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FDA Approves Gene Therapy Trial for Rare Neurological Disorder

5/7/2025

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Summary

Story summary

The FDA has greenlit BlackfinBio Limited's groundbreaking Phase 1/2 clinical trial for an innovative AAV gene therapy aimed at Hereditary Spastic Paraplegia Type 47 (SPG47). Set to unfold at Boston Children’s Hospital, this trial seeks to assess the therapy's safety and effectiveness, offering hope for a cure for this rare and debilitating neurological condition.

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Story summary

The FDA has greenlit BlackfinBio Limited's groundbreaking Phase 1/2 clinical trial for an innovative AAV gene therapy aimed at Hereditary Spastic Paraplegia Type 47 (SPG47). Set to unfold at Boston Children’s Hospital, this trial seeks to assess the therapy's safety and effectiveness, offering hope for a cure for this rare and debilitating neurological condition.