ICMR releases draft guidelines to treat rare genetic diseases

New Delhi [India], Dec 13, (ANI): The Indian Council of Medical Research (ICMR) on Friday released the draft guidelines for ensuring that rare genetic therapies and clinical trials can be performed in an ethical, scientific and safe manner in India.
"National guidelines for the treatment of patients suffering from rare genetic diseases" will assist medical experts to get genetic therapy product development and conduct clinical trials.
About 70 million Indians suffer from inherited genetic diseases or 'rare diseases' (RD), according to the apex medical research institute.
These include hemophilia, thalassemia, sickle-cell anemia certain forms of muscular dystrophies, retinal dystrophies such as retinitis pigmentosa, corneal dystrophies, primary immunodeficiency (PID) in children, lysosomal storage disorders such as Pompe disease, Gaucher's disease, haemangioma, cystic fibrosis among others.
Though it affects a small percentage of the population, the disease has vast, debilitating and life-threatening effects of the patients, many of whom are in the pediatric age group.
"These national guidelines provide the general principles for developing gene therapy products (GTPs) for any human ailment and provides the framework for human clinical trials which must follow the established general principles of biomedical research for any human applications. The guidelines cover all areas of GTP production, pre-clinical testing, and clinical administration, as well as long term, follow up," said (Prof) Dr Geeta Jotwani, a scientist at the ICMR.
"We recognised the huge burden of genetic diseases in India. There was a need to accelerate the development of advanced therapeutic options for rare genetic diseases. These guidelines will serve as an important resource and roadmap for those in the field trying to develop gene and cell therapies," said Dr Jotwani.
The guidelines cover all considerations for chemistry, manufacturing and control, quality assurance, product attributes for GTP, including personnel training and infrastructure requirements, she said.
All GTP research pertaining to human applications must be conducted within the principles of these guidelines in a scientific and ethical manner following all regulatory requirements as laid down for all forms of GTP, said the official.
Recently, the Central Drugs Standard Control Organization (CDSCO) in the New Drugs and Clinical Trials Rules, 2019 (NDCTR), has defined a rare disease drug or "orphan drug" for the untreatable disorder. (ANI)