Derek Jackson, vice president of cell and gene therapy product development at Pacira BioSciences, joins The Issue to take the program in a different direction.

We often focus our discussions on CGT for rare diseases. Most of the time, the teams of scientists working on the program don’t have any personal experience with the disease from their family, friends, or themselves.. but today we will talk about gene therapy development for a much more common disease: knee osteoarthritis. Moreover, the product we will be discussing is not only non-rare but also non-AAV.

Music by: https://www.steven-obrien.net/
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Bright New Morning - Steven O'Brien

(Used for free under a Creative Commons Attribution 4.0 License: https://creativecommons.org/licenses/by/4.0/)

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Dr. Ben Hurlbut, professor at Arizona State University's Lincoln Center for Applied Ethics, joins host Dr. Emily Walsh Martin for a critical conversation in the cell and gene therapy industry in which they discuss how ethics informs and impacts the field and offers a deep dive into the ethical, medical, and social considerations around germline gene editing.

Music by: https://www.steven-obrien.net/
--------------------------
Bright New Morning - Steven O'Brien

(Used for free under a Creative Commons Attribution 4.0 License: https://creativecommons.org/licenses/by/4.0/)

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Drs. Beverly Davidson and Bryan Simpson join Molecular Therapy EiC Dr. Roland Herzog to discuss their recent paper, Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in the brain, which was published in Molecular Therapy.

ASGCT is proud to present this episode of the Molecular Therapy Podcast in partnership with our upcoming Advancing Gene and Cell Therapy for Cancer conference, which will be held on October 16 and 17 in Philadelphia and online. This landmark hybrid event brings together innovative treatment approaches and an unparalleled opportunity to immerse yourself in the latest gene and cell therapy research for cancer. Learn more and register today at ASGCT.org/Advancing.

In This Episode:

Dr. Roland Herzog
Editor-in-Chief of Molecular Therapy and Professor of Pediatrics, Riley Children's Foundation Professor of Immunology, and Director of the Gene and Cell Therapy Program at Indiana University

Dr. Beverly Davidson
Chief Scientific Strategy Officer and Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at Children’s Hospital of Philadelphia

Dr. Bryan Simpson
Preclinical Team Lead at Latus Bio

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

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Join host Emily Walsh Martin, PhD, and David Altshuler, MD, PhD, Chief Scientific Officer of Vertex Pharmaceuticals. David came to Vertex after a lengthy career in medicine and academic research. David and Emily discuss how David and the team at Vertex think about innovation in drug development and how that impacts how they select programs and approaches for the diseases they focus on. They also cover what he’d love to see happen with new cell and gene platform technologies being brought forward.

Now is the time to register for the ASGCT Policy Summit, held in Washington DC on September 23-24. Whether you attend in person or virtually, you will not want to miss this year’s program. Great sessions are planned on reimbursement, Medicaid, venture capital, strategic investment outlooks, and the evolution of the regulatory landscape. Advanced registration ends August 23, so register today!

ASGCT.org/PolicySummit

Music by: https://www.steven-obrien.net/
--------------------------
Bright New Morning - Steven O'Brien

(Used for free under a Creative Commons Attribution 4.0 License: https://creativecommons.org/licenses/by/4.0/)

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

See omnystudio.com/listener for privacy information.

Join ASGCT and the Molecular Therapy family of journals for a conversation between Drs. Paloma Giangrande and Marcin Kortylewski. They discuss a recent article published in Molecular Therapy Nucleic Acids by Dr. Kortylewski and colleagues titled "Oligo-PROTAC strategy for cell-selective and targeted degradation of activated STAT3."

If you enjoy today’s conversation, you’ll also enjoy the upcoming ASGCT Policy Summit in Washington, DC, September 23-24.

This can't-miss event brings together policymakers and gene and cell therapy experts, including FDA leaders Julie Tierney and Dr. Nicole Verdun, to discuss the latest policies impacting this rapidly evolving field.

Register now at https://www.asgct.org/PolicySummit for invaluable insights on navigating the regulatory landscape.

In This Episode:

Dr. Paloma Giangrande
Editor-in-Chief of Molecular Therapy Nucleic Acids and Chief Technology Officer at Eleven Therapeutics

Dr. Marcin Kortylewski
Professor, Department of Immuno-Oncology at City of Hope

'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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Dr. Daniel Stone speaks with Drs. Mario Mietzsch and Robert McKenna from the University of Florida to discuss a recent article published in the Biomanufacturing in Gene and Cell Therapy special issue of Molecular Therapy Methods & Clinical Development by Drs. Mietzsch, McKenna, and colleagues titled Production and characterization of an AAV1-VP3-only capsid: An analytical benchmark standard.

If you enjoy today’s conversation, you’ll also enjoy the upcoming ASGCT Policy Summit in Washington, DC, September 23-24.

This can't-miss event brings together policymakers and gene and cell therapy experts, including FDA leaders Julie Tierney and Dr. Nicole Verdun, to discuss the latest policies impacting this rapidly evolving field.

Register now at https://www.asgct.org/PolicySummit for invaluable insights on navigating the regulatory landscape.

In This Episode:

Dr. Daniel Stone
Associate Editor-in-Chief of Molecular Therapy Methods & Clinical Development and Senior Staff Scientist, Infectious Disease Sciences, Vaccine and Infectious Disease Division at Fred Hutch Cancer Center

Dr. Mario Mietzsch
Assistant Scientist, Department of Biochemistry and Molecular Biology, University of Florida

Dr. Robert McKenna
Professor and Director of the Center for Structural Biology, Department of Biochemistry and Molecular Biology, University of Florida

'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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In this Very Special Episode, Emily Walsh Martin, PhD explores the cutting-edge transposase-based technology presented by SalioGen at the ASGCT Annual Meeting in which they achieved gene insertion without double-strand breaks. Join us as Jason Cole, CEO at SalioGen, takes us on a captivating journey through the company's groundbreaking work, including their initial data in Stargardt disease using LNP delivery to photoreceptors.

But that's not all! Discover Jason's unconventional path from small molecules to genetic medicines and gain insights into the strategic value of using a target product profile to guide development decisions.

Finally, we delve into the transformative potential of genetic medicine, bridging the gap from bench to bedside to payer-to-patient access.

And before we dive in, a quick reminder: The ASGCT Policy Summit (Sept 23-24 in DC or online) is an absolute must for R&D scientists, analytical experts, clinical researchers, and program managers seeking a comprehensive understanding of genetic medicine development. Don't miss this crucial conversation between regulators, payors, patients, caregivers, and developers.

ASGCT.org/PolicySummit

Music by: https://www.steven-obrien.net/
--------------------------
Bright New Morning - Steven O'Brien

(Used for free under a Creative Commons Attribution 4.0 License: https://creativecommons.org/licenses/by/4.0/)

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

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Mohamed Abou-el-Enein, MD, PhD, MSPH, the new editor-in-chief of Molecular Therapy Methods & Clinical Development (MTMCD), sits down with Scientific Editor Rory Bricker-Anthony, PhD, to discuss his vision for the journal.

If you enjoy today’s conversation, you’ll also enjoy the upcoming ASGCT Policy Summit in Washington, DC, on September 23 and 24.

This can't-miss event brings together policymakers and gene and cell therapy experts, including FDA leaders Julie Tierney and Nicole Verdun, PhD, to discuss the latest policies impacting this rapidly evolving field.

Register now at ASGCT.org/PolicySummit for invaluable insights on navigating the regulatory landscape. Virtual attendance options are also available.

Dr. Abou-el-Enein has served as MTMCD's associate editor-in-chief for the past two years, which will allow him to make a smooth transition in leadership. His vision for MTMCD is consistent innovation, evolution, and growth. Dr. Abou-el-Enein firmly believes that MTMCD has the potential to become a leading voice in the gene and cell therapy landscape. He is eager to leverage his experience and expertise to expand the journal’s reach and impact.

Dr. Abou-el-Enein is an associate professor of clinical medicine (oncology), pediatrics, and stem cell biology and regenerative medicine at USC Keck School of Medicine as well as executive director of the USC/CHLA cell therapy program. He is an internationally recognized expert in planning and executing clinical development programs, designing and operating academic GMP facilities, and translating cell and gene therapy products from preclinical research to clinical applications. His research laboratory is dedicated to pioneering advancements in CAR T-cell therapies, including optimizing engineering techniques and devising novel strategies for allogeneic applications and genetic delivery tools to increase accessibility and scalability.

'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.