The FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have jointly proposed a new submission program aimed at simplifying the approval pathway for gene therapies targeting ultra-rare diseases. This effort formalizes prior pilot efforts to expedite regulatory review for therapies serving exceptionally small patient populations. Complementing this, the NIH’s National Center for Advancing Translational Sciences awarded $350,000 in an STTR Phase 1 grant to Neucore Bio for advancing targeted exosome RNA therapies against Charcot-Marie-Tooth disease. Together, these initiatives exemplify a strategic push to streamline development and regulatory oversight for innovative rare disease treatments.