GeneQuine develops drugs that are based on gene therapy. The concept of gene therapy is to introduce genetic material into the patient’s own cells in the body. The cells produce then a therapeutic protein according to the template that the introduced genetic material (DNA) provides
GeneQuine is focused on the development of gene therapy agents for the treatment of osteoarthritis. Osteoarthritis is a degenerative joint disorder characterized by cartilage loss and inflammation. Patients affected by osteoarthritis experience joint pain as well as swelling and stiffness of the joints leading to … Read more
Gene therapy is a well-suited approach for the treatment of SMA due to the monogenic nature of the disease—meaning it’s caused by the deletion of or mutations in a single gene. AVXS-101 is our clinical-stage, proprietary gene therapy candidate of a one-time, intravenous treatment for SMA Type 1—designed to prevent further muscle degeneration caused by SMA through:
Delivery of a fully functional human SMN gene into target motor neuron cells
Production of sufficient levels of SMN protein required to improve motor neuron function
Rapid onset of effect in addition to … Read more
Tim Miller, Ph.D., President & CEO Dallas, TX
PlasmaTech Biopharmaceuticals/Abeona Therapeutics is focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases. PlasmaTech’s lead program is a gene therapy for Sanfilippo syndrome (MPS IIIA and IIIB) in collaboration with patient advocate groups, researchers and clinicians. In addition, the company is pursuing two additional proprietary platforms, Salt Diafiltration (SDF™) Process and Polymer Hydrogel Technology (PHT™), and is active in the development and commercialization of human plasma-derived therapeutics, including its proprietary
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
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 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
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
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