Keyword: Spark Therapeutics
The round sets up the JLABS resident to use advances in synthetic biology to create more targeted, controllable cell and gene therapies.
Just over a week ago, Spark Therapeutics was riding high on positive data for its hemophilia B gene therapy. It’s now fallen back to earth after spooking investors with the results of a trial in hemophilia A.
Patients with hemophilia B have seen their symptoms controlled for up to a year with a single dose of a gene therapy developed by Spark Therapeutics, almost entirely eliminating the need for treatment with clotting factors.
The mid-development tweaks have the blessing of regulators, enabling uniQure to start a pivotal trial next year with its fast-track statuses intact.
After decades of research, a gene therapy to correct a specific genetic mutation is on the brink of being approved by the FDA.
Spark Therapeutics’ inherited eye disorder treatment voretigene neparvovec faces an FDA advisory committee this week.
Allergan CFO Hilado and Gilead COO Young will retire from their positions and Rubius has poached a GSK SVP as CMO.
Nightstar will complete a phase 3 trial of its choroideremia gene therapy and advance two other eye disease candidates through early-stage studies.
The takeover will see Regenxbio pay about $86 million to add two of early-stage gene therapies to its pipeline.
The split gives uniQure full rights to AMT-060 but leaves it without a partner to cofund R&D as it closes in on the start of a pivotal trial.