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.

ASGCT Podcast Network

Patient Advocacy with Leah Myers + Jenny Burke

In this episode, Molecular Therapy Editor-in-Chief Dr. Joseph Glorioso and Dr. Rita Perlingeiro (University of Minnesota) will discuss an article recently published in Molecular Therapy titled, “Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies.”

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

In this episode,&nbsp;Molecular Therapy&nbsp;Editor-in-Chief Dr. Joseph Glorioso and Dr. Rita Perlingeiro (University of Minnesota) will discuss an article recently published in&nbsp;Molecular Therapy&nbsp;titled, &ldquo;<a href="https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(25)00543-X">Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies</a>.&rdquo;
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>

CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies with Joseph Glorioso and Rita Perlingeiro

In this episode of The Issue, Emily Walsh Martin is joined by Dr. Ralph Brandenberger, Master Principal; Head of CMC at Dark Horse Consulting for a discussion on due diligence – the most important time in a company’s existence, be it for funding, partnership, or acquisition. Listen in as Ralph and Emily peel back the curtain on what to expect, how to prepare, and how to stay calm, cool, and collected under scrutiny!

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, Emily Walsh Martin is joined by Dr. Ralph Brandenberger, Master Principal; Head of CMC at Dark Horse Consulting for a discussion on due diligence &ndash; the most important time in a company&rsquo;s existence, be it for funding, partnership, or acquisition. Listen in as Ralph and Emily peel back the curtain on what to expect, how to prepare, and how to stay calm, cool, and collected under scrutiny!
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 – Prepared under pressure: Navigating the due diligence process with Dr. Ralph Brandenberger

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>

Patient Advocacy with Leah Myers + Jenny Burke

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