Register: Aligning With the FDA on a Regulatory Pathway To Avoid Decision Day Surprises

Date: Thursday, March 26, 2026 2–3pm EDT
Event Location: Virtual

During the past seven months, biopharma companies have had to contend with unprecedented change within the upper echelons of the FDA. Among other challenges, executives at small and medium companies have reported communication issues, misaligned expectations and media leaks while attempting to get their products across the regulatory finish line.

Notably, many of these investigational therapies have targeted rare diseases affecting primarily children—products that FDA Commissioner Marty Makary and Center for Biologics and Research Director Vinay Prasad have vowed to expedite to the market.

At the same time, the FDA has outlined its commitment to aligning with drug sponsors on the regulatory approval process, with Makary and Prasad stating in a JAMA article published in June that they intend to provide “the regulatory navigation that [small] companies may lack internally.”

It’s clear that both the FDA and biopharma companies developing medicines for rare diseases want the same thing but there remains a disconnect. This panel discussion will look at how to leverage FDA frameworks, define expectations and adopt modern technologies, including AI and in silico models, to increase the likelihood of success.

Key Takeaways:

• Define success early with in silico models to secure an accelerated pathway
• Understand and determine the most appropriate pathway—novel or traditional—for your therapeutic candidate
• Jointly leverage AI systems for an efficient and comprehensive review process
• Identify the FDA’s current thinking on cell and gene therapy to align primary and secondary endpoints for success

Stacey Frisk

Executive Director | Rare Disease Company Coalition

Stacey Frisk is a policy and regulatory affairs leader dedicated to advancing evidence-based policies that promote innovation in rare diseases. She currently serves as Executive Director of the Rare Disease Company Coalition (RDCC), the leading voice of rare disease innovators. In this role, she advocates for policies that promote efficient, patient-centered, and fit-for-purpose approaches to the development and review of rare disease therapies.

With more than a decade of experience spanning industry, patient advocacy, and clinical research, Stacey has developed expertise in translating complex regulatory challenges into actionable policy solutions. At Sarepta Therapeutics, she served as a global regulatory strategist focused on gene therapies to treat ultra-rare neuromuscular disorders, directed regulatory policy and intelligence, and led initiatives to integrate patient data and perspectives into development and regulatory strategies.

Stacey is most passionate about shaping health policies that improve the lives of people with small and complex conditions and ensuring the voices of patients and families remain at the heart of drug development and regulatory decision-making.

Rahul Gupta, MD, MPH, MBA

President | GATC Health

Dr. Rahul Gupta is President of GATC Health, where he leads the company’s work applying AI and multiomics to accelerate drug discovery and development. From 2021 to 2025, he served as Director of the White House Office of National Drug Control Policy, the first physician to hold this Cabinet-level role. In that position, he directed a $44 billion national strategy and advanced innovative approaches to addressing the overdose crisis. His career spans medicine, public health, and biotechnology, making him a trusted leader at the intersection of science, policy, and innovation.

Linda Marbán, PhD

CEO | Capricor Therapeutics

Dr. Marbán is our Chief Executive Officer and has served in that capacity and on the Board since November 2013. Dr. Marbán is a co-founder of Capricor, Inc. (wholly-owned subsidiary of Capricor Therapeutics, Inc.) and has been with the Company since 2005 and became its Chief Executive Officer in 2010.

Dr. Marbán has been in the biotechnology field for more than 20 years and brings extensive experience across research, product development and business development to the Company. From 2003-2009, Dr. Marbán held various senior roles at Excigen, Inc., a gene therapy biotechnology company, where she was responsible for operations and business development and where she oversaw the development of a biologic pacemaker for the heart.

Prior to Excigen, Dr. Marbán worked in academic science, first at the Cleveland Clinic Foundation working on the development of contractile dysfunction in heart failure due to myocarditis, followed by a postdoctoral fellowship at Johns Hopkins University. While at Johns Hopkins, she advanced to the rank of Research Assistant Professor in the Department of Pediatrics, specializing in the mechanism of the biophysical properties of cardiac muscle. Her tenure at Johns Hopkins ran 2000 to 2003. Dr. Marbán earned a Ph.D. from Case Western Reserve University.

Matthew Winton, PhD

Chief Commercial and Business Officer | CervoMed

Dr. Matthew Winton is Chief Commercial and Business Officer at CervoMed, a clinical-stage company developing therapies for age-related neurologic disorders. He brings nearly two decades of global biotechnology experience leading organizations through scientific breakthroughs, commercial growth, and market transformation.

Before joining CervoMed, Dr. Winton was Chief Operating Officer at Inozyme Pharma, where he led commercial readiness and enterprise operations, playing a key role in the company’s successful acquisition by BioMarin Pharmaceuticals.

During nearly a decade at Biogen, Dr. Winton held multiple senior leadership roles, including Senior Vice President and Head of the U.S. Multiple Sclerosis Franchise and Vice President and Head of the U.S. Spinal Muscular Atrophy Franchise. He built and scaled commercial organizations, managed multi-billion-dollar neurology portfolios, and guided the launch and growth of SPINRAZA™, the first approved treatment for spinal muscular atrophy.

He holds a Ph.D. in Neuroscience from the Université de Montréal, an M.B.A. in Health Sector Management from Boston University, and a B.S. in Biology and Psychology from York University. He completed his postdoctoral fellowship at the Center for Neurodegenerative Disease Research (CNDR) at the University of Pennsylvania.

Heather McKenzie

Senior Editor

Heather McKenzie is a professional journalist with more than five years’ experience in the biopharmaceutical industry. Since joining BioSpace, she has written more than 300 features and breaking news articles with a particular focus in neuroscience, rare disease and regulatory policy. She has also traveled internationally to cover global biotech hubs such as Israel. In previous roles, she has covered current affairs, sports, education and politics. She previously spent eight years as a senior content producer for executive-level business conferences in the pharma/biotech, legal, energy and business strategy sectors. In her free time, Heather enjoys creative writing, spending time with family and playing with her energetic Russian Blue cat Roofus. She hails from Toronto and has also lived in Chicago and Chesapeake, Virginia. You can reach her at heather.mckenzie@biospace.com.