Hot Topic: Because Ellie Can’t Wait: Breaking Barriers in Advanced Therapy Development

Advanced therapies—cell, gene, and nucleic acid–based modalities—hold transformative potential for rare diseases and high-burden conditions. Yet despite scientific breakthroughs, translation to patients remains hindered by delays in development, regulatory uncertainty, technical bottlenecks, and feasibility challenges. Each barrier represents lost time for patients who cannot afford to wait.
Inspired by the patient voice, “Because Ellie Can’t Wait” reframes development urgency as a collective responsibility. This symposium will bring together regulators, industry leaders, and academic innovators to identify actionable strategies for accelerating advanced therapy development while maintaining safety, quality, and equity.
Gap: In rare diseases, obtaining sufficient high-quality patient samples is often the greatest barrier to translational progress. Individual sponsors repeatedly struggle with limited biospecimens, leading to underpowered studies and difficulty validating assays.Impact: Slow, fragmented development timelines with redundant sampling requests that impose undue burden on small patient populations.Opportunity: Establish patient sample repositories and pre-competitive sharing frameworks to maximize the value of scarce materials.

Preclinical and translational data—especially on immunogenicity, PK/PD, and manufacturing challenges—are siloed within companies. Lessons from prior health authority interactions are rarely shared.2. Lack of Data Sharing Across SponsorsGap: Preclinical and translational data—especially on immunogenicity, PK/PD, and manufacturing challenges—are siloed within companies. Lessons from prior health authority interactions are rarely shared.Impact: Each sponsor reinvents the wheel, leading to redundant studies, delayed submissions, and variable quality of IND packages.Opportunity: Create cross-company data-sharing initiatives and anonymized case study repositories to build collective learning in rare diseases.

3. Inefficient Preclinical Development PathwaysGap: Without shared benchmarks, sponsors often over-collect exploratory biomarkers or design redundant animal studies to “cover bases” for regulators.Impact: Wasted resources, delays in patient access, and poor translatability due to over-reliance on non-predictive models.Opportunity: Develop harmonized translational strategies and decision-based frameworks that define what is essential versus optional.4. Fragmented Health Authority EngagementGap: Sponsors interact with regulators in isolation, and experiences with flexibility or expectations (e.g., FDA vs. EMA) are not systematically shared.Impact: Missed opportunities for global alignment, duplication of work, and slower rare disease therapy approvals.Opportunity: Establish structured mechanisms for sponsors to share ano nymized learnings from regulatory interactions, accelerating collective progress.