Biologics New and Improving

BPI Contributor

March 1, 2009

5 Min Read


By far the most successful applications of biotechnology have been in the medical field. The vaccine industry is undergoing a complete transformation thanks to biotechnology. And cutting-edge research is giving us whole new ideas about disease therapy using nucleic acids and regenerative medicine.

Proteins and Other Therapeutics

Cancer has been a primary target for many MAb “magic bullets” and a major research area for life scientists over the past quarter-century. Oncologists have identified ∼200 cancers that affect human beings, some more dangerous than others, some more treatable. The newest mechanisms of action in oncology research include oxidative stress, epigenetics, cancer metabolism, and enzyme inhibition. Some of these are already being tested in clinical trials.

RNA Rx: Gene therapy is based on DNA, but RNA therapy may be the next step. Recent discoveries reveal additional possibilities to target human diseases using treatments such as microRNAs and small RNA-induced gene activation (RNAa).

Regenerative Medicine

The next futuristic concept in biotechnology is the potential to regrow damaged or diseased cells, tissues, organs, and even whole limbs using stem-cell technology. Treating serious illnesses and injuries by using the human body’s own natural regenerative capabilities may be on the horizon.

Regenerative medicine is the newest frontier in health care, and many questions yet remain — beyond the ethics of embryonic stem cells. Targeting stem cells in vivo is the newest idea. A few companies are pursuing conventional therapeutics to target stem cells instead of using cells as a therapies themselves.

Important discoveries are pointing to the chance for modulating cell fate for therapeutic purposes. Whether the aim is to regenerate or repair diseased or damaged tissue, produce personalized regenerative medicines, or create better drug development and research tools, the key is safe and effective modulation of cell fate. At the 2009 BIO International Convention, many researchers and executives leading the development of this technology will meet to discuss current approaches and explore future directions.



Neurogenesis: Depression affects millions of people all over the world, but current drugs used to treat it are inadequate for many potential patients. The 1998 discovery that humans produce new brain cells over their lifetimes (neurogenesis) sparked a new wave of scientific research. Stress appears to suppress neurogenesis in adults, probably due to increased glucocorticoid release — leading some researchers to suggest induced neurogenesis as a treatment for depression. Although clinical-stage research is still in its infancy, such drugs could completely change the way depression and other central nervous system diseases are treated.


The recent rebirth of the vaccine industry is in many parts attributable to advances in biotechnology. Emergence of molecular approaches to diagnosis and treatment of disease has led many people to believe that the era of “blockbuster drugs” is giving way to a new age of specialty products that target genetically defined niche populations. In fact, however, some see a new generation of blockbusters: vaccines.

Harriet Robinson, senior VP of research and development at Geovax Inc., is organizing a session on this topic at the 2009 BIO International Convention. “In this session,” she says, participants will discuss “challenges and setbacks for HIV/AIDS vaccine and highlight ongoing clinical-stage development of vaccines for both prevention and treatment.”

DNA Vaccines: Gene-based vaccines offer potential advantages in development, manufacturing, dosing, safety, and efficacy when compared with conventional live-attenuated, killed pathogen, cell-based, and protein subunit vaccines. Foremost is DNA’s ability to elicit broad, antigen-specific humoral and cellular immune responses without the potential safety issues associated with live pathogens. But researchers have as yet been unable to induce sufficient and consistent immune responses. Even so, this robust platform for rapid, cost-effective vaccine development could ultimately displace many existing vaccines as well as facilitate development of new ones for unmet medical needs.

Recent advances in delivery systems, antigen discovery and optimization, and novel vaccine adjuvants have created renewed interest in the potential of DNA vaccination for numerous disease indications being developed by leading research institutions and companies around the world.

Vaccines As Therapeutics: Novel antigen delivery systems, nanoparticles, RNA targeting, and tolerance-inducing protocols are helping researchers develop therapeutic vaccines to combat autoimmune diseases. The goal is to generate safer, more effective, and more durable therapeutics for many disorders such as type I diabetes, multiple sclerosis, and lupus erythematosus.



As of press time, the exhibit hall product focus zones were only beginning to fill up. For updates, visit the BIO exhibitor floor-plan at

The Therapeutics Zone

These exhibitors will showcase products and services related to RNA interference, gene silencing, human therapeutic proteins, and stem cell therapies.

Exhibitors (as of 1 February 2009)

RNL Bio (Booth #591)


Drug Discovery and Development Sessions: Monday 18 May 2009

Mastering Your (Cell) Fate: Stem Cells, iPSCs, and the Future of Medicine

Drug Discovery and Development Sessions: Tuesday 19 May 2009

The Fountain of Youth: Drugs Targeting Pathways Regulating Aging

Exciting Science Sessions: Tuesday 19 May 2009

Induced Pluripotent Stem Cells Revisited: Where Are We Now, and Where Are We Going?

Exciting Science Sessions: Wednesday 20 May 2009

RNA Rx: The Next Wave of Therapeutics Beyond RNAi

Neurogenesis: Enhancing Brain Cell Growth for the Treatment of Depression

Exciting Science Sessions: Thursday 21 May 2009

The New Frontier of Vaccines for Autoimmune Diseases: Development of Novel Tolerance-Inducing Immunotherapeutics

Fulfilling the Promise of Regenerative Medicine

The “Hottest” New Mechanisms of Action (MOAs) in Cancer

Vaccines Sessions: Wednesday 20 May 2009

Renewing the Promise of DNA Vaccines: Strategies to Enhance Delivery, Efficacy, and Immunogenicity

Blockbusters Aren’t Dead; They’re Just Called Vaccines

Vaccines Sessions: Thursday 21 May 2009

HIV/AIDS Vaccines, Preventative and Therapeutic Approaches to a Global Pandemic

Breaking Tolerance to Boost Vaccine Efficacy

   For complete session information, visit

Vaccines offer tremendous potential to treat a broad range of diseases and generate large amounts of income for the companies involved. The variable nature of vaccine efficacy presents their primary challenge. Scientists at the 2009 BIO International Convention will show true progress on overcoming the obstacles to realizing the industry’s high hopes.

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