Sponsor Pipeline

n-Lorem Foundation Clinical Trial Pipeline

Data updated daily from ClinicalTrials.gov — last refresh 2026-08-16
16
Active Trials
0
Recruiting
1
Disease Areas
What Changed in the Last 30 Days
First PostedNew Trial FilingPhaseStatus
2026-08-03 NCT07743268 Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS Phase 1/2 Enrolling by Invitation
Active Trials by Phase
Phase 1/2
16
Active Trials
NCT IDTitleConditionStatus
Phase 1/2
NCT07177196 Personalized Antisense Oligonucleotide Therapy for a Single Participant With PRPH2… Retinal Dystrophy Active, Not Recruiting
NCT07197268 Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene… Bainbridge-Ropers Syndrome Active, Not Recruiting
NCT07197294 Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3… Neurodevelopmental Disorder With or Without Variable Brain Abnormalities Active, Not Recruiting
NCT07226297 Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation… Charcot-Marie-Tooth Disease Type 2D Enrolling by Invitation
NCT07423494 Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS… Amyotrophic Lateral Sclerosis Not Yet Recruiting
NCT07474298 Personalized Antisense Oligonucleotide for A Single Participant With PACS1 Gene Mutation… Schuurs-Hoeijmakers Syndrome Not Yet Recruiting
NCT07221760 Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene… Dentatorubral-Pallidoluysian Atrophy Active, Not Recruiting
NCT06706388 Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene… Dentatorubral-Pallidoluysian Atrophy Active, Not Recruiting
NCT07588581 Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA) Active, Not Recruiting
NCT06392126 Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS Amyotrophic Lateral Sclerosis Active, Not Recruiting
NCT06816498 Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1… Autosomal Dominant Leukodystrophy Active, Not Recruiting
NCT06977451 Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS Amyotrophic Lateral Sclerosis Active, Not Recruiting
NCT07084311 Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation Dentatorubral-Pallidoluysian Atrophy Active, Not Recruiting
NCT07095686 Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS Amyotrophic Lateral Sclerosis Enrolling by Invitation
NCT07703462 Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS Amyotrophic Lateral Sclerosis (ALS) Enrolling by Invitation
NCT07743268 Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS Amyotrophic Lateral Sclerosis (ALS) Enrolling by Invitation
Disease Areas
ALS (6)
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