Biotech Companies Race to Bring Gene Therapies to Market, While States are too Slow to Adopt

(Credit: Sgt. Eric T. Sheler/ U.S. Air Force.  Wikimedia Commons)

Children born with spinal muscular atrophy, a rare muscle-wasting disease, would probably not be alive today if it wasn’t for an experimental treatment that tweaked their genes shortly after they were born. 

According to MIT Technology Review, many of the children can now both speak and sit on their own, and some are even walking. This wouldn’t be possible if they had not received treatment. 

Biotech company AveXis, who’s behind the revolutionary drug, is seeing best results in those who are treated at the earliest stage of life. This means that it’s critical to identify patients as soon as possible, ideally at birth. 

But whilst AveXis and other biotech companies are rushing to bring transformative gene therapies to market, states are too slow on adopting screening tests for genetic diseases that are, increasingly, treatable.


AveXis’s gene therapy works by using an engineered virus that delivers healthy copies of the SMN1 gene to cells throughout the body. The new gene then starts making a protein that’s essential for the survival of motor neurons. 

If you wait, however, “the children have limited motor neurons for the gene therapy to get into and work effectively,” AveXi’s chief medical officer, Sukumar Nagendran tells MIT. 

However, MIT’s Emily Mullin reports that a national committee that oversees newborn testing have voted to recommend spinal muscular atrophy to be added to the universal screening panel. Next step is to get US Secretery for Department of Health and Human Services, Alex Azar, to sign off on it. 

But, even if he does, spinal muscular atrophy screening for every newborn won’t be a done deal. A recommendation is just that —a recommendation.