A Single Shot Cures a Child's Deafness

Feb 1, 2024
A Single Shot Cures a Child's Deafness

A Young Boy Heard Sound For the First Time After Receiving a Single Shot from Precision Genetic Medicine Company Akouos

“There’s no sound I don’t like, they’re all good.”

These words could have been said by any of us. Without context, they are unremarkable.

And yet, they are profound. For they were said by an 11-year-old boy — Aissam Dam — who was born deaf.

Since birth, Aissam was diagnosed with a genetic condition known as otoferlin gene-mediated hearing loss. It is a condition caused by one single mutation of the otoferlin gene which affects hearing.

This condition affects about 200,000 people worldwide. Those that have this mutation have been deaf for their entire lives…

Until now.

A Single Shot

On October 4, 2023, Aissam underwent a surgery to inject a gene therapy into his inner ear.

It was just a single shot…

Administered into the inner fluid of his ear, it carried a version of the otoferlin gene that did not have the mutation. This is known as an in vivo gene therapy, where the therapy is injected directly into a patient.

The results were just published a few days ago by the Children’s Hospital of Philadelphia, where the procedure was performed.

The results were a breakthrough in the world of gene therapy and genetic editing.

Seen below, in the middle of the picture, is Aissam. He can now hear.

Aissam Dam's Deafness is Cured with a Single Shot
Source: Children’s Hospital of Philadelphia

I cheered when I read the results.

Not only is this a heartwarming story that has profound implications for those who have hearing loss caused by genetic mutations, it is also a huge win for an area of biotechnology that I have been actively researching and studying now for almost a decade.

And the company behind this breakthrough is also one that I know well, Akouos (pronounced “A-koo-os”).

Akouos was a company that I identified well before its IPO in June of 2020. And I recommended the company to the readers of one of my past paid research services.

Akouos was primarily developing gene therapies for hearing loss. And it was focused on what I consider to be the low-hanging fruit in the world of genetic editing — disease caused by single genetic mutations.

Akouos’ most advanced clinical therapy has been AK-OTOF, designed to replace the mutated otoferlin gene with a healthy version.

With a healthy version of the otoferlin gene, the correct protein is produced so that the inner ear functions properly for hearing.

And up until a few days ago, that was just a theory.

Now we know it works. And it really is that simple.

Once the therapy was administered into Aissam’s ear — just under a portion of his eardrum — Aissam’s own hair cells began constructing the proteins required for hearing.

This now opens the door for future therapies that might help more than 450 million people around the world with disabling hearing loss.

And it seems I wasn’t the only one who saw the potential.


Eli Lilly clearly saw the potential of what Akouos was working on, too.

So back in December of 2022, it was no surprise when I saw the news that Lilly announced its $487 million acquisition of the gene therapy company.

Lilly clearly had an insider’s view of the value of Akouos’ therapeutic pipeline, as its acquisition was pre-emptive, well before the start of its clinical trials.

Akouos (AKUS) Share Price, November 2021-November 2022

Akouos (AKUS) Share Price, November 2021-November 2022

From the chart above, some big money knew an acquisition was in play.

Akouos rose from its 2022 summer lows around $2.60 to close around $13.29. That’s a jump of more than 5-times in just the span of a few months.

Even more notable was that it happened in the depths of one of the longest biotech bear markets in history.

Eli Lilly’s offer represented a 174% premium over Akouos’ 30-day average share price when accounting for the total value of the acquisition offer. And that was after the share price had already risen from $2.60 to around $7 a share.

And despite Eli Lilly’s size, worth almost $600 billion, the Akouos announcement as a fully owned subsidiary of Eli Lilly still propelled Eli Lilly to all-time highs.

Eli Lilly (LLY) Share Price, 1-Year Chart

Eli Lilly (LLY) Share Price, 1-Year Chart

Obviously, catalysts like an acquisition offer or successful trial results can have a remarkably positive impact on the share price in the biotech industry.

And as we can see from the chart above, it is quite normal to see a run-up in share price in advance of the big news becoming public.

While the Akouos acquisition by Eli Lilly happened during a very slow period in the biotech industry, that environment is changing.

A Remarkable Period of Time

Deal flow has started to pick up.

We explored the increase in M&A (mergers and acquisitions) deals in my January 23, 2024 issue of Outer Limits. For those interested in the biotech industry, it is a must read.

Breakthroughs like the one we just learned about — a single shot that gave hearing to an 11-year-old deaf child — are not just a catalyst for Akouos, but for the enter biotech industry working on gene therapies and genetic editing.

And this development comes on the heels of the incredible news that came at the end of last year… concerning another gene therapy.

We celebrated those developments in the December 20, 2024 issue of Outer Limits.

It was a similar development to Akouos’ victory…

In this case, genetic editing company CRISPR Therapeutics (CRSP) received regulatory approval in the U.K. for its CRISPR-based gene therapy for sickle cell disease, as well as for transfusion dependent beta thalassemia. This came on November 16, 2023.

That news was quickly followed up on December 8, 2023. The FDA approved the same therapy for sickle cell disease for patients 12 years and older in the U.S. market.

And then on December 15, 2023, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of the same therapy for use in Europe.

What’s exciting about all this is that we’re just at the beginning.

We are entering a remarkable period in time where genetic editing technology can be used to cure all human disease caused by genetic mutations.

The industry has largely focused its initial sights on less complex genetic mutations. This is smart as it improves the likelihood of success.

Developing these gene therapies for simpler mutations will build a foundation for the entire industry… upon which biotech companies can begin taking on more complex genetic diseases using different kinds of genetic editing technologies, paired with different enzymes that are optimized for each individual disease.

These latest developments effectively mean that all of the most promising public and private biotech companies operating in this space are in play…

Some for their intellectual property portfolios, like Editas Medicine (EDIT).

And others for their therapeutic pipelines, like Intellia Therapeutics (NTLA), Caribou Biosciences (CRBU), CRISPR Therapeutics (CRSP), Beam Therapeutics (BEAM), and numerous private early stage biotech companies.

And that means plenty of great investment opportunities.

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