Medical Science 6

Antisense (How we do it at n-Lorem)

September 21, 2022

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How we make the impossible possible

We’d like to thank our sponsor, Argonaut Manufacturing Services. Argonaut provides contract manufacturing and sterile fill-finish services for biopharmaceutical, diagnostics, and life science organizations. Argonaut’s expertise in sterile fill-finish is the last step in the complex process of providing personalized antisense oligonucleotide (ASO) medicines to nano-rare patients.

When considering if the technology he led the creation of could be a viable way to develop a drug for a single patient, addressing their specific mutation, Dr. Crooke felt that he had to try, patients were dying without access to any treatment because they were just too rare. So began n-Lorem. As expected, developing an optimal ASO for a single patient is not an easy or simple process. Quality must lead every step and only optimal ASOs can reach patients.

Learn our science and processes behind the discovery and development of personalized ASO medicines for each of our nano-rare patients. n-Lorem’s mission is to provide hope and potential help to nano-rare patients – for free, for life; a mission that was impossible just a few years ago. This enables the non-profit approach that we believe is the only way to address the needs of nano-rare patients. Thanks to our partners like Argonaut, we are able to provide hope and potential help to patients today and tomorrow.

On This Episode We Discuss:

  • The difficulty of ASO site screening and how we make it quick
  • How we use our databases to predict how the next ASO will behave
  • Submitting an application to n-Lorem
  • The n-Lorem Access to Treatment Committee
  • Discovering and developing ASOs under FDA guidance
  • What problems can arise?
  • Our promise

Watch on YouTube:

The host of the show is Dr. Stanley Crooke, a scientist, a physician, an entrepreneur and the father of antisense technology. Dr. Crooke is responsible for driving the development of antisense or ASO technology, an RNA-targeted technology responsible for the commercialization of three best- and first-in class medicines and more than 40 drugs in development. In 2020, Stan formed n-Lorem to use this powerful technology to develop experimental personalized ASO medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life.

Credits

Hosted by: Dr. Stan Crooke.
Videographer: Jon Magnuson of Mighty One Productions.
Producers: Kim Butler, Colin Delaney, Kira Dineen, Jon Magnuson, Andrew Serrano and Amy Williford

More from n-Lorem:

See what else we are up to on Twitter, Instagram, Facebook, LinkedIn, YouTube and our website, nlorem.org. If you enjoy this episode, please rate and review us, as it helps others find our podcast. Questions/inquiries can be sent to podcast@nlorem.org.

Download Script:

For more information on the covered topics or to follow along with this Intro to Medical Science episode, download the supporting transcript.

Script

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Cardiovascular System Part 1: Blood

We cannot do
this alone

Together we are changing the world—
one patient at a time

We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.

We need your support

Join us on our Corps of Discovery of the mind and heart. Help us bring hope and potential help to nano-rare patients today. For free, for life.
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