Gene Therapy for Cystic Fibrosis Moves Forward in Development

Currently, about 90% of cystic fibrosis (CF) patients have a treatment option. This percentage jumped from 50% to 90% upon the approval of Trikafta. However, the remaining 10% of CF patients have nonsense mutations and live without a viable therapy. Boehringer Ingelheim aims to change this with their acquisition of the gene therapy BI 3720931.

Updates on BI 3720931

This lentiviral gene therapy is still in preclinical development, and it was created to give patients a functioning version of the CFTR gene. Researchers’ goal is to develop it as an inhalable agent rather than an injectable one, as this formulation requires less work from the patient. According to Boehringer, it has shown efficiency in its preclinical trials.

The company also announced that it licensed the rights to this research last Tuesday. They’ll pay a fee to IP Group and Oxford Biomedica, with the latter receiving further payments if certain milestones are achieved.

Hopefully, this gene therapy continues to advance through clinical trials, as it would offer a treatment to a population that currently lacks one.

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.

You can find the source article here.

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