From Lab to Patient: Key Takeaways from the 2026 Cell and Gene Therapy Symposium
- Cardiovascular care, still largely phenotype-driven, has an opening to follow oncology and rare disease toward genetically tailored treatment.
- The legal framework behind drug regulation hasn’t changed, but FDA’s approach to cell and gene therapy has, with a steady stream of guidance and added regulatory flexibility.
- Moving therapies from lab to clinic depends on academic and industry collaboration and on sustained funding for high-risk trials that venture capital often avoids.
- Patients should be partners from the start of development, with outcomes that matter in daily life, not only a cure.
- Access to CAR-T and gene therapies hinges on cost, local infrastructure at safety-net hospitals, and education for both patients and physicians.
- AI can lower manufacturing costs and improve consistency, but consent, data privacy, and equity must be addressed, and AI should remain a tool rather than a decision-maker.
Cell and gene therapies are changing what’s possible for patients with some of the hardest diseases to treat. At the 2026 Cell and Gene Therapy Symposium, researchers, clinicians, regulators, industry leaders, and patient advocates came together to take stock of that progress and the work still ahead, from the regulatory path and the “valley of death” to cost, access, and the ethics of AI.
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Keynote Speaker Eric Adler, MD Clinical Professor and Medical Director, Heart Transplant Program UC San Diego |
The day opened with a keynote from Eric Adler, MD, Clinical Professor and Medical Director of the Heart Transplant Program at UC San Diego. Adler laid out the scale of the challenge: cardiovascular disease causes more than 20 million deaths each year and is the leading cause of death worldwide. More than a million Americans are hospitalized for heart failure annually, and those hospital stays drive a large share of the cost.
“This gigantic proportion of the costs we spend are on these hospitalized patients, where it can be ten, twenty, fifty thousand dollars a day in the hospital. If you add that up times a million patients, you can see how we have this urgent issue in front of us.”
Oncology and rare disease now use genetic insights to tailor treatment. Cardiology, Adler noted, still relies largely on phenotype-based therapy, sorting patients by whether the heart is pumping well and prescribing accordingly. He sees an opportunity to catch up by learning from peers in oncology and immunology.
At UC San Diego, genetic testing in heart failure clinics is already part of that shift. Specific genetic results can signal when a patient needs a defibrillator or early transplantation, and that information has helped prevent deaths. Adler credited sustained support from organizations like the California Institute for Regenerative Medicine (CIRM) for advancing this work, and named patients themselves as “silent architects” of the progress.
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Rethinking the Path for Rare Disease Therapies
The first panel, “Rare Disease & Regulatory Updates,” examined the real-world hurdles facing rare disease therapies across regulation, manufacturing, financing, and patient access. Moderator Jason Conaty, PhD, JD, Counsel at Hogan Lovells Cadwalader, opened by pointing to the sharp rise in approved cell and gene therapy products over the past few years and the need to build infrastructure for orphan and ultra-rare diseases.
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Moderator Jason Conaty, PhD, JD Counsel, Hogan Lovells Cadwalader |
Panelists
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Regulation is adapting. Melissa Mendoza, JD, who previously worked at the U.S. Food and Drug Administration and is now at Hogan Lovells Cadwalader, explained that the legal framework governing drug regulation has stayed largely the same. What has changed is how FDA approaches cell and gene therapy, issuing guidance tailored to these products and offering flexibility for innovative treatment designs.
Experience builds agility. Don Fink, PhD, described how regulators grow more agile as they gain familiarity with new biological products. He advocated for conditional authorization, similar to emergency use authorization, so reimbursement can support development while trials continue.
Shared data can speed development. Lisa McGinley, PhD, presented CIRM’s new Rare Disease Acceleration Through Platform Innovation and Delivery (RAPID) Program, which aims to accelerate in vivo genetic therapies by funding multiple candidates, leveraging shared data, and encouraging early engagement with FDA on platform-based approaches.
New business models can keep therapies alive. Paul Ayoub, PhD, introduced Rarity PBC, a public benefit corporation built to sustain rare disease development over the long term. By maintaining infrastructure and reusing existing processes, Rarity aims to lower costs and advance therapies that might otherwise be shelved.
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Advancing Gene Editing from Discovery to Clinic
The “Advances in Gene Editing” panel focused on bringing cutting-edge therapies into clinical practice while keeping patient access and safety at the center. Saul Priceman, PhD, of the University of Southern California moderated a discussion with Justin Eyquem, PhD, of UCSF; Lior Goldberg, MD, of City of Hope; Tippi MacKenzie, MD, of UCSF; and Brett Staahl, PhD, of Scribe Therapeutics.
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Moderator Saul Priceman, PhD Associate Professor, Department of Medicine, University of Southern California |
Panelists
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Eyquem described how his team reprograms immune cells with gene-editing tools to enhance T-cell function, with a focus on simpler delivery methods that could make these therapies more affordable and widely available.
Goldberg stressed the urgency of progress in pediatric oncology, where combination therapies are needed over monotherapies and underfunding remains a persistent challenge.
MacKenzie shared her genome surgery work at UCSF, which centers on earlier diagnosis and bespoke genomic therapies to treat genetic conditions sooner and improve outcomes.
Staahl offered an industry view, explaining how Scribe uses CRISPR-based editing with CasX enzymes to pursue precision and scalability, including work on LDL cholesterol.
Much of the conversation centered on the “valley of death” between laboratory discovery and a clinically viable therapy. Panelists agreed that partnerships between academia and industry are essential to crossing it. Eyquem called for sustained funding for trials that can fail and adapt, noting limited venture capital interest in high-risk, high-reward projects. MacKenzie added that academia has a moral responsibility to push rare disease therapies forward.
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Speaker Catherine Ames Patient Advocate |
Patient advocate Catherine Ames opened the afternoon with her own story. In 2018, while still in college, she began experiencing debilitating symptoms and was soon diagnosed with several complex conditions, including IgG2 subclass immune deficiency, postural orthostatic tachycardia syndrome, and Ehlers-Danlos syndrome. She turned to writing, launching a column called “Chronically Catherine” that connected her with a wider community of people living with chronic illness and gave her a platform for advocacy.
Ames urged the scientists in the room to involve patients early in drug development. The outcomes that matter most to patients, she explained, often include reducing brain fog, pain, and other symptoms that shape daily life, not only finding a cure. She also pointed to a persistent gap: many patients still struggle to get a proper diagnosis, which is the first step to accessing any treatment. Her message was clear: Patients are essential partners in therapeutic development, and their insights belong at the table from the outset.
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Moderator Shyam Patel, PhD Associate Vice President of Patient Access, California Institute for Regenerative Medicine (CIRM) |
Panelists
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Partnerships build trust. Adam Clark of the Emily Whitehead Foundation emphasized collaboration among patients, providers, researchers, industry, and advocacy groups. Engaging these stakeholders, he said, builds the trust needed for programs that meet real patient needs.
Infrastructure matters close to home. Lynda Polgreen, MD, a pediatric endocrinologist at Harbor-UCLA Medical Center, described the resource constraints safety-net hospitals face in delivering advanced therapies. Her team recently received a CIRM Community Care Center of Excellence grant to build local infrastructure, including upgraded pharmacy facilities and expanded ambulatory care. The goal is to reach patients who are functionally isolated from leading medical centers despite living nearby.
Simpler manufacturing can bring therapies closer. Alun Carter, PhD, of Miltenyi Biotec shared how the company is developing platforms that simplify manufacturing and make decentralized production more feasible, while maintaining safety.
Education and navigation help patients follow through. Clark stressed that physician education matters as much as patient education, since misinformation can keep patients from life-saving therapies. Panelists also highlighted the role of nurse navigators and peer connections in guiding patients through a complex process.
Cost remains the biggest barrier. Insurance covers FDA-approved treatments, but the financial burden often remains out of reach for underserved patients. Panelists pointed to decentralized manufacturing and direct in vivo cell therapies as paths to lower costs, and hinted at a potential California pilot to develop new ways of financing these therapies.
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Moderator Randy Berholtz Life Sciences Entrepreneur, Venture Capitalist, and Board Member |
Panelists
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AI can lower costs. Alive Xiao, Co-Founder and CEO of Angelonia AI, outlined AI’s potential in therapeutic design and manufacturing. By improving batch consistency and manufacturing reproducibility, AI could reduce production costs and make these therapies more accessible.
Ethics must keep pace. Robert A. Bohrer, Professor Emeritus of Law at California Western School of Law, stressed the need to address consent, data privacy, and the use of genomic data to ensure equitable access and prevent misuse. Panelists raised concerns that AI could widen disparities in care for marginalized communities.
Regulation is still taking shape. FDA’s current guidance on AI takes a risk-based approach, and frameworks are being developed to clarify how AI informs decisions and how errors are addressed. Panelists noted that existing guidelines remain in draft form and that regulators will need to adapt quickly.
Keep the human element. Fr. Peter Fonseca offered a bioethical and religious perspective, arguing that AI should remain a tool rather than an autonomous decision-maker and that its use must respect human dignity. The question, he suggested, is not only what AI can achieve but what it should achieve when treating patients.
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Looking Ahead
Across every session, one theme held steady: progress in cell and gene therapy depends on the convergence of science, regulation, and new business models, and on keeping patients at the center of all three. The breakthroughs are real and accelerating. Turning them into widely accessible treatments will take continued collaboration among researchers, clinicians, regulators, industry, and the patients whose lives these therapies are meant to change.
FAQ: Cell and Gene Therapy Symposium 2026
It’s the gap between a promising laboratory discovery and a clinically viable therapy. Symposium panelists pointed to academic and industry partnerships, along with sustained funding for high-risk trials, as the keys to crossing it.
The legal framework for drug regulation has stayed largely the same, but FDA has issued guidance tailored to cell and gene therapies and offered flexibility for innovative treatment designs.
Cost, limited infrastructure at safety-net hospitals, and gaps in patient and physician education. Panelists pointed to decentralized manufacturing and in vivo therapies as ways to lower costs.
AI can improve therapeutic design and make manufacturing more consistent and reproducible, which could reduce costs. Panelists stressed that consent, data privacy, and equity must be addressed as it is adopted.
The California Institute for Regenerative Medicine funds research and infrastructure across the field, from cardiovascular genetics research at UC San Diego to a Community Care Center of Excellence at Harbor-UCLA Medical Center.