Diego Garzon | VP, Corporate Development
Apertura Gene Therapy

Accomplished biopharmaceutical executive with expertise in R&D, clinical operations, medical affairs, sales, and business development. Led and supported over $1.7 billion in transactions from sourcing to closing. Proven problem solver in CNS, oncology, nucleic acid therapeutics, and gene therapy, with a strong track record of building and leading teams in biotech and pharma companies of all sizes.

Appearances:

Day 2 - World Orphan Drug Congress USA 2025 @ 11:20

Panel: Early engagement and partnership between advocacy and industry

This session will examine the critical role of early-stage collaboration between advocacy groups and industry stakeholders in the development of orphan drugs and treatments for rare diseases. With the unique challenges posed by these conditions—including small patient populations, high development costs, and regulatory complexities—early engagement between patient advocates and pharmaceutical companies is essential to drive innovation, expedite regulatory pathways, and improve access to life-saving treatments.

Key topics will include the role of early dialogue in shaping clinical trials, the impact of advocacy efforts on regulatory approvals, and strategies for addressing the financial and logistical barriers that often impede progress in rare disease treatments. Participants will leave with practical strategies for fostering effective partnerships and lessons on how to leverage collaboration for meaningful impact in the orphan drug space.

Moderator: Vanessa Boulanger, Director of Patient Engagement, Boehringer Ingelheim

Mandy Rohrig, Associate Director of Patient Advocacy, BridgeBio Pharma

Diego Garzon, VP, Corporate Development, Apertura Gene Therapy

Allyson Berent, Chief Science Officer, Foundation for Angelman Syndrome Therapeutics, Foundation for Angelman Syndrome Therapeutics