BioMarin Pharmaceutical Inc. (NasdaqGS: BMRN ) BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company’s product portfolio comprises three approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan® (sapropterin dihydrochloride) Tablets, a product for the treatment of phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of peripheral arterial disease and sickle cell disease, and PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase 1 clinical development for the treatment of PKU.

        We also have a fully paid-up, non-exclusive license to more than 20 U.S. and more than 50 foreign granted patents directed to various display library technologies as a result of our acquisition of Cosmix Verwaltungs GmbH, or Cosmix, which covers other rights related to our Extreme Diversity platform. These include patents that have been granted in the U.S., Canada, China, Europe, India, Israel, Japan,

Hi Josh, Very nice summary of epigenetic cancer theory. Weinberg first presented that theory @ 1983 and showed demethylation of cancer cells. Major theory is cancer due to new mutations genes and minor theory due to epigenetic changes due to demethylation. Plants started @ 500 million years ago and began changing atmosphere from CO2 rich to more oxygen. Idea that original life resembled cancer from astrophysicist, (don’t remember name) but you mentioned you were big fan of his. Intuitively, hard to understand how cancer could be so metabolically robust if had to rely on de novo mutations. On the other hand, if cancer was a life form that had evolved over a billion years; then makes sense that cancer is such a robust life form. The other intuitive problem with mutation theory is as cancer becomes more undifferentiated, they all look the same. One would think if each cancer was it’s own set of new and different random mutations, cancers would all look very unique. As regards poorly differentiated cancer, microscopically, you’ve seen one cancer, you’ve seen them all. As regards continuing need for oxygen free growth; human fetus needs that ability in first week of life, until implantation in uterus and finally gets an oxygen rich blood supply. A four day old fetus looks a lot more like cancer than adult tissue.

Hale, A. E., White, K. & Chan, S. Y. Hypoxamirs in Pulmonary Hypertension: Breathing New Life into Pulmonary Vascular Research. Cardiovasc. Diagn. Ther. 2, 200–212 (2012).

Alimera Sciences, Inc. ( NasdaqGM:ALIM ) based in Alpharetta, Georgia, is a biopharmaceutical company that specializes in the research, development and commercialization of prescription ophthalmic pharmaceuticals. Presently the Company is focused on diseases affecting the back of the eye, or retina. Its advanced product candidate Iluvien(R) is an intravitreal insert containing fluocinolone acetonide, a non-proprietary corticosteroid with demonstrated efficacy in the treatment of ocular disease. Iluvien is in development for the treatment of diabetic macular edema (DME), a disease of the retina that affects individuals with diabetes and can lead to severe vision loss and blindness

HIF-dependent gene regulation was performed by a dual reporter gene assay, using a firefly luciferase construct and a reference Renilla luciferase construct pRL-TK (Promega, Madison, WI). A putative promoter of miR-322 containing the consensus sequence of the HRE was constructed by PCR from rat genomic DNA by using the primers: 5- CCG CTC GAG TAC TTA GTT TAA CTA CAG ACT C -3 (forward) and 5- CGA CGC GTG GGG CCG CTC TGG GGT ACC TGC -3 (reverse). Luciferase activity was measured in cell extracts with a Lumat LB9508 luminometer (Berthold, Bad Wildbad, Germany).

FDA approval could be more complicated because they need to target one disease in particular though, and they would probably need to focus on a very serious disease to justify gene therapy.

Taylor, G. A. et al. Behavioral characterization of P311 knockout mice. Genes, brain, and behavior. 7, 786–795 (2008).

        Under the Pediatric Research Equity Act of 2003, an NDA or supplement thereto must contain data that are adequate to assess the safety and effectiveness of the drug product for the claimed indications in all relevant pediatric subpopulations, and to support dosing and administration for each pediatric subpopulation for which the product is safe and effective. With enactment of the Food and Drug Administration Safety and Innovation Act, or the FDASIA, in 2012, sponsors must also submit pediatric study plans prior to the assessment data. Those plans must contain an outline of the proposed pediatric study or studies the applicant plans to conduct, including study objectives and design, any deferral or waiver requests, and other information required by regulation. The applicant, the FDA, and

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NAD+/NADH ratio upregulates fission and hence mitophagy, so this is affected by the CR pathway, although there is likely additional benefit to upregulating it directly. But I believe it also upregulates DNA repair, which is something different.

Catalent announces investment for its Zydis ODT technology, offering increased drug load and taste-masking capabilities.