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Rare Diseases And Orphan Drugs: Gene Therapy Business Developments #2

May 21, 2013

Gene therapy for rare genetic diseases is hot news in biotechnology. Biotechnology companies that develop and commercialize gene therapy is a rapidly growing field of medicine, especially for rare diseases. Gene therapy involves introducing genes into the body to treat diseases. The objective of gene therapy is to provide genes that correct or supplant the disease-controlling functions of cells that aren’t functioning or doing their job properly.

This is a second in a series of Blog Posts this week on the topic of recent business developments for gene therapy in the rare disease and orphan drug space.

I – Batten Disease And The University of North Carolina

Taylor’s Tale, a non-profit, tax-exempt entity (501(c)(3)), raises funds for research and promotes public awareness of Batten Disease. Taylor’s Tale is collaborating with an international coalition to help fund a gene therapy project, “Global Transfer for Batten Disease”, at the Gene Therapy Center at the University of North Carolina at Chapel Hill (UNC) :

 .. is a parallel study in infantile (INCL, CLN1 mutation) and late infantile (LINCL, CLN2 mutation) Batten disease. The project aims to test a global gene delivery platform in mouse models for INCL and LINCL, to see if it imparts a therapeutic benefit over previous gene therapy approaches. If these studies are successful, existing large animal biodistribution studies would pave a clear path forward for human translation following appropriate toxicology studies.”

The Principal Investigator, Steven Gray, PhD, is a member of the research team at the Gene Therapy Center at UNC. Dr. Gray is leading the Global Gene Transfer for Batten Disease Project, which is modeled after a project at UNC that is focused on the ultra-rare childhood disease Giant Axonal Neuropathy (GAN).

II – UniQure And Glybera

The European regulatory approval of UniQure’s orphan drug Glybera for the treatment of Lipoprotein Lipase Deficiency (LPLD) in late 2012, is the gene therapy that has received most of the recent media attention because :

•   It is the 1st gene therapy approved for sale in the western world
•   High cost of treatment estimated to cost close to $1 million/patient when it launches in the Summer of 2013 in Europe.

Facts about Glybera

•   Receives FDA Orphan Drug Designation (ODD) May 21, 2007
•   Receives European Commission (EC) orphan designation on March 8, 2004
•   EC approves Glybera’s marketing authorization on October 25, 2012, for LPLD under exceptional circumstances in all 27 EU member states
•   1st in a class of gene therapy products to receive approval in the Western world to treat orphan diseases
•   As part of the approval, it will be offered through “dedicated centers of excellence with expertise in treating LPLD & by specially trained doctors to ensure ongoing safety of this novel treatment paradigm”
•   Company getting ready to apply for regulatory approval in the US, Canada, and other markets.

III – Fabry Disease

A pan-Canadian study headquartered in Toronto, is “believed to be the first gene therapy clinical trial for Fabry Disease in the world.” The study is funded by the :

•   Canadian Institutes of Health Research
•   Kidney Foundation of Canada.

Patients who have Fabry Disease have a change in a gene called GLA and cannot make enough enzyme to break down a fatty substance called Gb3. Gb3 builds up and can lead to kidney, heart, or brain problems, and shortens a person’s life. About 400 Canadians suffer from Fabry Disease. The current treatment is to have Enzyme Replacement Therapy (ERT) every two weeks, which costs approximately $250,000/patient/year.

For this gene therapy study, researchers hope to remove stem cells from a Fabry Disease patient’s blood and insert a working copy of a new gene into them using a specially engineered virus. The plan in the final phase is to transplant the stem cells back into the patient with the hope that it will make the missing enzyme.

Please Note: “ExRNA Carrtoon” National Institute of Health [Public domain] | Wikimedia Commons.

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

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