Personalized Treatment Products
Haplomics is building on its discoveries of multiple distinct genetic haplotypes of FVIII, its growing patent portfolio, and its research relationship with its world class collaborators, to bring personalized (“haplotype-matched”) replacement FVIII products to the market. These personalized replacement FVIII products will be in the form of bio-therapeutics and companion diagnostics that include: (i) an extended spectrum of haplotype-pure FVIII concentrates (both plasma-derived and recombinant) for low immunogenicity protein replacement therapy; and (ii) FVIII-peptide and FVIII-protein-based drugs for pharmacogenetically-guided immune-tolerance induction (ITI) therapy. These products have the potential to reduce the incidence of inhibitor development in African American Hemophilia A (HA) patients by half and affect a general reduction in the frequency of inhibitor development for all HA patients.
Engineered Gene Repair
Using the latest genetic engineering tools, Haplomics has developed proprietary genetic engineering methodologies to repair the gene mutations (the errors in the DNA) of hemophilia patients. Haplomics is using state-of-the-art technology to genetically repair a cultured cell-line derived from a hemophilia patient with the most common gene mutation, the Intron 22 Inversion (I22I) mutant F8 gene. The I22I mutation occurs in 45% of all severe HA patients. With the repair, HA I22I patients should be able to tolerate receiving injected replacement FVIII. This initial genetic repair should result in patients producing clotting factor from their own cells which their immune system will recognize as self-protein and thereby prelude the development of inhibitors. Coupling this therapy with the haplotype-matched FVIII products mentioned above is expected to prove particularly effective.
Cure for Hemophilia A
Haplomics’ primary mission extends beyond improvements in the treatment of hemophilia to development of a cure. Ultimately, Haplomics believes that it can affect precise repairs to the I22I mutant F8 gene so that HA patients will begin producing sufficient functional FVIII on their own to stop bleeding and no longer require treatments at all, i.e., a cure. Success with repairing the I22I mutation is expected to lead to the ability to repair the other common hemophilia F8 gene mutations, curing patients with these variants as well.
For more about hemophilia, click here.
For more about inhibitor development, click here.