Business Wire

Updated Preclinical Data Support Potential of First AAV-Based Gene Therapy as a Treatment for Adrenomyeloneuropathy

PHILADELPHIA--(BUSINESS WIRE)--SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, today presented updated ...
Business Wire

SwanBio Therapeutics Announces New Preclinical Data Demonstrating Proof of Principle for SBT101 Gene therapy as a Potential Treatment of Adrenomyeloneuropathy

PHILADELPHIA--(BUSINESS WIRE)--SwanBio Therapeutics, a gene therapy company focused on developing treatments for patients with genetically defined neurological conditions, today announced new ...
Medscape

Adrenoleukodystrophy: Recent Advances in Treatment and Disease Etiology

X-linked adrenoleukodystrophy (X-ALD) is a debilitating disease caused by mutations in the ABCD1 gene. Approximately 30% of affected boys develop the progressive ...
Monthly Prescribing Reference

Skysona Gets Accelerated Approval for Early, Active Cerebral Adrenoleukodystrophy

Skysona is a one-time gene therapy designed to add functional copies of the ABCD1 cDNA into a patient’s own hematopoietic stem cells, resulting in the production of functional ALDP. Treatment with ...
Yahoo Finance

Spur Therapeutics Publishes Preclinical Proof-of-Concept Data for SBT101, its Gene Therapy Candidate in Adrenomyeloneuropathy (AMN)

LONDON, Oct. 31, 2024 (GLOBE NEWSWIRE) -- Spur Therapeutics today announced the publication of preclinical proof-of-concept data for SBT101, its potential first-in-class gene therapy program for the ...
Wired

Gene Therapy Halts Fatal Brain Disease

Scientists have used gene therapy to halt the progression of adrenoleukodystrophy, a fatal neurodegenerative disease caused by a single defective gene, in two seven-year-old boys. It took more than a ...