Gene therapy for spinal muscular atrophy secures funding to begin clinical trials

A Dallas biotech startup has raised money to begin clinical trials for what it hopes could be a treatment for the top genetic cause of infant mortality. AveXis is developing a viral gene therapy for infants with spinal muscular atrophy. With a new round of funding led by PBM Capital, the company is hoping to […]

A Dallas biotech startup has raised money to begin clinical trials for what it hopes could be a treatment for the top genetic cause of infant mortality.

AveXis is developing a viral gene therapy for infants with spinal muscular atrophy. With a new round of funding led by PBM Capital, the company is hoping to begin a Phase 1 clinical trial in SMA patients before the end of the second quarter. CEO John Carbona declined to disclose the size of the round but said it will “greatly assist us for the remainder of 2014.”

SMA is a degenerative disease caused by a defect in the SMN1 gene, which codes a protein necessary for the survival of motor neurons. Degeneration of those neurons in the spinal cord and brain stem cause weakness in muscles that control head, neck and limb movement as well as breathing and swallowing.

The company’s approach is to deliver functioning copies of the SMN gene to cells so they can produce adequate SMN proteins. It does that using a viral vector licensed from the company ReGenX.

The upcoming trial will enroll nine infants with type 1 SMA, which occurs when symptoms appear before a child reaches 6 months of age, at The Research Institute at Nationwide Children’s Hospital, the company said.

In most cases, children with type 1 die within a few years from complications. Types 2, 3 and 4 are characterized by later onset but also carry shorter life expectancies.

No treatments currently exist for SMA, which affects about one in every 6,000 babies. Instead, the approach is to manage symptoms and prevent complications.

But a number of companies are working alongside AveXis toward this market. French pharmaceutical company Trophos recently completed a pivotal trial of its small molecule drug in Europe, and Isis Pharmaceuticals is right behind in Phase 2 development of an SMA drug. PTC Therapeutics/Roche and Pfizer are also in the game.

Four-year-old AveXis got fast-track designation for its candidate from the FDA last fall.

And if you’re wondering about the name, AveXis says it comes from letters from the words adeno-associated virus, vector, helix and Isis, goddess of motherhood.

[Image credit: Flickr user Lisa Williams]