Approval of new drug that can fix faulty genes

According to George Dvorsky in We come from the future: The idea of treating genetic diseases by replacing a defective gene with a working copy has been around for nearly three decades, but the road to creating an actual therapy that's safe and effective has proven to be a long and difficult one. Now, thanks to the efforts of the Dutch biotech company uniQure, the Western world's first therapy to correct errors in a person's genetic code has finally been approved. While the therapy is extremely expensive and limited to a rare genetic disorder, the approval is set to open the door for similar interventions.

The drug is called Glybera and it's intended for the treatment of a genetic disorder called Lipoprotein Lipase Deficiency (LPLD), a rare, inherited disease that makes it impossible to metabolize fatty acids found in blood, what results in inflammation of the pancreas. The disorder affects about 1 in every one million people.

"This provides higher benefit to patients than the classical protein replacement strategy" he told Reuters. The European Commission gave uniQure its consent to start selling Glybera next summer.

Glybera works by introducing a normal, healthy LPL gene into the patient's body so that it can make functional LPL protein. The LPL gene is packaged in a delivery vector derived from adeno-associated virus (AAV), serotype 1, which has a natural propensity towards muscle cells.

The company is also currently working on regulatory approval for Glybera in Canada and the United States.

In future, it's thought that similar therapies will be able to remedy other genetic disorders, including Severe Combined Immunodeficiency Disorder (the so-called "bubble boy" disease).

It's worth noting that Glybera is not the world's first gene therapy drug. Back in 2003, China's Shenzhen SiBiono GeneTech won approval for a gene therapy drug for head and neck cancer (though not for use in Europe or North America).

Sources: Reuters, uniQure. Here is a link to the article in We come from the future.

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Nanobiotechnology - New comers

Crysalin Ltd - a breakthrough technology for the multi-billion dollar drug discovery industry

Background
Structure based drug discovery (SBDD) – in essence, computer aided design for pharmaceutical drugs - is routinely used in large pharmaceutical and biotechnology companies, with billions of dollars being deployed to accelerate the identification and optimisation of new drug candidates. Crysalin, spun-out from the University of Oxford in 2007, has developed a technology to address the major bottleneck in SBDD; specifically the provision of rapid and cost effective solutions for determining 3-D molecular structures for all proteins.

A New Technology for Protein Structure Elucidation
Crysalin has developed and patented lattice-based nanomaterials that demonstrate significant advantages in enabling protein structure elucidation. Current methods, specifically crystallisation, face numerous hurdles because the production of suitable crystal structures remains complex, time consuming and uncertain. In addition, whole classes of therapeutically relevant proteins, for example ion channel and G-protein coupled receptors which have been implicated in Alzheimer's and Parkinson's, are not amenable to crystallisation techniques.

Crysalin's technology enables structural elucidation of proteins by imposing order on target proteins, rendering them amenable to X-ray diffraction and electron microscopy techniques for resolving 3-D molecular structure. The approach relies on the design and production of protein-based building blocks that self assemble into 1-, 2-, or 3 dimensional arrays. The crystals produced according to this design are stabilised by intermolecular forces that are substantially stronger than those that stabilise conventional protein crystals.

The methodology is equally applicable to all protein classes and therefore will provide the biggest impact where current technologies have failed. To date, Crysalin has received seed investment for IP Group plc, Ora (Guernsey) Ltd. and the Oxford University Challenge Seed Fund (UCSF) to exemplify the technology.

NanoBiorCorp