Powering prescription: Mitochondria as "Living Drugs" - Definition, clinical applications, and industry advancements.

Mitochondria's role as engines and beacons of metabolism and determinants of cellular health is being redefined through their therapeutic application as "Living Drugs" (LDs). Artificial mitochondrial transfer/transplant (AMT/T), encompassing various techniques to modify, enrich, or restore mitochondria in cells and tissues, is revolutionizing acellular therapies and the future of medicine. This article proposes a necessary definition for LDs within the Advanced Therapeutic Medicinal Products (ATMPs) framework. While recognizing different types of LDs as ATMPs, such as mesenchymal stem cells (MSCs) and chimeric antigen receptor T (CAR T) cells, we focus on mitochondria due to their unique attributes that distinguish them from traditional cell therapies. These attributes include their inherent living nature, diverse sources, industry applicability, validation, customizability for therapeutic needs, and their capability to adapt and respond within recipient cells. We trace the journey from initial breakthroughs in AMT/T to the current state-of-the-art applications by emerging innovative companies, highlighting the need for manufacturing standards to navigate the transition of mitochondrial therapies from concept to clinical practice. By providing a comprehensive overview of the scientific, clinical, and commercial landscape of mitochondria as LDs, this article contributes to the essential dialogue among regulatory agencies, academia, and industry to shape their future in medicine.

Scientific venturing for early-stage professionals (ESPs), a key concept in the innovation ecosystem.

Early-stage professionals (ESPs) and senior scientists who want to transition from academia to the industry need support to develop new skills and know-how to endeavor this challenge. However, this topic is significantly underserved in the field of cell and gene therapy, slowing down ESPs' potential to make this step. The authors of this article, members of the ESPs in the South and Central America Subcommittee at the International Society for Cell and Gene Therapy, propose the concept of "scientific venturing," which stands for the process by which scientists become entrepreneurs or part of a company. In our article, we provide key aspects to understand this concept, considering key personality traits that need to be developed and a discussion about the "innovation ecosystem." Later, we consider how scientific venturing may result in an increase in difficulty in nascent innovation ecosystems such as Latin America, in comparison with those more advanced and mature in high-income countries. Finally, we provide key information for the ESPs and other professionals about the stages of private and public investment, including information about the resources needed for the sustainability of companies and startups. Understanding what scientific venturing involves for ESPs is key to taking advantage of the maturity of an innovation ecosystem, its network, and available opportunities.