Host Emily Walsh Martin welcomes Dr. Jim Burns, CEO of Ensoma, to discuss the company's novel technological approach to treating both rare diseases and solid tumor oncology. Dr. Burns provides a detailed overview of Ensoma's platform and its application in their ongoing clinical trials, including the recently announced first patient infusion for Chronic granulomatous disease. Learn how this initial trial is set to inform future therapeutic opportunities for indications with high unmet medical need.

Music: ‘Bright New Morning’ by Steven O’Brien – released under CC-BY 4.0. https://www.steven-obrien.net/

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Join all four Editors-in-Chief of the Molecular Therapy family of journals for a special 2025 Retrospective. Editors Dr. Glorioso, Dr. Giangrande, Dr. Abou-el-Enein, and Dr. Cripe dive into the most exciting advancements in gene and cell therapy from the past year.

Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

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Editor-in-Chief of Molecular Therapy, Dr. Joseph Glorioso, is joined by Rachael Nimmo, PhD, former director of the Cell Technology Group at Oxford Biomedica, and Kyriacos Mitrophanous, PhD, Chief Innovation Officer at Oxford Biomedica, to discuss an article recently published in Molecular Therapy by Nimmo and colleagues titled “Efficient in vivo generation of CAR T cells using a retargeted fourth-generation lentiviral vector.”

Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

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Join Dr. Paloma Giangrande, Editor-in-Chief of Molecular Therapy Nucleic Acids, as she discusses a recently published MTNA article, Controlling CRISPR-Cas9 genome editing in human cells using a molecular glue degrader, with its authors Drs. Krishanu Saha and Namita Khajanchi (UW-Madison).

If you enjoy this episode, check out our hybrid event this fall that will allow you to engage with pioneering researchers in gene editing! Breakthroughs in Targeted In Vivo Gene Editing will be held in San Diego and virtually November 20-21, 2025.

Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

Show your support for ASGCT!: https://asgct.org/membership/donate

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Join Dr. Mohamed Abou-el-Enein, Editor-in-Chief of Molecular Therapy Methods & Clinical Development, as he discusses a recent article published in MTMCD with co-authors Drs. Daniel Stone, Harrison Dulin, and Keith R. Jerome from the Fred Hutchinson Cancer Center.

The article, “Serum factors create species-specific barriers to hepatic gene transfer by lipid nanoparticles in liver-humanized mice,” will appear in MTMCD’s upcoming special issue, Lipid nanoparticles for cell and gene therapy.

If you enjoy this episode, check out our hybrid event this fall that will allow you to engage with pioneering researchers in gene editing! Breakthroughs in Targeted In Vivo Gene Editing will be held in San Diego and virtually November 20-21. Register to secure your spot or submit an abstract through Sept. 12 for the opportunity to present your research!

Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

Show your support for ASGCT!: https://asgct.org/membership/donate

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On this episode of Soundbites of the Annual Meeting, we talked to Kimberly Haugstad, CEO of RareRising and a longtime rare disease advocate who is also the parent of a child with severe hemophilia.

In her conversation with ASGCT's Communications Committee Chair, Lynnea Olivarez, Kimberly discusses:

• why patients, as the "lived experience experts" are so important to rare disease communications

• advice for scientists who would like to engage more directly with patient communities

• what's giving her hope right now

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This episode is the fourth in a series hosted by Molecular Therapy Editor-in-Chief Joseph Glorioso, PhD, that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past 25 years.

In this episode, Dr. Glorioso will discuss the review, How to democratize cell and gene therapy: A global approach, with author Rayne Rouce, MD, Texas Children’s Hospital.

If you want to learn more about cell and gene therapy access, register for the ASGCT Policy Summit, September 25-26, in Washington, D.C.! We’ll hear from policymakers, industry leaders, and science stakeholders, who will explore solutions to the challenges of accessing therapeutic treatments.

Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.

On this episode of Soundbites of the Annual Meeting, we talked to Mimi Lee, MD, PhD, a physician-scientist and program manager at the Advanced Research Projects Agency for Health (ARPA-H).

In her conversation with ASGCT's Communications Committee Chair, Lynnea Olivarez, Dr. Lee discusses:

• ARPA-H's unique approach to funding and structuring CGT development

• breaking down silos to improve health outcomes for all
• the role that CGT will play in prevention, rather than just treatment, of diseases

Listen and subscribe to the ASGCT Podcast Network so you don't miss any episodes!

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.