Gene therapy upstart Audentes bags a new program in buyout

Late last year San Francisco-based gene therapy upstart Audentes Therapeutics gathered a $42.5 million venture round from some A-list investors with plans to use the money to bankroll some new deals. Today, it made good on the plan and pulled back the covers from a buyout that gives the little biotech player a third preclinical program as it tackles a hot field.

Audentes says it has completed a deal to acquire Cardiogen Sciences, grabbing a lead program for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia (CASQ2-CPVT) with plans to hustle it into clinical development.

CASQ2 encodes a protein called calsequestrin 2, which Audentes says plays a key role "in the physiology of calcium release in cardiac muscle cells, and which is required to maintain normal heart rhythm."

That makes it a prime target for gene therapy, using an adeno-associated viral vector (AAV) to deliver a corrective gene to fix the mutation that triggers the disease. As of now, there are only some 6,000-plus diagnosed patients, without any treatment to turn to, though Audentes believes that number doesn't reflect the fact that the illness is often misdiagnosed.

There's no word in the release what Cardiogen is being sold for. But it's relying on mouse model results--one treatment cured the rodents--to prove its case for now, which would limit any cash consideration.

Gene therapy has been sizzling hot for the past couple of years. Pioneers like bluebird ($BLUE) helped set the pace, raising significant sums and inspiring more startups like Spark ($ONCE). Deerfield Management led the latest B-round financing for Audentes, with participation from Sofinnova Ventures and Venrock, two stalwarts in the industry. Current investors OrbiMed, 5AM Ventures and Versant Ventures also participated. 

"Expanding our portfolio through this acquisition aligns strongly with our strategy of developing AAV gene therapies for serious, rare diseases that have compelling pre-clinical proof-of-concept data and the potential for accelerated clinical development," says Matthew Patterson, the CEO at Audentes. Cardiogen is a fledgling, founded just last year by Dr. Louis Lange to translate the scientific discoveries of Dr. Silvia Priori and her team at the Fondazione Salvatore Maugeri in Italy.

Audentes already has two other preclinical candidates in the pipeline: AT001 for X-linked myotubular myopathy and AT002 for Pompe disease.

- here's the release