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India’s first indigenous CRISPR therapy, Birsa-101, signals a decisive shift toward affordable gene-editing solutions for Sickle-cell Disease
Marking a significant leap forward in medical innovation, India has developed its own CRISPR (clustered regularly interspaced short palindromic repeats)-based therapy — Birsa-101 — to treat Sickle-cell Disease. Named after the notable tribal leader, Bhagwan Birsa Munda, Birsa-101 offers promise as an affordable therapy for a genetic condition that primarily affects vulnerable communities in India. Clinical trials are set to commence next year, with participants likely to be from central and eastern India.
Sickle-cell disease (SCD) is a group of genetically inherited disorders that affect the ability of haemoglobin – a protein in red blood cells – to carry oxygen in the body. Typically, red blood cells (RBCs) with normal haemoglobin have a disc shape, enabling them to move through blood vessels and deliver oxygen efficiently. In the case of SCD, a genetic mutation in the gene (HBB gene) encoding a component of haemoglobin changes its structure, and the RBCs become crescent-shaped or sickle-shaped. These sickle RBCs are rigid and cannot move easily, impeding oxygen supply to the body.
SCD develops when an individual inherits two mutant copies of the HBB gene (one from each parent). Symptoms include painful episodes or crises that require hospitalisation, anaemia, swelling, frequent infections, and other severe complications such as stroke, vision problems, and kidney disease. Individuals who inherit one mutant copy of the HBB gene typically do not develop symptoms, but they can pass on the trait to their children.
Developing an indigenous gene therapy is part of India’s National Sickle Cell Anaemia Elimination Mission, launched in 2023, which aims to eliminate SCD by 2047.
Approximately 7.7 million people live with SCD globally, with sub-Saharan Africa accounting for 80 percent of global cases. SCD accounted for over 81,000 deaths in 2021, substantially contributing to the under-5 mortality burden. In the Indian context, the burden of SCD varies across geographies. On the other hand, the prevalence of sickle-cell trait among tribal groups ranges from 1-40 percent, and the prevalence of SCD is 1 percent. Notably, 73 percent of Indians with the trait belong to indigenous communities, representing 8.6 percent of the population.
Treatment can be administered through medicines such as hydroxyurea, blood transfusions, and antibiotics to reduce infections, while bone marrow transplantation is the only curative option. Against this backdrop of persistent disease burden, gene therapy is emerging as another curative option for SCD. Research into CRISPR began in 1987, but its application as a gene-editing tool was first demonstrated in 2012. The field advanced significantly thereafter, culminating in the 2020 Nobel Prize in Chemistry awarded to Emmanuelle Charpentier and Jennifer Doudna for developing the CRISPR-Cas9 gene-editing technology.
The first gene-editing therapy for treating SCD — Casgevy — was approved by the United States (US) Food and Drug Administration (FDA) in 2023. Owing primarily to high licensing fees, CRISPR-based gene-editing therapy for SCD costs reportedly INR 20-25 crore per patient. The newly developed indigenous platform is expected to cost around INR 50 lakh. Developing an indigenous gene therapy is part of India’s National Sickle Cell Anaemia Elimination Mission, launched in 2023, which aims to eliminate SCD by 2047.
Birsa-101 was developed by the Centre for Scientific and Industrial Research Institute of Genomics and Integrative Biology (CSIR-IGIB) and is based on CRISPR technology. Earlier this month, a formal technology transfer and collaboration agreement was finalised between CSIR-IGIB and the Serum Institute of India (SII). Steps to commence clinical trials are already in progress with CSIR-IGIB collaborating with several institutes, including the Department of Biotechnology, the Department of Science and Technology, the Drug Controller General of India, CSIR, and the Ministry of Tribal Affairs.
Earlier this month, a formal technology transfer and collaboration agreement was finalised between CSIR-IGIB and the Serum Institute of India (SII). Steps to commence clinical trials are already in progress with CSIR-IGIB collaborating with several institutes.
Participants from Madhya Pradesh, Chhattisgarh, and Jharkhand will be included in the clinical trials, which are set to begin in 2026. Clinical trials will be conducted collaboratively by CSIR-IGIB, SII, and All India Institute of Medical Sciences (AIIMS), Delhi, enabling the clinical translation of the indigenous technology into a scalable and affordable therapeutic option for India. Further, thalassemia — a genetic blood disorder characterised by reduced or absent haemoglobin production — is also a target for this technology. IGIB and SII are developing a parallel therapy for thalassemia, using a mechanism similar to that employed for treating SCD.
As CRISPR-based therapies such as Birsa-101 advance, responsible science communication becomes essential. Researchers and institutions must prioritise transparent updates, accessible explanations, and proactive engagement with communities regarding gene therapies. For instance, securing informed consent in vulnerable communities requires culturally grounded communication, translation into local languages, and sustained dialogue with community leaders and frontline health workers. Strengthening public dialogue and ensuring responsible media coverage will be key to countering misinformation and building confidence in gene-editing technologies as they move from laboratories to real-world use.
Gene-editing therapies demand specialised infrastructure, trained personnel, and strict biosafety and handling protocols. The patient pathway is equally complex, necessitating clinical assessments, preparatory measures, and long-term monitoring. While premier healthcare centres can support this continuum of care, replicating such capacity in remote and tribal districts—where SCD is most prevalent—remains a significant challenge.
Gene-editing therapies demand specialised infrastructure, trained personnel, and strict biosafety and handling protocols.
From a geopolitical perspective, the indigenous production of a gene therapy for SCD marks a notable shift towards self-reliance in biotechnology and potentially positions India as a manufacturer of affordable gene therapy for low- and middle-income countries (LMICs). In addition, the parallel development of the platform for thalassemia indicates a strong possibility of a shared gene therapy ecosystem in India – spanning across diagnostics, manufacturing, and clinical trials – paving the way to expand treatment options for other genetic disorders.
Collectively, Birsa-101 represents not only a major scientific milestone but also a broader shift in India’s biotechnology capabilities. By advancing an indigenous and affordable CRISPR platform, India has begun laying the foundation for long-term leadership in gene therapies. As clinical trials progress, sustained investment, community trust-building, and equitable access will determine how effectively these innovations benefit the populations that need them most.
Lakshmy Ramakrishnan is an Associate Fellow with the Centre for New Economic Diplomacy, Observer Research Foundation.
The views expressed above belong to the author(s). ORF research and analyses now available on Telegram! Click here to access our curated content — blogs, longforms and interviews.
Lakshmy is an Associate Fellow with ORF’s Centre for New Economic Diplomacy. Her work focuses on the intersection of biotechnology, health, and international relations, with a ...
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