You are invited to attend a presentation by:
ORPHAGENIX, a private biopharmaceutical company
Monday, May 12, 2008, 6.30 PM
at the Valley Forge Suites Hotel (directions below)
To register please click here: www.rxpcci.com
and follow directions
ORPHAGENIX is a privately held therapeutics company in Wilmington, DE with the vision to correct single point mutations in genes through the Company's proprietary gene editing technology, Targeted Gene Alteration. Several Orphan Diseases such as Sickle Cell Disease, Alpha-1
Antitrypsin Deficiency, and Spinal Muscular Atrophy are ideal targets for gene editing since correcting a single point mutation in the gene could lead to a cure for these diseases. These initial targets were selected from over 4,000 monogenetic diseases, and the Company is now ready to enter development with the lead target candidate. OrphageniX also acquired proprietary guanosine-rich oligonucleotide (GRO) technology focused on Cancer and Huntington's disease utilizing guanosine-rich DNA aptamers. Targeted Gene Alteration was developed to correct inborn base pair errors utilizing unique oligonucleotide sequences that exploit the innate gene repair machinery in the nucleus of the cell. The oligonucleotides enter the cell, bind to the complementary sequence and create one mismatch at the mutation site. The mismatch directs endogenous DNA repair enzymes to correct the single point mutation. Cells that are repaired with TGA can grow and divide with normal transcription and regulation. Correcting enough cells can lead to a cure.
PROGRAM
Cocktails and Dinner (6.30)
(There will be a cash bar and a special two-entree buffet menu)
Michael Herr, President and CEO, will deliver the Company's "Elevator" Pitch to the Group (8.00)
A Panel will address 3 Major Issues for the Company (8.15),
these are:
1. What pre-clinical (and/or clinical) data sets provide OrphageniX with a substantial valuation increase?
2. Which delivery technologies/companies would be ideal partners for OrphageniX in each target area (SCD, SMA, A1ATD)?
3. OrphageniX also has a straightforward cancer program based on a DNA aptamer that induces apoptosis (not gene correction). What is the best way to monetize the GRO asset?
Open discussion (9.00):
What is the perception of Targeted Gene Alteration compared to siRNA and whole gene transfer technologies with respect to its "readiness" as a therapy?
Remember to register by clicking on www.rxpcci.com and then following directions
Dinner prices are a flat $40, parking is free!
Lifetime dues for new members remain at $100. Join on May 12 and dinner is on us!
PCCI meetings are livelier and more interesting than ever. All agree the Group has much to offer, both to the outside world, and to each other. So bring a business colleague!
The Valley Forge Suites Hotel provides an excellent facility, more room and a fine menu!
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Pennsylvania Bio Program "The Finance Cycle of a Bioscience Firm: A Case Study"
June 10, 2008
Desmond Hotel
Malvern, PA
Breakfast: 7:30 a.m.
Program: 8:15 a.m. - 10:00 a.m.
John Gill, President & CEO, TetraLogic Pharmaceuticals
Federica O'Brien, Vice President and CFO, Cardiokine, Inc.
Barbara Schilberg, Managing Director & CEO, BioAdvance
Registration Fees:
Pennsylvania Bio Members: $35
Non-Members: $60
Register at www.pennsylvaniabio.com/
















