The CFRI CF Community Voices Podcast

Selective Targeting Distal Small Airways by AAV Vectors (...) – Xiaopeng Li, PhD (Audio)

Dr. Xiaopeng Li, Associate Professor in the Department of Pediatrics and Human Development in the College of Human Medicine at Michigan State University, presents results from CFRI-funded research on selective targeting distal small airways by adeno-associated virus (AAV) vectors. He discusses findings from CF pig models that support AAV4 as a suitable viral vector for CF gene therapy to target small airway secretory cells. This presentation was part of CFRI's Virtual 35th National CF Education Conference.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Joints in Cystic Fibrosis - Dr. Beth Clarke

Many individuals with cystic fibrosis suffer from chronic joint pain. In this podcast, recorded at CFRI's CF Adult Retreat in August, 2022, Dr. Beth Clarke, from the University of Glasgow in Scotland, discusses the causes of joint pain and cystic fibrosis associated arthritis (CFA), exercise and CFA and strategies to address this pain.

If you watch this episode, please let us know what you think at https://www.surveymonkey.com/r/KGB7PKY

CF Community Voices is made possible through grants from Chiesi USA, Genentech, Vertex Pharmaceuticals, Gilead Sciences, and Viatris.

CFRI’s 35th Cystic Fibrosis Education Conference - Awards Celebration on July 30, 2022

At CFRI's virtual Conference Awards Celebration on July 30, 2022, we honored these extraordinary individuals: Richard Moss, MD - Paul M. Quinton Cystic Fibrosis Research Legacy Award; Sonya Haggett, LCSW - CFRI Partners in Living Award In Memory of Anabel Stenzel; Anna Payne – Dave Stuckert Memorial Volunteer of the Year Award; Dennis Nielson, MD, PhD - Professional of the Year Award.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Keynote Remarks by Janet Woodcock, MD - CFRI’s 35th Cystic Fibrosis Education Conference

Dr. Janet Woodcock, Principal Deputy Commissioner of the US Food and Drug Administration, delivered Keynote Remarks at the opening of CFRI’s 35^th National CF Education Conference on July 30, 2022.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

mRNA therapy for People with Cystic Fibrosis: Next Steps – Manu Jain, MD, MSCI

Dr. Manu Jain, Professor of Medicine and Pediatrics at Northwestern University’s Feinberg School of Medicine and Director of the Adult CF Program at Northwestern, presents a comprehensive overview on mRNA therapy for people with cystic fibrosis, a mutation-agnostic treatment that could work for any person with CF regardless of their CFTR mutation. Dr. Jain provides an update on the principles behind mRNA therapy, the hurdles it faces to become a viable treatment option, and the various programs in clinical development. This presentation was part of CFRI's Virtual 35th National CF Education Conference on July 30, 2022.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Advances in Gene Therapy for Cystic Fibrosis – Paul McCray, MD

Dr. Paul McCray, Professor of Pediatrics, Division of Pulmonary Medicine, and Professor of Microbiology and Immunology at the University of Iowa, presents an overview on advances in gene therapy for cystic fibrosis, with a focus on gene therapy for lung disease treatment and prevention. Dr. McCray explores the different gene addition and gene editing strategies under investigation and reviews the challenges of delivery to the lung. This presentation was part of CFRI's Virtual 35th National CF Education Conference on July 30, 2022.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

The Power of Science, Community and Desperation to Speed Lifesaving Breakthroughs for the Final 10% – Emily Kramer-Golinkoff, MBE

Emily Kramer-Golinkoff, an adult with cystic fibrosis and the Co-Founder of Emily's Entourage, an innovative 501(c)3 foundation that accelerates research for new treatments and a cure for nonsense mutations of CF, describes her experience growing up with CF and the heart-shattering realization that she is a part of the approximately 10% of the CF community that is ineligible for CFTR modulators. She discusses the promise of science and innovation, each individual’s power to create transformative change, and the unique solidarity and commitment of the whole CF community to create a better future for everyone with CF. This presentation was part of CFRI's Virtual 35th National CF Education Conference on July 31, 2022.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

CFRI’s 35th Cystic Fibrosis Education Conference - Awards Celebration on July 30, 2022 (Audio)

At CFRI's virtual Conference Awards Celebration on July 30, 2022, we honored these extraordinary individuals: Richard Moss, MD - Paul M. Quinton Cystic Fibrosis Research Legacy Award; Sonya Haggett, LCSW - CFRI Partners in Living Award In Memory of Anabel Stenzel; Anna Payne – Dave Stuckert Memorial Volunteer of the Year Award; Dennis Nielson, MD, PhD - Professional of the Year Award.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

Keynote Remarks by Janet Woodcock, MD - CFRI’s 35th Cystic Fibrosis Education Conference (Audio)

Dr. Janet Woodcock, Principal Deputy Commissioner of the US Food and Drug Administration, delivered Keynote Remarks at the opening of CFRI’s 35^th National CF Education Conference on July 30, 2022.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

mRNA therapy for People with Cystic Fibrosis: Next Steps – Manu Jain, MD, MSCI (Audio)

Dr. Manu Jain, Professor of Medicine and Pediatrics at Northwestern University’s Feinberg School of Medicine and Director of the Adult CF Program at Northwestern, presents a comprehensive overview on mRNA therapy for people with cystic fibrosis, a mutation-agnostic treatment that could work for any person with CF regardless of their CFTR mutation. Dr. Jain provides an update on the principles behind mRNA therapy, the hurdles it faces to become a viable treatment option, and the various programs in clinical development. This presentation was part of CFRI's Virtual 35th National CF Education Conference on July 30, 2022.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.