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

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

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

New Frontiers in Hemophilia Treatments with Lindsey George, MD

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>

Cas9-CLIPT-mediated integration of large transgenes with Krishanu Saha, Anna Tommasi, and Dan Cappabianca

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

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&rsquo;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:&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>

New Frontiers in Hemophilia Treatments with Lindsey George, MD

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