Gene Therapy

Interesting take on gene therapy in general–nothing hearing specific.
After reading it, a take I had was that if there are major breakthroughs with gene therapies with hearing, it’s going to be very expensive. Hearing aids will look like a bargain.


A clinical trial for a gene therapy treatment for some forms of profound congenital hearing loss named CFG166 (Novartis is the company involved) was due to complete in December 2019. No word yet on how that went.


I have been actively following not only this but other trials. They fall into 3 categories. Hearing hair cell regeneration, improved neural transmission of hair cell impulses and hearing regions in the brain. The CGF166 trials (Hair cell regeneration) should be out soon, but I can only assume that no news is at best mixed news. Research is much like politics. Breaking news gets out ahead of a final report.

Here is a great article that summarizes the work that has been going on.

It is a little dated (2015) but still relevant on who is doing what in this effort. I have NO doubt that like so much in the filed of genetic modification that permanent solutions to hearing loss will come within the next 10 years. There is simply too much work going on.

The Hearing Health Foundation is another very good resource to stay informed on recent research. They fund much through their Hearing Restoration Project.

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You may be right, but I have considerable doubt. A tangential story. I have an autistic son diagnosed in the mid 90’s. Around that time the human genome was sequenced and there were predictions about having the genetics of autism sorted out and many treatments available. We’re now like 25 years later and although there has been some progress in studying the genetics of autism, there are no genetic or specific medical treatments available. It’s all about behavioral training and targeting bothersome symptoms. I hope they are more successful with hearing, but I’m not holding my breath.

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I agree with @jcech that we will see treatments within ten years. We’ve already seen doubling of word recognition scores in 4 out 6 subjects with moderate to severe ssn loss in the last Frequency Therapeutics trial. Audion will report on their phase 2 clinical trial in March or April. There is a lot going on.

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I had a look at a recent paper titled “Hearing Protection, Restoration, and Regeneration: An Overview
of Emerging Therapeutics for Inner Ear and Central Hearing Disorders”.

The authors found 43 companies working on therapies for hearing loss and various inner ear disorders with 76 active programs. The majority of those programs were for otoprotective solutions. Only four were related to gene therapy. The otoprotective programs were furthest along the pipeline.

I’m sure there are more programs than that (I can think of a couple that they missed off the top of my head). We can take it for granted that the vast majority of those programs won’t make it to the clinic, but it’s still a sizable number.

Just read today that Novartis has suspended their CGF166 trial due to less than expected improvement. Apparently candidates were selected how had profound hearing loss and improvement was recorded in only a few.

Perhaps more will come out and they may restart them after review of the results so far.

Are you sure that’s not old news? They suspended trials a few years ago and then resumed them. I couldn’t find a link on Google for anything recent. If this current trial was unsuccessful, that would be the end of CFG166, I would have thought.

This is the only instance I can find, and no source is provided so…?

Don’t know. The trial was due to finish in December 2019, so there
s really nothing to suspend.