EP 215: Vectorized RNAi and the next frontier of gene silencing with Rachel Salzman of Armatus Bio
Description
Summary:
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.
Show Notes:
0:00 Intro to The Genetics Podcast
01:00 Welcome to Rachel
01:35 Background of Armatus Bio and the mechanism behind its vectorized RNAi platform
05:31 Advantages of vectorized RNAi over conventional RNAi approaches
08:07 How microRNAs work and how engineered versions enable highly specific gene silencing
10:28 Current preclinical progress at Armatus and next steps toward first-in-human trials
14:06 Lessons on making smart risk decisions in rare disease drug development
17:26 Reflections on two decades of progress, setbacks, and realities in gene therapy
22:07 Hemophilia as a case study in gene therapy missteps and overlooked patient and market realities
25:21 Challenges around product purity and the need for financial innovation
29:23 Why AAV purity is so difficult to achieve and where pre-competitive collaboration could drive improvement
33:32 Rachel’s path from veterinary medicine to gene therapy through a family genetic diagnosis
35:58 Founding the Stop ALD Foundation and advancing lentiviral gene therapy into first-in-human use
39:58 The future potential of vectorized RNAi
42:00 Closing remarks
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