Gene therapy now consistently and effectively treating hemophilia

blood cells by RATusus CC BY e

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion and analysis.

Bill Maurits has hemophilia B, which means his body doesn’t produce enough factor IX, a protein that clots blood. He’s at risk for bleeding and his joints are damaged from all the bruises.

In April, Maurits, an engineering designer, joined a study in which he was dosed with viruses packed with a correct version of the gene that codes for factor IX. On June 11 at the European Hematology Association’s meeting in Copenhagen, the Philadelphia company that ran the gene-therapy study, Spark Therapeutics, is presenting results on four patients, him included.

In all four, factor IX activity has reached about 30 percent of average. That’s enough to prevent bleeding when you get hit by a baseball or twist your ankle. It’s also been enough to let Maurits go without factor IX replacements since April. “There’s no other explanation than ‘It worked,’” says Maurits.

Sure, gene therapy has been tried before. What’s different is that Spark’s therapy so far appears to work well every time it’s attempted—a consistency that’s eluded previous efforts.

Read full, original post: Gene Therapy Is Curing Hemophilia

Outbreak
Outbreak Daily Digest
Biotech Facts & Fallacies
Talking Biotech
Genetics Unzipped
Infographic: What are mRNA COVID-19 vaccines and how do they work?

Infographic: What are mRNA COVID-19 vaccines and how do they work?

As of 1 December 2020, thirteen vaccines have reached the final stage of testing: where they are being given to ...
favicon

Environmental Working Group: EWG challenges safety of GMOs, food pesticide residues

Known by some as the "Environmental Worrying Group," EWG lobbies ...
m hansen

Michael Hansen: Architect of Consumers Union ongoing anti-GMO campaign

Michael K. Hansen (born 1956) is thought by critics to be ...
News on human & agricultural genetics and biotechnology delivered to your inbox.
glp menu logo outlined

Newsletter Subscription

Optional. Mail on special occasions.
Send this to a friend