This episode, recorded at the ASGCT 2026 Annual Meeting in Boston, focuses on Molecular Therapy Advances’ upcoming special issue, Beyond prevalance: How rare diseases are reshaping therapeutic innovation. Listen as Editor-in-Chief of Molecular Therapy Advances, Dr. Mohamed Abou-el-Enein, discusses the evolution of therapeutic development for rare diseases with guest editors Drs. Jennifer Adair, Claire Booth, Terence Flotte, and Donald Kohn.

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

This episode, recorded at the ASGCT 2026 Annual Meeting in Boston, focuses on&nbsp;Molecular Therapy Advances&rsquo; upcoming special issue, <a href="https://www.cell.com/molecular-therapy-family/advances/special-issues/call-for-papers/rare-diseases">Beyond prevalance: How rare diseases are reshaping therapeutic innovation</a>.&nbsp;Listen as Editor-in-Chief of&nbsp;Molecular Therapy Advances, Dr. Mohamed Abou-el-Enein, discusses the evolution of therapeutic development for rare diseases with guest editors Drs.&nbsp;Jennifer Adair, Claire Booth, Terence Flotte, and Donald Kohn.
Music:&nbsp;'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.&nbsp;<a href="https://www.scottbuckley.com.au/">www.scottbuckley.com.au</a>

The American Society of Gene & Cell Therapy is the premier membership organization for professionals working in cell and gene therapy. On this network, we're sharing exciting stories from our members and the greater scientific community.

The American Society of Gene &amp; Cell Therapy is the premier membership organization for professionals working in cell and gene therapy. On this network, we're sharing exciting stories from our members and the greater scientific community.

ASGCT Podcast Network

How rare diseases are shaping therapeutic innovation with Jennifer Adair, Claire Booth, Terry Flotte and Donald Kohn

Molecular Therapy Oncology has released a new special collection, Advancements in Pediatric Cancer Therapy. Listen in as guest editor Nirali Shah and Editor-in-Chief Timothy Cripe discuss articles in this special collection, which covers the history, current landscape, and future direction of pediatric cancer therapy.

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

Molecular Therapy Oncology has released a new special collection, <a href="https://www.sciencedirect.com/special-issue/10X0KZR6L40">Advancements in Pediatric Cancer Therapy</a>. Listen in as guest editor Nirali Shah and Editor-in-Chief Timothy Cripe discuss articles in this special collection, which covers the history, current landscape, and future direction of pediatric cancer therapy.
Music:&nbsp;'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.&nbsp;<a href="https://www.scottbuckley.com.au/">www.scottbuckley.com.au</a>

Advancements in Pediatric Cancer Therapy with Nirali Shah and Timothy Cripe

In this episode of The Issue, Sara Johnson Davis, Senior Director of Government Affairs at Ardelyx and former Vice President, Head of Patient Advocacy & Policy at Fulcrum Therapeutics, joins host Sanjay D’Souza, PhD, to discuss the essential role of patient advocacy in cell and gene therapy.

The conversation covers the evolving role of patient advocacy in drug development, from early research and clinical trial design to policy, commercialization, and access. Sara shares lessons from the sickle cell disease community, reflects on the transition of gene editing from experimental science to approved therapy, and explains why trust, infrastructure, and policy are essential to ensuring transformative treatments actually reach patients.

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

In this episode of The Issue, Sara Johnson Davis, Senior Director of Government Affairs at Ardelyx and former Vice President, Head of Patient Advocacy &amp; Policy at Fulcrum Therapeutics, joins host Sanjay D&rsquo;Souza, PhD, to discuss the essential role of patient advocacy in cell and gene therapy.
The conversation covers the evolving role of patient advocacy in drug development, from early research and clinical trial design to policy, commercialization, and access. Sara shares lessons from the sickle cell disease community, reflects on the transition of gene editing from experimental science to approved therapy, and explains why trust, infrastructure, and policy are essential to ensuring transformative treatments actually reach patients.
Music:&nbsp;&lsquo;Bright New Morning&rsquo; by Steven O&rsquo;Brien &ndash; released under CC-BY 4.0.&nbsp;<a href="https://www.steven-obrien.net/">https://www.steven-obrien.net/</a>

From Innovation to Access: Patient Advocacy as a Strategic Partner with Sara Johnson Davis

Recorded live at the ASGCT 2026 Annual Meeting in Boston, Le Cong, PhD joins hosts Jason West, PhD and Sanjay D’Souza, PhD for a discussion on his work in genome engineering and the early development of CRISPR/Cas9. The three then dive into Dr. Cong’s use of artificial intelligence tools developed and currently used by his lab. Dr. Cong is a 2026 recipient of ASGCT’s Outstanding New Investigator Awards.

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

Recorded live at the ASGCT 2026 Annual Meeting in Boston, Le Cong, PhD joins hosts Jason West, PhD and Sanjay D&rsquo;Souza, PhD for a discussion on his work in genome engineering and the early development of CRISPR/Cas9. The three then dive into Dr. Cong&rsquo;s use of artificial intelligence tools developed and currently used by his lab. Dr. Cong is a 2026 recipient of ASGCT&rsquo;s Outstanding New Investigator Awards.
Music:&nbsp;&lsquo;Bright New Morning&rsquo; by Steven O&rsquo;Brien &ndash; released under CC-BY 4.0.&nbsp;<a href="https://www.steven-obrien.net/">https://www.steven-obrien.net/</a>

How rare diseases are shaping therapeutic innovation with Jennifer Adair, Claire Booth, Terry Flotte and Donald Kohn

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