Akouos — A New Company Focused on Genetic Hearing Loss

Discussion in 'Research News' started by Aaron123, Nov 30, 2017.

    1. Aaron123

      Aaron123 Member

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    2. Samir
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      Good find Aaron! :) You are on fire lately! :eek: Keep them coming! (y)
       
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    3. Makrohn
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      Makrohn Member Benefactor Advocate

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      Aaron123

      Aaron123 Member

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      Here's a bit more information on Akouos. They received SBIR (small business innovation research) funding this year and are developing a minimally invasive delivery system presumably to deliver gene therapy to the inner ear: https://www.sbir.gov/sbirsearch/detail/1305311
       
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    5. Paulmanlike

      Paulmanlike Member Podcast Patron Benefactor Hall of Fame

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      They just keep coming man, could treating hearing loss pharmaceutically (is that a word?) be the new laser eye surgery that's a multi million or billion dollar market?

      Nice find Aaron, although which will come first? Treating noise induced deafness or genetic defect deafness?
       
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    6. Benoves

      Benoves Member Benefactor

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      This one looks serious. Thanks for tagging me. Looks like not many companies are targeting this? Is this different from wat audion/frequency are doing?

      Hope they wil find a cure in our lifetime, for hearing loss and tinnitus. Better be soon. Untill then i hope to habituate.
       
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    7. Enclave

      Enclave Member

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    8. IvanRus

      IvanRus Member

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      https://cen.acs.org/business/start-ups/Akouos-raises-50-million-develop/96/web/2018/08

      Yes, this is another startup that managed to attract 50 million dollars of investment.
      With their focus on genetic hearing loss, I have some misunderstandings, I will contact them.
      Probably, they focus on a new method of regeneration - viral regeneration of hearing. They hope to launch a genetically modified virus that will restore hearing.

      Reference:
      Today there are 4 main directions for the possibility of regeneration of the inner ear, this is:
      1) Treatment with stem cells;
      2) Gene therapy;
      3) Cell therapy;
      4) Viral therapy.
       
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    9. Samir
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    10. Samir
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      Samir Manager Staff Benefactor Ambassador Advocate

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    11. FGG
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      FGG Member Podcast Patron Benefactor Ambassador Hall of Fame Advocate

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      This company is about to offer an IPO with the ticker: AKUS, if anyone is still following them.

      Their AAV platform is promising for genetic hearing loss but they appear to be looking into inner ear cell (hair and possibly support cell via their website) regeneration, too.
       
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    12. FGG
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    13. FGG
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      Anyone else listen to their webcast?

      In the foreseeable future, it looks like they are focused on genetic hearing loss. Of note, the company has apparently invented their own new surgical approach to get their drug into the cochlea uniformly (has to be done surgically with AAV drugs). Though the specifics were confusing because it sounded like he said it was an Oval window approach, then said Round Window but I believe he misspoke the second time and he meant Oval Window because he described moving the stapes footplate in the approach.

      It was an interesting talk and I think the company will do well even if it's not directly applicable to most of us in the immediate future. It's obviously good to have more of this research in general, both for the millions who suffer genetic hearing loss, and because the tech may have broader applications. E.g.. The flat epithelia problem of profound long term hearing loss might be improved with something like AAV transduction (which Akouous sounds like they are leaders on) of remaining cell populations (fibroblasts). That's pretty long term though.
       
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    14. Thuan

      Thuan Member

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      I haven't watched the webcast. Forgot about it. But, can you explain why this is a novel approach? Why can't we just use a needle to inject the drug to the round window (without structural compromise of and around the stapes in the oval window) and allow the drug to diffuse naturally in the cochlear medium?

      I have always thought that if the drug is effective but the mechanism of delivery is the bottle neck, then there is always a surgical option, albeit quite invasive, to directly inject into the cochlea.
       
    15. tommyd87

      tommyd87 Member

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      There
      There is a newsletter you can sign up to on their website too which they release each quarter.

      6C526BAA-2A01-461C-AEE1-B9864EE12178.png

      The above image is a screenshot of their various planned treatments and their current position.

      While obviously they are currently focused on genetic conditions, it is clear that they have made some progress in all aspects that they are looking to treat.

      I would be interested in learning whether some of their treatments might overlap and actually treat multiple conditions. Eg whether you could use the autosomal dominant disorder treatment as a substitute for the hair cell treatment or similar.
       
    16. FGG
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      Briefly, the difference is AAV is a viral vectored therapy and it needs to be directly injected into to its target rather than diffusing in.
       
    17. wwtsai
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      wwtsai Member Podcast Patron Benefactor

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      Akouos provided their 3Q 2020 financial results and some updates. Probably the most notable update is:

      "Continued progress towards IND filing in 2021 to enable a Phase 1/2 clinical trial for AK-OTOF, a gene therapy intended for the treatment of hearing loss due to mutations in the OTOF gene – Through a targeted delivery of a proprietary ancestral AAV, known as AAVAnc80, containing the OTOF gene, Akouos aims to restore otoferlin expression, potentially restoring physiologic hearing and providing long-lasting benefits to individuals with OTOF-mediated hearing loss. Akouos plans to submit an IND application to the FDA in 2021 to conduct a Phase 1/2 clinical trial. The planned Phase 1/2 clinical trial consists of two parts. The first part is a dose escalation phase designed to assess the safety, tolerability and bioactivity of AK-OTOF, administered to trial participants through a single unilateral intracochlear injection. The second part is a cohort expansion phase designed to assess safety and efficacy."
      Source: https://investors.akouos.com/news-r...d-quarter-2020-financial-results-and-provides
       
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    18. FGG
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      Akouos just released these two presentations:

      https://akouos.com/wp-content/uploa...reg-R-Workshop-Presentation_Akouos_FINAL5.pdf

      And

      https://akouos.com/wp-content/uploa...nference-Michelle-V-Presentation_vFINAL25.pdf

      Of note, they discuss their surgical method which I was really curious about. After a surgical approach, they inject directly through the round window and then vent the Stapes through fenestration. Looks interesting and gets their AAV genetic deafness drug through the entire cochlea. They propose that it's safe but would like to see the data (my guess is the Stapes is handled with very slow microdrilling).
       
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    19. wwtsai
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      Would a method like this possibly remedy the issues other firms like Frequency Therapeutics are having with drug delivery? I know everyone's coming up with their own methods, but all of these firms attend/speak at the same conferences every year. It'd be great to see them bouncing ideas and methods off of each other to improve their own products.
       
    20. FGG
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      FGG Member Podcast Patron Benefactor Ambassador Hall of Fame Advocate

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      Theoretically, if only a surgical approach would work, this should work imo.

      AAV drugs have to be applied directly into the cochlea so they can't use an intratympanic approach for genetic deafness gene therapies.
       
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