Gene Therapy: One Down, But One to Go

Roche is discontinuing its investigational hemophilia A gene therapy called SPK-8016. (SPK refers to Spark Therapeutics, which is carrying out trials) But clinical trials for SPK-8011 are still underway. SPK-8011 aims to deliver a healthy version of the gene that provides the instructions for producing functional factor VIII. SPK-8011, also known as dirloctogene samoparvovec, has been tested previously in a clinical trial to assess its safety and efficacy. Data showed an increased factor VIII activity in 21 of 23 patients, with an average bleeding rates decreased by more than 90% after up to five years of follow-up. SPK-8011 is now moving into Phase 3 testing, according to Roche. For more info click here.

Source: Laurie Kelley, Communique, August 2023