Category Archives: Genetic Disease

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

Top 17 Cancer Drugs on Breakthrough Therapy List

The FDA is evaluating proposals for new indications for at least 17 oncology medicines, according to Friends of Cancer Research. The list of therapies includes novel agents and existing drugs whose uses could be expanded if the agency approves the applications.

The breakthrough therapy program is designed to streamline drug development for therapies to treat serious and life-threatening illnesses. The FDA grants the status based on whether a therapy would provide a substantial improvement over existing options based on preliminary clinical evidence.

The Friends advocacy group helped promote the concept … Read more