Dutch gene therapy maker uniQure has announced a strategic reorganization that will cut both its workforce and its research and technology investments.
The biotech is looking to cut operating expenses while locking in resources to advance its prioritized clinical-stage programs as rapidly as possible to proof-of-concept, shared uniQure CEO Matt Kapusta. uniQure will discontinue more than half of its research and technology projects in order to focus R&D efforts on programs that leverage the company's CNS and liver-targeted gene therapy expertise.
More specifically, uniQure plans to focus resources on four clinical-stage programs:
-AMT-130 for the treatment of Huntington’s disease
-AMT-260 for the treatment of refractory mesial temporal lobe epilepsy
-AMT-162 for the treatment of SOD1-ALS, a rare form of ALS
-AMT-191 in Fabry disease
The reorg will also involve the elimination of 114 positions, which represents approximately 20% of the total workforce and 28% of the workforce not committed to Hemgenix.
Hemgenix, approved last November, is the first gene therapy for adults with Hemophilia B. It is also one ofthe most expensive drugs in the world, priced at $3.5 million per dose. uniQure sold the rights to Australian drugmaker CSL Behring in 2020 for $450 million, while the drug was still in phase 3 trials, but the deal stipulated that uniQure would complete the phase 3 trial and scale up manufacture for early commercial supply.
uniQure's reorg plan — which will bring cost savings of $180 million to extend cash runway into the second quarter of 2027 — will also consolidate all GMP manufacturing into the company's Lexington, MA manufacturing facility. However, according to uniQure, commercial manufacturing of Hemgenix for CSL Behring will be unaffected by these actions.
