We love to hear from our listeners. Send us a message. On this episode of Cell & Gene: The Podcast, Host Erin Harris talks to Ralf Schmid, Ph.D., Associate Director of Preclinical Research at Novartis Biomedical Research, about the evolving use of large animal models in gene therapy development. Dr. Schmid discusses the current reliance on non-human primates (NHPs), their growing logistical and ethical challenges, and the emerging interest in alternatives like genetically engineered pigs and...
Jul 17, 2025•25 min•Ep. 107
We love to hear from our listeners. Send us a message. In this episode of Cell & Gene: The Podcast, George Magrath, CEO of Opus Genetics, shares with Host, Erin Harris, how the company is advancing gene therapies for inherited retinal diseases (IRDs), with a focus on LCA5, a rare and severe form of childhood blindness. Built on the foundational science of Dr. Jean Bennett and leveraging the legacy of Luxturna, Magrath shares how Opus is developing subretinal AAV-based therapies targeting sev...
Jul 03, 2025•20 min•Ep. 106
We love to hear from our listeners. Send us a message. In this episode of Cell & Gene: The Podcast, Host Erin Harris interviews Dr. Gloria Matthews, Chief Medical Officer of Aegle Therapeutics, a clinical-stage regenerative medicine company focused on developing therapies for rare and severe dermatologic and immunologic disorders. Dr. Matthews explains how Aegle is pioneering the use of extracellular vesicles (EVs) derived from mesenchymal stem cells, which offer advantages over traditional ...
Jun 19, 2025•16 min•Ep. 105
We love to hear from our listeners. Send us a message. On this episode of Cell & Gene: The Podcast, Host Erin Harris welcomes Monika Swietlicka, Principal, Regulatory Strategy at Halloran Consulting Group to discuss the key regulatory challenges facing cell and gene therapy companies. Swietlicka highlights manufacturing complexity and clinical trial design limitations. On gene editing, she notes that the FDA is focused on off-target effects and demands robust data, including functional valid...
Jun 05, 2025•25 min•Ep. 104
We love to hear from our listeners. Send us a message. Host Erin Harris talks to Generate:Biomedicines' EVP of R&D, Dr. Alex Snyder about the convergence of AI, machine learning (ML), and synthetic biology in the development of next-generation therapies. They cover how AI is transforming drug discovery by enabling the rapid design and optimization of therapeutic candidates, particularly in complex fields like immuno-oncology and cell therapy. Dr. Snyder shares how that in the context of CAR-...
May 22, 2025•19 min•Ep. 103
We love to hear from our listeners. Send us a message. In this episode of Cell & Gene: The Podcast, Host Erin Harris sits down with Peter Altman, Ph.D., CEO of BioCardia, to discuss the company’s evolution from a cardiac biotherapeutic delivery firm to a developer of autologous and allogeneic cell therapies for cardiovascular and pulmonary diseases. Altman highlights BioCardia’s CardiAMP program, a precision medicine approach that pre-screens patients based on their cell profiles to improve ...
May 08, 2025•18 min•Ep. 102
We love to hear from our listeners. Send us a message. In this episode of the Cell & Gene podcast, Host Erin Harris speaks with Alicia Zhou, Ph.D., CEO of the Cancer Research Institute (CRI), about CRI’s mission to advance cancer immunotherapy and the critical role of federal funding, particularly from the NIH, in supporting basic and translational cancer research. Dr. Zhou highlights CRI’s focus on funding early-stage science and clinical trials to move immunotherapy toward curing all cance...
Apr 24, 2025•38 min•Ep. 101
We love to hear from our listeners. Send us a message. On the 100th episode of Cell & Gene: The Podcast, Host, Erin Harris, sits down with Audrey Greenberg, Founder and CEO of AG Capital Advisors. Audrey is the award-winning executive, board director, and strategic advisor with a proven track record of launching, scaling, and successfully exiting multi-billion-dollar ventures. They explore the biggest challenges and opportunities facing CGT biotech leaders today; the funding crisis in biotec...
Apr 10, 2025•28 min•Ep. 100
We love to hear from our listeners. Send us a message. In this episode of Cell & Gene: The Podcast, Host Erin Harris dives into the world of retinal gene therapy with Kenji Fujita, M.D., Chief Medical Officer of Atsena Therapeutics. Atsena develops treatments for inherited retinal diseases, including X-linked retinoschisis (XLRS), a rare genetic condition that currently has no FDA-approved therapy. They talk through the impact of XLRS on patients, the challenges of gene delivery to the retin...
Mar 27, 2025•15 min•Ep. 99
We love to hear from our listeners. Send us a message. In this episode, Host Erin Harris sits down with Dr. Simrit Parmar, Founder of Cellenkos, to explore how the company is pioneering umbilical cord blood-derived Regulatory T cell (Treg) cell therapies for autoimmune and inflammatory diseases. Dr. Parmar shares the vision behind Cellenkos, the advantages of cord blood-derived Tregs over other sources, and how their CRANE technology platform enhances precision in targeting diseases. They also d...
Mar 13, 2025•35 min•Ep. 98
We love to hear from our listeners. Send us a message. Omkar Kawalekar, Ph.D., Senior Manager, NextGen Therapies Manufacturing & Supply Chain Lead, Deloitte Consulting joins Host Erin Harris to share his expert take on how distributed manufacturing models address capacity constraints and supply chain risks, the role of automation in reducing batch-to-batch variability, as well as the power of digital technologies in optimizing production processes. He also covers the key considerations for C...
Feb 27, 2025•29 min•Ep. 97
We love to hear from our listeners. Send us a message. In this episode, Erin Harris sits down with Faraz Ali, CEO of Tenaya Therapeutics, to explore the cutting-edge world of gene therapies for heart diseases. As Tenaya makes significant strides in developing innovative treatments, Ali shares insights on Tenaya's gene therapy platform, their in-house manufacturing capabilities, and the potential impact of their therapies on prevalent heart conditions. They also discuss the complexities of commer...
Feb 13, 2025•36 min•Ep. 96
We love to hear from our listeners. Send us a message. Helen Sabzevari, Ph.D., President and CEO of Precigen, joins Host, Erin Harris to discuss how the company utilizes gorilla adenovirus vectors. These vectors offer significant advantages in delivering large genetic payloads, a crucial factor in developing effective gene therapies. Sabzevari also discusses Precigen's recent BLA submission for PRGN-2012, which includes Phase 1/2 pivotal study results where over 50% of patients achieved Complete...
Jan 30, 2025•27 min•Ep. 95
We love to hear from our listeners. Send us a message. Christopher Horan, Chief Technical Operations Officer, Artiva Biotherapeutics joins Cell & Gene: The Podcast Host, Erin Harris, to discuss the advantages natural killer (NK) cells have offer over T cells in terms of safety and efficacy for autoimmune disease. They take a deep dive into the key factors that make NK cells promising for ‘off-the-shelf’ cell therapy products. They cover Artiva's "manufacturing first" approach to enabling sca...
Jan 16, 2025•24 min•Ep. 94
We love to hear from our listeners. Send us a message. Cell & Gene: The Podcast Host Erin Harris talks to Christopher Choi, Ph.D., MBA, SVP Industry Partnership, Director of GMP and Cell Manufacturing Facility, and Associate Professor of Oncology at Roswell Park Comprehensive Cancer Center about the Center's Buffalo, NY-based cell and gene therapy hub slated to open in early 2025. They cover the increasingly important role of automation and digital technologies in CGT manufacturing. Choi als...
Jan 02, 2025•15 min•Ep. 93
We love to hear from our listeners. Send us a message. Cell & Gene: The Podcast Host Erin Harris talks to Lothar Roessig, M.D., SVP, Integrated Product Team Lead, Congestive Heart Failure at Asklepios BioPharmaceutical, Inc. (AskBio) about the biggest challenges in developing gene therapies for cardiovascular diseases, specifically congestive heart failure (CHF). They discuss AB-1002, the gene therapy the company is developing for the treatment of CHF and why a gene therapy approach is signi...
Dec 19, 2024•21 min•Ep. 92
We love to hear from our listeners. Send us a message. On this episode of Cell & Gene: The Podcast, Host Erin Harris, invites BlueRock Therapeutics' Dr. Amit Rhakit to take a deep dive into therapy options for Parkinson's disease (PD). They discuss how BlueRock's bemdaneprocel's mechanism of action differs from traditional PD treatments. They cover the limitations of current PD medications, how cell therapies might offer a different approach to long-term symptom management, and how BlueRock ...
Dec 05, 2024•31 min•Ep. 91
We love to hear from our listeners. Send us a message. Rita Johnson-Greene, COO at Alliance for Regenerative Medicine (ARM) joined Cell & Gene: The Podcast Host, Erin Harris, on site during 2024 Meeting on the Mesa to record their discussion. Johnson-Greene shared information about the Joint Clinical Assessment (JCA) in Europe and its potential impact on cell and gene therapies, how globalization impacts patient access, an update on ARM's Grow Internship Program, as well as a few key takeawa...
Nov 21, 2024•22 min•Ep. 90
We love to hear from our listeners. Send us a message. On this episode of Cell & Gene: The Podcast, Host Erin Harris talks to Dennis Williams, Senior Vice President of Late Stage Development at Adaptimmune. Williams shares valuable insights into the groundbreaking world of TCR T-cell therapies, with a particular focus on the TECELRA trial. The trial led to the FDA approval of Tecelra, marking a significant milestone as the first engineered T-cell therapy for solid tumors. Williams delves int...
Nov 07, 2024•21 min•Ep. 89
We love to hear from our listeners. Send us a message. This episode of Cell & Gene: The Podcast was recorded on site during ARM's 2024 Meeting on the Mesa in Phoenix. Tune in to hear Host Erin Harris talk to Miguel Forte, M.D., Ph.D., CEO of Kiji Therapeutics, President of ISCT, and Executive Committee Member of ARM about Kiji's clinical plan and why iPSC-MSC therapy for inflammatory diseases shows great promise. They also cover this year's conference and what to expect during and after the ...
Oct 24, 2024•25 min•Ep. 88
We love to hear from our listeners. Send us a message. Carsten Linnemann, Ph.D., CEO of Neogene Therapeutics and Head of Oncology Cell Therapy Clinical Development, AstraZeneca joins Cell & Gene: The Podcast’s Erin Harris to discuss T-cell receptor (TCR) therapies for solid cancers. They talk through key challenges and opportunities in developing TCR therapies targeting neoantigens as well as the potential benefits of TCR-T therapies for cancer treatment. Linnemann shares his thoughts on the...
Oct 10, 2024•14 min•Ep. 87
We love to hear from our listeners. Send us a message. Ray Therapeutics' Co-Founder and CEO, Paul Bresge, joins Host Erin Harris to talk about optogenetics and how the company’s optogenetics approach differs from other gene therapies on the market developed to restore vision in retinal diseases like retinitis pigmentosa. Subscribe to the podcast! Apple | Spotify | YouTube...
Sep 25, 2024•22 min•Ep. 86
We love to hear from our listeners. Send us a message. Tessera Therapeutics' CEO, Dr. Michael Severino, joins Host Erin Harris to discuss Gene Writing, the biotech's genome engineering technology that writes therapeutic messages into the genome to treat diseases at their source. Severino discusses why delivery has always been a challenge for gene editing technologies and why Gene Writing is different when it comes to efficient delivery. Subscribe to the podcast! Apple | Spotify | YouTube...
Sep 12, 2024•28 min•Ep. 85
We love to hear from our listeners. Send us a message. Allogene Therapeutics' EVP of R&D and CMO, Dr. Zachary Roberts, penned an original article for Cell & Gene titled, The Path Forward for CLL is Allogeneic. In his article, Dr. Roberts explains that chronic lymphocytic leukemia (CLL) is the most common leukemia in the U.S. And that while CLL remains a disease that is managed, it is not one that is often cured. In this episode, Host Erin Harris follows up Roberts' article with in-depth ...
Aug 29, 2024•25 min•Ep. 84
We love to hear from our listeners. Send us a message. Applications (BLAs) of recent approvals and complete responses for novel products or rare diseases as well as BLAs currently under review. Benton shares information about FDA Draft Gudiances, including Platform Technology Designation Program for Drug Development: Guidance for Industry. She also provides a regulatory outlook for CGT developers. Subscribe to the podcast! Apple | Spotify | YouTube...
Aug 15, 2024•28 min•Ep. 83
We love to hear from our listeners. Send us a message. Debra Miller, CEO and Founder and Dr. Michael Kelly, Chief Scientific Officer at CureDuchenne talk to Host Erin Harris about the FDA approval for the expansion to the label indication for ELEVIDYS from Sarepta Therapeutics as well as how CureDuchenne works with various biotechs developing gene therapies for Duchenne muscular dystrophy (DMD). Miller and Kelly also share their advice and recommendations for companies developing drugs for DMD -...
Aug 01, 2024•30 min•Ep. 82
We love to hear from our listeners. Send us a message. Lucas Harrington, Co-Founder and Chief Scientific Officer of Mammoth Biosciences, joins Erin Harris to shed light on the biotech's proprietary ultracompact CRISPR systems to develop potential long-term curative therapies for patients with life-threatening and debilitating diseases. They discuss why in vivo delivery continues to be a bottleneck against the clinical advancement of gene-editing-based medicines and what ultracompact CRISPR syste...
Jul 18, 2024•21 min•Ep. 81
We love to hear from our listeners. Send us a message. KSQ Therapeutics' CSO, Micah Benson, Ph.D., joins Erin Harris to discuss how Tumor-Infiltrating Lymphocytes (TILs) as a treatment modality have the potential to treat a variety of solid tumor types. Benson explains KSQ's Phase 1/2 clinical study, KSQ-001EX, which consists of TILs in which the SOCS1 gene is inactivated by CRISPR/Cas9 gene editing. In addition to solid tumors, Benson also addresses the therapeutic potential for autoimmune dise...
Jul 04, 2024•36 min•Ep. 80
We love to hear from our listeners. Send us a message. Strand Therapeutics' CEO and Co-Founder Jake Becraft joins Erin Harris to discuss genetic regulation and how the company's programmable mRNA constructs combine genes for self-replication derived from RNA viruses with genetically programmed logic circuits that control the location, timing, and intensity of expression of therapeutic proteins within the patient’s body, enabling precise and controlled delivery. They also cover the role synthetic...
Jun 20, 2024•28 min•Ep. 79
We love to hear from our listeners. Send us a message. 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. Regarding delivery, Ko explains the advantage of the mRNA approach over the AAV approach. Subscribe to the podcast! Apple | Spotify | YouTube...
Jun 06, 2024•15 min•Ep. 78
