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).
Apertura Gene Therapy has entered into a cooperative research and development agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development and the National Human Genome Research Institute.
Atsena Therapeutics Inc. has selected a lead clinical candidate for ATSN-401, a gene therapy for Stargardt disease. ATSN-401 is now advancing in IND-enabling studies.
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.
Opus Genetics Inc. has secured U.S. FDA alignment on an eight-patient phase III trial of its lead gene therapy, OPGx-LCA5, for an ultra-rare form of inherited childhood blindness.
The gene therapy specialist formerly known as EG427 has a new name and fresh momentum, after closing a €33 million (US$37.7 million) series C that will further advance clinical development of the lead program in chronic neuro-urology indications.
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.
With more and more approved products, cell and gene therapies (CGTs) are moving from the initial stronghold in academic medical centers, and these complex biologics are now available across a wider network of treatment centers.
Spinal cord traumatic injury can lead to loss of motor function and progressive development of muscle spasticity and rigidity. Researchers from the University of California San Diego and collaborating institutions investigated a novel gene-delivery-based antispasticity strategy.
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