India’s First siRNA Drug Trial Approved for Rare Eye Disease Treatment


Biocon and Quark Pharmaceuticals’ novel siRNA (small interfering RNA) drug candidate-QPI-1007 for ocular neuroprotection got approval from the Drug Controller General of India (DCGI) to conduct the first-ever clinical trial of a siRNA therapy in India.

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The first patient in India in the pivotal global Phase II/III clinical trial of siRNA candidate, QPI-1007 for the treatment of patients with rare ocular disorder, nonarteritic ischemic optic neuropathy (NAION) is being randomly allocated.

nonarteritic ischemic optic neuropathy

Figure 1. The optic nerve head in acquired optic neuropathies (photo credit: Nat Rev Neurol. 2010 Apr;6(4):221-36. doi: 10.1038/nrneurol.2010.5).

Small (or short) interfering RNA (siRNA) is the most commonly used RNA interference (RNAi) tool for the triggering of short-term silencing of protein-coding genes. siRNA is a synthetic bidirectional RNA which has been designed specially to target a specific mRNA for degradation.

RNA interference principle

Figure 2. The mechanism of RNA interference (photo credit: Nat Rev Drug Discov. 2009 Feb;8(2):129-38. doi: 10.1038/nrd2742).

While siRNA provides the chance to induce powerful breaking down of a gene in a variety of cell lines, their use is limited only to the cells that are agreeable to the introduction of DNA into the cell of synthetic oligonucleotides. siRNAs can also be used for the breaking of non-protein coding genes, for example- the long noncoding RNAs (lncRNA) and holds various therapeutic potential.

QPI-1007 is a chemically modified version of double-stranded RNA molecule. The modification has been done by the Quark’s proprietary technology. QPI-1007 is being developed to serve as a neuroprotectant by temporary inhibition of the expression of caspase 2 which will ultimately stop the apoptotic death of retinal ganglion cells. QPI-1007 is designed for the treatment of NAION and other optic neuropathies. 

Biocon Chairperson & Managing Director Kiran Mazumdar-Shaw said, “Biocon is committed to develop innovative therapies that address unmet medical needs and the initiation of the Phase II/III study investigating QPI-1007 in NAION in India is an important step towards this goal. India has a significant NAION patient population and we are pleased to be the first biopharma company in the country to provide an siRNA-based therapy that is likely to benefit thousands of patients who either have no access to treatment or cannot afford it.”

The effect of QPI-1007 on visual function in the patients with acute NAION will  be determined by the QRK207 study. NAION is an unmet medical condition. The Phase II/III study will be conducted by Quark in collaboration with “Neuro-Ophthalmology Research Disease Investigator Consortium” (NORDIC).

Dr. Daniel Zurr, Chairman, and CEO of Quark stated that “siRNA therapeutics is a new class of medicine, with the power to bring in revolutions in the drug development and offer therapeutic precision and has widespread applicability.” He added, “QPI-1007 represents a novel therapeutic strategy for treating NAION and future plans are to develop it for additional optic neuropathies, including glaucoma, which, similar to NAION, are characterized by the death of retinal ganglion cells.”

In 2013, Biocon and Quark Pharma collaborated for the development, manufacturing and marketing of QPI-1007 in India and other key markets.

Featured image credit: Layers of eye © Sciencepics (Stock Photo ID: 83134274)

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