What if mRNA could be a drug – Moderna Therapeutics

Modified messenger RNA (mRNA) Therapeutics™ can be encoded for virtually any known protein and designed to be taken up by the cells in specific tissues and organs. Our novel chemistry enables mRNA to elude the body’s innate immune response. Once delivered, like native mRNA in healthy individuals, mRNA Therapeutics™ act as cellular software directing ribosomes to express proteins or antibodies within targeted tissues – and have the power to catalyze the expression of hundreds to thousands of proteins for each mRNA molecule.

mRNA drugs drive therapeutic benefit by virtue of … Read more

How CRISPR-Cas9 Works ?


Researchers are developing ways to use CRISPR to treat genetic conditions like sickle-cell anemia and cystic fibrosis, and are also experimenting with genetic changes that could eliminate viruses like HIV. Even though viruses aren’t genetic diseases, certain gene edits have been shown to prevent the virus from spreading to new cells and to “destroy inactive HIV residing in the human genome by altering critical viral genes,” according to a look at genome surgery in MIT Tech Review. Experts even think these types of changes could eventually help treat complex conditions … Read more

Gene Therapy Technology Companies

Gene therapy is getting major attention again this month with the recently announced deal between Bristol-Myers Squibb and uniQure and upcoming top-line data from Celladon’s Phase 2 CUPID 2 study in heart failure.

We believe this renewed attention to the space could be translated into growth opportunities for companies in the field as well as a potential boost for three Buy-rated names in our universe: Applied Genetic Technologies (ticker: AGTC), GenVec ( GNVC ) and Oxford Biomedica ( OXBDF ).

Earlier in April, Buy-rated Bristol-Myers ( BMY ) and uniQure … Read more

OvaScience’s – fertility technology

OvaScience’s proprietary technology platform leverages the breakthrough discovery of egg precursor (EggPCSM) cells. EggPC cells are immature egg cells found inside the protective ovarian lining that have the potential to be matured into new, fertilizable eggs. It was long believed a woman was born with a set number of eggs that die over time. The presence of EggPC cells changes that fundamental understanding about female biology, and opens up extraordinary possibilities to help women improve their fertility.

EggPC cells were first discovered by one of OvaScience’s scientific founders, Jonathan Tilly, … Read more

Last 20 Oncologic Drugs Approved

From January 1, 2009, to December 31, 2013, the US FDA approved 51 drugs in oncology for 63 indications. During this time, 9 drugs received more than 1 approved indication. The Table lists the last 20 drugs (total of 21 approvals) approved by the FDA and their median wholesale prices.

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