In 2019, we launched the Path to a Cure which centers around three core strategies to address the underlying cause of cystic fibrosis: repairing broken CFTR protein, restoring CFTR protein when none exists, and fixing the underlying genetic mutation to address the root cause of CF.

Watch our Path to a Cure research webinar to hear from Bill Skach, MD, EVP and chief medical officer, and JP Clancy, MD, VP of clinical research, discuss updates on the research underway to accelerate treatments, what the future holds, and more.

Originally recorded February 4, 2021.

Questions and Answers:

0:00 | Research Overview: Path to a Cure
45:05 | Is there hope cystic fibrosis will be cured in the next 10 years?
46:20 | Will MRNA be tested alongside CFTR modulator therapies?
47:05 | Is MRNA or RNA more applicable to cystic fibrosis?
48:18 | Can you estimate the time frame for Trikafta access being expanded? Will there be an expansion to theratyping?
49:27 | Does knowledge from the MRNA COVID-19 vaccine help with CF research?
50:45 | Are we studying aerosolized MRNA therapies in double delta mutations?
52:04 | What will the criteria be for those to receive the new genetic treatments?
53:35 | What delivery strategies are being explored for MRNA therapies?
54:53 | How much do CFTR modulators affect new clinical trial recruitment?
56:34 | What major benefit do you think we could see from the Eloxx treatment?
57:40 | Which drug currently in development are you most optimistic about?

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