Gene Therapy News

Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, and Synpromics Limited, a leading synthetic promoter development company backed by Calculus Capital, today announced they have entered into a broad, multi-target collaboration agreement.

Researchers have taken a significant step forward in developing gene therapy against a fatal neurodegenerative disease that strikes children. By delivering a working version of a gene to produce a key enzyme that is lacking in Batten disease, the scientists delayed symptoms and extended lifespan in dogs with a comparable disease.

The paper appears online today in Science Translational Medicine.

Researchers found inserting genetic material into intestinal cells to fix the disorder causing cystic fibrosis was effective in human cell cultures and mice, according to a new study.

Cystic fibrosis, which has no cure, is caused by mutations of the CFTR gene. The mutations cause cell proteins that channel chloride ions and water out of cells to malfunction, leading to the build-up of thick mucus that can clog airways or the gastrointestinal tract.