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
– Advantagene Inc.
– Bluebird Bio
– Human Stem Cells Institute
– Oxford BioMedica Plc
– Shanghai Sunway Biotech Co. Ltd.
– Sibiono GeneTech Co. Ltd.
– Spark Therapeutics, LLC
– UniQure N.V.
– Vical Inc.
– ViroMed Co. Ltd. dba VM BioPharma … Read more
http://www.genengnews.com/insight-and-intelligence/25-young-companies-in-the-money/77900274/#gsaccess… Read more
BioViva‘s CEO Liz Parrish is a fire-starter and probably not everyone’s favorite person. But if your goal is as bold as defeating aging then you’ll have to break some eggs. And so feel as you might but I have to admit that Parrish is definitely one of my favorite people – not only for the scientific work that she does but also because she is a fun, passionate and fully committed individual that inspires me. Thus it was no surprise that, despite some technical difficulties with my set up, I … Read more
A new genetic engineering technology called CRISPR/Cas9 has opened the door to unprecedented tinkering with the human genome. Nature takes a look at the scientific possibilities and the ethical dilemmas they raise.… Read more
Tim Miller, Ph.D., President & CEO
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
RNAi Lead products from top companies.
- ALN-TTR02 (patisiran)
- Vigil immunotherapy
- ALN-TTRsc (revusiran)
- SPC3649 (miravirsen)
Acuity Pharmaceuticals (now OPKO Health)
Alcon (subsidiary of Novartis)
Atugen AG (acquired by Silence Therapeutics)
Calando Pharmaceuticals (subsidiary of Arrowhead)
Cubist Pharmaceuticals (acquired by Merck)
Dharmacon RNAi Technologies (part … Read more