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

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

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.

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Patient Advocacy with Leah Myers + Jenny Burke

Dr. Marcin Kortylewski (Oligonucleotide therapies and applications Section Editor of Molecular Therapy Nucleic Acids) sits down with Dr. Norman Michael Drzeniek and Niklas Kotzian from the Berlin Institute of Health at Charité. Join them as they discuss their new article, mRNA-based CAR T cell engineering: Unmodified mRNA enables high CAR expression without innate immune activation in T cells.

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

Dr. Marcin Kortylewski (Oligonucleotide therapies and applications Section Editor of&nbsp;Molecular Therapy Nucleic Acids) sits down with Dr. Norman Michael Drzeniek and Niklas Kotzian from the Berlin Institute of Health at Charit&eacute;. Join them as they discuss their new article, <a href="https://www.cell.com/molecular-therapy-family/nucleic-acids/fulltext/S2162-2531(25)00359-2">mRNA-based CAR T cell engineering: Unmodified mRNA enables high CAR expression without innate immune activation in T cells</a>.
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>

mRNA-based CAR T cell engineering with Norman Drzeniek and Niklas Kotzian

In this episode, Melissa Majerol, Model Lead of the Cell and Gene Therapy Access Model at the Center for Medicare and Medicaid Innovation (CMMI) at CMS, breaks down how the CMMI Access Model program aims to simplify and accelerate patient access to innovative cell and gene therapies, starting with recently approved therapies in Sickle Cell disease. Listen in as we dive into the specifics of this new access model and its current status.

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

In this episode, Melissa Majerol, Model Lead of the Cell and Gene Therapy Access Model at the Center for Medicare and Medicaid Innovation (CMMI) at CMS, breaks down how the CMMI Access Model program aims to simplify and accelerate patient access to innovative cell and gene therapies, starting with recently approved therapies in Sickle Cell disease. Listen in as we dive into the specifics of this new access model and its current status.
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>

The Issue - Evolving Patient Access to CGTs via Innovation in Medicaid Reimbursement Models with Melissa Majerol

Dr. Daniel Stone (Associate Editor-in-Chief, Molecular Therapy Advances) sits down with Dr. Krishanu Saha, Anna Tommasi, and Dr. Dan Cappabianca from the University of Wisconsin-Madison. Join them as they discuss their team’s recent publication, Efficient nonviral integration of large transgenes into human T cells using Cas9-CLIPT, and learn more about their novel method to improve the manufacturing of multifunctional, genome-edited immune cell therapies.

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

Dr. Daniel Stone (Associate Editor-in-Chief, Molecular Therapy Advances) sits down with Dr. Krishanu Saha, Anna Tommasi, and Dr. Dan Cappabianca from the University of Wisconsin-Madison. Join them as they discuss their team&rsquo;s recent publication, Efficient nonviral integration of large transgenes into human T cells using Cas9-CLIPT, and learn more about their novel method to improve the manufacturing of multifunctional, genome-edited immune cell therapies.
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>

Patient Advocacy with Leah Myers + Jenny Burke

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