ASGCT Podcast Network

Molecular Therapy Editor-in-Chief Dr. Joseph Glorioso joins researchers Kathy Steece-Collier (Michigan State University), Jeffrey Kordower (Arizona State University), and Fredric Manfredsson (Barrow Neurological Institute) to discuss their groundbreaking work on RNA interference for Parkinson’s disease. This episode focuses on their recent article titled "Disease-modifying, multidimensional efficacy of putaminal CaV1.3-shRNA gene therapy in aged parkinsonism male and female macaques."

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

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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 academic innovators.

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

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

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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/

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

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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See omnystudio.com/listener for privacy information.

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

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

See omnystudio.com/listener for privacy information.

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

Sh...

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...

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:

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

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

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 scie...

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

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

Show your...

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:

Listen and subscribe to the ASGCT Podcast Network so you don't miss...

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’s Hospital of Philadelphia, and “The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy,” with author Jeff Chamberlain, PhD, University of Washington School of Medicine. 

If you enjoy this deep dive into res...

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:

"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 breakthrough: a child diagnosed with a rare genetic disorder had been successfully treated for the first time with a customized CRISPR gene editing therapy.

T...

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, Inc.; and The deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy; with author Dr. Mark Kay, Stanford University. 

If you enjoy this deep dive into...

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 field over the past 25 years,” with author Dr. Terry Flotte, UMass Chan Medical School, and “From concept to cure: The evolution of CAR-T cell therapy,” with author Dr. Carl June...

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 data at the ASGCT Annual Meeting, the premier event in cell and gene therapy, next month! Join us in New Orleans May 13-17, where we'll learn from more than 2,100 abst...

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.

Whether you’re from industry or academia, today’s conversation will help wrap all our heads around testing novel therapies for the first time in humans.

The ASGCT Annual Meeting is almost here...

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 event in our field and save $465 as an ASGCT member. Check out the full program, plan your trip, and register for the ASGCT Annual Meeting by visiting annualmeeting.asgct.org.

In This Episode

Paloma...

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 historical context that influences decision-making in clinical trials, and the unique considerations facing those who might choose to pursue cell and gene therapies. 

You can also hear Brendan in a panel discussion titled T...

Today's episode takes us into crucial territory where science meets ethics in gene therapy development. Following our recent compelling conversation with Ben Hurlbut on germline genetic editing, we're diving deeper into the ethical dimensions of bringing new treatments to patients.

We'll explore two critical challenges that development teams face: determining the initial dose for therapy and establishing acceptable parameters for off-target effects before clinical testing. While these decisions are grounded in scientific data, they carry profound ethical implications – especially considering that AAV-based therapies are currently "one and done" treatments, where redosing isn't yet possible.

Thomas We...

Join Dr. Paloma Giangrande, Editor-in-Chief of Molecular Therapy Nucleic Acids, as she discusses RNA exon editing with Ascidian Therapeutics' leadership: CSO Dr. Robert Bell and CEO Dr. Michael Ehlers. Their conversation explores Dr. Bell's recent MTNA publication "RNA Exon Editing: Splicing the Way to Treat Human Diseases."

Last Call: Submit your abstracts for ASGCT's 2025 Annual Meeting in New Orleans by January 31st, 11:59 PM ET. Present at cell and gene therapy's premier event and get published in Molecular Therapy. Details at annualmeeting.asgct.org/abstracts.

In This Episode

Paloma Giangrande, PhD
Editor-in-Chief, Molecular...

Sonal Gupta, MD, PhD, SVP and Head of Clinical Development at Affyimmune Therapeutics talks with host Emily Walsh Martin, PhD, to explore the challenges of cell therapy clinical trials and how to move patients quickly and thoughtfully through the steps required to receive therapy in aggressive solid tumor cancers. 

But first, "we’ve moved straight from pumpkin spice season into ASGCT Annual Meeting registration season," as Dr. Walsh Martin says. So hop online and join ASGCT, then register and take advantage of the incredible member discounts on Annual Meeting registration. We’ll see you in New Orleans, May 1...

This episode features a conversation between Dr. Timothy Cripe and Dr. Joseph Glorioso, who discuss an article published in Molecular Therapy Oncology by Dr. Glorioso and colleagues titled Oncolytic Herpes Simplex Viruses Designed for Targeted Treatment of EGFR-bearing Tumors.

Join the editor-in-chief of Molecular Therapy, Dr. Roland Herzog, and ASGCT this January for the next installment of Molecular Therapy Presents: Clinical Gene and Cell Therapy. This transformative field has grown from promising experimental treatments to approved medicines for a wide range of genetic and/or acquired diseases. This virtual event is free for ASGCT members to attend...

Host Emily Walsh Martin, PhD, is joined by Chris Choi, PhD, Roswell Park Comprehensive Cancer Center's Senior Vice President of Industry Partnerships and Technical Director of the center’s new Good Manufacturing Practice (cGMP) Facility. Dr. Choi is also an associate professor of oncology. This wide-ranging conversation covers cell therapy development and manufacturing and how the growing Roswell Park Center is aiming to support sponsors in the CAR T and cell therapy space. Emily and Chris also discuss their experiences on cross-functional teams and how a program's research, manufacturing, and clinical elements must be knit together for success.

...

This episode features a conversation between Dr. Roland Herzog and Dr. Joseph Glorioso. On January 1, 2025, Dr. Glorioso will begin his 5-year term as the Editor-in-Chief of Molecular Therapy, marking the end of Dr. Herzog’s successful tenure at the helm of the journal. Drs.Herzog and Glorioso engage in a wide-ranging conversation that touches on the history of Molecular Therapy, highlights from the gene and cell therapy field, and how Molecular Therapy will continue to grow and evolve under Dr. Glorioso’s leadership.

ASGCT is proud to present this episode of the Molecular Therapy Podcast in partnership with...

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: htt...

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/)

Show your support for ASGCT!: https://asgct...

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...

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...

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 evolv...

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...

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...

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 re...

At #ASGCT2024, Dr. Lam (CIRM) spoke during a symposium on developing CGTs with a DEI-forward approach. In this interview, she talks about  incorporating DEI into the review criteria for CIRM's funded programs, how to measure DEI, and what she'd like to see in the long term for patients.

Meeting registrants can watch this session (and all others) through June 21 on the virtual platform and the app!

Host: Lynnea Olivarez, incoming Communications Commmittee Chair

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As she begins her tenure as ASGCT President, Paula Cannon, PhD (USC), tells host Lynnea Olivarez, incoming chair of the Communications Committee, why she's excited to take the reins for the Society.

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Jef Akst, managing editor at BioSpace, talks about the major CGT issues she's been covering at #ASGCT2024 including regulatory considerations and AAV manufacturing. She also tells host Lynnea Olivarez what she hopes to be reporting on next year at the ASGCT Annual Meeting in New Orleans!

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Todd Carter, PhD (CSO at Voyager Therapeutics) talks with incoming Communications Committee Chair Lynnea Olivarez about changes in the approach to developing gene therapies for CNS disorders like Huntington's disease.

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ASGCT Board of Directors member Lindsey George, MD, (Children's Hospital of Philadelphia) discusses the dramatic advancements in available treatment options for patients with hemophilia and the responsibility of scientists and clinicians in guiding patients through these once-in-a-lifetime decisions.

Host: Lynnea Olivarez, incoming chair of the ASGCT Communications Committee

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Incoming ASGCT Communications Committee chair Lynnea Olivarez catches up with ASGCT Career Development Award winner Ashley Cooney, PhD, (University of Iowa) to discuss her work in vector-delivered airway gene transfer and use of saline solution to "dramatically increase" gene transfer.

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Leah Myers, Executive Director of the FamilieSCN2A Foundation, and Jenny Burke, who sits on the organization's board, talk about the importance of empowering patients and families affected by genetic diseases.

Host: Lynnea Olivarez

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Dr. Murry talks about his early work reprogramming injured heart cells, his vision for the future of organ regeneration, and the privilege of getting paid to be curious.

Host: Lynnea Olivarez

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"Storytelling is completely underrated in science," says Benjamin McLeod, a lab scientist-turned-communicator. Listen to him talk about the importance of presenting CGT research in a narrative format.

Host: Lynnea Olivarez

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In this episode, host Lynnea Olivarez talks with David Segal, PhD, an abstract session co-chair at the ASGCT Annual Meeting. Dr. Segal discusses new technologies that he says have moved from academia to industry, and, hopefully soon, to rare disease patients.

Miss a session? Catch it on demand on the virtual platform for 30 days following the conclusion of the meeting!

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ASGCT President Dr. Jeff Chamberlain cuts the ribbon at the ASGCT 27th Annual Meeting and, having planned much of this year's event, reflects on nearly three decades of ASGCT.

Host: Lynnea Olivarez, ASGCT Communications Committee

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Lynnea Olivarez, incoming ASGCT Communications Committee chair, will be live at the ASGCT 27th Annual Meeting talking to Society leaders, speakers, and abstract presenters about their contributions and what they're excited about at the Annual Meeting.

These bite-sized pods are perfect for your walk between the hotel and convention center or your commute to work during the Annual Meeting.

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

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Join host Chris Leidli as he sits down with Emily Walsh Martin, PhD, and Lynnea Olivarez to preview the most intriguing and promising abstracts presented at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting in Baltimore.

Emily and Lynnea share their thoughts on the breathtaking Presidential Symposium before discussing the rest of the program, including clinical implications, the potential impact on rare diseases, and more!

Looking for a curated guide through the Annual Meeting? Check out ASGCT's new Session Paths! These six staff-crafted programs provide pathways for some of the most...

Tune in for an exceptional discussion on chemistry, manufacturing, and controls with Dr. Susan D’Costa, Chief Technical and Commercial Officer at Genezen, a US-based CDMO for viral vectors. "Geek out" with Susan and Emily on the intricacies of defining your CMC strategy and ready yourselves for a tour-de-force of analytical and process development for viral vectors.

The future of cell and gene therapy is nearly here! The ASGCT Annual Meeting is less than a month away, and starting on April 22, you can download the abstracts collection to get a sneak peek at the cutting-edge research being pr...

Join Drs. Timothy Cripe and Marco Davila as they discuss a recent article published in the December issue of Molecular Therapy Oncology by Dr. Davila and colleagues titled, Bispecific CD33/CD123 targeted chimeric antigen receptor T cells for the treatment of acute myeloid leukemia.

Dr. Timothy Cripe: Editor-in-Chief of Molecular Therapy Oncology and Professor and Chief of Hematology/Oncology/BMT at Nationwide Children’s Hospital

Dr. Marco Davila, Rustum Family Endowed Chair in Translational Research, Senior Vice President and Associate Director for Translational Research, Department of Medicine and Immunology, Roswell Park Comprehensive Cancer Center

...

Join Emily Walsh Martin, PhD for an in-depth interview with Sabrina Mogle, co-founder of RareMoon Consulting. They talk about what drove her to dedicate her career to the orphan drug and advanced therapy space, and how helping clients navigate regulatory conversations on the path from idea to BLA keeps her motivated today. They also discuss her advice for clients in interacting with regulators given the evolving CBER and global regulatory landscape.

Do they make it through the whole episode without either providing the standard regulatory wisdom of  “it depends”? Listen to find out!

Have you regis...

Welcome a special ASGCT Annual Meeting preview hosted by Lynnea Olivarez, member of the ASGCT Communications Committee.

Today's guest is Emily Mullin, a staff writer at WIRED who covers biotechnology, medicine, and genetics. Emily is a stalwart on the CGT beat, and today, we're looking ahead to the major topics and themes that will be highlighted at the upcoming ASGCT Annual Meeting.

The ASGCT Annual Meeting is the field's flagship event, bringing together stakeholders from academia, industry, regulatory agencies, and more. It's a prime opportunity to exchange research, trends, and the latest in the field.<...

Dr. Roland Herzog sits down with Drs. David Schaffer and Adam Schieferecke to discuss a recent article published in the February issue of Molecular Therapy by Drs. Schaffer and Schieferecke and colleagues titled, Evolving membrane-associated accessory protein variants for improved adeno-associated virus production.

Read the full article: https://www.cell.com/molecular-therapy-family/molecular-therapy/abstract/S1525-0016(23)00679-2

Dr. Roland Herzog: Editor-in-Chief of Molecular Therapy and Professor of Pediatrics at Indiana University

Dr. David Schaffer: Hubbard Howe Jr. Distinguished Professor, Chemical and Biomolecular Engineering, Bioengineering, Molecular and Cell Biology, and the Helen Wills Neuroscience...

A special ASGCT Annual Meeting preview episode and crossover with the Rx for Biotech podcast featuring guest hosts and ASGCT Communications Committee members Chris Leidli and Catherine Gillespie. They interview ASGCT's Molecular Therapy Scientific Editor Rory Bricker-Anthony, PhD, and preview a scientific symposium at the upcoming ASGCT Annual Meeting on the impact of generative AI on cell and gene therapy (CGT), best practices, and real-world applications for communicators.

Dr. Bricker-Anthony, a speaker in the session, shares insight into what attendees can learn from the upcoming session, including current tools and applications in AI for CGT communications.

Today we have a very special guest on The Issue: Federico Mingozzi, PhD. Federico’s first ASGCT podcast could have easily been on Giants of Gene Therapy. Still, we're excited that he could join us for an in-depth chat about his career journey and AAV immunogenicity. Dr. Mingozzi has done seminal work throughout his career in AAV gene therapy, and his 2020 Molecular Therapy review of our current understanding of the immune reaction to these therapies is a must-read for anyone trying to get a handle on the very complicated landscape of immune reactions to viral vectors.

Have yo...

Join Drs. Paloma Giangrande (Editor-in-Chief of Molecular Therapy Nucleic Acids and Chief Technology Officer at Eleven Therapy) and Maxim Berezovski (Professor of Bioanalytical Chemistry at the University of Ottawa). They will discuss a recent article published in Molecular Therapy Nucleic Acids by Dr. Berezovski and colleagues titled, Discovery of DNA aptamers targeting SARS-CoV-2 nucleocapsid protein and protein-binding epitopes for label-free COVID-19 diagnostics.

Host: Dr. Rory Bricker-Anthony, Scientific Editor of the Molecular Therapy Family of Journals

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

Show your support for...

Episode 2 of The Issue on the ASGCT podcast network is an in-depth interview with Dr Tom McCauley, CSO of Omega Therapeutics. Tom and Emily talk about his path from condensed matter physics to leading teams of scientists exploring the next generation of molecular therapeutics. They also dive into their mutual experience in the early days of in vivo gene editing and the challenges one faces with the complexities of these products, particularly before there is much of a roadmap for development. Finally, they dive into the recent public Ad Comm and ultimate approval of Casgevy and how we hope...

This episode features a conversation between Dr. Richard Vile, Professor of Immunology at Mayo Clinic and Dr. Timothy Cripe, Editor-in-Chief of Molecular Therapy Oncology and Professor and Chief of Hematology/Oncology/BMT at Nationwide Children’s Hospital. They will discuss a recent article published in the June issue of Molecular Therapy Oncology by Dr. Vile and colleagues titled, "Trap and ambush therapy using sequential primary and tumor escape-selective oncolytic viruses."

Host: Dr. Rory Bricker-Anthony, Scientific Editor of the Molecular Therapy Family of Journals

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

Join Dr. Emily Walsh Martin for the first episode of her new podcast, The Issue on the ASGCT podcast network. Emily welcomes Michael Storey, PharmD (Nationwide Children's Hospital), to discuss the path from patient diagnosis to therapy administration for approved gene and cell therapies including the clinical and administrative steps along the way.

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

Welcome to the first episode of the Molecular Therapy Podcast! Join Dr. Roland Herzog, Editor-in-Chief of Molecular Therapy and Professor of Pediatrics at Indiana University, and Dr. Norbert Pardi, Assistant Professor of Microbiology at the Perelman School of Medicine at the University of Pennsylvania, as they discuss a recent article published in the September issue of Molecular Therapy by Dr. Pardi and colleagues titled, "Development of an mRNA-lipid Nanoparticle Vaccine Against Lyme Disease."

Host: Dr. Rory Bricker-Anthony, Scientific Editor of the Molecular Therapy Family of Journals

'Electric Dreams' by Scott Buckley - released under CC-BY 4.0.<...

Note: this episode is cross-posted with the Rx for Biotech podcast, hosted by Chris Leidli. 

Emily Walsh Martin, PhD, at Tremont Therapeutics Consulting, works with gene and cell therapy companies and investors who are seeking to advance novel therapies in the clinic. ASGCT’s Policy Summit is a big deal to her: It provides a full view of the clinical pipeline and helps her guide clients past the drug approval process and into considerations of patient access, manufacturing, and insurance reimbursement.

In this special episode, Dr. Walsh Martin shares her highlights from the fifth annual Poli...

Note: this episode is cross-posted with the Rx for Biotech podcast, hosted by Chris Leidli.

Francesca Cook is the Vice President, Pricing and Market Access, at REGENXBIO and past chair of ASGCT's Government Relations Committee. She’s attended and helped with ASGCT’s annual Policy Summit since its inception five years ago. In this episode, Cook shares her experience and advice around the event—coming up Sept. 18-19 (virtually + in Washington, DC) with its highest-profile line-up yet and more ways to interact with top CGT decision makers. Her interviewer is Chris Leidli, host of the Rx for Biotec...

Luigi Naldini, MD, PhD, discusses his intercontinental career and the future of gene editing and use of lentiviral vectors during a one-on-one conversation with ASGCT President Hans-Peter Kiem, MD, PhD. Welcome to the seventh episode of Giants of Gene Therapy!

Dr. Naldini is a Professor of Cell and Tissue Biology and of Gene and Cell Therapy at the San Raffaele University School of Medicine and Scientific Director of the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. He has achieved worldwide renown as the pioneer of lentivirus gene therapy, a tool that offers hope for...

Terry Flotte, MD, discusses the early days and possible future of gene therapy, the use of AAV to deliver GT to targeted sites in the body, and his inspiration behind becoming a physician, scientist, and medical school dean. He spoke one-on-one with ASGCT President Hans-Peter Kiem, MD, PhD. Welcome to the sixth episode of Giants of Gene Therapy!

Dr. Flotte is the Provost, Dean, and Celia and Isaac Haidak Professor at UMass Chan Medical School, with faculty appointments in the Department of Pediatrics and the Horae Gene Therapy Center. He is also a practicing pediatric pulmonologist and...

Jennifer Doudna, PhD, shares her personal journey to co-inventing CRISPR-Cas9 for gene editing and the promise of her discovery, during a one-on-one conversation with ASGCT President Hans-Peter Kiem, MD, PhD. Welcome to the fifth episode of Giants of Gene Therapy!

Dr. Doudna has been at UC Berkeley since 2002. She is a professor in the departments of Molecular and Cell Biology and Chemistry, the Li Ka Shing Chancellor’s Professor of Biomedical Science. She’s also the president and co-founder of the Innovative Genomics Institute. In 2020, Dr. Doudna earned the Nobel Prize in Chemistry for co-inventing CRISPR-Cas9 genome edit...

Drew Weissman, MD, PhD is a pioneer of mRNA technology, which has been used in more than 1 billion administered doses of COVID-19 vaccines. Weissman is the Roberts Family Professor in Vaccine Research, Director of Vaccine Research in the Infectious Disease Division, and the Director of the Institute for RNA Innovation at the University of Pennsylvania and the Perelman School of Medicine.

He co-developed the mRNA technology that made the COVID-19 vaccines possible. His inventions were used in the Pfizer and Moderna vaccines, and have the potential to treat a range of medical conditions. He is also promoting...

Carl June, MD, shares his scientific journey, from medical research officer in the U.S. Navy to leader behind the first FDA-approved gene therapy, during his one-on-one conversation with ASGCT President Hans-Peter Kiem, MD, PhD. Welcome to the third episode of Giants of Gene Therapy!

Dr. June is a professor in immunotherapy in the Department of Pathology and Laboratory Medicine at University of Pennsylvania's Perelman School of Medicine, the director of the Center for Cellular Immunotherapy, and the director of the Parker Institute for Cancer Immunotherapy at Penn. He was instrumental in bringing CAR T cells to...

Kathy High, MD, shares her personal and scientific journey, from hemophilia to retinal disease, during her one-on-one conversation with American Society of Gene & Cell Therapy President Hans-Peter Kiem, MD, PhD, in the second episode of ASGCT's Giants of Gene Therapy.

Dr. High is the president of therapeutics at AskBio. Before that, she was the co-founder, president, and head of research and development at Spark Therapeutics, where she oversaw the development, approval, and launch of Luxturna, the first AAV gene therapy approved for use in the United States.

Dr. High has published extensively in the space...

David Baltimore, PhD discusses the Nobel Prize, searching for answers on an HIV vaccine, and how gene therapy could change the course of history during his one-on-one conversation with American Society of Gene & Cell Therapy President Hans-Peter Kiem, PhD in the debut episode of ASGCT's Giants of Gene Therapy.

Baltimore needs very little introduction. He is president emeritus and distinguished professor of biology at Caltech and was awarded the Nobel Prize in Physiology or Medicine in 1975 at the age of 37 for “discoveries concerning the interaction between tumor viruses and the genetic material of the cell.”

Musi...

Hello, and welcome to the American Society of Gene & Cell Therapy Podcast Network! I’m Hans-Peter Kiem, current president of ASGCT, and I’m hosting our first program, Giants of Gene Therapy. I’ll be interviewing luminaries from the world of cell and gene therapy, from scientific pioneers to Nobel laureates, about a wide range of subjects near to their hearts and expertise.

Our first episode, with guest Dr. David Baltimore, will release near the end of October, and I hope that you’ll join us each month as we welcome a new guest to Giants of Gene The...