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