According to a story from Scimex, the Children’s Medical Research Institute in Sydney, Australia, has recently agreed to a new partnership with LogicBio Therapeutics, a biotechnology company from Boston, MA. The new agreement will allow the two organizations to work together in order to develop a gene therapy for a rare childhood liver disease called propionic acidemia.
About Propionic Acidemia
Propionic acidemia is a rare, genetic, metabolic disorder that first appears in childhood. This disorder is characterized by the deficiency of an enzyme necessary for the conversion of certain fats and amino acids into sugar so that it can be used as energy. This leads to a buildup of propionic acid in the body. Propionic acidemia is linked to mutations affecting both copies of either the PCCA or PCCB genes. Symptoms include poor feeding, poor muscle tone, vomiting, and lethargy. This disease can quickly become fatal due to infections, stroke, cardiomyopathy, or excess ammonia in the blood. Some patients may experience fairly normal development, but others develop lifelong learning disabilities. There are no approved treatments specifically for the disorder; management strategies include a low protein diet, antibiotics, and liver transplant. The disease is more common in Saudi Arabia. To learn more about propionic acidemia, click here.
What Could Gene Therapy Bring?
The development of a gene therapy for propionic acidemia could dramatically improve the lives of patients like Charlize Gravina, who is just three years old. Her twin brother Isaac was also born with the disorder. At age two, Isaac received a liver transplant in an attempt to treat the illness, but he died after experiencing complications following the operation. Even after that frightful outcome, Charlize has received two liver transplants to treat her own case. While she is still going strong, parents Julie and Paul are encouraged by the news of a new treatment for the disease being in development.
Professor Ian Alexander is playing a leading role in the new collaboration and he says that gene therapy could be a real live saver for patients facing this disorder. The partnership aims to bring down costs and improve patient access to gene therapy. Professor Alexander intends for the research to yield gene therapy approaches for many genetic disorders beyond just propionic acidemia.
