Preexisting Humoral Immunity to AAV in Subjects with MPS type III
Research type
Research Study
Full title
A Study of Pre-existing Humoral immunity to Adeno-Associated Vectors in Subjects with Mucopolysaccharidosis type III
IRAS ID
255805
Contact name
Suresh Vijay
Contact email
Sponsor organisation
Abeona Therapeutics Inc.
Duration of Study in the UK
0 years, 1 months, 1 days
Research summary
The study is non-interventional (i.e. no study drug is administered) and consists of a single clinical evaluation in which the subject will undergo a medical history review, a physical exam and a blood sample will be collected. The study population consists of subjects with Mucopolysaccharidosis type III (MPS III) who are potential candidates for the clinical trials ABT-001 and ABT-002 sponsored by Abeona Therapeutics.
Mucopolysaccharidosis III (MPS III or Sanfilippo syndrome) is a group of lysosomal storage diseases with an estimated incidence of 1 in 70,000 births and caused by the deficiency of enzymes involved in the metabolism of the glycosaminoglycan (GAG) called heparan sulphate. MPS III is caused by the build-up of these GAGs in many parts of the body. MPS III is divided into types IIIA, IIIB, IIIC, and IIID, which are distinguished by their genetic cause. These are severe progressive diseases mainly affecting the brain and spinal cord.
There is currently no treatment available for the disease. Gene therapy may offer a new treatment therapy option for MPS III. Adeno-associated virus (AAV) is a type of virus that has promising features as a vector (carrier to deliver the genetic material to the cell) for gene therapy. However some individuals may show pre-existing immunity to adeno-associated viruses (AAVs) and these subjects may be less likely to benefit from AAV vector-based therapies. The main purpose of this study is to provide information in regard to a subjects pre-existing immunity against adeno-associated vector serotype 9 (AAV9) by measuring total antibodies against AAV9 in a subjects blood sample. Identification of subjects with pre-existing AAV immunity may be an important consideration for the design of these current and future clinical trials.
This study will be conducted at up to 10 centres worldwide with 40-50 subjects taking part.
REC name
South Central - Oxford B Research Ethics Committee
REC reference
19/SC/0228
Date of REC Opinion
29 Apr 2019
REC opinion
Unfavourable Opinion