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A pipeline of therapies for cystic fibrosis

Melissa A Ashlock1, Robert J Beall, Nicole M Hamblett

  • 1Cystic Fibrosis Foundation Therapeutics, Inc., Bethesda, Maryland 20814, USA. mashlock@cff.org

Seminars in Respiratory and Critical Care Medicine
|September 18, 2009
PubMed

Insights

The Cystic Fibrosis Foundation (CFF) has invested significantly in developing new cystic fibrosis (CF) therapeutics. This research outlines the drug development process, highlighting the CFF

Area of Science:

  • * Drug discovery and development for genetic diseases.
  • * Clinical research coordination and funding models.

Background:

  • * Cystic Fibrosis (CF) therapeutics development requires multi-stakeholder collaboration.
  • * The Cystic Fibrosis Foundation (CFF) has a long history of funding CF research.

Purpose of the Study:

  • * To discuss the process of bringing therapeutic strategies into clinical development for CF.
  • * To examine the funding, management, and infrastructure supporting drug candidate progression.
  • * To highlight the importance of clinical trials and outcome measures for CF therapeutics.

Main Methods:

  • * Review of the Cystic Fibrosis Foundation's (CFF) historical investment and drug development pipeline.
  • * Analysis of the strategies and infrastructure supporting therapeutic progression.
  • * Discussion of clinical trial processes and efficacy assessment for CF drug candidates.

Main Results:

  • * Over $875 million invested by CFF since 1986, facilitating ~50 drug candidate assessments.
  • * A current pipeline of over 30 Foundation-funded therapeutic compounds.
  • * Detailed examination of the pathway from discovery to clinical availability for CF treatments.

Conclusions:

  • * The CFF's coordinated efforts have significantly advanced cystic fibrosis therapeutics development.
  • * Robust funding, management, and infrastructure are crucial for successful drug progression.
  • * The ongoing pipeline holds substantial potential for improving the lives of individuals with CF.

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