Add thelocalreport.in As A Trusted Source
India has taken a historic step in the field of genomics with the launch of its first indigenous CRISPR-based gene therapy for sickle cell disease (SCD), known as Birsa-101. This development could significantly reduce the cost of therapy and target one of the country’s most vulnerable populations.
Launched on November 19 by Union Science and Technology Minister Dr Jitendra Singh, the treatment was developed by the CSIR-Institute of Genomics and Integrative Biology (IGIB) in partnership with the Serum Institute of India (SII).
Birsa-101, named after tribal leader Lord Birsa Munda, uses enFnCas9, a specially designed CRISPR enzyme that can precisely correct the DNA mutation that causes sickle cell disease.
This therapy was designed with affordability in mind. The indigenous platform is expected to reduce the cost of CRISPR-based SCD treatment, which typically costs millions of dollars (about ₹20-25 crore, or $2-3 million), to around ₹50 lakh.
Dr. Singh described the gene-editing technique as a “precision genetic surgery” that could change the way many hereditary diseases are treated, including curing sickle cell disease.
The Union Minister underlined that the discovery has shown the country’s ability to create groundbreaking treatments at a fraction of the world’s cost. He emphasized that this discovery has important national implications, especially for the tribal people of central and eastern India, where the prevalence of the disease is highest.
Serum Institute will conduct upcoming clinical trials in collaboration after IGIB scales up the production process. The first phase, in partnership with AIIMS, New Delhi, is expected to begin next year. A pool of about 150 potential participants has already been assembled by IGIB from central and eastern India, especially the tribal areas of Madhya Pradesh, Chhattisgarh and Jharkhand.
After editing, patients will receive a single infusion, which will cause their bone marrow to stop making sickled red blood cells and start producing healthy cells.
How is sickle cell disease treated with Birsa-101?
A crippling systemic disease marked by chronic anemia, acute painful episodes, organ infarctions, and persistent organ damage, sickle cell disease is a long-term, single-gene disorder that significantly reduces life expectancy.
In fact, a person can be a carrier even without having any disease. If one parent has the disease and the other is a carrier, or if both parents are carriers, the child is more likely to have it.
Birsa-101 precisely pinpoints the genetic code mutation that causes the disease. Treatment should be administered as a single infusion; After this, the body should begin producing normal red blood cells instead of sickle-shaped cells.
Furthermore, the CRISPR Cas9 gene editing tool – the genetic scissors that earned the Nobel Prize for Chemistry in 2020 – is based on bacterial immune system proteins that can cleave genetic material into pieces. It is possible to use these proteins to precisely remove damaged genomic material.
If the treatment proves effective in curing the condition, it will initially be made available to people with severe sickle cell disease, perhaps with organ damage. Additionally, it may eventually be extended to children as well. The condition is estimated to affect 30,000 to 40,000 newborns in India every year.
According to the Indian Express, this approach differs significantly from the widely accepted treatment CasGevi, which modifies a gene to stimulate the body’s blood production process to produce more fetal hemoglobin, which does not have the same defect.
Thalassemia, another genetic blood condition in which the body produces little or no hemoglobin, can be treated with the same therapy. A similar mechanism will be used in the thalassemia treatment being developed by IGIB and SII.