In this episode, Dr. Paloma Giangrande, Editor-in-Chief of Molecular Therapy Nucleic Acids, speaks with Normand Allaire, Head of Genomics at the Cystic Fibrosis Foundation Therapeutics Lab. They discuss the article, Upregulation of a CFTR mRNA isoform has therapeutic potential for the treatment of 3’ CFTR PTC variants, which introduces an ASO-based therapeutic approach to cystic fibrosis for the 10% of patients who lack an approved treatment.

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

In this episode, Dr. Paloma Giangrande, Editor-in-Chief of Molecular Therapy Nucleic Acids, speaks with Normand Allaire, Head of Genomics at the Cystic Fibrosis Foundation Therapeutics Lab. They discuss the article, Upregulation of a CFTR mRNA isoform has therapeutic potential for the treatment of 3&rsquo; CFTR PTC variants, which introduces an ASO-based therapeutic approach to cystic fibrosis for the 10% of patients who lack an approved treatment.
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>

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

A CFTR mRNA isoform for the treatment of 3’ CFTR PTC variants with Normand Allaire

In this episode, recorded on-site at the ASGCT 2026 Annual Meeting, Molecular Therapy Advances Associate Editor-in-Chief Dr. Dan Stone speaks with Dr. Dan DuBreuil of Sanofi. They break down the science behind a recent paper detailing the development of a secretable frataxin, highlighting its potential to improve treatment efficacy for Friedreich’s ataxia.

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

In this episode, recorded on-site at the ASGCT 2026 Annual Meeting, Molecular Therapy Advances Associate Editor-in-Chief Dr. Dan Stone speaks with Dr. Dan DuBreuil of Sanofi. They break down the science behind a recent paper detailing the development of a secretable frataxin, highlighting its potential to improve treatment efficacy for Friedreich&rsquo;s ataxia.
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>

Development of a secretable frataxin for Friedrich’s ataxia with Dr. Dan DuBreuil

Recorded on site at the ASGCT 2026 Annual Meeting in Boston, this mini-series introduces listeners to ASGCT members and the work driving progress across the cell and gene therapy field. In each episode, members are invited to share their backgrounds, discuss the current state of the field, and reflect on the value of ASGCT membership. This series of brief, engaging conversations will help listeners get to know the people, perspectives, and ideas shaping the ASGCT community.

In this episode, host Ben McLeod of the ASGCT Communications Committee talks with Dr. Mark Deehan of MassGeneral Hospital. Dr. Deehan discusses being a new member of ASGCT and how receiving an ASGCT Career Development Award, funded by the Foundation for Angelman Syndrome Therapeutics (FAST), has advanced his lab work.

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

Recorded on site at the ASGCT 2026 Annual Meeting in Boston, this mini-series introduces listeners to ASGCT members and the work driving progress across the cell and gene therapy field. In each episode, members are invited to share their backgrounds, discuss the current state of the field, and reflect on the value of ASGCT membership. This series of brief, engaging conversations will help listeners get to know the people, perspectives, and ideas shaping the ASGCT community.
In this episode, host Ben McLeod of the ASGCT Communications Committee talks with Dr. Mark Deehan of MassGeneral Hospital. Dr. Deehan discusses being a new member of ASGCT and how receiving an ASGCT Career Development Award, funded by the Foundation for Angelman Syndrome Therapeutics (FAST), has advanced his lab work.
Music:&nbsp;'Origami' by Scott Buckley - released under CC-BY 4.0.&nbsp;<a href="https://url.us.m.mimecastprotect.com/s/IkczCERmXJSyRj4iNfxU7G5vB">www.scottbuckley.com.au</a>

Member Minutes: Mark Deehan, PhD

Recorded on site at the ASGCT 2026 Annual Meeting in Boston, this mini-series introduces listeners to ASGCT members and the work driving progress across the cell and gene therapy field. In each episode, members are invited to share their backgrounds, discuss the current state of the field, and reflect on the value of ASGCT membership. This series of brief, engaging conversations will help listeners get to know the people, perspectives, and ideas shaping the ASGCT community.

In this episode, host Ben McLeod of the ASGCT Communications Committee sits down with Chrissy Green, the co-founder and president of Finding Hope for Frizzle (FRRS1L). As a patient advocate, Chrissy discusses how her parent-led organization is working to develop a treatment for FRRS1L. In addition, she shares how the ASGCT Annual Meeting has given her access to individuals and organizations who can help with the development of the treatment.

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

Recorded on site at the ASGCT 2026 Annual Meeting in Boston, this mini-series introduces listeners to ASGCT members and the work driving progress across the cell and gene therapy field. In each episode, members are invited to share their backgrounds, discuss the current state of the field, and reflect on the value of ASGCT membership. This series of brief, engaging conversations will help listeners get to know the people, perspectives, and ideas shaping the ASGCT community.
In this episode, host Ben McLeod of the ASGCT Communications Committee sits down with Chrissy Green, the co-founder and president of&nbsp;<a href="https://www.frrs1l.org/" data-auth="NotApplicable" data-linkindex="0">Finding Hope for Frizzle (FRRS1L)</a>. As a patient advocate, Chrissy discusses how her parent-led organization is working to develop a treatment for FRRS1L. In addition, she shares how the ASGCT Annual Meeting has given her access to individuals and organizations who can help with the development of the treatment.
Music:&nbsp;'Origami' by Scott Buckley - released under CC-BY 4.0.&nbsp;<a href="https://url.us.m.mimecastprotect.com/s/IkczCERmXJSyRj4iNfxU7G5vB">www.scottbuckley.com.au</a>

A CFTR mRNA isoform for the treatment of 3’ CFTR PTC variants with Normand Allaire

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