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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