ICMR calls for proposals on 'Therapeutics for Inherited Rare Diseases'

The Indian Council of Medical Research has called for proposals to support research for developing newer therapeutics for inherited rare diseases.Inherited rare diseases are infrequently occurring disorders and have been defined by countries in terms of prevalence in their own population, the apex health research institute said in a call for proposal document.The WHO defines rare diseases as often debilitating lifelong disorders with a prevalence of one or less per 1,000 population.

The Indian Council of Medical Research has called for proposals to support research for developing newer therapeutics for inherited rare diseases.

Inherited rare diseases are infrequently occurring disorders and have been defined by countries in terms of prevalence in their own population, the apex health research institute said in a call for proposal document.

The WHO defines rare diseases as often debilitating lifelong disorders with a prevalence of one or less per 1,000 population. Only 5 per cent of inherited rare disorders have treatment among which most of the treatments are highly priced and often not available worldwide.

Access to and availability of medicines are important to reduce morbidity and mortality associated with rare diseases, the document said.

''Therefore, there is a need for research and development in intervention, technology transfer and indigenously manufactured therapeutics for rare diseases which could be achieved by collaborative efforts,'' it said.

The goal of this call is to support research and development research proposals towards developing intervention strategies on the aspects of inherited rare diseases such as discovery of newer therapeutic agents and approaches, development of therapeutics including repurposed drugs, biologics, genetic error corrective measures and management to improve quality of life of patients with rare diseases such as orthopaedic/surgical approaches and innovative management protocols, etc.

Only inherited rare diseases under the groups Storage Disorders, Small Molecule Inborn Errors of Metabolism, Primary Immunodeficiency Disorders (PID), Neuromuscular Disorders (NMD), Hematological Disorders (excluding Sickle Cell Diseases and Thalassemia), Skeletal dysplasia will be considered, the document said.

Approaches may include development of resources such as cellular and animal models, vectors for genetic error corrective measures, and mechanistic aspects that are required for drug development as part of the project, it said.

The projects should be time-bound, normally for a duration of 2-3 years. The last date for submission of proposals is January 25, it said.