Author Archives: Patricia Inacio PhD

Amicus Discontinues Gene Therapy Program for CLN6 Batten Disease

Amicus Therapeutics announced it will discontinue its investigational gene therapy program of AT-GTX-501 for late infantile neuronal ceroid lipofuscinosis 6 disease, also known as CLN6 Batten disease. The company is, however, advancing its gene therapy program for juvenile Batten disease, also known as CLN3 disease, according to a press release.

Rarebase Launches Collaborative Platform to Find New Therapies

Rarebase, a public benefit biotechnology company, has launched the Function therapeutic discovery platform specifically for rare neurological diseases, including Batten disease. Led by scientists, engineers, patients, and advocates, Function combines large-scale therapeutic screening with genetic screens, all conducted in nerve cells (neurons) grown in a dish. The combination…