Cell & Gene: The Podcast

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In episode 119 of Cell & Gene The Podcast, Host Erin Harris talks to Dr. Norman Putzki, Global Head Clinical Development, Novartis, about the FDA approval of Itvisma, now the only gene replacement therapy approved for children, adolescents, and adults with spinal muscular atrophy (SMA). Dr. Putzki walks us through the six-year development journey behind the STEER and STRENGTH Phase 3 programs. And we explore what the expanded age-range label means for patients who were previously left behind, why intrathecal, fixed-dose AAV delivery represents a pivotal advance for safety...

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In Episode 118, Host Erin Harris talks to Alexander Cryer, Ph.D., Instructor in Medicine at Mass General Brigham, about a proof of concept strategy that reprograms tumor cells with mRNA lipid nanoparticles to overactivate the cGAS-STING pathway, forcing cancer cells to produce and export large amounts of the innate immune agonist cGAMP to stimulate surrounding immune cells and drive anti-tumor immunity. Dr. Cryer explains the basic biology of cGAS-STING and how his team restored this pathway in tumor cells and leveraged intratumoral LNP delivery to overcome...

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On episode 117, Host Erin Harris welcomes Paul Perreault, Strategic Advisor, Board Member and former CEO of CSL, who shares insights from his decade leading the company through global expansion and the COVID-19 pandemic. Perreault highlights the importance of patient-centered culture, strategic focus, and adaptive leadership for cell and gene therapy innovators. He offers practical advice on building a values-driven culture that connects employees to patients, the importance of focusing on core competencies to attract capital, and the necessity of continuous leadership development, even advocating for mentorship...

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In episode 116 of Cell & Gene: The Podcast, Host Erin Harris talks to Dr. Arun Upadhyay, Chief Scientific officer, Head of Research and Development at Ocugen to discuss how the company’s modifier gene therapy platform is redefining treatment possibilities for inherited retinal diseases such as retinitis pigmentosa, Stargardt disease, and geographic atrophy. Dr. Upadhyay explains how Ocugen’s gene-agnostic approach differs from traditional single-gene therapies by targeting shared disease pathways to preserve photoreceptors and slow vision loss across diverse genetic mutations. He also shares key lessons lear...

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Episode 115 of Cell & Gene: The Podcast features Host Erin Harris' talk with Aliya Omer, Vice President and Global Head of Hematology and Cell Therapy at AstraZeneca. Omer shares valuable insights from her rich experience leading cell therapy development across multiple top companies. She highlights the critical importance of collaboration by breaking down silos across research, manufacturing, regulatory, and commercial teams to deliver innovative therapies efficiently. She also discusses AZ’s diverse and ambitious cell therapy portfolio, encompassing autologous CAR-T, TCR-T, in vivo gene therapies, and regulatory T...

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On episode 114 of Cell & Gene: The Podcast, Host Erin Harris talks to Brian Culley, CEO of Lineage Cell Therapeutics, about advancing cell therapy beyond oncology and into transformative treatments for conditions such as spinal cord injury and blindness. Culley shares how Lineage’s allogeneic, off-the-shelf approach, anchored by its OPC1 program, aims to replace lost or damaged cells to restore function, starting with patients who have plateaued after chronic spinal cord injury. He details the DOSED study’s innovative delivery device and thaw-and-inject formulation, both designed to s...

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Welcome to Episode 113 of Cell & Gene: The Podcast. Host Erin Harris is joined by Thorsten Gorba, Ph.D., VP Process Development at Aspen Neuroscience to explore how the company is advancing the field of cell therapy manufacturing. Aspen Neuroscience stands at the forefront of integrating machine vision and AI/ML to assess induced pluripotent stem cell (iPSC) colony quality by offering a transformative approach to move beyond the subjective nature of manual evaluation. Dr. Gorba discusses how these technologies bolster reproducibility and scalability and help reduce...

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On episode 112 of Cell & Gene: The Podcast, Michael Quigley, Ph.D., Chief Scientific Officer and Global Head of Research at Sanofi talks to Host Erin Harris about the establishment of Sanofi’s dedicated Genomic Medicine Unit (GMU). Dr. Quigley emphasizes in vivo delivery and process optimization to improve patient experience, scalability, and global access. They discuss the importance of partnerships with academia, industry, and regulators, and Dr. Quigley discusses how advances in AI are accelerating research efficiency, molecule optimization, and experimental design across Sanofi’s portfolio. He a...

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On episode 111 of Cell & Gene: The Podcast, Host Erin Harris talks to Ernexa Therapeutics' President and CEO, Sanjeev Luther, about how the company is advancing cell therapy innovation for cancer and autoimmune disease through engineered induced pluripotent stem cell-derived mesenchymal stem cells (iMSCs). Luther shares how Ernexa’s synthetic, standardized, off-the-shelf iMSCs are designed to overcome historic hurdles in MSC therapy, including variability, scalability, and manufacturing complexity, by leveraging master cell banks, precise gene editing, and novel product engineering. He also shares insights into the process of...

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This is Episode 4 of "FDA Fridays," a special series from Cell & Gene: The Podcast, Chief Editor Erin Harris speaks with regulatory affairs expert Daniela Drago, Partner, NDA Partners, about how shifting FDA and CBER priorities under the current administration could influence the trajectory of cell and gene therapy development. From the impact of leadership changes and evolving policy directives to the role of funding allocations and public health priorities, Drago sheds light on what CGT developers and sponsors should be watching most closely. She explores regulatory...

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In this episode of Cell & Gene Podcast episode, Host Erin Harris talks to Stanford School of Medicine Ph.D. student, Yuanhao Qu, about his work developing CRISPR-GPT, an AI-driven multi-agent system designed to automate genetic experimental design and data analysis, making CRISPR experiments more efficient and accessible, even for non-experts. Qu explains how CRISPR-GPT addresses key challenges such as guide design, delivery methods, off-target prediction, and protocol generation, and shares how collaborations with Princeton helped shape the tool’s architecture and evaluation. Qu also discusses Biomni, a...

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This is Episode 3 of "FDA Fridays," a 4-week special series from Cell & Gene: The Podcast. Host Erin Harris talks to Bambi Grilley, Ph.D., Professor of Pediatrics and the Director of Clinical Research and Early Product Development for the Center of Cell and Gene Therapy (CAGT) at Baylor College of Medicine and Chief Regulatory Officer for ISCT, a leading expert at the forefront of pediatric cell and gene therapy. Dr. Grilley shares her wealth of experience and unique perspective on the most pressing barriers to accessing...

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Episode 2 of FDA Fridays, the special series from Cell & Gene: The Podcast, features Host Erin Harris' discussion with regulatory expert Monika Swietlicka, Principal, Regulatory Strategy, Halloran Consulting Group about the key strategies and challenges in navigating FDA regulation for cell and gene therapies. Their conversation covers the importance of defining a therapy’s intended role (curative, chronic, or adjunctive) early in development, the different regulatory expectations based on disease context and patient population, and the crucial impact of unmet medical need on regulatory flexibility. Swietlicka emphasizes th...

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In Episode 109 of Cell & Gene: The Podcast, Host Erin Harris talks to Will Chou, M.D., CEO of Passage Bio about the promise and challenges of developing gene therapies for frontotemporal dementia (FTD), a neurodegenerative disease with strong genetic underpinnings. Dr. Chou discusses the biological basis of FTD and the complex scientific, manufacturing, and regulatory hurdles that come with targeting the brain. They discuss the latest interim results from the upliFT-D study of Passage Bio’s PBFT02, including safety and tolerability findings, dose cohort comparisons, and th...