Cell & Gene: The Podcast

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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-0001

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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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