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Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like MPS II moving into the spotlight.

Innovation drives speed in early phase drug development. ICON’s Accelerated Pharmaceutical Solutions integrate advanced formulation design, real-time dose flexibility, and cutting-edge CMC expertise to transform complexity into clarity. By uniting science, technology, and strategy, biotechs can move faster, smarter, and with confidence—turning obstacles into opportunities for breakthrough success.

Vinay Prasad, chief of the FDA’s Center for Biologics Evaluation and Research, is planning to publish a paper this month to outline his office’s thinking on accelerating gene editing reviews.

Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.

FDA Commissioner Marty Makary called these changes “common-sense reforms” that could expedite the development of cell and gene therapies.

After a prolonged funding chill, investors say 2026 is shaping up for continued dealmaking and diversified bets beyond oncology and immunology.