news

UPV/EHU-University of the Basque Country researchers are using nanotechnology for therapeutic purposes.

This shows the expression of the retinoschisis protein (green) after the transfection of a cell line of retina pigment epithelium (ARPE-19) with a lipid nanoparticle-based formulation with the RS1 gene....

“Donated lungs that have to be discarded because they are not good enough for transplant can now be repaired and made suitable for patients,”The Times has reported.

The study behind the news looks at an experimental gene therapy technique that has been tested on pig and human lungs. Under this technique, a gene known as IL-10 is introduced into the lung tissue cells to alter their behaviour. The research showed that in experimental conditions the gene combatted the damaging inflammation that sometimes causes problems in lung transplants.

In a breakthrough clinical trial on humans, researchers used gene therapy to encourage the heart to repair itself after cardiac damage.

Mounting evidence has pointed towards the heart having a regenerative potential but its capacity for regeneration is insufficient to replace the massive loss of cardiomyocytes following ischaemic damage.

SACRAMENTO, California, USA. Jacob Rutt is a bright 11-year-old who likes to draw detailed maps in his spare time. But the budding geographer has a hard time with physical skills most children take for granted ― running and climbing trees are beyond him, and even walking can be difficult. He was diagnosed with a form of muscular dystrophy known as Duchenne when he was two years old.

AMSTERDAM, The Netherlands, July 20, 2012

uniQure announced today that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion that recommends marketing authorization of Glybera® (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD) under exceptional circumstances. LPLD is a very rare, inherited disease. Patients with the disease are unable to handle fat particles in their blood plasma, which leads to recurring severe abdominal pain and pancreatitis.

NEW YORK – The stem cell and regenerative medicine industry is facing a serious funding problem. Just as the first therapies are reaching commercialization, biotechs in phase 2 and 3 are struggling to raise the funds needed to finish their studies. Over 300 biotechs, pharmas, investors, and suppliers will be meeting in Boston on September 20-21 at the Stem Cells USA and Regenerative Medicine Congress 2012.

The Stem Cells USA & Regenerative Medicine Congress is the region's largest commercial stem cells conference.

Most stem cell companies face two options - go bankrupt or find a partner with deep pockets. Despite there being hundreds of companies with promising technologies, most of them never pass Phase II trials due to lack of funding.

ALACHUA, FL May 16, 2012 - Florida Biologix®, a full-service biopharmaceutical development and contract manufacturing organization, provided cGMP manufacturing services to National Taiwan University Hospital under a contract signed in March 2008. Florida Biologix produced, purified, and tested a clinical batch of their novel gene therapy for a Phase I clinical trial of a rare genetic disease – AADC deficiency. The trial was led by Dr. Paul Wuh-Liang Hwu. Results of the clinical trial were published in the May 16th issue of Science Translational Medicine.

2 February, 2012. Alachua, FL:  Florida Biologix, with a proven track record of successful biopharmaceutical cGMP manufacturing campaigns and drug product fills for US clinical trials, announces that it now meets European GMP requirements for aseptic fill and finish.  These enhancements provides companies seeking high-quality aseptic fills for European clinical trials an outstanding and cost competitive provider for filling Phase I/II drug products into vials or other containers.

Gene therapy suffered another blow, when Dutch biotech company Amsterdam Molecular Therapeutics, AMT, got another negative opinion for it's lead drug Glybera. The opinion was already a second for Glybera, since the company got first negative opinion in July 2011 and asked for re-examination from EMA.

Interestingly, this time the company got a positive opinion from CAT, Committee of Advanced Therapies, which might indicate that the product was deemed safe and effective, but CHMP, The Committee for Medicinal Products for Human Use, voted to yield a negative decision.