The rare disease startup sector focuses on developing new therapies, diagnostics, and support solutions for conditions that affect small patient populations but often have severe, lifelong impacts. These startups work to solve unmet medical needs that traditional pharma has historically overlooked by using cutting-edge technologies such as gene therapy, RNA therapeutics, genomic diagnostics, AI-driven drug discovery, and digital patient support tools. Because rare diseases are frequently genetic and lack established treatments, companies in this sector often pursue highly targeted approaches, partner closely with patient communities, and follow accelerated regulatory pathways. The result is a fast-moving ecosystem that aims to deliver life-changing treatments, improve early diagnosis, and enhance care and quality of life for patients and families living with rare or ultra-rare conditions.