Cell & Gene Therapy Symposium 2025

California Life Sciences and the California Institute for Regenerative Medicine (CIRM) hosted their second annual Cell & Gene Therapy Symposium, convening leaders across academia, industry, policy, and patient advocacy to examine the promises and challenges of cell and gene therapies.

Across panel discussions exploring the scientific breakthroughs, commercial realities, patient perspectives, and policy imperatives that shape the ecosystem, several common themes emerged:

• Alignment of regulatory, commercial, and manufacturing strategy from the start is critical.
• Equity and access must be built in, not retrofitted.
• Standardization and scalability are crucial to moving from bespoke therapies to population-level impact.
• California’s leadership remains central to advancing science, policy, and equity simultaneously.

California’s unique role as a national and global leader, with its ecosystem of world-class academic medical centers, CIRM-backed infrastructure, and vibrant biotech community, position our state to define the future of cell and gene therapy. With our infrastructure, talent, and patient advocacy leadership, we have the power to not only sustain innovation but also define a model where these therapies are both scientifically transformative and equitably accessible.

Promises and Challenges of Cell and Gene Therapy

Paula Cannon, PhD, Distinguished Professor at the USC Keck School of Medicine and President of the American Society for Gene and Cell Therapy, opened the symposium by framing the cell and gene therapy (CGT) field as both a scientific revolution and an industry at a crossroads. She highlighted how genome editing, engineered immune cells, and novel delivery systems are transforming treatment options for diseases once considered untreatable, from rare monogenic disorders to complex conditions such as cancer and HIV.

CGT has moved from proof-of-concept to tangible clinical outcomes, with more therapies reaching approval globally. The expansion into programmable B cells and advanced CRISPR applications suggests therapies will become more durable, personalized, and potentially preventative, Dr. Cannon predicts. However, cost, complexity, and scalability are central hurdles to getting these groundbreaking treatments to patients that need them. Despite scientific breakthroughs, the price of therapies often exceed $1 million, putting sustainable reimbursement and equitable patient access into question.

With institutions like USC, City of Hope, Cedars-Sinai, Stanford, and the California Institute for Regenerative Medicine (CIRM), California remains a hub for discovery, translational research, and clinical development. Dr. Cannon noted that California’s ecosystem, with its combination of academic excellence, biotech innovation, and patient advocacy, is uniquely positioned to tackle access and affordability. To achieve these goals, she urged closer alignment between academia, industry, policymakers, and payers to ensure that innovation translates into real-world impact.

Commercial Headwinds

This session addressed the critical economic and commercial challenges shaping the future of cell and gene therapies. Panelists emphasized that while science is advancing rapidly, the industry has yet to establish a sustainable commercial model that ensures both profitability and patient access.

Manufacturing Innovations: How we Scale Up in an Affordable Way

This panel tackled one of the most pressing questions in the CGT field: how to build scalable, affordable, and sustainable manufacturing systems. The discussion explored not just technical barriers, but also economic, regulatory, and organizational challenges.

Key Themes

Rethinking “Scale”

• Panelists emphasized that scale is not one-size-fits-all. For rare diseases, speed and flexibility matter more than volume; for large-population indications (e.g., Parkinson’s), scalability must be built in from the start.
• Scaling requires parallel attention to analytics, supply chain, scheduling, and data systems, not just the cleanroom.

Selective Automation

• Automation is a strategic enabler but not universally applicable.
• Areas such as iPSC derivation, gene editing, and standardized operations (e.g., vector production, cell washing) are well suited for automation.
• However, for individualized or complex processes, selective automation and human oversight remain critical.

Fail Fast vs. Plan Smart

• Panelists debated the idea of “fail fast.” In biotech, failure carries heavy costs. Instead, the group stressed planning with the end in mind: early regulatory engagement, robust recordkeeping, and cross-functional input to reduce late-stage setbacks.
• Investors play a role by supporting long-term planning and helping companies build world-class teams, not just funding short-term milestones.

Phase-Appropriate Approaches

• Using phase-appropriate materials (e.g., research-grade plasmids for early trials) can save cost and time if regulators are engaged early.
• Basket trials and platform designations were highlighted as regulatory pathways to support rare and orphan indications with limited patient populations.

AI & Digital Tools

• AI is promising in predictive modeling of batch outcomes, image analysis, and regulatory documentation.
• Current limitations include small datasets and variable data quality; synthetic data and standardized processes may help bridge the gap.
• Panelists also noted immediate benefits of AI in administrative tasks (investigations, record review) and training (e.g., VR for aseptic techniques).

Patient Perspective

• A patient advocate challenged the field to look beyond “cures” and consider regenerative outcomes that restore function and quality of life.
• Simplifying delivery mechanisms (reducing reliance on chemo pre-conditioning or complex hospital care) was highlighted as critical for equitable access.

Panelists underscored that scaling CGT manufacturing is not about blanket automation, but about smart, situational innovation. Success will depend on early regulatory dialogue, cross-disciplinary integration, selective application of digital tools, and patient-centered design. For California, the convergence of biotech, AI, and clinical infrastructure provides a unique platform to set new global standards in scalable CGT production.

Patient Advocate Address

Adrienne Shapiro, Founder of Axis Advocacy, delivered a deeply personal and powerful account of dealing with sickle cell disease across five generations of her family. Her remarks underscored the human urgency behind scientific innovation, reminding the audience that progress in CGT is measured not just in regulatory approvals, but in lives extended.

Adrienne shared how her advocacy began as a mother and a caregiver, and has since flourished into becoming a national voice for patients. She credited CIRM with helping her find her voice and shaping her role as a bridge between patients, scientists, and policymakers. Adrienne then traced the arc of progress in sickle cell disease advocacy, from early clinical descriptions in 1910 and activism by the Black Panthers in the 1970s that drove the creation of federal sickle cell programs, to scientific advancements like newborn screenings, the introduction of hydroxyurea, and the first bone marrow transplants.

Throughout her address, Adrienne challenged the field to push beyond discovery towards affordable, scalable solutions that truly address patient needs. She urged inclusion of patients at every stage of development, from trial design to delivery models, arguing that therapies cannot succeed if they fail to reflect the lived experiences of those they aim to serve.

Adrienne’s address at the symposium reminded the audience that the ultimate measure of success in CGT is not just cures discovered, but cures delivered equitably. Her charge to the field: integrate patient voices from the start, commit to affordability and access, and use California’s leadership to set a standard where cures are not for the few, but for all.

The Role of Academic Medical Centers

This panel explored how academic medical centers (AMCs) serve as the critical engine for advancing cell and gene therapies (CGTs) from early discovery to patient access. Panelists highlighted the unique ability of AMCs to integrate discovery science, translational development, GMP manufacturing, regulatory engagement, and clinical trials — all within a single ecosystem.

Key Themes

Early Innovation & Risk-Taking

• Many groundbreaking therapies begin with philanthropic or foundation support at AMCs, which can back high-risk, high-reward ideas until they reach IND-enabling studies.
• Academic flexibility allows investigators to pursue ideas that may lack short-term commercial appeal but hold long-term clinical promise.

Common Pitfalls in Translation

• Panelists noted challenges such as starting with cell lines or processes that lack clinical viability, or developing protocols too complex to scale.
• The need for quality by design and early attention to identity, purity, and potency assays was emphasized. Engaging manufacturing teams before publication can prevent costly course corrections later.

Manufacturing & Platforms

• Discussion on the importance of platform processes — modular, phase-appropriate systems that accelerate first-in-human trials while being adaptable for commercial scalability.
• AMCs provide a natural space for building these translational “engines” with functional assays that support regulatory flexibility.

Integration of Science, Manufacturing, and Clinical Insight

• Dr. Porteus highlighted the “triangle” of discovery scientists, process engineers, and clinicians working side by side in AMCs, which is difficult to replicate in industry or CDMOs.
• Perseverance in academia allows therapies to mature through decades of iteration — exemplified by CAR-T and CRISPR progress.

Patient-Centered Clinical Innovation

• Dr. Wayne recounted the early pediatric CAR-T trials, including Emily Whitehead’s landmark case, as examples of AMC environments enabling rapid bedside-to-bench problem-solving.
• AMCs are also central to post-approval adoption, especially in states like California where Medicaid coverage requires negotiation between AMCs, government, and payers to ensure equitable access.

Industry Partnerships

• Panelists described evolving partnership models:
  • Resident partner programs where company staff embed in AMC GMP facilities.
  • Shared risk arrangements, including equity in exchange for services.
  • Licensing out validated targets or processes to industry for speed, with AMCs reinvesting through sponsored research.
• Despite Big Pharma retrenchment, AMCs provide continuity and credibility, often de-risking programs before handoff.

Beyond the Lab: Incentives & Access

• A recurring theme was the need to reward process engineering and cost reduction in academic promotion and funding systems.
• AMCs must also expand community access through hub-and-spoke models, ensuring patients outside major centers benefit from advanced therapies.

Academic medical centers are not just research hubs; they are “de-risking engines” that carry CGTs from idea to patient. Their ability to integrate discovery, manufacturing, regulatory strategy, and clinical expertise under one roof makes them indispensable to the CGT ecosystem.

In California, AMCs are uniquely positioned to set national standards in accelerating innovation, reducing costs, and expanding access. California’s AMCs — USC, Stanford, Cedars-Sinai, City of Hope, CHLA — represent a critical mass of translational capacity, supported by CIRM and a culture of patient engagement. The state’s role in pioneering Alpha Clinics and novel nonprofit models (e.g., Access for Kids) demonstrates how AMCs can push beyond discovery into equitable adoption frameworks.

Patient & Market Access

This session examined the barriers patients face in accessing cell and gene therapies (CGTs), from manufacturing delays to payer denials, and explored how California’s infrastructure can drive more equitable delivery.

Key Themes

Financial & Operational Strain on Academic Centers

• Panelists noted growing pressures on academic medical centers (AMCs), with shrinking budgets and slower patient throughput despite rising demand.
• Long waiting lists for CAR-T and gene therapy infusions are compounded by supply chain and manufacturing constraints, leaving eligible patients untreated.

Insurance & Reimbursement Barriers

• Malika described extensive payer delays, sometimes requiring months of appeals for a single pediatric patient. Denial and deferment have become routine payer strategies, creating life-threatening lags.
• Safety-net hospitals face particular strain, with limited margins and cash flow challenges when manufacturers demand payment before insurers remit.

Manufacturing as a Determinant of Access

• Feth emphasized that manufacturing bottlenecks, not demand, are the leading access barrier. Prolonged vein-to-vein timelines and limited production slots delay treatment.
• Decentralized and automated manufacturing technologies could enable care at community hospitals, reducing patient travel and expanding treatment beyond major AMCs.

CIRM’s Role in Access & Affordability

• CIRM integrates access requirements into its funding, mandating that grantees plan for affordability and delivery early in development.
• CIRM’s Community Care Centers of Excellence program will extend CGT clinical trial and treatment capabilities beyond major hubs, while training a broader workforce and partnering with local community organizations.
• The Patient Support Program now helps cover travel, meals, lodging, and childcare costs for California patients in trials, reducing financial barriers to participation.

Policy & Payment Models

• Panelists discussed outcome-based reimbursement models as a way to align cost with clinical benefit. While promising, these models risk placing AMCs in the role of financial intermediaries if payments are delayed or denied.
• Agreement that sustainable policy reform must address payer accountability, cost recovery, and manufacturing right-sizing alongside clinical innovation.

Access to CGTs remains constrained by reimbursement delays, manufacturing bottlenecks, and inequitable distribution of care. California is advancing solutions through funding accountability, infrastructure expansion, and patient support programs, but broader payer reform and scalable manufacturing innovation are needed to ensure therapies reach patients on time and at scale. The state’s policies can serve as a national model for embedding patient affordability and access guarantees into CGT funding and commercialization strategies.