Throughout its decades of operation, Orchard Therapeutics PLC – ADR (NASDAQ:ORTX) has made significant breakthroughs in gene therapy. It has had potential and reliable advancements in the development of cures for rare and often-fatal diseases. Its ex vivo autologous gene therapy approach has been used by thousands of scientists to a bold vision to a potentially life-transforming reality.
The company has evaluated the safety and efficacy of OTL-203, a treatment for mucopolysaccharidosis type I (MPS-I). Interim data from the ongoing proof-of-concept clinical trial is out, and the results are being shared virtually. This will happen during the 46th Annual Meeting of the European Society for Blood and Bone Marrow Transplantation (EBMT).
The Interim Data Supports Planned Initiation of Registration Trial in 2021
The ongoing proof-of-concept study completed enrollment in December 2019, and by then, eight patients with the severe Hurler subtype of MPS-I had received OTL-203. This was followed by six months follow up of the rest of the patients with the longest follow–up going up to 24 months.
The general results demonstrated tolerability and a safety profile consistent with the previous analyses. According to the CEO of Orchard, Bobby Gaspar, positive trends were observed in biomarkers and clinical measures even as the follow up of patients continue.
Gaspar has also highlighted the possibility of advancing the program, given the growing amount of positive data. The development and design of a registrational trial by a panel of disease experts is underway, which is likely to result in the study’s launch in 2021.
MPS-I Occurs At A Frequency of One in Every 100,000 Live Births
There are three subtypes of MPS-I, and Hurler syndrome is reportedly the most severe subtype. It is also the most common in about 60% of children born with MPS-I, and if untreated, they hardly live past 10years. More often than not, it is called by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme. This is what breaks down glycosaminoglycans, sugar molecules, also known as GAGs.
Victims of accumulation of GAGs suffer from loss of vision and hearing, skeletal deformity, neurocognitive impairment, pulmonary complications, and cardiovascular complications.