<p><strong>Summary: </strong></p><p>This week on The Genetics Podcast, Patrick is joined by Dr. Rachel Salzman, CEO of Armatus Bio. They discuss the promise of vectorized RNAi for autosomal dominant diseases, the key scientific and clinical hurdles in gene therapy, and Rachel’s lessons from two decades in the field.</p><p><strong>Show Notes: </strong></p><p><strong>0:00 </strong>Intro to The Genetics Podcast</p><p><strong>01:00</strong> Welcome to Rachel</p><p><strong>01:35</strong> Background of Armatus Bio and the mechanism behind its vectorized RNAi platform</p><p><strong>05:31</strong> Advantages of vectorized RNAi over conventional RNAi approaches</p><p><strong>08:07 </strong>How microRNAs work and how engineered versions enable highly specific gene silencing</p><p><strong>10:28 </strong>Current preclinical progress at Armatus and next steps toward first-in-human trials</p><p><strong>14:06 </strong>Lessons on making smart risk decisions in rare disease drug development</p><p><strong>17:26 </strong>Reflections on two decades of progress, setbacks, and realities in gene therapy</p><p><strong>22:07 </strong>Hemophilia as a case study in gene therapy missteps and overlooked patient and market realities</p><p><strong>25:21 </strong>Challenges around product purity and the need for financial innovation</p><p><strong>29:23 </strong>Why AAV purity is so difficult to achieve and where pre-competitive collaboration could drive improvement</p><p><strong>33:32 </strong>Rachel’s path from veterinary medicine to gene therapy through a family genetic diagnosis</p><p><strong>35:58 </strong>Founding the Stop ALD Foundation and advancing lentiviral gene therapy into first-in-human use</p><p><strong>39:58 </strong>The future potential of vectorized RNAi </p><p><strong>42:00 </strong>Closing remarks</p><p>Please consider rating and reviewing us on your chosen podcast listening platform! </p><p><a href="https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link"><u>https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link</u></a><br></p>

The Genetics Podcast

Sano Genetics

EP 215: Vectorized RNAi and the next frontier of gene silencing with Rachel Salzman of Armatus Bio

NOV 27, 202543 MIN
The Genetics Podcast

EP 215: Vectorized RNAi and the next frontier of gene silencing with Rachel Salzman of Armatus Bio

NOV 27, 202543 MIN

Description

<p><strong>Summary: </strong></p><p>This week on The Genetics Podcast, Patrick is joined by Dr. Rachel Salzman, CEO of Armatus Bio. They discuss the promise of vectorized RNAi for autosomal dominant diseases, the key scientific and clinical hurdles in gene therapy, and Rachel’s lessons from two decades in the field.</p><p><strong>Show Notes: </strong></p><p><strong>0:00 </strong>Intro to The Genetics Podcast</p><p><strong>01:00</strong> Welcome to Rachel</p><p><strong>01:35</strong> Background of Armatus Bio and the mechanism behind its vectorized RNAi platform</p><p><strong>05:31</strong> Advantages of vectorized RNAi over conventional RNAi approaches</p><p><strong>08:07 </strong>How microRNAs work and how engineered versions enable highly specific gene silencing</p><p><strong>10:28 </strong>Current preclinical progress at Armatus and next steps toward first-in-human trials</p><p><strong>14:06 </strong>Lessons on making smart risk decisions in rare disease drug development</p><p><strong>17:26 </strong>Reflections on two decades of progress, setbacks, and realities in gene therapy</p><p><strong>22:07 </strong>Hemophilia as a case study in gene therapy missteps and overlooked patient and market realities</p><p><strong>25:21 </strong>Challenges around product purity and the need for financial innovation</p><p><strong>29:23 </strong>Why AAV purity is so difficult to achieve and where pre-competitive collaboration could drive improvement</p><p><strong>33:32 </strong>Rachel’s path from veterinary medicine to gene therapy through a family genetic diagnosis</p><p><strong>35:58 </strong>Founding the Stop ALD Foundation and advancing lentiviral gene therapy into first-in-human use</p><p><strong>39:58 </strong>The future potential of vectorized RNAi </p><p><strong>42:00 </strong>Closing remarks</p><p>Please consider rating and reviewing us on your chosen podcast listening platform! </p><p><a href="https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link"><u>https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link</u></a><br></p>