File
| Title | Disorder | Modality | PI/Lead Investigator | Institution |
|---|---|---|---|---|
| IND-enabling studies for Aspartylglucosaminuria (AGU) to support the initiation of an AAV9/AGA gene transfer clinical trial | Aspartylglucosaminuria | Viral vector-based approaches (AAV9/AGA) | Steven J. Gray | University of Texas Southwestern Medical Center |
| Viral gene therapy for Menkes disease | Menkes Disease | Viral vector-based approaches (AAv9/ATP7A) | Stephen Gerard Kaler | The Research Institute at Nationside Childen's Hospital |
| Advancement of Prion Protein-Lowering Divalent siRNA Therapy for Prion Disease | Prion Disease | Oligonucleotide-based approaches (siRNA) | Eric Vallabh Minikel | Broad Institute, Inc. |
| Silence ALS: A Platform for the Discovery and Development of Antisense Therapeutics for Patients with Ultra-Rare Forms of ALS | Amyotrophic Lateral Sclerosis (ALS) | Antisense Oligonucleotides (ASOs) | Neil Alan Shneider | Columbia University Health Sciences |