Edgar Rodriguez

Edgar Rodriguez

Company: Lacerta Therapeutics

Job title: Chief Executive Officer & Co-Founder


Efficacy & Safety of a Tissue-Restricted AAV Vector Encoding Frataxin 11:30 am

Ubiquitous overexpression of the frataxin protein, which is deficient in Friedreich’s ataxia (FA), can lead to toxicity, negating the potential therapeutic benefit of a gene therapy approach to FA Tissue restricted expression of AAV-delivered frataxin is a viable approach to overcoming toxicities and achieve full therapeutic benefit The efficacy and safety of this approach, from…Read more

day: Day Two: Track A AM

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