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.
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:
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.
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
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:
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:
"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.”
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...
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 o...
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...
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 registr...
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 Gen...
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 sp...
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On Health Stuff, hosts Dr. Priyanka Wali and comedian Hari Kondabolu tackle all the health questions that keep you up at night with hilarity and humanity. Together they demystify the flashy trends, and keep you informed on the latest research. You can rely on Health Stuff to bring you real, uninhibited, and thoughtful health talk of the highest caliber, and a healthy dose of humor.
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