In this episode, host Emily Walsh Martin revisits her 2022 Caregiver’s Perspective interview with Amber Freed, founder of SLC6A1 Connect. Amber is a caregiver to her son Maxwell, who has since received treatment for SLC6A1. Emily and Amber discuss Maxwell’s treatment and the role a caregiver plays in that journey.

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

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Emily Walsh Martin, PhD, spoke with Amber Freed, founder and CEO of SLC6A1 Connect, a patient organization focused on accelerating the development of therapies for SLC6A1 patients like her son Maxwell.

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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

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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/

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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

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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/

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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

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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/

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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/membership/donate

See omnystudio.com/listener for privacy information.