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Tern Therapeutics is partnering with Andelyn Biosciences to develop its investigational gene therapy that targets vision loss in children with CLN2 Batten disease, also known as late-infantile Batten disease. Andelyn, a contract development and manufacturing organization specializing in cell and gene therapy, will participate in the…
Gene therapy targeting nerve cells in the gut improved nerve cell survival and intestinal function — specifically that of the bowel — while also increasing survival rates in a mouse model of Batten disease, a new study by researchers at Washington University School of Medicine (WashU Medicine) in St.
Treatment with the investigational therapy Batten-1 (miglustat) for 18 months, or about 1.5 years, stabilized motor symptoms in patients with juvenile Batten disease, according to the final results from a Phase 1/2 trial. The progression of motor symptoms was considerably slowed down, as evaluated using the modified Unified Batten Disease…
Theranexus has launched a global offering of around €4 million (about $4.5 million) to help fund the pivotal Phase 3 trial of the company’s investigational therapy Batten-1 for juvenile Batten disease. The Beyond Batten Disease Foundation (BBDF) acquired €1.15 million (about $1.3 million) in shares before the…
Treatment for up to six months with Batten-1 — Theranexus’ novel therapy for juvenile Batten disease — reduced nerve cell death and stabilized patients’ motor symptoms. That’s according to preliminary results from an ongoing Phase 1/2 clinical trial testing the experimental therapy. The results will be presented at the…
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