On Friday, September 25, 2026, John F. Crowley, President and CEO of the Biotechnology Innovation Organization (BIO), delivered the keynote address at the Emily Whitehead Foundation’s Believe Ball in Philadelphia, bringing his distinctive perspective on rare disease innovation to an audience of over 400 guests. The event, held at the Crystal Tea Room in the historic Wanamaker Building, assembled patients, caregivers, scientists, developers, manufacturers, and healthcare providers united by a shared mission to advance therapies for cancer and rare diseases. Crowley’s presence at this specific venue carries particular weight: Philadelphia is the city where Emily Whitehead made medical history in 2012 as the first child in the world to be saved by CAR T-cell therapy, a breakthrough that transformed treatment possibilities for young cancer patients.
Crowley brings to the stage not just professional authority but a deeply personal understanding of what it means to battle rare disease. In 1998, two of his own children received a diagnosis of Pompe disease—a severe and often fatal neuromuscular disorder that typically strikes in infancy. Rather than accept the medical status quo, Crowley co-founded Novazyme Pharmaceuticals to pursue an experimental treatment that would ultimately save his children’s lives. His journey from desperate parent to biotechnology entrepreneur later inspired the film “Extraordinary Measures,” a narrative that continues to resonate with audiences seeking hope in the face of genetic illness.
Table of Contents
- Why John Crowley’s Leadership Matters to the Rare Disease Community
- The Emily Whitehead Foundation and Its Mission in Advancing Cell Therapy
- CAR T-Cell Therapy and the Broader Landscape of Advanced Therapeutics
- Gathering the Biotech Community: What the Believe Ball Represents
- The Rare Disease Patient’s Perspective: Understanding the Stakes
- John Crowley’s Journey from Pompe Disease to Biotechnology Leadership
- The Biotechnology Innovation Organization and Its Role in Advancing Rare Disease Treatment
Why John Crowley’s Leadership Matters to the Rare Disease Community
Crowley’s rise from a crisis in his own household to leadership of BIO—the organization representing over 1,200 biotechnology companies, academic institutions, and related organizations—illustrates the power of personal experience in driving innovation policy. When he speaks about rare diseases and advanced therapies, he does so from lived experience, not abstract theory. This dual credibility as both a successful entrepreneur and a parent who has watched his children struggle with and overcome a life-threatening genetic disorder gives him unique standing in conversations about research funding, regulatory pathways, and healthcare access. At the Believe Ball, this perspective would have resonated particularly with attendees who understand the gap between hope and reality—those who have sat in hospital waiting rooms and fought for access to experimental treatments.
The connection between Crowley’s experience and the work being advanced through events like the Believe Ball is not coincidental. Both represent the conviction that rare disease patients deserve innovation and investment. Novazyme’s eventual success (the company was later acquired) proved that entrepreneurial approaches could translate research into therapies. Similarly, Emily Whitehead’s survival and the subsequent scaling of CAR T-cell therapy demonstrated what becomes possible when families, researchers, and the biotechnology sector unite around a single goal.
The Emily Whitehead Foundation and Its Mission in Advancing Cell Therapy
The Emily whitehead Foundation exists because one girl survived what should have been unsurvivable. In 2012, at age six, Emily Whitehead received experimental CAR T-cell therapy at Children’s Hospital of Philadelphia—a treatment that was so new it had never been attempted in a pediatric patient. Her leukemia cells were removed, genetically modified to help the immune system recognize and destroy cancer, and reinfused into her body.
She survived, and that survival opened a door. Today, CAR T-cell therapy is an FDA-approved treatment for certain blood cancers, and the foundation bearing Emily’s name works to advance access to these and other advanced therapies for patients who might not otherwise have options. The foundation’s work extends beyond celebrating past victories into the practical realm of supporting current patients and driving future innovation. The Believe Ball serves this dual purpose: it gathers the community of stakeholders who understand the science and the stakes—the researchers developing next-generation therapies, the manufacturers scaling up production, the healthcare providers administering treatments, and the patients and families whose lives depend on access. The event itself, by design, bridges the gap between scientific achievement and human need.
CAR T-Cell Therapy and the Broader Landscape of Advanced Therapeutics
CAR T-cell therapy represents one frontier in what researchers call “advanced therapies”—a category that includes gene therapies, cell therapies, and other cutting-edge approaches that work fundamentally differently from traditional pharmaceuticals. Rather than taking a drug that circulates through the bloodstream, advanced therapies often involve removing cells from a patient’s body, modifying them in a laboratory, and returning them to fight disease from within. The complexity is staggering: each treatment must be customized to the individual patient, manufacturing must meet exacting standards, and the immune response triggered by these therapies can be unpredictable and dangerous if not carefully managed. Emily Whitehead’s case exemplifies both the promise and the peril of this frontier.
Her treatment worked, but the intensity of her immune response—cytokine release syndrome—nearly killed her. She spent weeks in intensive care with high fevers and organ stress before her body stabilized and her cancer dissolved. Success in advanced therapy often means navigating this knife’s edge, where the treatment that saves you is also the treatment that could harm you. This is why events like the Believe Ball matter: they bring together the full ecosystem of people who understand these nuances and continue pushing for improvements in safety, efficacy, and access.
Gathering the Biotech Community: What the Believe Ball Represents
The decision to host the Emily Whitehead Foundation’s annual gala at the Crystal Tea Room in the Wanamaker Building is not arbitrary. Philadelphia is the home of Children’s Hospital of Philadelphia, where Emily Whitehead received her groundbreaking treatment. It is a city with a deep history in medical innovation, and a place where the bridge between laboratory discovery and patient outcome remains visible and tangible. By holding the Believe Ball in this location, the foundation anchors the event in its historical significance while inviting attendees to recognize that the work is ongoing. Over 400 guests attending the Believe Ball represents a powerful concentration of people who could collectively shape the future of rare disease treatment.
Scientists present their latest findings to manufacturers who must scale production. Healthcare providers discuss barriers to patient access with policy advocates. Patients and families share their stories with researchers who might otherwise encounter rare diseases only through journal articles. This mixing is essential, because the gap between a laboratory breakthrough and a living patient receiving treatment is vast and complex. It requires regulatory navigation, manufacturing innovation, healthcare infrastructure, insurance coverage, and persistent advocacy.
The Rare Disease Patient’s Perspective: Understanding the Stakes
For patients and families living with rare diseases, the stakes of events like the Believe Ball are existential. A rare disease, by definition, is often neglected by large pharmaceutical companies because the patient population is small and the profit margins are thin. When a family receives a diagnosis of a rare condition, they often face not only the medical challenge but also the economic and logistical reality that treatments may not exist, or may exist only in experimental form, or may require travel to distant medical centers. The conversation that happens at a gala might seem abstract compared to the lived reality of a child with a progressive neuromuscular disorder or cancer that has exhausted standard treatments.
Yet this is precisely where someone like John Crowley’s presence becomes meaningful. His story demonstrates that rare disease families cannot afford to accept inevitability. Crowley did not wait for pharmaceutical companies to solve his children’s problem; he created a company to do it. This entrepreneurial defiance in the face of medical tragedy has become a template for how rare disease communities mobilize. When 400 people gather at the Believe Ball—including venture capitalists, pharmaceutical executives, and policy makers—the conversation shifts from “your disease is too rare to matter” to “how do we scale this therapy, reduce its cost, and get it to more patients.” The question changes from whether it can be done to how it will be done.
John Crowley’s Journey from Pompe Disease to Biotechnology Leadership
Pompe disease, the condition that struck Crowley’s two children in 1998, is a lysosomal storage disorder caused by a deficiency in the enzyme acid alpha-glucosidase. Without this enzyme, glycogen accumulates in muscle and nerve cells, leading to progressive weakness, respiratory failure, and early death. In 1998, there was no treatment. Crowley’s response was not to grieve in isolation; it was to educate himself in biochemistry and genetics, connect with researchers working on potential therapies, and ultimately co-found Novazyme Pharmaceuticals. The company’s mission was singular: develop a treatment for Pompe disease.
After years of research and development, Crowley’s vision proved successful. The treatment improved his children’s conditions and outcomes dramatically. Novazyme was eventually acquired, and enzyme replacement therapy for Pompe disease is now an established treatment that has extended and improved the lives of many patients globally. Crowley’s transition from founder to BIO president reflects a deliberate choice to expand his advocacy beyond his own children’s disease. As head of BIO, he has become a voice for the entire biotechnology sector, advocating for policies that encourage innovation, streamline regulatory pathways, and ensure that advances in molecular medicine reach patients. His presence at the Believe Ball in 2026 signals that this commitment to rare disease innovation remains central to his leadership.
The Biotechnology Innovation Organization and Its Role in Advancing Rare Disease Treatment
The Biotechnology Innovation Organization represents over 1,200 member companies, research institutions, and related organizations working across every segment of the biotechnology industry. From startups developing novel gene therapies to large pharmaceutical companies manufacturing approved treatments, BIO serves as the collective voice advocating for policies that support innovation. In the rare disease space, BIO members are often the companies taking on what larger pharma considers too risky or unprofitable: developing treatments for conditions that affect only hundreds or thousands of people worldwide.
These companies operate on venture capital funding, grant money, and the hope that regulatory approval and patient access will eventually create a sustainable business model. The work is high-risk, high-cost, and utterly necessary for rare disease patients who would otherwise have no options. Crowley’s keynote at the Believe Ball would likely emphasize the intersection of innovation, regulation, and patient access—the three pillars that determine whether a breakthrough in the laboratory becomes a reality in the clinic. The Crystal Tea Room in Philadelphia, where Crowley addressed an audience of cancer and rare disease patients, caregivers, scientists, and industry leaders on September 25, 2026, became a focal point for a larger conversation about what it takes to transform medical catastrophe into medical victory.
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