BioWorld - Thursday, July 16, 2026

Gene therapy, BioWorld Science
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Illustration of human brain and dna
Neurology/psychiatric

Voyager’s gene therapy reduces tau levels in AD models

July 15, 2026
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At the Alzheimer’s Association International Conference, researchers from Voyager Therapeutics Inc. presented preclinical efficacy data for VY-1706, a blood-brain barrier-penetrant AAV9 gene therapy designed to reduce tau levels in models of Alzheimer’s disease (AD).
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Drug design, drug delivery & technologies

ARPA-H program supports advances for rare genetic diseases

July 10, 2026
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The Advanced Research Projects Agency for Health (ARPA-H), an agency within the U.S. Department of Health and Human Services, has announced the teams for the THRIVE (Treating Hereditary Rare diseases with In Vivo prEcision genetic medicines) program. With a commitment of up to $160 million over 5 years, THRIVE aims to accelerate solutions for rare genetic pediatric diseases across multiple technological approaches, clinical trial designs and deployment models.
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Art concept for gene therapy research
Genetic/congenital

AAVOlig001-ARSA shows promise in preclinical MLD study

July 7, 2026
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Metachromatic leukodystrophy (MLD) is a rare inherited lysosomal storage disorder characterized by progressive neurodegeneration resulting from loss of arylsulfatase A (ARSA) activity. Researchers at Kazan Federal University reported preclinical efficacy data for a gene therapy candidate in a porcine model of MLD.
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3D rendering of adeno-associated viral vector
Ocular

CSF-delivered AAV-based gene therapy rescues ocular symptoms in model of Tay-Sachs disease

June 23, 2026
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Deficiencies of the enzyme β-N-acetylhexosaminidase (Hex) cause rare, autosomal recessive, fatal, neurodegenerative lysosomal storage disorders called GM2 gangliosidoses, including Tay-Sachs disease (TSD) and Sandhoff disease. Hex enzyme is a heterodimer encoded by HEXA (α subunit) and HEXB (β subunit), whose mutations result in TSD and Sandhoff disease, respectively.
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