About
Gene Therapy for Rare Disease
Areas of Focus
Categories
Virus TechnologyGene Therapy, RNA & Peptide TherapySan Diego & Southern California
Clinical Trials (4)
View allTotal Trials
4
As Lead Sponsor
3
As Collaborator
1
Total Enrollment
58
By Phase
PHASE3: 1PHASE1/PHASE2: 2
By Status
RECRUITING: 3NOT_YET_RECRUITING: 1
Clinical Trials Pipeline4 trials
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Phase 1/2
Phase 2/3
N/A
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
2032
2033
Today
Hover over trial bars to see details
Recent Trials
NCT06692712Lead Sponsor
Phase 3, Open-label Study to Assess the Efficacy and Safety of a Single Lumbar Intrathecal Administration of MELPIDA in Individuals with Hereditary Spastic Paraplegia Type 50 (SPG50) Versus Matched Prospective Concurrent Controls.
PHASE3Not yet recruiting
24 participants
Started: Mar 1, 2025 · Completed: Jun 1, 2032
NCT06609889Collaborator
A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease
PHASE1/PHASE2Recruiting
10 participants
Started: Dec 3, 2024 · Completed: Oct 31, 2028
NCT06151600Lead Sponsor
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J
N/ARecruiting
20 participants
Started: Jul 29, 2024 · Completed: Mar 1, 2028
NCT05518188Lead Sponsor
Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
PHASE1/PHASE2Recruiting
4 participants
Started: Feb 15, 2023 · Completed: Oct 1, 2030
Locations (1)
Headquarters
Encino, CA, United States
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