Apertura and NIH institutes to test gene therapy for NPC1

Apertura has entered a CRADA with various NIH institutes for a gene therapy to treat Niemann-Pick Disease Type C1 (NPC1). The Ara Parseghian Medical Research Fund will provide financial backing for the multi-year research effort. Under the agreement, researchers plan to use Apertura’s TfR1 CapX adeno-associated virus capsid. Credit: H_Ko / Shutterstock.com. Apertura has entered a cooperative research and development agreement (CRADA) with various NIH institutes for a gene therapy to treat Niemann-Pick Disease Type C1 (NPC1). The company signed the agreement to test the investigational therapy with Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)…

Source: Pharmaceutical Technology