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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 significantly different from traditional therapies.
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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 is doing traditional dopamine replacement therapy differently.
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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 takeaways from the event that sector professionals can anticipate impacting the CGTs in the next 12 months and beyond.
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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 into the complexities of developing this innovative treatment for synovial sarcoma, a rare and aggressive form of soft-tissue cancer. He also highlights the unique challenges and promising outcomes associated with TCR T-cell therapies, offering a comprehensive understanding of this cutting-edge approach in cancer immunotherapy.
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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 event.
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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 future landscape of cell therapy in oncology and beyond. Previous episodes of Cell & Gene: The Podcast have focused on TCR therapies, including Episode 50, featuring Affini-T Therapeutics’ Co-Founder, CEO, and President Dr. Jak Knowles. Be sure to check out that episode, too.
https://www.cellandgene.com/doc/targeting-oncogenic-drivers-for-solid-tumor-cancers-with-affini-t-therapeutics-dr-jak-knowles-0001Subscribe to the podcast!
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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.
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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.
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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 questions around existing issues with current CAR T approaches, "auto versus allo," and why allogeneic is the path forward. They also discuss how and why allogeneic therapies are truly scalable.
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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.
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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 - while much progress has been made, more innovation is needed.
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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 systems are designed to do.
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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 disease.
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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 biology plays to genetically program mRNA to deliver therapies.
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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.
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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. She takes a deep dive into the pediatric patient journey, and she shares her expectations for ISCT 2024 Vancouver.
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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. He talks about his time and learnings as President Elect, President, and Immediate Past President of ISCT. He covers Cellicon Valley and Philadelphia's success as on-going CGT hub, and much more.
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Dr. Marcus Conant is CMO at Addimmune, a biotech startup developing a cell therapy for HIV that spun out of cell and gene therapy biotech American Gene Technologies (AGT). He spent his career on the front lines of HIV treatment and research and remains an advocate for the HIV patient. He formed the Kaposi’s Sarcoma Research & Education Foundation in 1982, which later became the San Francisco AIDS Foundation. On this episode, Dr. Conant shares they why behind cell and gene therapy to treat HIV, and he explains Addimmune’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.
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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. They cover the important role induced pluripotent stem cells' (iPSCs) play in the trial, and they also discuss the company's investigational cell therapy, bemdaneprocel.
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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. They also talk through Vor’s decision to build an internal cell therapy manufacturing facility co-located with their Cambridge, MA-based headquarters.
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