Roche backs AveXis in a $65M round for SMA gene therapy

Gene therapy biotech AveXis raised $65 million in Series D cash to advance a potential one-time treatment for the rare spinal muscular atrophy, a debilitating disease with no approved therapies.

The round, led by T. Rowe Price-managed funds, brought back previous investors Deerfield Management, Roche ($RHHBY) Venture Fund and Venrock, joining new backers including Janus Capital, RA Capital and Foresite Capital.

With the proceeds, AveXis plans to focus on scAAV9.CB.SMN, a Phase I gene therapy for SMA Type 1. The disease results from a deficiency in the SMN protein, leading to the destruction of motor neurons and gradual disability. AveXis' treatment, armed with technology licensed from ReGenX, is designed to deliver a corrective copy of the gene responsible for SMN production, using a harmless virus to fix the defect at the heart of SMA.

For AveXis, the latest fundraise follows a $10 million C round closed in January, led by Deerfield and Roche. And the company believes its growing investor syndicate serves as an affirmation of its pipeline promise, CEO Sean Nolan said.

"This points to the broad enthusiasm surrounding our SMA clinical program and the progress we have made to date," Nolan said in a statement. "We are now well-positioned to continue development of our novel gene therapy for patients suffering from SMA Type 1, a rare genetic disease and the most common genetic cause of infant mortality."

Roche Venture Fund's investment follows its parent company's sweeping approach to SMA, including a 2011 partnership deal with PTC Therapeutics ($PTCT) worth up to $490 million and a January buyout of Trophos for as much as $545 million.

Elsewhere in SMA, Isis Pharmaceuticals ($ISIS) posted an encouraging update from its ongoing midstage program earlier this summer, pressing forward with a Biogen ($BIIB)-partnered treatment. And Pfizer ($PFE) nixed a $70 million collaboration with Repligen ($RGEN) in January, handing back an early-stage SMA treatment and an undisclosed number of preclinical candidates.

There are currently no approved treatments for the disease, which affects between one in 6,000 to one in 10,000 children around the world.

- read the statement