| Scribe Therapeutics | Brett Staahl | A First-in-Class CIRSPR-CasX Gene Editing Therapy for Lowering Lp(a) to Prevent
Cardiovascular Events |
$12,714,480 |
| 4D Molecular Therapeutics, Inc. | Dr Melissa Calton | Gene Therapy for Alpha-1 Anti-Trypsin Deficiency |
$5,915,073 |
| AcuraStem Incorporated | Mr. Samuel V. Alworth MS, MBA | Late-stage development of AS-241, an UNC13A Targeting Antisense Oligonucleotide treatment for Amyotrophic Lateral Sclerosis, for IND-enabling studies |
$7,500,000 |
| Cure Rare Disease | Dr. Tahseen Mozaffar | Advancement of a myotropic, liver-detargeting therapy for LGMD2i/R9 |
$7,292,297 |
| Scribe Therapeutics | Brett Staahl | A first-in-class CRISPR-CasX gene editor silencing APOC3 transcription for the treatment of Severe Hypertriglyceridemia |
$13,000,000 |
| Savanna Biotherapeutics, Inc. | Dr. Sunil Gandhi Ph.D | Microglia replacement therapy for CSF1R-related Leukoencephalopathy |
$12,993,456 |
| Cedars-Sinai Medical Center | Dr. David Joseph Lefer | TY1 and Semaglutide to Treat Cardiometabolic HFpEF |
$10,571,220 |
| University of California, San Diego | Dr. Stephanie Cherqui | CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia |
$7,423,504 |
| Cedars-Sinai Medical Center | Dr. Alessandra Ciullo | Noncoding RNA drug TY2 for arrhythmogenic cardiomyopathy |
$10,419,929 |
| Navega Therapeutics | Ana Maria Moreno | Development of an AAV Epigenetic Gene Therapy for Intractable Chronic Pain Disorders |
$9,486,864 |
| Scripps Health | Dr. Darryl D. D’Lima | Stem Cell-Based Cartilage Tissue Regeneration |
$12,715,000 |
| Stanford University | Dr. Bertha Chen | Autologous iPSC-derived progenitor smooth muscle cells for treatment of urinary incontinence |
$7,275,596 |
