Cell & Gene: The Podcast

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Synthetic Biology And Programmable mRNA
Explore genetic regulation, synthetic biology's role in genetically programming mRNA to deliver therapies, and how Strand Therapeutics is enabling precise and controlled delivery with these mRNA constructs.  Continue Reading..
  • Elixirgen Therapeutics' Aki Ko joins Erin Harris to detail the company's Bobcat mRNA™ therapeutic and how it differs from traditional exon-skipping drugs and AAV-microdystrophin in treating DMD.

  • Dr. Bambi Grilley is 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 she is Chief Regulatory Officer at ISCT. Dr. Grilley talks to us about her role as CRO at ISCT and the global regulatory landscape for cell and gene therapies.

  • In anticipation of ISCT 2024 in Vancouver, Canada, Dr. Bruce Levine is back on Cell & Gene: The Podcast to share information about the event as well as his expertise on the future of CAR-T therapy for solid tumors, autoimmune disease, and more.

  • On this episode of Cell & Gene: The Podcast, Addimmune CMO, Dr. Marcus Conant, shares the why behind cell and gene therapy to treat HIV, and explains the company's lead asset, AGT103-T, which is designed to provide broad protection using silencing RNA to inhibit binding/entry of the virus, halt replication, and prevent escape.

  • BlueRock Therapeutics' SVP Head of Development, Dr. Ahmed Enayetallah, joins Cell & Gene: The Podcast Host, Erin Harris, to discuss the company's phase I clinical trial for Parkinson’s disease, which continues to show positive trends at 18 months.

  • In 2021 on Episode 13, Vor Bio’s President and CEO, Dr. Robert Ang shared data about the company’s lead engineered hematopoietic cell (eHSC) product candidate. Dr. Ang is back to talk to Cell & Gene: The Podcast Host, Erin Harris, about how Vor has treated 8 patients demonstrating clinical proof of concept and is also in the clinic with a CAR-T that could be used in combination with shielded stem cell transplants.

  • Orca Bio's CMO, Dr. Scott McClellan, joins Erin Harris on this episode of Cell & Gene: The Podcast to discuss how and why toxicities continue to stymie allogeneic cell therapies. They cover Orca Bio's plan to potentially lowering the risk of GvHD as well as Orca-T, the investigational high-precision allogeneic cell therapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies.

  • On this episode of Cell & Gene: The Podcast, Host Erin Harris talks to Life Biosciences' CSO, Sharon Rosenzweig-Lipson, Ph.D., about the Boston-based biotech's cellular rejuvenation therapies for the treatment of age-related diseases.

  • Rare Disease Day is February 29, 2024, and in honor of this important day, Cell & Gene: The Podcast Host Erin Harris sits down with Jackson Laboratory’s (JAX) Rare Disease Translational Center (RDTC)'s VP, Cat Lutz.

  • On this episode of Cell & Gene: The Podcast, Kite Pharma's Global Head of Technical Operations, Christopher McDonald talks to Host Erin Harris about Kite's recent FDA approval of manufacturing process change resulting in reduced median turnaround time for Yescarta. They talk through the most significant steps Kite took to reach this approval, lessons learned from having worked with the FDA, scaling up technologies for cell therapy manufacturing, and more. 

  • The Dedham Group's Jennifer Klarer, M.Sc.Eng., Partner and Head of Cell & Gene Therapy, joins Erin Harris on this episode of Cell & Gene: The Podcast for a deep dive into pricing and value for CGTs. Klarer shares a detailed breakdown of the most misunderstood aspects of cell and gene therapy pricing, value, and affordability. She details how manufacturers can get ahead of addressing time-to-treatment issues typically experienced at launch. They cover obtaining adequate investment dollars, and much more.

  • Paul Lammers, M.D., M.Sc., CEO of Triumvira Immunologics joins Cell & Gene: The Podcast to talk to Host Erin Harris about TAC, the company's proprietary T cell Antigen Coupler, which has both autologous and allogeneic approaches. They also discuss targeting relapsed or refractory HER2-positive solid tumors and CLDN18.2-positive solid tumors. And they cover a realistic outlook on the evolution of cancer treatment.

  • Kristin Yarema, Ph.D., CEO, Poseida Therapeutics sits down with Cell & Gene: The Podcast Host Erin Harris to discuss four of the biggest challenges the CGT sector faces when it comes to the development of off-the-shelf therapies. Kristin shares how 2024 might see some real progress in allogeneic therapies. 

  • A first-time attendee of Meeting on the Mesa discusses his experience and delves into the cell therapy used to treat corneal endothelial disease.

  • Vittoria Biotherapeutics’ CEO shares the company's mission to expand the applications for CAR-based therapeutics by employing novel cell engineering and gene editing technologies.

  • Astellas' Richard Wilson is Erin Harris' guest on Cell & Gene: The Podcast to discuss deals and collaborations in gene therapy. 

  • What does the future of AAV gene therapy hold and how can you achieve scalability in manufacturing with consistent yield and product purity?

  • Hear from speakers on the Cell & Gene: The Podcast about Meeting on the Mesa 2023, the events new location for 2024, major topics and trends at this year's event, and ARM's GROW internship program.

  • Explore the biggest and most troubling challenges facing the treatment of Type 1 Diabetes (T1D) and the future of cell and organ transplantation in treating T1D as well as the latest innovations in biopreservation.

  • Discuss the evolution of allogeneic, in vivo CAR-T cell therapeutics, recent progress in lentiviral vector manufacturing, and The Colorado Laboratory and Innovation Manufacturing Building.

  • Learn about base editing and prime editing and their potential to meet unmet medical need from the FDA's Center for Biologics Evaluation and Research.

  • Listen to a discussion on the near-term future of CAR-T, along with a deep dive into various innovations and patient access progress being made in both solid tumors and hematologic malignancies.

  • Experts discuss the evolution of genome editing, its present state, as well as its undeniable potential.

  • Stephan Krause, Ph.D., Executive Director, AS&T, CT Global Quality, at Bristol Myers Squibb, discusses the major themes and topics from the Advanced Therapy Medicinal Products Conference, as well as his focus areas for cell therapy in 2023 and beyond.

  • How is RNA technology shaping the future of gene therapy? Find out the role AI is playing along with how new technologies are effecting the CGT sector.

  • What is prime editing and why is it considered a major technological advancement in gene editing?

  • Dive into the theme of the 2023 PDA ATMP conference, along with discussing CGT manufacturing strategy with the President and CEO of Parenteral Drug Association.

  • Cell and gene therapy expert Dr. Usman "Oz" Azam provides his valuable perspective on the therapy space and answers questions about the FDA, solid tumor cancer treatment, and much more.

  • Hear from the CEO of a therapeutics company about how they are engineering immune cells to target oncogenic driver mutations, such as KRAS, to minimize potential tumor escape mechanisms.

  • Parviz Shamlou, Vice President and Executive Director at Thomas Jefferson University joins Cell & Gene: The Podcast to talk to Host Erin Harris about Jefferson Institute for Bioprocessing (JIB), the full-service development and training organization specializing in biopharmaceuticals and biologics. Shamlou explains how JIB will move forward cell and gene therapies as well as JIB’s partnership with NIBRT (National Institute for Bioprocessing Research & Training), and much more.

  • Shankar Ramaswamy, M.D., CEO & Co-Founder, Kriya Therapeutics joins Cell & Gene: The Podcast's Erin Harris for a discussion on the evolving role computational biology plays in gene therapy development. They also discuss the company's lessons learned from building its own North Carolina-based in-house manufacturing facility as well as Ramaswamy's insight on the future of gene therapy. 

  • Dr. Linda Marbán, Ph.D., CEO of Capricor Therapeutics, a clinical-stage biotech focused on the development of cell and exosome-based therapeutics for the treatment and prevention of serious diseases, joins Cell & Gene: The Podcast's Erin Harris to discuss the current state and the near-term future of exosomes. Marbán discusses Capricor’s exosome program and the company’s proprietary allogeneic cardiosphere-derived cells (CDCs) and engineered exosomes. She also covers the company's HOPE clinical trial series' progress to treat Duchenne muscular dystrophy (DMD).

  • In December 2022, Atara Biotherapeutics’ Ebvallo received European Commission approval as the first-ever therapy for adults and children with EBV+ PTLD. The ground-breaking approval represents the first approval of an allogeneic T-cell immunotherapy ever, globally. Atara Bio's CEO, Pascal Touchon, talks to Cell & Gene: The Podcast's Erin Harris about the biopharma's path to regulatory approval, commercialization plans for Ebvallo, an in-depth look at EBV+ PTLD, the connection between EBV and serious diseases like multiple sclerosis (MS), and much more.

  • Cell & Gene: The Podcast Host, Erin Harris, welcomes Romesh Subramanian, Ph.D., CEO of Ascidian Therapeutics, a Boston-based biotech focused on rewriting RNA to the pod. During the episode, Subramanian defines RNA exon editing, and they discuss the differences between RNA exon editing and gene editing. With current programs in ophthalmology, neurological and neuromuscular disorders, as well as rare diseases, Subramanian explains the overwhelming potential of RNA exon editing.

  • When it comes to manufacturing, many cell and gene therapy companies struggle to automate the diverse processes needed to scale manufacturing. London-based PA Consulting's Associate Partner, Paolo Siciliano, talks to Cell & Gene: The Podcast's Erin Harris about how lean, small biotechs with limited resources and limited funds can strive for automation success. They also discuss what cell and gene therapy companies should do today to address tomorrow’s scalability challenges and what the long-term effects on the business may be from such upfront work.

  • On this episode of Cell & Gene: The Podcast, Host Erin Harris talks to Andy Ramelmeier, EVP Head of Technical Operations at Sangamo Therapeutics, a biotech based in Brisbane, California that applies cell and gene therapy to combat Hemophilia A and other genetic diseases. Ramelmeier explains the why behind the company's decision to build its own Phase 1/2 cGMP AAV in-house facility. He also talks broadly about manufacturing cell and gene therapies and the potential and realistic solutions to needed to drive down manufacturing costs.

  • Miguel Forte is Entrepreneur in Residence at AdBio, a VC firm based in France, and President Elect of ISCT. Forte and Cell & Gene: The Podcast host, Erin Harris, break down market outlook perspective for the cell and gene therapy space. He provides valuable tips to employ when seeking VC partnership, and he covers ISCT's primary focus areas and what to expect from ISCT 2023 Paris.

  • Mustang Bio's Robert Sexton, VP Program and Alliance Leadership, explains the why behind the need for this role in any CGT company. Having worked for Novartis, Sanofi, Legend Biotech, and more, with responsibilities having included quality, operations, and order management, Sexton is currently responsible for aspects of CMC. As such, he addresses the industry's top CMC challenges as well as sound advice for future scale up. 

  • Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER) within the Food and Drug Administration (FDA) sits down with Cell & Gene: The Podcast host Erin Harris to discuss the most anticipated areas of innovation in clinical development, and he offers recommendations to companies in their engagement with the FDA in order to make manufacturing more streamlined and cost effective. Dr. Marks also provides advice to listeners about how and when to engage with CBER, and much more.

  • Shoreline Biosciences' Chief Scientific Officer Dr. Robert Hollingsworth shares his insight on intelligently designed allogeneic off-the-shelf, standardized, and targeted iPSC-derived natural killers (NK) and macrophage cellular immunotherapies. He explains the company's preclinical testing on a potential NK cell therapy for acute myeloid leukemia. We discuss how the field recognizes the importance of off-the-shelf NK cell therapy not only for cost reduction but also for improving safety measures. As such, Dr. Hollingsworth talks through why NK cells are necessary to move immunotherapies forward.

  • BlueRock Therapeutics' President and CEO Seth Ettenberg, Ph.D. talks to Cell & Gene: The Podcast listeners about the company's Phase 1 Clinical Trial for Advanced Parkinson’s Disease. Ettenberg also covers the most promising therapeutic applications for iPSCs currently in development as well as the major regulatory challenges that the field faces for their clinical use. We also cover how far iPSCs have come and what future progress may entail.

  • Orgenesis' CEO Vered Caplan explains the business model behind providing cell and gene therapies that may be manufactured at the point of care. She details how working with hospitals and healthcare providers to enable them to participate in CGT development on their sites and providing these therapies in-house by adapting them to closed system manufacturing rather than relying on the typical biotech model is an important step forward in CGT manufacturing.

  • Capstan Therapeutics' CEO, Laura Shawver, Ph.D., talks us through the company's in vivo CAR therapies and what led to the in vivo cell engineering breakthrough that has been decades in the making. Shawver explains the promise of harnessing mRNA and targeted LNP delivery to train a patient’s body to make CAR-T cells in vivo. She also details best practices to garner funding and investment partnership.

  • PepGen, a Cambridge, MA-based biotech, is advancing oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases. SVP Head of Clinical Development Dr. Michelle Mellion explains PepGen's trial of PGN-EDO51, the company’s lead product candidate for the treatment of DMD patients whose mutations are amenable to an exon 51 skipping approach. She also explains how the delivery of oligonucleotide therapies differ from other techniques in genetic medicine, and much more.

  • On the heels of Congress' reauthorization of the Prescription Drug User Fee Authorization (PDUFA) Act, BridgeBio's Chief Regulatory Affairs Officer, Adora Ndu, explains the Commitment Letter, the programs that may be rolled out under PDUFA VII, why right-sizing CBER will help the cell and gene sector going forward as well as how sponsor companies can prepare for 2023 from a regulatory perspective.

  • Arrowhead Pharmaceuticals is a biopharmaceutical company based in Pasadena, CA that develops medicines that treat intractable diseases by silencing the genes that cause them. Javier San Martin, Arrowhead's CMO, talks to Cell & Gene: The Podcast listeners about the similarities and differences between RNAi and CRISPR. He also explains the roadmap for RNAi as well as the company's pipeline development strategy, which includes pre-clinical to Phase 3 trials.

  • Will Junker, Head of Manufacturing Quality at Kite Pharma, talks to Cell & Gene: The Podcast listeners about the challenges associated with capacity planning, whether to partner with a CDMO, and why it's important to prioritize inventory over time-to-market. We also discuss why developing a better understanding of the vector manufacturing process is critical to bringing continuous and secure supply to market. 

  • With 20+ gene therapy targets in Novartis Gene Therapies’ pipeline, President Chris Fox and her team are hyper-focused on newborn screenings as the pathway to helping more and more patients. The company's initial gene therapy, Zolgensma, for spinal muscular atrophy (SMA) has been approved in more than 40 regions and countries and has been used to treat more than 2,300 patients worldwide. Fox details what’s next on the commercialization front for the therapy, and she shares advice with Cell & Gene: The Podcast listeners about what it takes to commercialize a therapy.

  • Sernova Corp. is a clinical-stage regenerative therapies company currently focused on insulin-dependent diabetes. CEO and President, Philip Toleikis, Ph.D., details the company's on-going clinical trial to treat Type 1 diabetes (T1D) as well as the very real potential to progress from simply masking symptoms to having a functional cure for T1D. Toleikis also provides his take on the near-term future of regenerative medicine.


Erin Harris

Cell & Gene, the most valuable resource for delivering in-depth content from authoritative authors and sources to professionals in the CGT sector, brings you Cell & Gene: The Podcast. Join us as Cell & Gene Chief Editor, Erin Harris interviews industry and academic leaders on what they’re working on and how it's moving the sector forward. Each episode of Cell & Gene: The Podcast will explore approved and pipeline therapies, new trends in manufacturing, the long, regulated road to commercialization, and everything in between.