Significantly increase the brain volume of children with the latest clinical results of AAV gene therapy

On July 26, 2021, Abeona Therapeutics announced that in pediatric patients with type A Sanfilippo syndrome (MPS IIIA, also known as mucopolysaccharidosis type IIIA), its new gene therapy ABO-102 is 1/ The Phase 2 Transpher A clinical trial achieved positive results
.

Magnetic resonance imaging (MRI) data showed that at the 24th month, compared with untreated patients, ABO-102 had a clinically significant increase in the brain gray matter, corpus callosum, and amygdala in three patients

MPS IIIA is a rare and fatal hereditary neurodegenerative disease.
There is currently no approved treatment
.

Due to genetic mutations, patients lack the SGSH enzyme responsible for decomposing mucopolysaccharides (GAG, also known as "glycosaminoglycans"), which causes mucopolysaccharides to accumulate in lysosomes and cause cell dysfunction

ABO-102 is a gene therapy under development using adeno-associated virus 9 (AAV9) as a vector
.

ABO-102 uses self-complementary (self-complementary) AAV9 vector and is administered as a single-dose intravenous infusion

This ongoing open-label, dose-escalation, two-year Phase 1/2 clinical trial of Transpher A, the primary endpoint is the improvement of patients' neurodevelopment and safety
.

Secondary endpoints include behavioral assessment, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate (HS) levels in plasma and urine, and brain and liver volume

Previous trial results showed that within 18 months to 2 years after treatment, three patients in cohort 3 (aged 27 months, 19 months, and 12 months) who received treatment showed sustained neurocognitive ability Improve
.

In addition, in all cohorts (n=14), HS, a biomarker of enzyme activity in the central nervous system of patients, showed a rapid and sustained decrease

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Reference materials:

[1] Abeona Therapeutics Announces New MRI Data Showing Increased Brain Volume in Young Patients with Sanfilippo Syndrome Type A (MPS IIIA) After Treatment with ABO-102 Gene Therapy.