AVROBIO Announces Preclinical Gene Therapy Data for Pompe Disease at American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

May 18, 2022

AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced preclinical data demonstrating the efficacy and safety of AVR-RD-03 gene therapy in a mouse model of infantile onset Pompe disease. The results, which will be presented today during the “Disease models and Clinical Applications: Musculo-skeletal Diseases” poster session at the American Society of Gene and Cell Therapy (ASGCT) annual meeting in Washington, D.C., demonstrate that a gene therapy using hematopoietic stem cells (HSC) significantly reduced toxic accumulation of glycogen in a mouse model of Pompe disease, including in cardiac and skeletal muscle as well as the central nervous system (CNS). Eight months post infusion, substrate levels in multiple treated tissues were nearly indistinguishable from normal mice.

Research Tools

All-In-One Screener Stock Ideas Stock List Guru List Guru Real-Time Picks Insider List Insider Trades Economic Indicators Sector & Industry Performance DCF Calculator Discussion Board

Product

Pricing Plans Excel Add-In Google Sheets Add-on Data API Stock Comparison Table Manual of Stocks Mobile App 中文

Education

Financial Glossary Tutorials FAQ Schedule Free Session Buffett Indicator Shiller P/E Yield Curve Today U.S. Inflation Rate Global Market Valuation Fed Net Liquidity Buffett Assets Allocation

Company

About GuruFocus Career Contact Us ETF Advertise Site Map Term of Use Privacy Policy Referral Program Partner Program

Survey

We'd love to learn more about your experiences on GuruFocus.com and how we can improve!

Take Survey

Disclaimers

GuruFocus.com is not operated by a broker or a dealer. It has an affiliated registered investment adviser, which serves as the subadviser to an exchange traded fund. This investment adviser does not provide advice to individual investors. Under no circumstances does any information posted on GuruFocus.com represent a recommendation to buy or sell a security. The information on this site, and in its related newsletters, is not intended to be, nor does it constitute investment advice or recommendations. The individuals or entities selected as "gurus" may buy and sell securities before and after any particular article and report and information herein is published, with respect to the securities discussed in any article and report posted herein. Gurus may be added or dropped from the GuruFocus site at any time. In no event shall GuruFocus.com be liable to any member, guest or third party for any damages of any kind arising out of the use of any content or other material published or available on GuruFocus.com, or relating to the use of, or inability to use, GuruFocus.com or any content, including, without limitation, any investment losses, lost profits, lost opportunity, special, incidental, indirect, consequential or punitive damages. Past performance is a poor indicator of future performance. The information on this site, and in its related newsletters, is not intended to be, nor does it constitute investment advice or recommendations. The information on this site is in no way guaranteed for completeness, accuracy or in any other way. The gurus listed in this website are not affiliated with GuruFocus.com, LLC. Stock quotes are provided by QuoteMedia, Inc. (CSI). Company fundamental data is provided by Morningstar. Analyst estimates data is sourced from both Refinitiv and Morningstar, with priority given to Refinitiv data. Data is updated daily.