India has come close to developing a gene therapy for sickle cell disease, officials say.

India has come close to developing a gene therapy for sickle cell disease, officials say.

This microscope photo provided by the Centers for Disease Control and Prevention shows crescent-shaped red blood cells from a patient with sickle cell disease.

This microscope image provided by the Centers for Disease Control and Prevention shows the crescent-shaped red blood cells of a patient with sickle cell disease. | Photo credit: AP

India is close to developing a gene therapy for sickle cell disease, a genetic blood disorder that is highly prevalent among scheduled tribes, officials of the Union tribal affairs ministry said on June 19.

Tribal Affairs Ministry Secretary Vibhu Nair said the government hoped to hear “good news” on laboratory tests by January 2025. All India Institute of Medical Sciences (AIIMS) Director M. Srinivas said researchers were working to develop gene therapy using the gene-editing tool CRISPR-Cas9.

“We hope that in the next six months to one year we should be able to use this method to treat SCD — making India one of the first countries to do so,” Srinivas said.

He was speaking at a national conference on creating awareness about sickle cell disease organised by the Ministry of Tribal Affairs in collaboration with Birsa Munda Centre, AIIMS.

Union Tribal Affairs Minister Jual Oram, in his address at the inauguration of the conference, lauded the efforts but said it is important to involve and coordinate with grassroots health workers like ASHA and Anganwadi workers for proper implementation of these schemes.

“They are the ones who will do the heavy work on the land,” Mr Oram said.

Officials of the Ministry of Tribal Affairs said Hindu The “good news” that Mr. Nair was referring to was related to the trials currently being conducted by the Council of Scientific and Industrial Research-Institute of Genomics and Integrative Biology (CSIR-IGIB).

“After this, the trials will move to the next phase and eventually will be conducted on patients,” a senior official said.

This comes just months after the US Food and Drug Administration approved the CRISPR-Cas9 technology for cell-based gene therapy to treat sickle cell disease in December 2023.

Officials in the tribal affairs ministry said one of the main challenges for India is to find a way to make this therapy cost-effective.

Developing gene therapies using CRISPR has been part of India’s mission to eliminate sickle cell disease by 2047. A government dossier on the mission, launched by Prime Minister Narendra Modi in July 2023, says the technology has the “potential to be a single-dose cure for blood disorders such as sickle cell anaemia”.

Ministry officials said that a part of this mission also includes conducting more than 7 crore tests among the vulnerable tribal population in 17 states and union territories, out of which three crore tests have been conducted so far.

The CRISPR-Cas9 system consists of an enzyme that behaves like molecular scissors that can be directed to cut a piece of DNA at a precise location. This will then allow a guide RNA to insert an altered genetic code at the sites of the incision. While there are a few ways to effect such changes, the CRISPR system is considered the fastest and most versatile.

Addressing the gathering of doctors, experts and health professionals, Shri Oram said that the Central Government is committed to work on the Mission to Eliminate Sickle Cell Disease and called upon the officials of all Ministries and Departments to ensure that the grassroots level workers are involved in the implementation process and he himself should also join them.

Following the address by senior officials and the minister, a series of technical panel discussions were also held on identification and screening of sickle cell disease, management of the disease and other issues. District centres from around 350 districts across the country were participating in the conference virtually, officials said.

Apart from the gene therapy being developed by India, the Mission to Eliminate Sickle Cell Disease also includes the development of two coded formulations – AYUSH-RP and AYUSH-SC3 – for management of the disease through a systemic drug development process, for which continuous trials will be conducted by the Central Council for Research in Ayurvedic Sciences in collaboration with the Indian Council of Medical Research.

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