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Rare Diseases And Orphan Drugs: New Startup And Other Happenings (06.05.13)

June 5, 2013

The following Blog Post discusses a new startup and other happenings in the rare disease and orphan drug space:

I – RNAi Delivery Startup – Arcturus Therapeutics

Arcturus Therapeutics, a San Diego-based company is founded in early 2013.  The aim of Arcturus Therapeutics is to “pursue RNA interference (RNAi) solutions for rare diseases for which there is no adequate treatment.” On June 3, 2013, Arcturus Therapeutics raises $1.3 million in a seed funding round led by several high net-worth private investors from the US and Canada. This new round of funding will support:

•   Purchases of capital equipment
•   Further development of intellectual property of RNAi delivery technologies
•   RNAi target selection, design, and in-vitro proof of concept studies.

According to a article, Joseph Payne, Arcturus Therapeutics’ President and CEO, says that the company’s flagship product would target a rare liver disease. The article goes on to review the technique of RNA interference:

“ … uses small snippets of RNA to silence a cell’s expression of genes that viruses and tumors need to survive. One especially problematic step for researchers and pharmaceutical companies, though, has been delivering those snippets to necessary cells in the body.”

Global Industry Analysts, a publisher of market research, estimates that the RNAi technologies market will reach approximately $4.04 billion by 2017.

II – Mayo Clinic And WES Service

Since September 2013, the Mayo Clinic, offers a Whole-Exome Sequencing (WES) service for patients with unknown or rare diseases and advanced cancer. The Mayo Clinic is contracting the sequencing to Baylor College of Medicine and Foundation Medicine, with the goal of doing in-house sequencing by the end of 2013. Between 30 – 35 patients have already made use of the WES service. Some interesting facts about the WES service :

•   For cancer, the diagnostic rate is approximately 40%, with a diagnostic rate slightly higher for unknown/rare diseases (the # of patients is too small to be significant)
•   Average out-of-pocket expense after insurance is $7,000 – $11,000 depending on the service and the patient’s condition
•   Turnaround time is 1 – 2 months, which will be reduced over time as more sequencing is done in-house.

Researchers recently completed the world’s first WES of small intestine neuroendocrine tumors (carcinoids). These types of tumors are the most common cancer of the small bowel and doesn’t respond well to chemotherapy. What is important about this research is that the “sequence analysis revealed six pathways as potential drug targets for this type of cancer …. a very important step in achieving targeted therapies and individualized treatment …” – personalized medicine.


EU-LIFE, a new alliance of ten European leading life-science institutes, officially launches in Barcelona, Spain, in late May 2013. The purpose of the new alliance is to raise “the international profile of European research and to develop coordinated responses to the challenges facing the sector.”  EU-LIFE provides the following numbers :

•   Scientists & support staff: 7,039

•   Publications: 2,551

•   Coordinated EU research projects: 59.

It will be interesting to see if this newly formed European alliance gets involved with rare diseases and/or orphan drugs over time.

Please Note: “Global Thinking” by Benjamin D. Esham [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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