Cell & Gene: The Podcast

EPISODES

Inside RNAi With Arrowhead Pharmaceuticals' Javier San Martin

Thursday, September 29, 2022

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...  Continue Reading..

• Planning For Manufacturing Capacity With Kite Pharma's Will Junker

  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. 

• Commercialization Learnings With Novartis Gene Therapies' President Chris Fox

  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.

• A Stem Cell-Derived Treatment For T1D

  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.

• Best Practices For Partnering With A CDMO

  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 episode, biotechs will learn how to reduce risk, lower costs, and how to better manage expectations when it comes the biotech / CDMO relationship. 

• An Interview With Dr. Carl June

  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.

• Inside Gamma Delta T Cells with TC BioPharm’s CEO, Bryan Kobel

  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.

• Moving On From AAV With Poseida Therapeutics' CEO, Mark Gergen

  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.

• Inside The New Hybrid Roles For Cell Therapy Clinical Trials With Immatics' Michael Mehler

  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 main aspects of a site operations lead and why the role is imperative to commercial success as well as how he sees the role evolving in the near term. He also explains what size CGT companies will benefit most from these new hybrid roles.

• Allogeneic RNA Cell Therapy Program With Cartesian Therapeutics' Dr. Metin Kurtoglu

  Cartesian Therapeutics’ Dr. Metin Kurtoglu joins Cell & Gene: The Podcast to explain their autologous RNA cell therapies, Descartes-08 for Myasthenia Gravis and Descartes-11 for multiple myeloma. He also sheds light on Descartes-25, their allogeneic RNA cell therapy, and the reasons for the move to "off-the-shelf.” Hear why the future of RNA cell therapy is not confined to rare and fatal diseases and subscribe so you never miss an episode!

• Gene Therapy's Commercial Manufacturing Issues with Encoded Therapeutics' Andy Stober

  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.

• Inside CAR-T Therapy For Ovarian Cancer with Anixa Biosciences' Amit Kumar, Ph.D.

  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 therapy may evolve in the short term, and much more.

• Pivoting to Allogeneic CAR-T Therapies with Celyad Oncology’s CMO, Dr. Charlie Morris

  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.

• Rocket Pharmaceuticals CEO Dr. Gaurav Shah On FDA's Clinical Holds On Gene Therapy Trials

  In a new episode from Cell & Gene: The Podcast, Rocket Pharmaceuticals CEO Dr. Gaurav Shah offers his take on why gene therapy developers have been plagued by clinical holds, whether the FDA’s adeno-associated virus (AAV)-focused CTGTAC meeting's takeaways will hold up throughout 2022, and more. Listen now and subscribe so you never miss an episode!

• Inside Solid Tumor Therapies With BlueSphere Bio’s CEO Dr. David Apelian

  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.

• Restoration Of Immune Balance With Anish Suri, Ph.D., President And CSO At Cue Biopharma

  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.

• REGENXBIO's Nina Hunter, Ph.D. Details The Accelerated Approval Pathway For Gene Therapies

  REGENXBIO's VP of Regulatory and Science Policy, Nina Hunter, Ph.D., shares potential solutions to gene therapies' regulatory roadblocks and why the accelerated approval pathway is so pertinent.

• Selecta Biosciences' Dr. Carsten Brunn On Gene Therapy With AAV Vectors

  Selecta Biosciences' President and CEO Dr. Carsten Brunn details the company's ImmTOR platform, which can mitigate unwanted immune responses that cause immune-related toxicities that impose significant treatment challenges with current therapies.

• Cellevolve Bio's Dr. Derrell Porter On The Need For Diversity And Inclusion In Biotech

  Cellevolve Bio's founder and CEO. Dr. Derrell Porter shares his experience with starting up a cell therapy company and why we, as an industry, need to advocate for more Black leaders and leaders of color in the biotech industry.

• RNA's Advantages Over Other Cell Therapies With Cartesian's CSO

  Dr. Michael Singer, CSO at Cartesian Therapeutics, details why RNA has advantages over other cell therapies, the company's three RNA trials, and the therapeutic benefit of multiple modifications.

• Vor Biopharma's CEO On Genome Engineering Of Hematopoietic Stem Cells

  Vor Biopharma's CEO Dr. Robert Ang explains the company's lead engineered hematopoietic cell (eHSC) product candidate, genome engineering of HSCs, and the cell manufacturing process needed to leverage genome engineering technologies for the cells they're creating.

• AVROBIO on Patient Advocacy's Role in Clinical Trial Development & Recruitment

  Fernanda Copeland, Global Head, Patient Advocacy & Engagement at AVROBIO, explains how to incorporate community education, patient feedback, and more to improve clinical trial development and recruitment.

• Understanding CAR-M Technology With Carisma Therapeutics' Dr. Debora Barton

  Carisma Therapeutics’ Dr. Barton breaks down the latest in CAR-M technology as well as the company's Phase 1 First-in-Human Study of Adenovirally Transduced Anti-HER2 CAR Macrophages in Subjects with HER2 Overexpressing Solid Tumors.

• Inside The Gene Therapy For Coronary Artery Disease With XyloCor Therapeutics' Albert Gianchetti

  XyloCor Therapeutics' EXACT clinical trial is a Phase 1/2 multicenter, open-label, single arm, dose escalation trial for coronary artery disease — the most common type of heart disease — with gene therapy at its center. 

• Advancing Patient Engagement With Novartis Gene Therapies' Dr. Amy Nicole Nayar

  Dr. Amy Nicole Nayar, VP of U.S. Patient Advocacy and Government Affairs at Novartis Gene Therapies explains what patient engagement should encompass in the cell and gene field as well as the unique challenges cell and gene therapy companies face where a patient engagement officer provides essential help. 

• BridgeBio's Approach To Gene Therapy & Nearing The Clinic With Dr. Eric David

  BridgeBio's CEO of Gene Therapy, Dr. Eric David, explains the company's gene therapy programs for Canavan disease and congenital adrenal hyperplasia, their manufacturing plans, and the platform underpinning their approach to gene therapy.

• Inside Spark Therapeutics' Investigational SPK-8011 For Hemophilia A With Dr. Tiffany Chang

  Spark Therapeutics' Clinical Development Lead, Hematology, Dr. Tiffany Chang explains the company's data from its ongoing Phase 1/2 clinical trial of investigational SPK-8011 for hemophilia A, the largest gene therapy trial in this disease to date as well as the misconceptions and unknowns about the disease. 

• Gene Therapies For Spinal Cord-Related Disorders With SwanBio's CSO

  Karen Kozarsky, Ph.D., Co-Founder and CSO of SwanBio explains how the company is pioneering a deep and varied pipeline of gene therapies for genetically defined neurological diseases with a focus on the spinal cord.

• Chronic Lymphocytic Leukemia: A Patient's Perspective

  Bob Levis was diagnosed with Chronic Lymphocytic Leukemia in 2002 and was one of the early experimental trial participants at Penn Medicine for Dr. Carl June’s CAR-T therapy. Bob shares his remarkable journey with CLL as well as his important work with the CLL Society. 

• State-of-the-Art GMP Manufacturing RNA Cell Therapies With Cartesian's CEO

  Dr. Murat Kalayoglu, CEO at Cartesian Therapeutics, details the cell and gene therapy sector's advancements in RNA cell therapy and the why behind the company's decision to build its own wholly-owned GMP manufacturing facility.

• Cell & Gene Therapy Firsts With Dr. Bruce Levine

  Dr. Bruce Levine, Barbara and Edward Netter Professor in Cancer Gene Therapy at the University of Pennsylvania, explains his road to adoptive immunotherapy trials, overcoming the gap between academia and industry, and how Philadelphia earned the Cellicon Valley name for its continued innovation in cell and gene therapy, and more.

• Hospital Systems & Complex Therapies With University Of Pennsylvania Health System's Kevin Mahoney

  On this episode of Cell & Gene: The Podcast, University of Pennsylvania Health System’s CEO Kevin Mahoney talks to Host Erin Harris about how hospital systems must evolve to meet the needs of cell and gene therapy patients as these complex therapies are commercialized. They discuss what comprises UPHS and the ongoing innovation it brings to advanced therapies as well as how the Health System stays patient-focused at all costs.

• The Future Of Tissue-Based Therapies

  During this first episode of Cell & Gene: The Podcast, Host Erin Harris talks to Enzyvant’s CEO Rachelle Jacques about the biotech’s lead asset, investigational regenerative therapy RVT-802 for congenital athymia. Jacques provides an update on FDA approval and details what’s next for RVT-802. She explains tissue-based therapies — how far they’ve come and the work that’s left to be done. And, they discuss what being patient-focused means in the world of rare diseases.