Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for life- threatening paediatric rare genetic diseases. We are currently conducting clinical trials using adeno-associated virus serotype 9 (AAV9)-based vectors for correction of enzymatic defect in lysosomal storage disorders. These studies may be of interest to your patients. Other AAV-based gene therapies have received marketing approvals by EMA and FDA (Glybera and Luxturna), and approval of an AAV9-based gene therapy for spinal muscular atrophy type 1 is expected soon, collectively underscoring the feasibility and overall safety of this approach.

We are actively recruiting participants for two ongoing phase I/II clinical trials for Sanfilippo syndrome (MPS III types A and B) employing a single intravenous infusion of AAV9. With clinical follow up of 2 years (median follow up of over 13 months), a single dose of ABO-101 (Sanfilippo B) or ABO-102 (Sanfilippo A) demonstrated rapid and sustained reduction of enzymatic substrates (heparan sulfate) and reduction of liver volume to near normal for a total of over 14 subjects treated. There have been no treatment-related serious adverse events reported to date. Early signs of neurodevelopment benefit appear to be emerging, especially in younger, higher functioning children.

Abeona’s ABO-102 and ABO-101 clinical programs are enrolling patients with a confirmed MPS IIIA/B diagnosis aged 6 months to 2 years, or children older than 2 years with a minimum cognitive function of at least 60% of normal for age. Further details on the inclusion and exclusion criteria can be found in Clinicaltrials.gov registers on the following links:

  • ABO-101 clinical trials.gov link here.
  • ABO-102 clinical trials.gov link here.Abeona Therapeutics is planning to activate a phase I/II clinical trial for Neuronal Ceroid Lipofuscinosis (CLN) type 1 targeting Infantile, Late infantile and Juvenile onset forms of CLN1 disease. The ABO-202 trial design will mimic the one for the Sanfilippo syndrome programs with the exception that this will be a dose escalation in which each cohort will include a combination of intrathecal and intravenous dose administrations. More information will be provided as soon as we have precise stating timlines on submissions for USA and Europe.