2012 BIO Investor Forum: Presenting Companies Making News

Need a heart valve? Perhaps a bit more cartilage in your knees? Better yet, how about new blood vessels to bypass the old blocked ones? Amazingly, the days of relying upon animal or human cadavers’ tissue grafts to address these medical needs may possibly be drawing to a close.

This is not Star Trek and this is not the Walt Disney’s optimistic science of Tomorrowland. As detailed in the Wall Street Journal last week, medical researchers are already conducting R&D focused on producing living tissue from inkjet printers.

San Diego-based Organovo introduced the first commercial 3-D bioprinters in 2010. These medical printers aim to use an ink made of living cells to build up tissue structures, layer by layer, into human tissue.

While this machine does exist and has many potential uses, it will likely be several years before we begin to see clinical trials involving the production of personalized tissue, such as of blood vessels, skin or muscle.

Organovo is just one example of the 110 innovative life science companies that will be participating in next month’s 11th Annual BIO Investor Forum – held October 9-10, 2012 at the Palace Hotel in CA.

Hoping to learn about more of the “fierce” companies participating in this year’s Investor Forum?  FierceBiotech recently came out with their annual Fierce 15 list and two BIO Investor Forum presenting companies made the list.

bluebird bio, based in Cambridge, MA, is in the process of developing innovative gene therapies for severe genetic disorders. According to FierceBiotech, they are positioned at the crossroads of a resurgent gene therapy technology and the hot development space for new drugs that may treat rare diseases.

Just weeks ago, the company announced that it won orphan status in the US and Europe for its lead program, a gene therapy that is in development to treat adrenoleukodystrophy (ALD).

Prosensa, a Dutch biopharmaceutical company focused on the discovery, development and commercialization of RNA modulating therapies focuses on genetic disorders with a large unmet medical need, primarily neuromuscular disorders.

Prosensa and GlaxoSmithKline (GSK) will report Phase III results for PRO051/GSK-2402968 – an exon 51 skipping RNA drug for the treatment of Duchenne Muscular Dystrophy (DMD) – in the second half of 2013.

Bluebird BIO CEO, Nick Leschly and Prosensa CEO, Hans GCP Schikan will discuss their R&D activities focused on rare diseases at a Therapeutic Workshop offered at the Investor Forum.

Raring to Go: The Race to Treat Ultra-Rare Diseases

The signing of FDASIA by President Obama in July reinforces the country’s commitment to fostering innovation for rare diseases.  With a record 460 medicines in late-stage clinical trials, biopharmaceutical companies have embraced rare disease medicine and are rapidly developing the pipeline.  This panel will provide an in-depth analysis of this thriving market opportunity by featuring companies specializing in the ultra rare disease market alongside clinical and policy/regulatory experts in the field.

Wednesday, October 10th, 2:00 p.m. – 2:55 p.m.

Moderator: Ellen Licking, Senior Writer & Analyst, Real Endpoints

Panelists: E. Cartier Esham, PhD, Senior Director, Emerging Companies Health Affairs, BIO; Emil Kakkis, MD, PhD, Chief Executive Officer & President, Ultragenex Pharmaceutical; Nick Leschly, CEO, bluebird bio and Hans GCP Schikan, PharmD, CEO, Prosensa

The forum, or BIF as we call it around here, is an international investor event that explores trends and opportunities in life sciences. Broadly speaking, the event features venture-stage growth and emerging public companies.

To learn more about the Therapeutic Workshops or BIO Investor Forum, please visit here. In the meantime, be sure to check back often as I continue to plug away at my company snapshot campaign.

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