A gene therapy to correct a mutation that causes maple syrup urine disease prevented newborn death and normalized growth in a calf as well as in mice.

The "AAV Vectors in Gene Therapy - Pipeline Insight, 2025" report has been added to ResearchAndMarkets.com's offering.The Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline signifies a ...

In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants ...

As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare diseases.

Ferriero et al. show that liver fibrosis reduces the efficiency of AAV-based gene therapy by limiting liver uptake and altering vector distribution to other organs. The impact of fibrosis varies ...

Detailed price information for Solid Biosciences Inc (SLDB-Q) from The Globe and Mail including charting and trades.

The two deaths occurred in children in Russia and Kazakhstan, with the liver failure starting five to six weeks after administration of the adeno-associated virus-based treatment. Both children ...

Pfizer has ended the global commercialisation of its haemophilia B (factor IX deficiency) gene therapy Beqvez, citing soft demand for gene therapies among haemophilia patients and physicians.

AAV-SLB101, which was rationally designed to target integrin receptors, and has shown enhanced cardiac and skeletal muscle transduction with decreased liver targeting in nonclinical studies.

Ferriero et al. show that liver fibrosis reduces the efficiency of AAV-based gene therapy by limiting liver uptake and altering vector distribution to other organs. The impact of fibrosis varies ...

Reported positive initial data from INSPIRE DUCHENNE trial of next-generation Duchenne gene therapy candidate, SGT-003; In mid-2025, ...

A study led by UMass Chan Medical School researchers has demonstrated that a gene therapy to correct a mutation that causes ...