Golden Ticket: Competition to Create Genetic CF Treatments

Many of us are familiar with the famous story written by Roald Dahl titled Charlie and the Chocolate Factory; we know the tale of the golden ticket and Charlie’s journey to Willy Wonka’s factory. The Cystic Fibrosis Foundation loves this story too. In fact, they’ve adapted the idea of the golden ticket and applied it to the development of genetic therapies for cystic fibrosis (CF).

The foundation has teamed up with Bakar Labs of the University of California, Berkeley to create the Golden Ticket competition. Starting on May 2nd, researchers focusing on genetic therapies (e.g., gene delivery, gene insertion, gene therapy, gene editing) may apply. Judges will receive applications until May 30th, and then they’ll invite the finalists to present their work in-person on July 20th of this year.

You may be asking an important question at this point: what is this metaphorical golden ticket? Well, three winners will receive support from Bakar labs alongside a year of free laboratory space. This means the winners will have access to the Cystic Fibrosis Foundation’s large network, infrastructure, and expertise. Some examples include patient registries, help in designing clinical trials, the Therapeutics Development Network, experts, advice, and more.

This is huge and could greatly advance research into treatments for cystic fibrosis. Hopefully, this competition will push drug development and get viable treatment options to patients sooner.

About Cystic Fibrosis (CF)

CF is a rare, genetic disorder that is characterized by the buildup of sticky, thick mucus that damages the respiratory and digestive systems. This mucus clogs the airways, which then causes breathing issues, trapped bacteria, lung damage, frequent infections, and respiratory failure. Beyond its damage to the lungs and airways, it also stops the release of digestive enzymes, stopping patients from absorbing the nutrients they need. Other symptoms include:

  • Shortness of breath
  • Constipation
  • Stuffy nose
  • Difficulty with weight gain
  • Exercise intolerance
  • Persistent coughing and wheezing
  • Male infertility
  • Salty-tasting skin

These symptoms are the result of a mutated CFTR gene, which is passed down in an autosomal recessive pattern.

Find the source article here.

Share this post

Share on facebook
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email