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Monday, May 12, 2008
Embassy Suites, Chesterbrook, Pennsylvania (directions)
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OrphageniX, Inc.
A private biopharmaceutical company
About
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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.
3 Major Issues
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- What pre-clinical (and/or clinical) data sets provide OrphageniX with a substantial valuation increase?
- Which delivery technologies/companies would be ideal partners for OrphageniX in each target area (SCD, SMA, A1ATD)?
- 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
- What is the perception of Targeted Gene Alteration compared to siRNA and whole gene transfer technologies with respect to its "readiness" as a therapy?
Program:
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6:30 - Cocktails & Dinner (Cash bar and special 2-entree buffet menu)
8:00 - Michael Herr, President and CEO will deliver the Company's "Elevator" Pitch to the Group
8:15 - A Panel will address 3 Major Issues for the Company
9:00 - Open discussion: members and guests
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