“However, the new gene therapy has shown promising results during the first phase trial on five patients, aged between 22 and 41. The annualised bleeding rate has been observed to be zero for all five participants over a cumulative follow-up of 81 months. The therapy will be very useful for some blood disorders that were incurable earlier,” he said.
Describing the human gene therapy, Swain said, stem cells were extracted from the blood of the patients and after certain gene corrections, the cells were again transplanted with a lentiviral vector in the respective patients.
“Gene therapy provides the body with the ability to produce factor VIII, ensuring that there is no need for repeated infusions,” he said.
Gene therapy using viral vectors has emerged as a paradigm-changing therapeutic option for haemophilia as it will address the disease by introducing a normal copy of the defective gene. “A distinctively prepared lentiviral vector having no capacity for new infection and self-replication has been used in the gene therapy,” Swain said.
