Tuesday, February 25, 2014

Gene Therapy: Hopes to deliver!!!


The field of gene therapy has still been evolving and improving for several years, after suffering a setback due to the death of a SCID (Severe Combined Immune Deficiency) patient, as a result of adverse effect of therapeutic gene transfer. At present, several new companies, in partnership with leading gene therapy researchers, have been coming up with novel treatments for various diseases, not just restricting  to genetic disease, but extending the potential to different cancers.

Here, is a list of few of the Gene therapy companies, which are potentially set-up for changing the medical treatment scenario in coming years:

1. SPARK Technologies, Philadelphia, the roots of the origin of this company lies with Children's hospital of Philadelphia. This company is likely to complete the clinical trial phase of Leber's congenital Amaurosis, till 2015.

2. Uniqure1 is a Dutch origin company. One of it's drugs, Glybera is currently at marketing stage for treatment of LPL enzyme deficience and therapy of several other diseases like hemophilia, porphyria, parkinsonism are at various stages of clinical trial.
3. Lysogene, Paris - working with lysosomal disorders

4. Editas medicine, Cambridge aims at bringing genome editing technology to human therapeutics.

5. Voyager therapeutics, Cambridge is targeting to develop treatment for several central nervous system diseases.

6. Advantagene1, Massachusetts developed a drug named rostAtak for prostate cancer.

7. GlaxoSmithKline1 - ADA deficiency

8. Vical1 - Melanoma (Phase 3 trial)

9. Applied genetic technologies1 for alpha1 anti-trypsin deficiency

10. Blue bird bio1 - Adrenoleukodystrophy (ald) and beta-thalassemia

11. Celladon1 - Cardiomyopathies

12. Ceregene1 - Parkinson's disease, Alzheimer's disease.

13. Genzyme1, Cambridge - Parkinson's disease

14. Oxford Biomedica1 - Parkinson's disease

15. Shenzen SiBiono Gen-Tech, China1 - Gendicine for lung cancer

 1Moran N. First gene therapy nears landmark European market authorization. Nat Biotechnol. 2012 Sep;30(9):807-9. doi: 10.1038/nbt0912-807.