ASGCT Podcast Network

Host Dr. Emily Walsh Martin welcomes Dr. Niki Paulk, CEO and Founder of Siren Biotechnology, for a timely conversation on turning scientific promise into commercial success. Dr. Paulk offers perceptive insights into the complex world of biotech entrepreneurship, covering essential fundraising strategies from Seed and Series Rounds to Community Rounds and beyond. This episode is a must-listen for both industry professionals and acad...

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 the...

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 ve...

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! B...

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...

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 l...

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 y...

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 C...

On this episode of Soundbites of the Annual Meeting, we talked to Stephen Kaminsky, PhD, professor of research in genetic medicine at Weill Cornell Medical College, and lead author of a phase 1 clinical trial of a new vaccine for cocaine addiction.

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

• How the vaccine works and how its approach is different from other treatmen...

This episode is the third 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 reviews, “Current trends in gene therapy to treat inherited disorders of the brain,” with author Beverly Davidson, PhD, The Children&rs...

During this episode of Soundbites of the Annual Meeting, we spoke with P.J. Brooks, PhD, who is Deputy Director of the Division of Rare Diseases Research Innovation, part of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH).

In his conversation with ASGCT's Communications Committee Chair, Lynnea Olivarez, Dr. Brooks talked about:

• The impact of developing cell and gene t...

"Certainly, I've been anxious about data...I did an experiment, I put a lot of time into it, I'm hoping to get good results...we all know that feeling, right? The stakes are just so different here...this kid's life depends on whether this experiment worked."

--Kiran Musunuru, MD, PhD

One of the highlights of the ASGCT 28th Annual Meeting was the announcement on May 15 of a historic medical break...

In honor of Molecular Therapy’s 25th anniversary, this episode is the second in a series hosted by Dr. Joseph Glorioso, Editor-in-Chief of Molecular Therapy, 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 reviews, AAV vector development, back to the future, with author Sheila Mikhail; M34, I...

In honor of Molecular Therapy’s 25th anniversary, this episode is the first in a series hosted by Dr. Joseph Glorioso, Editor-in-Chief of Molecular Therapy, that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past 25 years by expert authors. In this episode, Dr. Glorioso will discuss two of these reviews, “Gene therapy then and now: A look back at changes in the ...

On this episode of The Issue, Host Emily Walsh Martin, PhD, is tackling her absolute favorite subject: the importance of long-term data!

Emily is joined by Dolan Sondhi, PhD, of Weill Cornell Medical College, to discuss her recent paper, “Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease.”

We hope to see all of your long-term da...

Today, Niki Gallo, principal consultant of regulatory affairs at Halloran Consulting, joins host Emily Walsh Martin for a deep dive into timeline management pitfalls, the nuances of INDs, and the rapidly evolving FDA landscape. Niki brings to the program her extensive experience shepherding innovative therapeutics through the regulatory process and timely insights into the complexities of transitioning from the bench to the bedside...

Drs. Ayşegül Yildiz and Tim Beissert join Dr. Paloma Giangrande to discuss a recent article published in Molecular Therapy Nucleic Acids by Drs. Yildiz. Beissert, and colleagues titled “Trans-amplifying RNA expressing functional miRNA mediates target gene suppression and simultaneous transgene expression”.

Are you registered for ASGCT’s 2025 Annual Meeting in New Orleans? Join us May 13-17 for the premier eve...

Join host Emily Walsh Martin and guest Brendan Hayes, senior director of education and innovative therapies at the National Bleeding Disorders Foundation, formerly known as the National Hemophilia Foundation. for an illuminating conversation about trust, innovation, and the power of patient advocacy in advancing cell and gene therapies.

Together, they dive into how patient communities shape the development of new therapies, the his...