Finding studies
Finding studies
Take this into the appointment.
Saves the questions and what to expect into your notes, next to the visit they belong to.
Alicia Gomez
CONTACT
clinicaltrials@aspatx.com
CONTACT
Lead
Aspa Therapeutics
Canavan disease is an ultra-rare, profoundly disabling and fatal disease with no approved therapy. The Sponsor is developing BBP-812, an investigational gene therapy product for systemic delivery in participants with Canavan disease. BBP-812 is a recombinant adeno-associated virus serotype 9 (rAAV9) vector engineered to deliver the aspartoacylase (ASPA) transgene under control of a ubiquitous promoter to restore ASPA expression in both neuronal and non-neuronal cell types.
Age
0–2
Sex
ALL
Healthy volunteers
Not accepted