Cell & Gene: The Podcast

Dr. Peter Marks, Director of CBER at the FDA, talks about innovations in clinical development, engaging with the FDA for streamlined manufacturing, and the importance of early communication. He discusses plans for 2023, personal hobbies like sailing, and the dedication to advancing cell and gene therapies.

We love to hear from our listeners. Send us a message.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 necessary to move immunotherapies forward.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

We love to hear from our listeners. Send us a message.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.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

We love to hear from our listeners. Send us a message.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. Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

We love to hear from our listeners. Send us a message.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.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

We love to hear from our listeners. Send us a message.PepGen, a Cambridge, MA-based biotech, is advancing oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases has focused on delivery. 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.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

We love to hear from our listeners. Send us a message.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.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

We love to hear from our listeners. Send us a message.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.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

We love to hear from our listeners. Send us a message.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.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

President Chris Fox of Novartis Gene Therapies discusses the company's focus on newborn screenings and the commercialization of Zolgensma for SMA. She shares insights on patient advocacy, work-life balance, and personal experiences like parenting and working out.