Category Archives: Rnai

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

Cystic fibrosis Cure with RNAi Technology – ProQR

What is cystic fibrosis?
Cystic fibrosis (CF) is a genetic disease affecting more than 70,000 patients worldwide. CF patients have viscous mucus accumulating in their vital organs. The mucus clogs tubes and organs thereby disrupting several processes in the body.

Pancreatic enzymes, necessary to digest food, are blocked from entering the intestines, so CF patients are not able to digest food by themselves. The thick layer of mucus in the lungs is a great environment for destructive bacteria. The thick mucus makes it hard to clear the lungs from these … Read more

CRISPR/Cas9 System for Targeted Genome Modification

The CRISPR/Cas9 system, Origene’s latest tool in genome editing, is a complete system for highly specific gene knockout with low cell toxicity. The CRISPR/Cas9 technology uses co-expression of a Cas9 protein with a guide RNA vector expressed from the human U6 polymerase III promoter. Cas9 unwinds the DNA duplex and cleaves both strands upon recognition of a target sequence by the guide RNA.

CRISPR/Cas9 Kit Features:
Complete kit for gene knockout using CRISPR
Two guide RNA vectors in for efficient cleavage
Knockin GFP-Puro for selection
pCas-Guide-scramble included as a negative … Read more

CureVac – Messenger RNA (mRNA) Technology

CureVac is pioneering the use of natural and chemically unmodified mRNA as a data carrier to instruct the human body to produce its own proteins capable of fighting a wide range of diseases. CureVac’s novel technology is the broadest and most advanced mRNA therapeutic platform and allows for rapid, low-cost production of multiple drugs and vaccines. Additionally, CureVac’s mRNA vaccines are thermostable, which eliminates the demand for cold-chain storage and infrastructure, a major challenge in the vaccine supply of most developing countries.

CureVac’s RNA Technology – Inspired by Nature


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Novartis Sold its RNAi Technology to Arrowhead

Arrowhead acquires patents as well as intellectual property rights to Alnylam’s RNAi tech, 30 gene targets Novartis has picked from its partnership and a pipeline with three candidates that had generated preclinical data. And it follows their 2011 deal to buy out Roche’s spurned RNAi work, providing some added boasting rights to being a leader in the field.

“This is an important deal for us. Novartis has been working in the RNAi field for over a decade and their developments in proprietary oligonucleotide formatting and modifications are some of the … Read more

The CRISPR/Cas9 System & Genome editing

Clustered regularly interspaced short palindromic repeats (CRISPR) and a CRISPR associated protein (Cas9) comprise a system adapted from bacterial DNA known as CRISPR/Cas9. The CRISPR/Cas9 system act as an adaptive immune system allowing bacteria to recognize foreign DNA and cut it, destroying an invading virus or pathogen. CRISPR/Cas9 has been adapted to other organisms to make specific cuts in their genomic DNA.


CRISPR/Cas9 technology holds the potential to “edit” a patient’s genome, restoring genes with disease-causing mutations to their normal healthy sequence, deleting disease-causing genes, or improving genes … Read more