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Rare Diseases And Orphan Drugs: The Meaning Behind Company Names #1

January 22, 2015

This is the first Blog Post in a series that examines the meaning behind company names in the rare disease and orphan drug space. Medical terminology can be traced to Greek and Latin origins. How about biotechnology and pharmaceutical company names ? This Blog Post takes a look at several companies that have interesting name derivations :

•   Atterocor

•   Audentes Therapeutics

•   AveXis

•   Abeona Therapeutics.

I – Atterocor

Atterocor, created in 2012 as a University of Michigan spinoff, is a drug development company focusing on the accelerated development of a novel treatment for an aggressive rare cancer, Adrenocortical Carcinoma (ACC). ACC is often fatal because it is usually diagnosed in the later stages of the disease. It affects about 1,000 patients in the United States. ACC is a cancer of the adrenal cortex that occurs when cancer cells form in the outer layer (cortex) of the adrenal gland. The cancer frequently impacts the body’s secretion of adrenal hormones and patients are often diagnosed when they seek medical attention due to symptoms associated with excess hormone production. Surgery is not a viable treatment option for the majority of ACC patients and other treatment options are extremely limited.

According to a recent online article : “Atterocor — “attero” is Latin for weaken or destroy, and “cor” is short for cortex ….”. Thus, Atterocor, the name of the new company that is to destroy, to weaken, or to ruin the cortex – the cancer of the adrenal cortex.

Atterocor’s lead product candidate, ATR-101, is currently in Phase I human trials for ACC. ATR-101 receives ODD from the European Medicines Agency (EMA) and in March 2012, receives FDA ODD for ACC.

Atterocor FDA ODD Database Record

Generic   Name: (N-[2,6-bis(1-methylethyl)-pheyl-N’-[[1-4-dimethyl-amino)phenyl]cyclopentyl]methyl]urea,   hydrochloride salt
Trade   Name: n/a
Date   Designated: 03-09-2012
Orphan   Designation: Adrenocortical carcinoma
Orphan   Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor:   Atterocor, Inc. 820 Heatherway Ann Arbor, MI 48104


II – Audentes Therapeutics

Audentes Therapeutics is a San Francisco-based biotechnology company founded in November 2012. The company develops new treatments for rare muscle diseases through the use of Adeno-Associated Virus (AAV) gene therapy technology. “Audentes” comes from the Latin word audeo, which means “to dare”, “to be brave or bold”.

Audentes Therapeutics has two lead rare disease programs in preclinical studies that are based on AAV gene therapy technology :

•   AT001 for X-linked Myotubular Myopathy (XLMTM)

•   AT002 for Pompe Disease.

Audentes Therapeutics FDA ODD Database Record

In December 2014, the FDA grants Audentes Therapeutics ODD for its product candidate At001, a gene therapy product for XLMTM:

Generic Name: Non-replicating adeno-associated viral vector, serotype 8, expressing human myotubularin gene
Trade Name: At001
Date Designated: 12-03-2014
Orphan Designation: X-linked myotubular myopathy
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Audentes Therapeutics, Inc. 101 Montgomery St., #2650 San Francisco, CA 94104


III – AveXis

AveXis, a synthetic biology platform Dallas-based company formed in July 2013, announces in October 2014, that the FDA grants ODD to the company’s gene therapy product, scAAV9.CB.SMN (called chariSMA), for the treatment of Spinal Muscular Atrophy (SMA). chariSMA is developed at Nationwide Children’s Hospital (Columbus, Ohio) and licensed to AveXis for development and commercialization.

According to AveXis’ website, the name AveXis has an interesting derivation:

•   Av = Adeno-Associated Virus (AAV)

•   ve = vector

•   X  = DNA helix

•   is = Isis (goddess of children, nature and magic).

AveXis FDA ODD Database Record

Generic Name: Adeno-associated virus serotype 9 expressing the human Survival Motor Neuron gene
Trade Name: n/a
Date Designated: 09-30-2014
Orphan Designation: Spinal Muscular Atrophy
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: AveXis, Inc. One Energy Square Suite 604 Dallas, TX 75206


IV- Abeona Therapeutics

Abeona Therapeutics is created in March 2013 with the goal of developing therapies for patients with Lysosomal Storage Diseases (LSDs). Abeona Therapeutics, who in partnership with scientists at Nationwide Children’s Hospital, and with financial support from multiple patient advocacy groups, are collaborating to develop gene therapies for children with the rare LSD, Sanfilippo (SF) Syndrome or Mucopolysaccharidosis III (MPS III).

Abeona in Roman Mythology is the Goddess of Departures. She protects children as they step away from home for the first time, keeping them safe as they venture into the world. Her name comes from the Latin verb abeo, “to depart, go away, or go forth”.

Abeona Therapeutics FDA ODD Database Record

In April 2014, Abeona Therapeutics receives FDA Orphan Drug Designation for their investigational gene therapies for both Sanfilippo Syndrome Types A and B:

 # Generic Name/ODD Date Sponsor Company Indication
1 Recombinant AAV9 expressing human sulfoglucosamine sulfohydrolase/ 04.29.14 Abeona Therapeutics MucopolysaccharidosisType III-A(Sanfilippo SyndromeType A)
2 Recombinant AAV9 expressing human alpha-N-acetylglucosaminidase/ 04.30.14 Abeona Therapeutics MucopolysaccharidosisIII-B(Sanfilippo SyndromeType B)


Please Note: “Jigsaw” by User: Amada44 (Own work) [Public domain] | Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

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