Basic Information

Gene symbol ABCD1 Synonyms ABC42, ALD, ALDP, AMN Type of gene protein-coding
Description ATP binding cassette subfamily D member 1

GTO ID GTC3047
Trial ID NCT05394064
Disease Adrenomyeloneuropathy
Altered gene ABCD1
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment SBT101
PhasePhase1|Phase2
Recruitment statusRecruiting
TitleA Phase 1/2 Randomized, Blinded, Dose-escalation Study to Evaluate the Safety and Efficacy of Intrathecal Administration of AAV9-ABCD1 Gene Therapy (SBT101) in Adult Patients With Adrenomyeloneuropathy
Year2022
CountryNetherlands|United States
Company sponsorSwanBio Therapeutics, Inc.
Other ID(s)SBT101-CT101|2021-004410-19
Vector information
Vectoradeno-associated virus
ConstructrAAV9-ABCD1
Vector typerecombinant adeno-associated virus serotype 9 (rAAV9) vector
Transgene/Inserted genea functional copy of the human adenosine triphosphate (ATP)-binding cassette transporter subfamily D member 1 (hABCD1) gene

Clinical Result

Cohort 1
Administration route intrathecal infusion
Age Adult, Older_Adult
References : SwanBio Therapeutics Announces FDA Investigational New Drug Clearance for First AAV-Based Gene Therapy for the Treatment of Adrenomyeloneuropathy

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