Rare Diseases And Orphan Drugs: Gene Therapy Business Developments #1

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. There are two types of gene therapy :

•   Somatic gene therapy that introduces therapeutic genes at the tissue or cell level to treat a specific individual
•   Germ-line gene therapy that inserts genes into reproductive cells/embryos to correct genetic defects that can be passed on to future generations.

Innovative gene therapy, which has the “potential to cure lethal diseases by enabling normal genes to take over for defective ones” gained momentum in 2012 with the :

•   Orphan designation by both the FDA and European Medicines Agency (EMA) for bluebird bio’s gene therapy product for Adrenoleukodystrophy (ALD)

and the
•   European Union (EU) approval of orphan designated Glybera – the “1^st gene-therapy medicine approval in the Western world”, for the treatment of a lipoprotein lipase deficiency.

This is a first 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. Future Blog Posts will discuss and review gene therapy for such rare diseases as :

•   Batten Disease
•   Canavan disease
•   Giant Axonal Neuropathy (GAN).

I – Bluebird bio

Bluebird bio, a Cambridge, Massachusetts biotechnology company, develops next generation products based on gene therapy to treat severe genetic and rare diseases. The company has two clinical stage products in development for the following rare diseases :

•   Childhood Cerebral Adrenoleukodystrophy (CCALD)
•   Beta-Thalassemia/Sickle Cell disease.

Also, bluebird bio recently partnered with Celgene, to form a global collaboration to “discover, develop, and commercialize novel disease-altering gene therapies in oncology”. On May 14, 2013, bluebird bio files a $86.25 million IPO, with the plan to list on The Nasdaq Global Market under the symbol “BLUE”.  J.P. Morgan and Bank of America Merrill Lynch are leading the offering.

II – Lentigen

Lentigen is a biologics company (Gaithersburg, Maryland) developing and commercializing breakthrough treatments for human disease based on Lentiviral Vectors (LV), the company’s technology platform. LV is a method of delivering genetic sequence information into cells to reprogram their function. Lentigen receives FDA Orphan Drug Designation in January 2013 for the novel gene therapy, P140K  Methylguanine Methyltransferase (MGMT) transduced human CD34 cells (LG631-CD34), for bone marrow protection in the treatment of glioblastoma multiforme.

III – Lysogene

Lysogene, founded in 2009 by Karen Aiach, is a clinical stage biotechnology company, specializing in gene therapy that targets severe genetic pathologies of the central nervous system in children. The company announces in May 2013, that its lead gene therapy product SAF-301 for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA or Sanfilippo Type A Syndrome) receives FDA Orphan Drug Designation (ODD) on May 6, 2013. In September 2010, the European Commission grants SAF-301 orphan designation for the same indication. SAF-301 is an adeno-associated viral vector serotype rh.10 carrying the human SGSH and SUMF1 cDNAs. It is currently under investigation in a Phase I/II clinical trial (NCT01474343) conducted in France. This is an “ … open-label, single arm, monocentric, Phase I/II clinical study …. with direct injection of the investigational medicinal product SAF-301 to both sides of the brain …”.

MPS IIIA is an inherited disease caused by the lack of an enzyme called N-Sulfoglucosamine Sulfohydrolase, which is required for the break down of a substance in the body called heparin sulphate. As a result of not having the capability to break down heparin sulphate, there is a gradual build up the substance in the cells of the body, particularly in the brain, resulting in a range of symptoms – learning disabilities and behavioural  problems. MPS IIIA is usually diagnosed in children – life expectancy is between 10 and 20 years old.

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

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