| Phase 1/2 |
| NCT07743268 |
Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS |
Amyotrophic Lateral Sclerosis (ALS) |
Enrolling by Invitation |
2026-08-03 |
| NCT07703462 |
Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS |
Amyotrophic Lateral Sclerosis (ALS) |
Enrolling by Invitation |
2026-07-14 |
| NCT06392126 |
Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS |
Amyotrophic Lateral Sclerosis |
Active, Not Recruiting |
2026-07-13 |
| NCT06816498 |
Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1… |
Autosomal Dominant Leukodystrophy |
Active, Not Recruiting |
2026-07-13 |
| NCT06977451 |
Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS |
Amyotrophic Lateral Sclerosis |
Active, Not Recruiting |
2026-07-13 |
| NCT07084311 |
Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation |
Dentatorubral-Pallidoluysian Atrophy |
Active, Not Recruiting |
2026-07-13 |
| NCT07095686 |
Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS |
Amyotrophic Lateral Sclerosis |
Enrolling by Invitation |
2026-07-13 |
| NCT07588581 |
Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation |
Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA) |
Active, Not Recruiting |
2026-05-15 |
| NCT06706388 |
Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene… |
Dentatorubral-Pallidoluysian Atrophy |
Active, Not Recruiting |
2026-04-09 |
| NCT07221760 |
Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene… |
Dentatorubral-Pallidoluysian Atrophy |
Active, Not Recruiting |
2026-04-07 |
| NCT07474298 |
Personalized Antisense Oligonucleotide for A Single Participant With PACS1 Gene Mutation… |
Schuurs-Hoeijmakers Syndrome |
Not Yet Recruiting |
2026-03-16 |
| NCT07423494 |
Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS… |
Amyotrophic Lateral Sclerosis |
Not Yet Recruiting |
2026-02-20 |
| NCT07226297 |
Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation… |
Charcot-Marie-Tooth Disease Type 2D |
Enrolling by Invitation |
2025-11-10 |
| NCT07197268 |
Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene… |
Bainbridge-Ropers Syndrome |
Active, Not Recruiting |
2025-09-29 |
| NCT07197294 |
Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3… |
Neurodevelopmental Disorder With or Without Variable Brain Abnormalities |
Active, Not Recruiting |
2025-09-29 |
| NCT07177196 |
Personalized Antisense Oligonucleotide Therapy for a Single Participant With PRPH2… |
Retinal Dystrophy |
Active, Not Recruiting |
2025-09-16 |