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Orphan Drugs And Rare Diseases: Top US Life Science Start-Ups

November 5, 2014




BioSpace recently publishes an online list of the “Top 30 Life Science Start-ups To Watch in the U.S.” The US life science companies are launched no earlier than 2011 and are headquartered in the US. The companies are then “weighted by a number of categories and ranked in a cumulative fashion based on the points awarded each category”.

BioSpace Categories & Methodology Used For Ranking

•    Collaborations: 2 points awarded if company is a spinout from well-known company/institution, signs developmental/commercial agreements/partnerships. Two points awarded per collaboration

•    Editorial: Editor awards up to 10 points to companies for interesting areas of science and technology or for working in a broad and open market

•    Finance: 1 point awarded per $10 million in seed financing/money raised

•    Pipeline: 2 points if company has compound/device in ongoing clinical trial & 2 points for each compound/device

•    Sales: 5 points added if company has product to service to sell.

Source Of Chart Data

•   Columns 1 – 3 are from online BioSpace article

•   Column 4 is from the Orphan Druganaut Blog

•   Column 5 is from online FierceBiotech article.

Top US Life Science Start-Ups To Watch In Orphan Drug/Rare Disease Space

Rank # Company Name/ Year Founded # of BioSpace Points (43 is Highest Score) Orphan Drug/ Rare Disease Programs FierceBiotech’s 2014 Fierce 15 Biotech Companies
3 Spark Therapeutics/ 2013 33 Gene Therapy Programs in Hemophilia B, RPE65-related blindness, & inherited retinal dystrophies X
4 Audentes Therapeutics/ 2013 25 Gene Therapy Programs for Pompe Disease & X-Linked Myotubular Myopathy (XLMTM)
8 Dimension Therapeutics/ 2013 22 Gene Therapy Programs for Hemophilia A & B X
12 Navitor Pharmaceuticals/ Launches June 2014 16 Novel drugs targeting cellular nutrient signaling via mTORC1 pathway for several rare disorders X
13 Arcturus Therapeutics/ 2013 15 Developing RNA medicines for TTR-mediated Amyloidosis
18 Scioderm/ 2012 11 FDA Breakthrough Therapy Designation for lead product SD-101 for Epidermolysis Bullosa (EB)
23 Global Blood Therapeutics/ 2012 6 Lead Program for Sickle Cell Disease (SCD)
29 Cydan/ 2013 3 Orphan drug accelerator to partner with rare disease stakeholders to pick most promising rare disease programs to start companies around


Please Note: “3D Bar Graph Meeting” by “The Gold Guys Blog” [CC-BY-SA-2.0] via Wikimedia Commons.

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