The project is the result of an initiative of Spanish people affected by this rare disease. FARA, one of the main patients' associations in the United States now joins the endeavour The gene therapy-based research project to tackle ...
Tags: FARA, CBMSO, GENEFA Platform, IRB
A new gene therapy that successfully treated a rare eye disease in clinical trials could prove the key to preventing more common inherited causes of blindness, researchers say. In six male patients, doctors used a virus to repair a ...
A new, preliminary treatment involving triple-gene therapy appears safe and effective in helping to control motor function in Parkinson's disease patients, according to new research. The therapy, called ProSavin, works by reprogramming ...
Preliminary research shows that gene therapy might one day be a powerful weapon against leukemia and other blood cancers. The experimental treatment coaxed certain blood cells into targeting and destroying cancer cells, according to ...
Tags: gene therapy, leukemia, blood cancer
VBL Therapeutics has secured fast track status from the US Food and Drug Administration (FDA) for its lead oncology drug VB-111, for prolongation of survival in patients with Recurrent Glioblastoma Multiforme (rGBM). VB-111 was already ...
Editas Medicine, a transformative genome editing company, today announced it has secured a $43m Series A financing led by Flagship Ventures, Polaris Partners and Third Rock Ventures with participation from Partners Innovation Fund. ...
Tags: Editas Medicine, genome editing
Nuvilex, an international biotechnology company providing cell and gene therapy solutions for the treatment of diseases, has acquired the exclusive worldwide rights to use the cellulose-based live-cell encapsulation technology for the ...
Tags: Nuvilex, Live-Cell Encapsulation Technology, biotechnology, cell, gene
Fidelity Biosciences, a subsidiary of Fidelity Investments, and US-based Regenx Biosciences have announced the formation of Dimension Therapeutics, a gene therapy company focused on developing novel treatments for rare diseases. Dimension ...
Tags: Biosciences, Health, Medicine
US-based biotechnology company Veritas Bio received a patent related to in vivo delivery of nucleic acids from the European Patent Office. Inventor Dr Pachuk noted the allowed claims cover transfecting nucleic acids into skin or muscle ...
The parents of a Naperville boy with Duchenne muscular dystrophy are hoping sales of a product they invented and recently launched will help provide research funds and renewed hope for him and others with the rare disorder. Marty and Geri ...
Tags: toy
Biotechnology company bluebird bio has formed a broad collaboration with Celgene to develop new disease-altering gene therapies in oncology. The multi-year research and development collaboration aims to apply gene therapy technology to ...
New research from Mayo Clinic and Banner Sun Health Research Institute has demonstrated that testing a portion of saliva gland could be a way to diagnose Parkinson's disease. The study, which enrolled 15-patient, collected biopsies from ...
The US Patent and Trademark Office (USPTO) has granted a notice of allowance (NOA) to Sanovas' minimally invasive nested balloon catheter for localized drug delivery. The patent will protect an integral part of the company's NanoVas ...
Tags: USPTO, NOA, minimally invasive nested balloon catheter, medical product
The US FDA has granted orphan drug designation to Milo Biotechnology's AAV1-FS344 to treat Becker and Duchenne muscular dystrophy. AAV1-FS344 is myostatin inhibitor delivered through gene therapy to increase muscle strength in patients. ...
Tags: FDA, orphan drug designation, US
Baxter International has collaborated with Chatham Therapeutics for the development and commercialization of factor IX hemophilia B gene therapy treatment. According to the agreement, the companies will investigate the next generation of ...
Tags: IX hemophilia B gene therapy, Biological Nano Particles, clinical trials