We love to hear from our listeners. Send us a message. 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 engine...
Apr 13, 2023•28 min•Ep. 47
We love to hear from our listeners. Send us a message. 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-...
Mar 30, 2023•41 min•Ep. 46
We love to hear from our listeners. Send us a message. 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 over...
Mar 16, 2023•21 min•Ep. 45
We love to hear from our listeners. Send us a message. 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 addr...
Mar 02, 2023•32 min•Ep. 44
We love to hear from our listeners. Send us a message. On this episode of Cell & Gene: The Podcast, Host Erin Harris talks to Andy Ramelmeir, 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. Ramelmeir 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 th...
Feb 16, 2023•22 min•Ep. 43
We love to hear from our listeners. Send us a message. 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. Subscribe to the podcast! Apple | Spotify | YouTube...
Feb 02, 2023•27 min•Ep. 42
We love to hear from our listeners. Send us a message. MustangBio'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. Subscribe to the p...
Jan 19, 2023•36 min•Ep. 41
We love to hear from our listeners. Send us a message. 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 t...
Jan 05, 2023•29 min•Ep. 40
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 f...
Dec 22, 2022•27 min•Ep. 39
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 e...
Dec 08, 2022•29 min•Ep. 38
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 ...
Nov 24, 2022•37 min•Ep. 37
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...
Nov 10, 2022•40 min•Ep. 36
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 ...
Oct 27, 2022•31 min•Ep. 35
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 | ...
Oct 13, 2022•28 min•Ep. 34
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 ...
Sep 29, 2022•33 min•Ep. 33
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! Ap...
Sep 15, 2022•23 min•Ep. 32
We love to hear from our listeners. Send us a message. 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...
Sep 01, 2022•20 min•Ep. 31
We love to hear from our listeners. Send us a message. 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. Subscribe to the...
Aug 18, 2022•28 min•Ep. 30
We love to hear from our listeners. Send us a message. Emily Moran, VP of Vector Manufacturing and Avi Nandi, VP of Process Development at the Center for Breakthrough Medicines share key considerations for biotechs looking to partner with a CDMO. During this epsiode, biotechs will learn how to reduce risk, lower costs, and how to better manage expectations when it comes the biotech / CDMO relationship. Subscribe to the podcast! Apple | Spotify | YouTube...
Aug 04, 2022•28 min•Ep. 29
We love to hear from our listeners. Send us a message. Dr. Carl June, an HIV gene therapy pioneer and the father of CAR-T cell therapy, joins Cell & Gene: The Podcast to discuss the current state of CAR-T therapy, what's on the short-term horizon for immunotherapy, on-going manufacturing capacity issues, an update on Emily Whitehead, the first child to receive CAR-T cell therapy on an experimental basis, and more. Subscribe to the podcast! Apple | Spotify | YouTube...
Jul 21, 2022•28 min•Ep. 28
We love to hear from our listeners. Send us a message. TC BioPharm's CEO Bryan Kobel explains gamma delta t cells (GDT) in depth as well as the advantages of using GDT cells as a cell therapy vehicle. He discusses why the publicly-traded, clinical-stage cell therapy company conducts and manages all clinical trials in house as well as the unique challenges and benefits that come along with doing so. Subscribe to the podcast! Apple | Spotify | YouTube...
Jul 07, 2022•33 min•Ep. 27
We love to hear from our listeners. Send us a message. Poseida Therapeutics is a broad platform technology company led by CEO, Mark Gergen. During this episode, Gergen explains how and why the company is moving away from AAV delivery in favor of nanoparticle delivery. He shares some of the biggest hurdles the CGT sector faces when it comes to the development of off-the-shelf therapies as well as the manufacturing challenges Poseida has had to overcome and how they did it. Subscribe to the podcas...
Jun 23, 2022•28 min•Season 1Ep. 26
We love to hear from our listeners. Send us a message. Autologous cell therapy clinical trials and products are very complex, and the stakeholder investment is diverse. Immatics' Director, Clinical Operations, Michael Mehler, explains why new hybrid roles operate in clinical operations, clinical supply chain, strategic operations, and medical affairs to help bridge the gap between the sponsor company and the clinical site for clinical trials in an autologous setting. Mehler talks through the mai...
Jun 09, 2022•24 min•Season 1Ep. 25
We love to hear from our listeners. Send us a message. Dr. Metin Kurtoglu, COO at Cartesian Therapeutics, explains the clinical-stage biotech's autologous RNA cell therapies, Descartes-08 for Myasthenia Gravis and Descartes-11 for multiple myeloma. He also explains Descartes-25, their allogeneic RNA cell therapy and the reasons for the move to "off-the-shelf," and why the future of RNA cell therapy is not confined to rare and fatal diseases. We also discuss post-care for patients having received...
May 26, 2022•20 min•Season 1Ep. 24
We love to hear from our listeners. Send us a message. Encoded Therapeutics' Chief Manufacturing Officer Andy Stober explains the unique commercial manufacturing issues in gene therapy and how to address them. Stober also shares some of the lessons he's learned, which may educate the next generation of gene therapy scientists to accelerate future manufacturing success. We also talk about what developers need to consider regarding the ethical responsibilities in gene therapy. Subscribe to the pod...
May 12, 2022•27 min•Season 1Ep. 23
We love to hear from our listeners. Send us a message. Amit Kumar, Ph.D., President and CEO at Anixa Biosciences details the company’s progress on its Phase 1 trial for ovarian cancer. We also discuss why the company’s ovarian cancer program is integral for initial proof of concept for other solid tumor indications. He covers why CAR-T therapy uses an existing hormone/hormone receptor relationship to create a targeted approach to treat ovarian cancer. Dr. Kumar shares how the field of cell thera...
Apr 28, 2022•19 min•Season 1Ep. 22
We love to hear from our listeners. Send us a message. Celyad Oncology’s CMO, Dr. Charlie Morris, shares the benefits and challenges of the allogeneic approach versus the autologous approach and why a non-gene edited shRNA approach may result in better efficacy and safety for CAR-T therapies. Subscribe to the podcast! Apple | Spotify | YouTube...
Apr 14, 2022•25 min•Season 1Ep. 21
We love to hear from our listeners. Send us a message. Rocket Pharmaceuticals CEO Dr. Gaurav Shah offers his take on why gene therapy developers have been plagued by clinical holds, whether the FDA’s AAV-focused CTGTAC meeting's takeaways will hold up throughout 2022, and more. Subscribe to the podcast! Apple | Spotify | YouTube...
Mar 31, 2022•27 min•Season 1Ep. 20
We love to hear from our listeners. Send us a message. BlueSphere Bio's CEO Dr. David Apelian explains the difficulties associated with treating solid tumor cancers as well as what the CGT sector needs to get right in the short term and why. Subscribe to the podcast! Apple | Spotify | YouTube...
Mar 17, 2022•25 min•Season 1Ep. 19
We love to hear from our listeners. Send us a message. Cue Biopharma's Anish Suri, Ph.D., explains why the clinical-stage biopharma is engineering a novel class of injectable biologics to selectively engage and modulate targeted T cells directly within the body to transform the treatment of cancer. Subscribe to the podcast! Apple | Spotify | YouTube...
Mar 03, 2022•19 min•Season 1Ep. 18
