Since the first gene therapy trials were conducted 25 years ago, there have been high expectations from the public, and much attention from investors, that previously incurable diseases would be cured by gene therapy. Still, despite numerous gene therapy clinical trials for many different indications, there are no approved gene therapy drugs in the United States. In 1999, one gene therapy patient died during clinical trials, the first ever. This highly publicized event led to heightened regulatory scrutiny over all such trials. Then in 2003 and 2005, three subjects developed leukemia as a direct consequence of gene therapy; one of them eventually passed away. The regulatory response stemming from these incidents led to greater regulatory oversight in gene therapy, as compared to other investigational drugs and biologics…
Category: <span>Cell & Gene Therapy</span>
Acute Renal Failure (ARF) is a severe inflammatory disease state often accompanied by multiple organ failure (MOF). ARF is precipitated by many factors such as blood loss, surgery, sepsis, toxins, trauma, and is most often linked to the loss of kidney tubule function. Proximal tubule cells are specifically injured in acute renal failure. Current therapies for ARF involve conventional kidney support with hemodialysis or hemofiltration. These therapies offer replacement of normal renal functions such as waste removal, fluid, and electrolyte balance, but they cannot provide vital endocrinological and metabolic functions of a healthy kidney. Despite advances in synthetic materials and extracorporeal circuits for hemodialysis and hemofiltration, ARF is associated with a high mortality rate ranging between 55–70 percent…
The Intraocular Delivery of Neuroprotective Factors to the Retina Using Encapsulated Cell Technology
Ophthalmic disorders are a group of diseases with a rapidly increasing frequency associated with an increase in the aged population. Patients with potentially blinding diseases have become one of the largest segments of the healthcare field, with more than 50 million patients in the United States alone. Their sight is threatened by diseases such as age-related macular degeneration (AMD), diabetic retinopathy (DR), glaucoma, or retinitis pigmentosa (RP). Until recently, there were essentially no effective treatment options to halt the progression of chronic, potentially blinding diseases. Biotechnological advances have resulted in the development of a variety of promising new protein factors that, if delivered to diseased cells of the retina, hold promise for treatment by interrupting or reversing the disease process…
The United States Pharmacopeia (USP) is a 184-year-old organization that has been in the forefront of technology since its inception. From publishing a manual about how to prepare therapeutic potions, USP has evolved into a compendium of standards and information on manufactured pharmaceutical products, with more than 4,000 monographs covering drug substances and biologics, and their dosage forms, excipients, and nutritional supplements. It is not surprising that the USP initiative in cell and gene therapy and tissue engineering has closely followed the emergence of these technologies…
Neurodegenerative diseases such as Parkinson’s disease and multiple sclerosis, along with injuries such as stroke affect millions of individuals worldwide and costs healthcare systems billions of dollars each year in North America alone. The diseases result from the death of specific cell types within the central nervous system. Current treatment efforts have focused primarily on alleviating symptoms using pharmaceuticals. However, recent advances in our understanding of these conditions, coupled with advances in biology, genomics, transplantation, and biochemical engineering are making cell therapy (the transplantation of viable cells to replace dead cells) more attractive as a potential avenue of treatment…
Current in vivo gene therapy (GT) approaches are beginning to demonstrate significant clinical and safety limitations that may ultimately reduce their therapeutic utility. In particular, the potential for systemic toxicity due to the antigenicity of the gene transfer vector, the prospect of insertional mutagenesis/oncogenesis during gene transfer, and the possibility of germ line transfer of the transgene are issues raising concern. One promising alternative to gene therapy that mitigates these clinical and safety issues is gene-based cell therapy (GBCT), in which autologous cells are removed from a patient and modified ex vivo for a desired characteristic prior to reimplantation. By transferring the transgene ex vivo, many of the issues surrounding the in vivo use of the transfer vectors are reduced and issues surrounding germ line transfer can be practically eliminated…