Gene therapy: News digest for 7/31/16

Spinal Muscular Atrophy

The FDA has granted a breakthrough therapy designation to AVXS-101 (AveXis, Inc.) for the treatment of spinal muscular atrophy (SMA) type 1 in pediatric patients.

 Brain Cancer (Glioblastoma)

Kingston, Ontario, band and Canadian rock icons the Tragically Hip launched their 15-date summer tour in Victoria, BC, this past weekend, performing in support of their 14th studio album, Man Machine Poem, and starting on what for the group’s fans, the band members themselves and literally all of Canada will surely be an emotion-laden trip …
Researchers show a new strategy, based on targeting specific surface molecules, that eliminates most of the cancer cells in a mouse model of glioblastoma.

Blood Cancer

Spark, Pfizer receive US FDA Breakthrough Therapy status for SPK-9001 to treat haemophilia B

pharmabiz.comSpark Therapeutics and Pfizer Inc. announced that the US Food and Drug Administration (FDA) has granted breakthrough therapy designa…
Read more about Ziopharm Oncology’s Phase 1 adoptive cellular therapy study with CD33 CAR-T

Metabolic Disorders

“We are excited by the promise of Selecta’s immune modulating therapies to enhance gene therapies for rare and serious diseases, like MMA and OTC,” said Werner Cautreels, PhD, Chairman, CEO and President of Selecta Biosciences. “Selecta’s novel approach is important for addressing the immunogenicity related to the administration of gene therapy vectors, and our collaboration agreements with the NHGRI, MEE, and ICGEB are expected to enable the development of these novel treatments.” Selecta’s SVP-Rapamycin (SEL-110) demonstrated in animal models an ability to mitigate the formation of antibodies against gene therapy vectors and lessen inflammatory immune responses against transduced cells. By avoiding these undesired immune responses, SVP-Rapamycin (SEL-110) immunotherapy could have the potential to keep patients eligible for subsequent gene therapy doses, a feature not available with current gene therapies. SEL-212, the combination of SVP-Rapamycin (SEL-110) with pegsiticase, a uricase enzyme


Last week, The US National Institutes of Health (NIH) awarded $30 million to six research collaborations in annual funding to help advance science towards a cure for HIV.

Personalized Medicine
Read how researchers are using animal models to improve understanding and treatment outcomes in ALS with personalized medicine, gene therapy and biomarkers.

Clinical  Trial Mortality

Loss of lives in clinical trials has companies, and investors, proceeding with caution.


A new gene-editing technique with potential to revolutionize cancer treatment has U.S. and Chinese scientists in a race to test it on humans.


Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out /  Change )

Google+ photo

You are commenting using your Google+ account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s