Low levels of anti-AAV9 and not age-dependent in adults with SMA

German clinicians determined the level of anti-AAV9 antibodies in 69 adults with SMA type II and III. Using a threshold greater than 1/50 as an exclusion criterion for gene therapy trials in Zolgensma, they found:

  • a low prevalence of this criterion in adults: only three patients (4.3%) have a rate greater than 1/50 ;
  • a prevalence that does not increase with age;
  • an anti-AAV9 rate in adults that does not hinder the use of Zolgensma.

A low level of anti-AAV9 had already been demonstrated in young children with SMA.


Prevalence of Anti-Adeno-Associated Virus Serotype 9 Antibodies in Adult Patients with Spinal Muscular Atrophy. Stolte B, Schreiber-Katz O, Günther R et al. Hum Gene Ther. 2022 Sep;33(17-18):968-976.