Blind World

New Hope for Families Dealing With Alzheimer's, AIDS and Age-Related Macular Degeneration.

May 4, 2004.
Press Release.
SOURCE American Association of Pharmaceutical Scientists.

Key Health Issues Presented at This Year's National Biotechnology Conference of the American Association of Pharmaceutical Scientists (AAPS) Will be Held May 16-18 in Boston

Pharmaceutical science leaders from the FDA, research institutions and top pharmaceutical companies will gather at the 2004 AAPS National Biotechnology Conference, AAPS' spring annual meeting. Research presented will highlight advances in treatment methods for AIDS, Alzheimer's and age-related blindness, providing patients and families with innovative biotechnology-based therapies.

In response to AAPS members' needs, the National Biotechnology Conference is an annual event that incorporates all aspects of the pharmaceutical sciences and provides an open forum for discussion of hot industry topics. The conference will take place in Boston, a popular biotechnology hub, at the Westin Copley Place, May 16 -18. The following are topics of interest:

Key Opinion Leaders Take the Stage to Discuss Advances in Research -- In its commitment to provide cutting-edge educational workshops and networking opportunities, AAPS will host sessions featuring key opinion leaders from top research firms and institutions.

Out of the Darkness: Treating a Major Cause of Blindness -- The topic of Ribonucleic Acid Interference (RNAi) treatments will take center stage at the conference. RNAi is a novel technique using short molecules of RNA (siRNAs) that, when introduced into a human cell, can "turn off" or block the function of a specific gene. This naturally occurring cellular mechanism has great promise to treat diseases such as cancer, Hepatitis C, HIV and certain eye diseases. RNAi was named Science magazine's "Breakthrough Technology of 2002" and one of Technology Review's "Top 10 Emerging Technologies for 2004." Dr. Nassim Usman of Sirna Therapeutics will present results from the company's work on an RNAi treatment for Age-Related Macular Degeneration (AMD), the leading cause of irreversible blindness in people over the age of 55. Dr. Usman will provide a timetable for the first projected RNAi clinical trials in humans.

Combining Forces: RNAi and Gene Therapy Unite for HIV Treatment -- With an estimated 40 million people worldwide living with HIV, effective treatment is in great demand. Dr. John Rossi, Professor and Chair of the City of Hope's Beckman Research Institute's Department of Molecular Biology, will be presenting conclusions to his pre-clinical data for HIV treatment. Expanding on current HIV treatments, which use chemotherapy cocktails to attack the virus at all stages, Dr. Rossi's approach uses the latest techniques in biotechnology. Using RNAi and gene therapy, his method attacks the viral life cycle to prevent the spread of the virus to other cells and make the cells resistant to HIV.

Potential "Biogeneric" Opportunities for the Generic Pharmaceutical Industry -- In the next few years, over $10 billion in biologics, including such products as insulin and growth hormone, lose their patent protection and become available for generic competition. While industry leaders are trying to protect their assets, the growing market could provide great opportunities for generic manufacturers. Current guidance for submitting a generic biologics are vague, and the FDA wants to proceed carefully to ensure safety and efficacy. Under current guidelines, the typical cost savings associated with traditional generic drugs may also not be possible. On Tuesday, May 18 from 1:00 pm - 3:30 pm, representatives from the FDA, USP and the pharmaceutical industry will participate in an open discussion about the regulatory issues concerning generic biologics. Media is invited to attend this lively discussion or schedule an interview with session moderator Dr. Robert Bell of Drug and Biotechnology Development, LLC.

Stem Cell Research Takes a Turn -- "Sonic Hedgehog" may sound like the name of the popular video game, but it is also the name of a protein signal that could possibly cure Alzheimer's disease. Dr. David Schaffer and his team at the University of California, Berkeley use this protein to stimulate adult stem cells to divide into the much-needed neurons that are lost in the disease. In this revolutionary method, the body will be able to turn its own stem cells into neural cells, a perfect tissue match is achieved and the need for embryonic stem cells is eliminated. Where current treatments focus on saving remaining neurons, this new method allows the body to make the neurons it so desperately needs and could cut back on the current yearly care costs of over $100 billion for the 4.5 million Americans suffering from Alzheimer's.

Dr. William Haseltine of Human Genome Sciences will be on-site and available for interviews following his presentation "The Impact of Genomics on Biotechnology." Dr. Haseltine's presentation will be the highlight of the "Future of Biotechnology" session.

About AAPS The American Association of Pharmaceutical Scientists (AAPS) is a professional, scientific society of more than 11,500 members employed in academia, industry, government and other research institutes worldwide. Founded in 1986, AAPS provides a dynamic international forum for the exchange of knowledge among scientists to enhance their contributions to health. AAPS offers timely scientific programs, on-going education, information resources, opportunities for networking and professional development. For more information about AAPS, visit us at

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