2012: A Critical Year for Biotechnology

Posted under biotech regulation, Blog, Companies, Diagnostics, economy, FDA, Food and Drug Administration, Funding, Medical Devices, Medical Supply, Pharmaceuticals, Public Policy, regulation, Startups, therapeutics, Universities, Videos by biotechnow@bio.org (Biotechnology Industry Organization)

In 2012, we must continue to look for ways to reduce risk; enhance reward; reduce uncertainty; and reinforce the position of the industry as a major contributor resolving society’s most pressing needs. Policies to encourage investment in innovation and to speed discovery to scientific breakthroughs simply must be the priority in 2012.

The industry relies on a policy and regulatory environment that encourages innovation for much needed treatments and cures for some of the most devastating and life threatening diseases. BIO will continue to engage with leaders in Washington to ensure that policies support an economic environment that encourages innovation and enables our members to saves lives and transform our world.

BIO has developed a comprehensive national policy strategy – Unleashing the Promise of Biotechnology – designed to incentivize investors, strengthen small business, and promote innovation in the U.S.

The creation of a 21^st century Food and Drug Administration (FDA) is critical right now. Congress must provide the FDA with the resources it needs to keep pace with rapidly evolving biomedical science and fulfill its vital health and safety mission. The FDA must recognizes its national role in advancing innovation by reviewing innovative products in a timely manner and promoting a consistent and science-based decision making process that is reflective of patient needs.

Strengthening the FDA’s review process is also a focus of our ongoing discussions with policymakers about the next renewal of the Prescription Drug User Fee Act (PDUFA V) to get back-to-basics for patients based on principles that are science-based and transparent. The review process should be well-managed to appropriately balance benefits and risks, enhance public trust, and increase patient access to new medicines.

The Therapeutic Discovery Project (TDP) program, enacted in 2010, is an example of the type of policies necessary to spur continued medical innovation, while at the same time protecting and growing high-paying U.S. jobs. The TDP program provided $1 billion in research grants and credits for small biotech companies pursuing new therapies for diseases such as Alzheimer’s, HIV/AIDs, Parkinson’s and MS. BIO is now calling on Congress and the Administration to work together to extend and expand the Therapeutic Discovery Project in early 2012 to support continued American innovation and further accelerate the development of life-saving cures.

Furthermore, current tax law does not do enough to foster investment in health care, green technology, or energy-focused biotechnology companies. Given the economic and societal benefits of ensuring a robust biotech industry, it is imperative that Congress and the Administration adopt policies that recognize the unique financial structure and capital needs of biotech companies.

Other BIO policy priorities include ensuring the National Institutes of Health has sufficient funding to sustain the public-private collaboration that is transforming biomedical discoveries into innovative treatments for patients.

In addition, along with our state affiliates, we will continue to work with states governments seeking to grow their biotech sectors as part of their economic development and job creation strategies.

Foundation Fighting Blindness Invests in Gene Therapy Research Projects

Posted under blindness, Blog, clinical trial, Companies, Diagnostics, Foundation Fighting Blindness, Funding, gene therapy, Health, Medical Devices, Medical Supply, Pharmaceuticals, Startups, therapeutics, Universities, Videos by biotechnow@bio.org (Biotechnology Industry Organization)

Recently, the Foundation Fighting Blindness announced an $8.25 million investment in six new gene therapy research projects targeted to have treatments ready for clinical trials within three years. The projects include the following:

• The Institut de la Vision in Paris and the Friedrich Miescher Institute in Basel, Switzerland, are developing a gene therapy that may revive degenerating cones and potentially enable them to regain their ability to respond to light and provide vision. The treatment also has the potential to improve the health of cones and extend their lifespan significantly. This therapeutic approach holds the potential to benefit people affected by a range of conditions, because it works independently of the underlying disease-causing genetic defect. Resurrecting cones may be able to improve an affected individual’s well being, because these cells provide central, daytime and detailed vision that is critical for independent living.
• The Oklahoma University Health Sciences Center, which in collaboration with Copernicus Therapeutics, is developing a nanoparticle gene therapy delivery system. Nanoparticles are tiny manmade particles, 1/12,000^th the diameter of a human hair, which can potentially penetrate retinal cells, possibly making them effective for delivery of therapeutic genes. They may provide advantages in certain cases over viral gene delivery technologies currently used in retinal disease therapies. Perhaps most beneficial is their potential to deliver large genes — genes that exceed the capacity of viral delivery systems — for treating some diseases.
• AGTC, a clinical stage biotechnology company, funds will support researchers at Oregon Health & Science University’s Casey Eye Institute and the University of Florida in their pre-clinical work to evaluate a gene therapy treatment for X-linked retinoschisis, a blinding disease that affects over 35,000 patients in the U.S. and Europe.
• The Massachusetts Eye and Ear Infirmary and the University of Florida are investigating gene therapy for two different LCA-causing genes.
• The University of Pennsylvania is working on choroideremia gene therapy led by Dr. Jean Bennett, who is also one of the lead investigators on the landmark LCA gene therapy clinical trial that has restored vision in more than 40 patients.

The Foundation Fighting Blindness is a national nonprofit dedicated to advancing sight-saving research. The Foundation funds research toward preventions, treatments and cures for vision-robbing retinal degenerative diseases including macular degeneration, retinitis pigmentosa, Usher syndrome, and several other rare conditions. These newly-funded projects build on the success of landmark gene therapy clinical trials now underway that have restored some vision in children and young adults who were virtually blind.