell and Gene therapy (CGT), though originally started with monogenic diseases and cancers, now has progressed into the aging-related diseases globally, with clinical trials now spanning into dementias, heart failure and autoimmune diseases. While advancements in the field are remarkable and now include in-vivo therapies, viral-vector free (LNP and others) therapies, newer gene editing techniques (especially base and prime editing), and many others, the biotechnology arena is facing a challenge more than ever with regards to funding and business development. The root cause of these challenges is not only the political turmoil and financial heuristics, but also the fundamentals of clinical trial designs which absorb the bulk of drug and CGT development costs (>70% of the total drug development costs). Dr. Lasagna from New York had proposed the phases of clinical trial design in 1970s (phase I, II and III), which is still considered to be gold standard by most of pharmaceutical/biotechnology companies, academics, and regulators alike.
In the current era of healthcare technology which includes AI/machine learning based approaches for drug development and decentralized clinical trials, we must exploit the technologic advancements in order to advance the field, else we risk the future of next generations who are trying to design and develop new CGT and drugs, which is critically required to combat the escalating burden of aging-related diseases (including cancers). Herein, I present a new paradigm of smart CGT development and digital clinical trials, as we cannot keep on continuing the same Lasagna model of clinical trials from the 1970s era, which is unsustainable in the current millennium (based on Einstein’s quote “insanity is doing the same thing over and over again and expecting different results”)
