Significant advancements in regenerative medicine have the potential to transform pediatric healthcare. Improvements in diagnostics (e.g., genome sequencing) as well as the development of novel therapeutics (e.g., cell/gene therapies, ex-vivo/in-vivo gene editing) are creating a renaissance in potentially curative treatments. This provides hope for patients and families with limited therapeutic interventions, with more than 34 regenerative medicines already approved by the US Food and Drug Administration for human use. These treatments have targeted conditions like inherited retinal disorders, spinal muscular atrophy, and sickle cell disease. Developing gene therapy and gene editing therapeutics for infants involves unique considerations to ensure safety and efficacy in this vulnerable population. Adeno-associated viruses (AAVs) serve as a preferred vector for gene delivery due to their ability to infect humans without causing significant disease. In addition, clustered, regularly interspaced short palindromic repeats (CRISPR) technology offers unprecedented precision in gene editing. However, there is a critical need to establish improved safety profiles, with a focus on immunogenicity, genotoxicity, and the development of new delivery systems. The International Neonatal Consortium (INC) is working to streamline regulatory paths for neonatal therapies, highlighting the need for collaborative efforts from researchers, clinicians, regulators, and families to overcome these challenges. Ethical, financial, and logistical considerations are all equally important, particularly in ensuring equitable access to these potentially transformative treatments. Moreover, the importance of long-term follow-up to monitor safety and efficacy is emphasized, along with the need for careful patient selection and study design. This rapidly evolving field demands ongoing innovation, careful ethical consideration, and ecosystem-wide collaboration to realize its full promise for the future of child health.