ViroStatics and Vichem Chemie Announce Strategic Partnership to Identify and Develop Kinase Inhibitors for the Treatment of HIV/AIDS and Other Chronic Diseases

SASSARI, Italy, PRINCETON, N.J. & BUDAPEST, Hungary–(Business Wire)–
ViroStatics, srl, an Italian-US biopharmaceutical company today announced the
formation of a strategic partnership with Vichem Chemie of Budapest, Hungary to
identify, synthesize, screen, and develop the next generation of
AntiViral-HyperActivation Limiting Therapeutics (AV-HALTs) for the treatment of
HIV/AIDS and other chronic diseases.

Vichem Chemie is a Hungarian biotech research company focusing upon the field of
kinase inhibitors, one of the fastest growing segments within the pharmaceutical
industry with applications in oncology, autoimmune diseases and other unmet
medical needs. ViroStatics has brought the first-in-class AV-HALT, VS411, into
Phase II development. AV-HALTS are a new class of drugs that combine direct
anti-viral activity with a reduction in the chronically elevated immune system
activation that is now known to play a major role in the progression of HIV
disease, ultimately leading to AIDS.

“We are extremely pleased to be working with Vichem Chemie to discover valuable
new AV-HALT drugs,” explained Franco Lori, MD, President and CEO of ViroStatics.
“Vichem`s expertise, know-how and credibility in the area of in vitro discovery
of novel drug families are well-known. Our new partnership brings together both
companies` substantial intellectual properties and significant libraries of
Kinase Inhibitors.”

“The expertise of ViroStatics in translating basic discoveries into clinical
applications -from the bench to the bed side – is well appreciated,” added
Gyorgy Keri, PhD and CEO of Vichem Chemie. “The Companies` strengths are both
complimentary and synergistic. We look forward to a successful partnership to
identify new and exciting compounds that ViroStatics will develop into important
new therapies against HIV/AIDS and other chronic diseases.”

“The new class of AV-HALTs represents a novel approach to the treatment of HIV
based upon the most recent discoveries in the field of viral pathogenesis,” said
Richard B. Pollard, MD, Professor of Medicine and Chief of the Division of
Infectious Diseases, University of California Davis Health System. “ViroStatics
was the first to achieve Proof-of-Principle for the AV-HALTs with the two-drug
combination product, VS411. The collaboration between ViroStatics and Vichem
Chemie is expected to lead to the second generation of AV-HALTs where both
antiviral activity and reduction of excessive immune activation are combined in
a single molecule.”

About Vichem Chemie

Vichem Chemie was established in 1999. Its predecessor, Biosignal LTD, was
founded in 1991. Vichem performs advanced medicinal chemistry in the kinase
inhibitory field using its unique Nested Chemical Library (NCL) technology. NCL
includes more than 500 scaffolds and contains inhibitors against 124 kinases.
Utilizing the NCL, Master Key, Pharmacophore modelling, and Scaffold-hopping
technologies, Vichem provides patentable lead compounds against selected kinases
in five research cycles and performs selectivity profiling and off-target
identification with target fishing technology. Vichem has a
pharmacodiagnostics-based drug discovery program and develops preclinical
candidates with lead optimization for efficacy and ADMET. Vichem has
developmental candidates for HIV, Influenza, Tuberculosis and five oncological
kinase targets.

About ViroStatics

ViroStatics srl, an Italian pharmaceutical company with operations in Sassari,
Italy and Princeton, NJ, discovers and develops novel therapeutics to address
significant medical needs in HIV/AIDS, chronic infections and related fields.
The company is developing its lead product, VS411, as the first fixed-dose
combination of two drugs that not only decrease HIV replication but also protect
and conserve the immune system. ViroStatics recently announced its intention to
merge with Genetic Immunity to form Immunacia – a multinational
biopharmaceutical company with Business/Medical/Legal operations in the United
States and research facilities in Hungary and Italy – dedicated to developing
novel treatments that partner with the immune system to improve human health.

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Vichem Chemie Research Ltd.
Prof. György Kéri
Chief Executive Officer
Phone: +36-1-487-2080
Fax: +36-1-799-9990
gykeri@vichem.hu
www.vichem.hu
or
ViroStatics, srl
Michael Stevens, PharmD
+1-609-951-2206
Chief Development Officer
m.stevens@virostatics.com
www.virostatics.com

Copyright Business Wire 2010

‘DNA Sudoku’ to revolutionise genome sequencing, medical genetics

Washington, June 25 (ANI): Sudoku, the popular mathematics puzzle that has taken people by storm, is now set to revolutionize the world of genome sequencing and the field of medical genetics, according to a new study.

Researchers at Cold Spring Harbor Laboratory (CSHL) have combined 2,000-year-old Chinese math theorem with concepts from cryptologyto develop what they dubbed as the “DNA Sudoku”, because of its similarity to the logic and combinatorial number-placement rules used in the popular game.

The strategy allows tens of thousands of DNA samples to be combined, and their sequences – the order in which the letters of the DNA alphabet (A, T, G, and C) line up in the genome – to be determined all at once.

The accomplishment is quiet contrary to past approaches that allowed only a single DNA sample to be sequenced at a time.

It also has an upper hand on current approaches that, at best, can combine hundreds of samples for sequencing.

“In theory, it is possible to use the Sudoku method to sequence more than a hundred thousand DNA samples,” said CSHL Professor Gregory Hannon, leader of the team that invented the “Sudoku” approach.

With such efficiency, the approach promises to reduce costs dramatically.

The new method has tremendous potential for clinical applications. It can be used, for example to analyse specific regions of the genomes of a large population and identify individuals who carry mutations that cause genetic diseases – a process known as genotyping.

The key to the team’s innovation is the pooling strategy, which is based on the 2,000-year-old Chinese remainder theorem.

The method is currently best suited for genotype analyses that require only short segments of an individual’s genome to be sequenced to find out if the individual is carrying a certain variant of a gene or a rare mutation.

However, with the improvement in sequencing technologies and researchers gaining the ability to generate sequences for longer segments of the genome, Hannon envisions wider clinical applications for their method such as HLA typing, already an important diagnostic tool for autoimmune diseases, cancer, and for predicting the risk of organ transplantation.

The report will be published as the cover story in the July 1 issue of the journal Genome Research.(ANI)

Scientists move a step closer to creating ideal neural cells for clinical use

Washington, April 14 (ANI): Scientists at the Burnham Institute for Medical Research (Burnham) have come up with a way to convert human embryonic stem cells (hESCs) into neural progenitor cells, which may be ideal for transplantation.

Lead researcher Dr. Alexei Terskikh says that the new method to create these committed neural precursor cells (C-NPCs) does not produce mutations in the cells, and may be useful for clinical applications.

When the C-NPCs created using the Terskikh protocol were transplanted into mice during an experiment, they became active neurons and integrated into the cortex and olfactory bulb.

The researchers say that the transplanted cells did not generate tumour outgrowth.

“The uniform conversion of embryonic stem cells into neural progenitors is the first step in the development of cell-based therapies for neurodegenerative disorders or spinal injuries.

Many of the methods used to generate neural precursor cells for research in the lab would never work in therapeutic applications. This protocol is very well suited for clinical application because it is robust, controllable and reproducible,” said Dr. Terskikh.

According to the researcher, extensively moving cells from plate to plate to expand the numbers of neural precursor cells limits the plasticity of the cells, can introduce mutations and may lead to the expression of oncogenes.

Dr. Terskikh claims that the new protocol avoids this by using efficient conversion of hESCs into primary neuroepithelial cells without the extensive passaging.

During the course of the study, the researchers were able to rapidly neuralise the hESCs by culturing them in small clusters in a liquid suspension. They revealed that the cells developed the characteristic “rosettes” seen in neuroepithelial cells.

The C-NPCs were later cultured in monolayers. Immunochemical and RT-PCR analysis of the cells demonstrated that they were uniformly C-NPCs.

Immunostaining and imaging showed that the cells could be differentiated into three distinct types of neural cells.

The researchers later showed that the C-NPCs differentiated into neurons after transplantation into the brains of neonatal mice.

The study has been published in the journal Cell Death and Differentiation. (ANI)

Gene therapy may safely regenerate gum tissue

Washington, April 8 (ANI): University of Michigan scientists have moved a step closer to using gene therapy to regenerate tooth-supporting gum tissue.

The researchers say that they have developed a method of gene delivery that appears safe.

According to them, their work assuages one of the biggest safety concerns surrounding gene therapy research and tissue engineering.

The most notable incident highlighting the safety concerns of gene therapy research and treatment occurred several years ago when a teenager died when given the adenovirus during a gene therapy clinical trial at the University of Pennsylvania.

The U-M researchers say their approach, though involves the adenovirus, differs because they use lower dose and put the genes on a localized area rather than inject them into the blood vessels, where they can then travel through the bloodstream and result in unexpected and sometimes fatal reactions.

“What the U-M study showed is (the topical method) is very well contained and doesn’t distribute throughout the body,” said William Giannobile, a professor at the U-M School of Dentistry.

“This approach alleviates the safety concern about negative reactions within the body.

“When the teenager died, it got into his bloodstream and he reacted to it. It was tragic. This is the first study of periodontal disease therapy that demonstrates the distribution of these genes is very safe, suggesting that it could be used in the clinic for clinical application.

“Our study doesn’t look at all the safety concerns, but certainly this is very important to the field. The two clinical applications to date where it shows potential are periodontal disease and diabetic wounds. Maybe the reason for this is that both diseases result from a compromised or a defective healing environment,” Giannobile added.

The researcher revealed that the next step would be to use the new gene delivery approach in human clinical trials.

The planning stages for these studies will commence in the next year.

A paper on this work is scheduled to appear in the May issue of the journal Human Gene Therapy. (ANI)

Fibre-optic probe made for space program may revolutionise cataract detection

Washington, January 13 (ANI): A compact fibre-optic probe developed in America for the space program has now shown some promise to detect cataract early, something that can significantly reduce the incidence of vision loss.

Researchers from the National Eye Institute (NEI), part of the National Institutes of Health, joined hands with their colleagues from the National Aeronautics and Space Administration (NASA) to develop the simple, safe eye test for measuring a protein related to cataract formation.

The researchers say that helping people know about subtle protein changes before a cataract develops may encourage them to reduce their risk of contracting the condition by making simple lifestyle changes, such as decreasing sun exposure, quitting smoking, stopping certain medications and controlling diabetes.

“By the time the eye’s lens appears cloudy from a cataract, it is too late to reverse or medically treat this process. This technology can detect the earliest damage to lens proteins, triggering an early warning for cataract formation and blindness,” said Manuel B. Datiles III, M.D., NEI medical officer and lead author of the clinical study.

According to the researchers, the novel device is based on a laser light technique called dynamic light scattering (DLS). It was initially developed to analyse the growth of protein crystals in a zero-gravity space environment, they reveal.

NASA’s Rafat R. Ansari, a senior scientist at the John H. Glenn Research Center and co-author of the study, is the man who brought it to the attention of NEI vision researchers that the technology could possibly have clinical applications too.

He conceived that idea after learning that his own father’s cataracts were caused by changes in lens proteins.

Several proteins are involved in cataract formation, but one known as alpha-crystallin serves as the eye’s own anti-cataract molecule. Alpha-crystallin binds to other proteins when they become damaged, thus preventing them from bunching together to form a cataract.

However, humans are born with a fixed amount of alpha-crystallin, and therefore a depleted supply cause by radiation exposure, smoking, diabetes or other causes can result in a cataract.

“We have shown that this non-invasive technology that was developed for the space program can now be used to look at the early signs of protein damage due to oxidative stress, a key process involved in many medical conditions, including age-related cataract and diabetes, as well as neurodegenerative diseases such as Alzheimer’s and Parkinson’s,” said NASA’s Dr. Ansari.

“By understanding the role of protein changes in cataract formation, we can use the lens not just to look at eye disease, but also as a window into the whole body,” the researcher added.

The recent NEI-NASA clinical trial looked at 380 eyes of people, aged 7 to 86, who had lenses ranging from clear to severe cloudiness from cataract. The researchers used the DLS device to shine a low-power laser light through the lenses.

The team had already determined alpha-crystallin’s light-scattering ability, which was then used to detect and measure the amount of alpha-crystallin in the lenses.

They observed that as cloudiness increased, alpha-crystallin in the lenses decreased.

According to the researchers, alpha-crystallin amounts also decreased as the participants’ ages increased, even when the lenses were still transparent, and that such age-related pre-cataract changes would remain undetected by currently available imaging tools.

“This research is a prime example of two government agencies sharing scientific information for the benefit of the American people. At an individual level, this device could be used to study the effectiveness of anti-cataract therapies or the tendency of certain medications to cause cataract formation,” said NEI director Paul A. Sieving, M.D., Ph.D.

The researchers reckon that the DLS technique may be helpful in looking at long-term lens changes due to aging, smoking, diabetes, LASIK surgery, eye drops for treating glaucoma, and surgical removal of the vitreous gel within the eye, a procedure known to cause cataracts within six months to one year.

They say that this approach may also be helpful in the early diagnosis of Alzheimer’s disease, in which an abnormal protein may be found in the lens.

Meanwhile, NASA will continue using the device to look at the impact of long-term space travel on the visual system.

“During a three year mission to Mars, astronauts will experience increased exposure to space radiation that can cause cataracts and other problems. In the absence of proper countermeasures, this may pose a risk for NASA. This technology could help us understand the mechanism for cataract formation so we can work to develop effective countermeasures to mitigate the risk and prevent it in astronauts,” Dr. Ansari said.

A research article describing the study has been published in the Archives of Ophthalmology. (ANI)