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:

• why patients, as the "lived experience experts" are so important to rare disease communications

• advice for scientists who would like to engage more directly with patient communities

• what's giving her hope right now

Listen and subscribe to the ASGCT Podcast Network so you don't miss any episodes!

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.

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.

If you want to learn more about cell and gene therapy access, register for the ASGCT Policy Summit, September 25-26, in Washington, D.C.! We’ll hear from policymakers, industry leaders, and science stakeholders, who will explore solutions to the challenges of accessing therapeutic treatments.

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

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.

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

• breaking down silos to improve health outcomes for all
• the role that CGT will play in prevention, rather than just treatment, of diseases

Listen and subscribe to the ASGCT Podcast Network so you don't miss any episodes!

Show your support for ASGCT!: https://asgct.org/membership/donate

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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:

• How the vaccine works and how its approach is different from other treatments
• Safety results from the unblinded data
• Implications this vaccine could have for other substance use disorders

Listen and subscribe to the ASGCT Podcast Network so you don't miss any episodes!

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.

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’s Hospital of Philadelphia, and “The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy,” with author Jeff Chamberlain, PhD, University of Washington School of Medicine.

If you enjoy this deep dive into research that is critical to the field, check out two more ASGCT events happening this fall: Advancing Cell and Gene Therapies for Cancer and Breakthroughs in Targeted In Vivo Gene Editing. During these events – in October and November, respectively – you’ll be able to hear directly from researchers about their own work related to these topics, or you can submit your own abstract for possible presentation. We hope to see you there!

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

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.

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:

• The impact of developing cell and gene therapies as platforms to treat more than one disease
• Projects he's involved in that are working towards the platform approach
• What's giving him hope right now about the future of the field

Listen and subscribe to the ASGCT Podcast Network so you don't miss any episodes!

Did you attend the 28th Annual Meeting?

Watch all sessions on demand on the virtual meeting platform through July 18!

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.

"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 breakthrough: a child diagnosed with a rare genetic disorder had been successfully treated for the first time with a customized CRISPR gene editing therapy.

The infant, KJ Muldoon, was born with a rare metabolic disease called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. When he was just six months old, KJ received his personalized therapy at the Children's Hospital of Philadelphia (CHOP), where ASGCT Members Kiran Musunuru, MD, PhD; and Rebecca Ahrens-Nicklas, MD, PhD; led the group of researchers who developed KJ's treatment.

KJ is now growing and thriving. He recently left the hospital where he has spent the majority of his life. And the day after presenting the research that led to this first-of-its-kind development, Dr. Musunuru told us that there was so much more to the story.

Listen to our first episode of Soundbites of the Annual Meeting with Dr. Kiran Musunuru.

Did you attend the 28th Annual Meeting?

• Watch all sessions, including Dr. Musunuru's presentation, on demand on the virtual meeting platform through July 18!
• Not a member? Join now and receive 19 months of membership for the price of 12.

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.

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, Inc.; and The deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy; with author Dr. Mark Kay, Stanford University.

If you enjoy this deep dive into research that is critical to the field, check out two more ASGCT events happening this fall: Advancing Cell and Gene Therapies for Cancer and Breakthroughs in Targeted In Vivo Gene Editing. During these events – in October and November, respectively – you’ll be able to hear directly from researchers about their own work related to these topics, or you can submit your own abstract for possible presentation. We hope to see you there!

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

Show your support for ASGCT!: https://asgct.org/membership/donate

See omnystudio.com/listener for privacy information.