Now Available: Breakthroughs in Muscular Dystrophy Abstracts

Read about the latest advancements in CGTs for muscular dystrophy from our collection of more than 60 pieces of original research! The collection is now available to download via a PDF and to sort via an Excel file on ASGCT.org.

View the abstracts PDF     Sort the abstracts via Excel     View plain language summaries

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Check out abstracts highlighted below!

• Oral abstracts will be presented 4-5:45 p.m. on Tuesday, Nov. 19.

• Poster abstracts will be presented concurrently in two rooms 5:45-7 p.m. on Tuesday, Nov. 19.

Oral Abstracts

Tuesday, Nov. 19, 4 – 5:45 p.m. CT

1. Editing of Postmitotic Muscle and Satellite Cells for Continuous Correction of Dystrophin Expression in Duchenne Muscular Dystrophy
Niclas Bengtsson, PhD, University of Washington

2. CSF Delivery of INS1201 AAV9-Micro-Dystrophin as a Potential Therapy for DMD
Gretchen Thomsen, PhD, Insmed, Inc.

3. Therapeutic Potential of ENTR-601-44, an Endosomal Escape Vehicle (EEVTM) - Oligonucleotide Conjugate for the Treatment of Exon 44 skip Amenable DMD
Mahasweta Girgenrath, PhD, Entrada Therapeutics Inc

4. Development of Gene Therapy for LGMD-R9
Evelyne Gicquel, PhD, Genethon

6. GNT0004, Genethon's AAV8 Vector-delivered Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy: First Data from Phase 1/2 Part of GNT-016-MDYF All-in-one Clinical Trial in Ambulant Boys
Serge Braun, PhD, AFM TELETHON / GENETHON

58. RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data
Jahannaz Dastgir, DO, REGENXBIO Inc.

5. The Venture Philanthropy Model of Drug Development - Working with Non-Profit Foundations and Governments from Concept to Drug Approval
Eric Hoffman, PhD, Binghamton University

View the abstracts PDF     Sort the abstracts via Excel     View plain language summaries

Register for #ASGCTBreakthroughs24