News

A single intravitreal injection — into the vitreous cavity, in the back of the eye — of a gene therapy was found to preserve the function and structure of the retina in a dog model of late infantile Batten disease. Eye inflammation was…

Treatment for up to two months with Batten-1 — Theranexus’ novel therapy for juvenile Batten disease, also known as CLN3 disease — was found to be generally safe and well-tolerated in patients ages 17 and older. That’s according to early data from an ongoing two-year Phase 1/2…

Alterations in producing, transporting, and degrading proteins, as well as in neuronal activity, are likely involved in juvenile Batten disease, according to data from lab-grown neurons from a patient with the most common disease-causing mutation. These findings point to previously unknown mechanisms that may lead to neurodegeneration among patients…

Both of Regenxbio‘s gene therapy candidates for neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as late infantile Batten disease, have advanced to in-human testing, the company reported. These clinical trials will evaluate the one-time experimental gene therapies in patients with the inherited disease, which typically occurs…

Researchers have proposed an alternate classification model for Batten disease types based on the site first affected by each type and how it progresses. They tested their hypothesis in two of the most common Batten types. A comprehensive analysis of published data so far showed infantile Batten disease…

Female mice with Batten disease caused by a mutation in the Cln8 gene had greater disease severity, including worse behavioral deficits and a shorter lifespan, than their male counterparts, a study has found. These female mice also had more activation in the brain of microglia, a type of cell that drives…

Researchers described the natural history of retinal degeneration in a mouse model of CLN7, a form of late-infantile Batten disease, that mimics human patient retinal disease progression. The study revealed a progressive loss or dysfunction of various types of nerve cells within the retina that relay signals from the…

A newborn with microcephaly (unusually small head) and difficult-to-treat seizures was diagnosed with CLN10 disease, or congenital Batten disease, after a genetic analysis confirmed a mutation in the CTSD gene, a case study reports. Researchers stressed the importance of offering genetic testing and counseling to families at increased risk of…

Theranexus and Inserm Transfert have entered an agreement to work toward the development of treatment candidates for neurological disorders such as Batten disease, the two companies announced. “This flagship agreement is perfectly in line with our strategy to develop medicinal products blocking the progression of rare neurological diseases…

Levels of fatty molecules called glycerophosphodiesters, or GPDs, are elevated in the blood and other bodily fluids of people with juvenile Batten disease — and may be a useful biomarker for diagnosing or treating the inherited disorder — a new study revealed. This finding “raises the potential to use…