Clinical Trial Update: Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation [Phase 1, Phase 2]

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Summary

Phase 1/2 trial update for a personalized antisense oligonucleotide therapy targeting ASXL3 in Bainbridge-Ropers Syndrome; currently active but not recruiting.

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Diseases / indications

• Bainbridge-ropers Syndrome

Drugs mentioned

• ASXL3-targeted antisense oligonucleotide (personalized therapy) (antisense oligonucleotide targeting ASXL3)

Snippet

Sponsor: n-Lorem Foundation | Conditions: Bainbridge-Ropers Syndrome | Phase 1, Phase 2 | Status: ACTIVE_NOT_RECRUITING