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Table 2 Details of the products, services or R&D activities of the OMPOs

From: The work, goals, challenges, achievements, and recommendations of orphan medicinal product organizations in India: an interview-based study

Organization Products/Services
APL APL has discovered drugs for Niemann-Pick disease (NPD) and focal segmental glomerulosclerosis (FSGS). It is also working on drugs for non-RD diseases. Two drugs are in the early discovery stage, one for non-alcoholic steatohepatitis (NASH) and the other for obesity. Further, it is looking at other drugs for metabolic syndrome.
DART Exon skipping therapy for muscular dystrophy.
Eyestem Cell therapy for ophthalmic diseases such as retinitis pigmentosa (RP) and macular degeneration (MD)
GLS Carrier screening test that includes 10,000 pathogenic genomic variants which cover 1700 diseases.
GROW Discovery-based clinical applications and gene therapy for RP.
GUaRDIAN R&D for molecular genetic diagnostics; identifying novel markers specific to a disease and population; education in clinical genomics.
IB Botanical drugs: 3 are in the pipeline:
1) a drug for Levodopa-induced dyskinesia (LID)
2) drugs for Huntington’s disease
3) drugs for Sumatriptan-resistant migraine patients.
MedGenome There are three business segments:
(i) Research services: mainly for pharmaceutical companies and research organizations which outsource work.
(ii) Genetic diagnostics: Primarily focused on monogenic diseases and oncology, as India has a huge population suffering from these diseases. It focuses on actionable mutations such as from lung, colorectal, and breast cancer. Identifying predisposition mutations and individuals-at-risk in families. Helping them to manage the disease, and increasing surveillance.
(iii) Data analytics and developing new databases. It has a fairly large bioinformatics team which develops algorithms and pipelines, and helps create unique databases.
It works with clients across the world on customized solutions, or licensing out technologies that it develops. It has a lab in the US which caters to the pharma and biopharma industry.
The first and third services are mainly handled by the US lab, whereas the second service is mainly handled by the India center.
Pristine Broadly, products are divided into 5 segments, of which segment (v) is for RD patients:
i) fortification of rice, flour, oil, milk; ii) pre-mixes of nutritional supplements; iii) animal feed pre-mixes for the poultry, equine and aquaculture industries; iv) Organic staples: Rice, millets, flours, oil seeds, cold pressed oil and organic infant food; (v) Food for special dietary uses; and (vi) Nutritional supplements for specific disease conditions. These supplements are categorized by the Food Safety and Standards Authority of India (FSSAI) as ‘Food for special medical purpose’. They are sold under the brand name ‘Balance Metanutrition’, and are for IEMs, diabetes, autism, cancer, Alzheimer’s.
RLS DNA-based diagnostics for maternal and child health conditions such as NIPT, microarrays, single gene disorders, clinical exome, whole exome, whole genome.
SMOI (i) Services related to mobility aids and accessories
(ii) A low-cost mobility device is in development
TBXT IoT- and AI-based wearable medical devices for neurological disorders. The device for epilepsy can 1) predict the occurrence of an epileptic attack; 2) assess the efficacy of medicine; and 3) predict any serious, potentially fatal, attack.
New Organizations
 CDD Collaborate with various research institutes across the country to fund research leading to the development of therapies for rare diseases, specifically GNE Myopathy.
 Genetico Software and database management system to assist geneticists in diagnosing RD patients. Some of the strategies include matching patients with similar clinical profiles to help achieve diagnosis and knowledge about specific RDs.