Keyword: Spark Therapeutics
After steering Spark's Luxturna through FDA approval and launch, COO John Furey is heading Imvax, which is developing a brain cancer vaccine.
After launching in late 2013 with $50 million and a clutch of gene therapies out of Children’s Hospital of Philadelphia, Spark Therapeutics hit the gas. It picked up another $73 million and a Big Pharma partner en route to its upsized IPO in 2015.
The update adds to the impression that the gene therapy is a threat to assets including BioMarin’s valrox and Spark Therapeutics’ SPK-8011.
UniQure is reportedly working with advisers to assess options including a sale or partnership but is yet to decide on which path to take.
An Italian team has developed a viral vector for hemophilia gene therapy and shown in monkeys that it may improve efficacy.
The update suggests FIX activity increases seen at six weeks are sustainable, giving uniQure a boost as it races Spark Therapeutics to market.
After refocusing on gene therapy, Axovant is appointing five new executives to posts ranging from clinical development to commercial operations.
The FIX-Padua therapy achieved FIX activity levels of 23% to 37%, suggesting it is several times more effective than unIQure's old asset.
Spark Therapeutics announced encouraging preclinical data on SPK-3006, its gene therapy to treat the muscle disorder Pompe.
ProQR Therapeutics reported interim phase 1/2 data for its LCA10 treatment showing it improved vision for the majority of patients.