genes

First miRNA drug enters human studies

Wed, 28 May 2008 06:59:54 -0400

Denmark's Santaris has kicked off a Phase I study of SPC3649, a potential treatment for hepatitis C infection. The trial marks the first time a microRNA drug has been tested in humans. SPC3649 will be tested for safety in 48 healthy volunteers before being administered to hepatitis patients in a Phase II trial.

MicroRNA is one of two types of RNAi found in all human cells and affects how genes are turned off and on (siRNA is the other). "The mechanism of action of this drug represents a potential breakthrough in medical science. The ability to switch off the functions of particular microRNAs may enable clinicians to modulate entire networks of genes associated with disease or ill-health," said Santaris' CEO.

- check out Santaris' release for more

SPOTLIGHT: Sangamo targets gene manipulation

Thu, 15 May 2008 06:59:53 -0400

Forbes has highlighted the development work of Sangamo BioSciences, which has a "hefty" pipeline of drug candidates in development that use zinc-finger DNA binding proteins to manipulate genes for a number of diseases which currently can't be treated. Report

Shares slip as developers see RNAi doubts

Wed, 02 Apr 2008 07:59:55 -0400

Shares of Opko and Alnylam have slid since a new study published in late March concluded that experimental drugs developed with the hot new science of RNA interference may work by activating the human immune system rather than by silencing select genes. That conclusion, notes New York Times biotech writer Andrew Pollack, suggests that the entire field could be much less precise than previously imagined, with unexpected consequences for patients' health that could threaten a variety of clinical programs. For now, the companies involved say that they have data demonstrating that their drugs are safe and work as intended. There are at least five RNAi therapies in clinical development now consisting of short sequences of double-stranded RNA.

- check out the New York Times article

Related Articles:
Alnylam trial shows RNAi works in humans. Report
Alnylam's makes progress with RNAi program. Report
Is Alnylam Roche's 'second Genentech'? Report
OPKO acquires rights Phase III drug. Report

Osmetech: biomarker license gives it diagnostics edge

Tue, 12 Feb 2008 06:59:54 -0500

Osmetech says its exclusive license for a key biomarker gives the company a big edge in identifying potential adverse reactions to warfarin, a commonly used therapy used to fight thrombosis. "This marker in conjunction with variants from the two genes (CYP2C9 and VKORC1) recommended by the FDA will improve patient outcomes by reducing the overall complications of warfarin therapy, reduce costs in the health system and help provide a new standard of personalized care, " says CEO James White said. ''The fact that Osmetech has an exclusive license to this marker for warfarin testing, we believe will help position us as the leading diagnostic company in this area."

- check out this release
- read the article from Thompson Financial

Related Articles:
Osmetech submits app for warfarin test. Report
Warfarin label to include genetic test suggestion. Report
Personalized medicine advances with new genetic test. Report

RNAi screening finds 273 proteins used by HIV

Fri, 11 Jan 2008 06:59:54 -0500

Harvard Medical School's Dr. Stephen J. Elledge and a team of scientists used RNA interference to screen thousands of protein-making genes, and have identified 273 proteins HIV needs in order to survive in human cells. The finding raises the possibility that the proteins may be targets for the treatment of AIDS. Previously, only 36 of proteins were known to play a role in HIV's survival.

Patients using current treatments often have to switch drug regimens as the virus mutates and becomes immune to older drugs. A therapy based on these new findings wouldn't have that same problem, since it would block the disease from entering human cells entirely. However, according to a Harvard press release, "the challenge will be to develop drugs that inhibit HIV by interacting with these human proteins without hurting our cells." Currently, only one drug prevents the virus from entering white blood cells--Pfizer's recently-approved Selzentry. The Harvard study was published in Science Magazine.

- see this press release on the findings
- read the New York Times article for more

Related Articles:
New HIV drugs offer breakthrough therapies. Report
Selzentry approval marks big win for Pfizer. Report

Genizon pockets $31M CAD in fifth round

Mon, 07 Jan 2008 06:59:55 -0500

Canada's Genizon BioSciences has garnered $31 million Canadian in its fifth round of financing. The lead investor is BTF B.V. of Haarlem, The Netherlands, which specializes in late-stage biotech companies.

"Genizon's outstanding DNA resources and a platform that is applicable to many stages of drug development make a compelling investment case," said Jan Mellegers, CEO of BTF. "The company has impressed us with its ability to not only discover many genes in each disease, but also to assemble these into GeneMaps that lead to new drug targets and biomarkers."

- here's the release

ALSO: ZyGem says it has closed its first round of financing. Release

Related Article:
Pfizer inks genetic license with Genizon. Report

More jaw-dropping scientific advances lay ahead

Wed, 02 Jan 2008 06:59:54 -0500

If the past year saw some incredible gains in biology, next year we should see even more sizeable advances. The sequencing of the human genome is now seven years old, but now we're witnessing new genetic discoveries almost daily. Genes that cause disease and fight disease are being identified and sized up by scientists around the globe. Stem cell research has made great strides and we've seen the first steps taken toward creating artificial life.

With a considerable amount of momentum, we're likely to take a few more baby steps in the direction of personalized medicine on the public level, with big strides seen in the clinic. For developers, the trend can only be ignored at their own risk. New therapies in the clinic more and more often will be tied to a genetic test.

Related Articles:
Scientists in a race to create artificial life forms. Report
The race is on to produce synthetic life. Report
$999 DNA test offers insights on disease risks. Report
Genetic test seen as big step to personalized meds. Report

In breakthrough, scientists reprogram skin cells into ESCs

Tue, 20 Nov 2007 06:59:56 -0500

On FierceBioResearcher.com today, you'll see a report that Dolly creator Ian Wilmut is giving up on cloning and focusing on harvesting stem cells from skin tissue for biomedical research work. You'll get a better understanding why he's making the switch with the publication today of two studies showing that skin cells were reprogrammed into replicas of embryonic stem cells by simply adding four genes. The approach, reported by two sets of scientists, allows researchers to create stem cells that act in the kind of blank-slate fashion of ESCs, without the controversial destruction of human embryos. There is the possibility that introducing the genes in the way that's being reported today could create risks, especially if a cancer gene is being added into the skin cell equation. But the scientists say that it won't take long to perfect the technology, opening up the entire field of stem cell research in one fell swoop.

"Everyone was waiting for this day to come," the Rev. Tadeusz Pacholczyk, director of education at the National Catholic Bioethics Center, told The New York Times. "You should have a solution here that will address the moral objections that have been percolating for years."

- read the article in The New York Times

Related Articles:
Dolly creator scraps cloning, takes new approach. Report
Ian Wilmut discusses how regulations affect stem cell science. Report
Mature stem cells prove effective in cloning. Report
Single-parent stem cells may skirt cloning controversy. Report
Researcher promises cloned embryonic stem cells. Report

Upstart biotech attracts A-list investors

Tue, 09 Oct 2007 06:59:57 -0400

An upstart biotech company focused on developing new therapies targeted at the genetic variations linked to dry age-related macular degeneration has raised $37 million in its first round. New Haven, CT-based Optherion is banking on scientific research that has unveiled the role of variations in the CFH and CFB genes in AMD. Variations in the CFH gene are linked to half of all patients and a combination of variations in the CFH and CFB genes are associated with almost three out of every four cases. Researchers will also work on a new therapy for Dense Deposit Disease, an end-stage renal disease with an orphan-sized market that is triggered by the same genetic variations they're targeting for AMD. The company attracted some marquee names in biotech, including Domain Associates, J&J Development Corp., Biogen Idec and Pappas Ventures.

- see the release on the first round

ALSO: The stem cell company Osiris Therapeutics has secured $30 million in new financing. "This transaction, along with our current cash position and existing financing vehicles, gives us greater flexibility as we advance our Phase III clinical programs toward market launch," said Osiris CFO Cary Claiborne. Release

Related Articles:
Biotech sees big money from corporate players. Report
Osiris Therapeutics blueprints $80M IPO. Report
Osiris gains $50M. Report
Osiris granted orphan status. Report

Nobel Prize goes to gene targeting trio

Mon, 08 Oct 2007 06:59:57 -0400

As a child, Mario Capecchi spent four years on the streets in his native Italy, surviving by stealing or begging for food. Today, he's one of three scientists--the other two are Oliver Smithies and Sir Martin Evans--who've won the Nobel prize for medicine, recognition for their collective work on gene targeting, a process that allows researchers to turn genes off and on in mice. Their work involved introducing a genetic alteration in embryonic stem cells which are then injected into a mouse embryo. That development has spurred a huge advance in understanding the role of genes and health--and advanced a myriad number of therapeutic programs.

- see the release from the Nobel Assembly
- read the article from the New York Times

Related Article:
Gene therapy makes a big comeback. Report

ALSO NOTED: Bavarian Nordic earns milestone payment; Cyclacel acquires Align Pharmaceuticals; and much more...

Mon, 08 Oct 2007 06:59:50 -0400

> Bavarian Nordic has earned a $50 million advanced payment for achieving milestones outlined in its contract to provide smallpox vaccine to the U.S. government. Report

> Coley Pharmaceuticals of Wellesley, MA has adopted a shareholder rights program. Release

> Cyclacel Pharmaceuticals has acquired Align Pharmaceuticals for about $3.5 million in cash. Release

> UK-based Shire Pharmaceuticals is selling a block of medicines to Almirall of Spain for $213 million. Report

> A Florida jury ruled for Merck in yet another Vioxx case, bringing the drug maker's record to 11-5 in trials related to the drug. Report

> Eli Lilly is owning up to Zyprexa's ills in new warnings added to the medicine's label. Report

And Finally...Too much debt? Blame it on your genes. New research indicates that our genes may play a key role in our economic behavior. Report

RNAi leaders Isis, Alnylam form new company

Fri, 07 Sep 2007 06:59:58 -0400

Isis Pharmaceuticals and Alnylam Pharmaceuticals--two leaders in the hot field of RNA--have teamed up to form Regulus Therapeutics, a company that will focus on developing new drugs based on micro-RNA. Micro-RNA is one of two types of RNA interference found in all human cells and affects how genes are turned off and on. MiRNAs regulate whole networks of genes; therefore therapeutics based on this technology represent a new approach to target the pathways of disease, according to the companies.

Both companies will grant Regulus licenses to their IP for miRNA therapeutic applications, as well as certain early fundamental patents in the miRNA field. Alnylam will make an initial investment of $10 million to balance venture ownership; thereafter Isis and Alnylam will share funding of Regulus. Regulus will be operated as an independent company with an independent Board of Directors and management team, which will include Nobel laureate David Baltimore.

"The emerging biology of microRNAs points to a completely new understanding of cellular mechanisms for regulation of gene expression," noted Baltimore. "I believe that microRNAs represent previously unexplored disease targets where pharmacological approaches could lead to the emergence of novel therapies for many human disorders. "

- see this release for more

Related Articles:
A billion-dollar lesson in RNAi economics. Report
Isis gains from big RNAi deal. Report
Roche harnesses Alnylam's RNAi tech in $1B deal. Report

Molecular diagnostics firm gains $34M in VC

Tue, 28 Aug 2007 06:59:55 -0400

Amsterdam-based Agendia, a molecular diagnostics company, has raised $34 million in its fourth venture round, adding ING to its list of backers. Current investors Van Herk Biotech, Gilde Healthcare Partners and Global Life Science Ventures also participated. The company developed the MammaPrint, a prognostic DNA microarray-based in vitro diagnostic test that measures the activity of 70 genes to assess the risk of breast cancer recurrence.

- check out the release on the latest round

Related Article:
Personalized medicine advances with Agendia's new genetic test. Report
Diagnostics tied to new drugs. Report

Protein Sciences takes on Big Pharma in vaccines race

Thu, 23 Aug 2007 06:59:57 -0400

Big Pharma has a big stake in creating new flu vaccines, but tiny Protein Sciences in Connecticut believes it can get a new vaccine approved in '08. The big trend in vaccines today is to move away from egg-based production and into cell-based production, and Protein Sciences' approach is to inject flu genes into caterpillar cells. The protein that is expressed is used in the vaccine. The company may be small, but it has some big names at the top. CEO Daniel Adams is a co-founder of Biogen and David Fedson comes from Sanofi-Aventis.

- read the report from the Wall Street Journal (sub. req.)

Related Article:
Vaccine "renaissance" attracts huge new venture investments. Report

A billion-dollar lesson in RNAi economics

Mon, 09 Jul 2007 00:01:39 -0400

Roche's billion-dollar deal for gene-silencing technology (below) underscores once again what a hot commodity RNAi tech has become. And prices are rocketing up fast. Merck paid $1.1 billion to buy Sirna, leaving CytRx and Alnylam in an incredibly influential position to dictate prices. A nonexclusive deal like Roche's shows just how influential that position has become. The reason for all the positive attention, of course, is that gene silencing is shaping up as a game changer in healthcare. For an outside observer, the premiums in this field are looking stratospheric. But big bets in biotech can be rewarded with even bigger global markets. With a wide variety of diseases triggered by the activity of specific genes, a mechanism to turn them off is worth every bit of the Nobel Prize handed out for the basic research last year. Barring a significant stumble in R&D, look for new work in the field to grow aggressively. And don't expect the buy-in price to go down anytime soon. - John Carroll

Press Release: Genizon BioSciences Secures $10 Million In Additional Financing

Maureen Martino — Mon, 05 Feb 2007 11:04:15 -0500

Genizon BioSciences Inc. Secures $10 Million In Additional Financing

MONTREAL -- Genizon BioSciences today announced the first closing of its Series D equity financing, in the amount of CDN$10 million. Investors include Pfizer Inc. and existing investor BTF B.V. of Haarlem, The Netherlands, a venture capital fund focused on late stage biotechnology companies. Pfizer's investment in Genizon is related to a license and collaboration agreement announced January 16, 2007.

Genizon will use the funds to conduct genome-wide association studies in the Quebec Founder Population and to enhance and commercialize discoveries already generated from previous studies.

"Genizon is poised to capitalize on its lead in genome-wide scanning and disease gene discovery, and this will be enabled by this financing," said John Hooper, Ph.D., President and CEO of Genizon. "Pfizer's investment validates Genizon's technology, as does Pfizer's collaboration in three of Genizon's programs. We are also pleased by the additional investment from BTF, which will be pivotal in adding value."

"We are excited about Genizon's development over the last year," stated Jan Mellegers, CEO of BTF. "This additional investment in the company reinforces our belief in the value being generated by Genizon's gene discovery programs and through its alliances."

Genizon conducts genome-wide association studies involving more than 40,000 members of the Quebec Founder Population, whose extensive genetic sharing and low genetic variability are ideally suited for the discovery of genes affecting people worldwide in the many common diseases with strong genetic components. Genizon has completed eight genome-wide association studies to date, with several planned for 2007.

About Genizon BioSciences

Genizon BioSciences Inc., founded in 1999, is the only source for GeneMaps--comprehensive maps of genes, genetic markers, biochemical pathways and drug targets that are unequivocally involved in causing human disease-accelerating the development of safer, more effective medicines. GeneMaps also lead to the development of predictive diagnostics that facilitate personalized medicine. Genizon's proprietary, automated gene discovery platform using genome-wide association studies involving thousands of members of the Quebec Founder Population provides unprecedented understanding of the genetic origins and mechanisms of common diseases, resulting in the best possible drug targets and genetic markers. The company's GeneMaps provide pharmaceutical partners with intellectual property that creates opportunities for leadership in many therapeutic areas by bringing to market better treatments that address the root causes of diseases. Genizon is conducting gene discovery programs in more than 25 common diseases. Genizon's genotyping and genetic analysis services also assist partners in rapidly generating statistically reliable gene discoveries from existing DNA collections.

Contact: Genizon BioSciences, Inc. Henry Bretecher, +1 514-270-3991 ext. 230 Chief Financial Officer henry.bretecher@genizon.com or Schwartz Communications Lauren Arnold or Wendy Mejia, +1 781-684-0770 genizon@schwartz-pr.com

Source: Genizon BioSciences, Inc.

Press Release: Lentigen Corporation and Wave Biotech Announce Formation of Collaboration

Maureen Martino — Fri, 26 Jan 2007 13:30:41 -0500

Lentigen Corporation and Wave Biotech Announce Formation of Collaboration

BALTIMORE and SOMERSET, N.J., January 25, 2007 -- Lentigen Corporation and Wave Biotech, LLC announced today that they have entered into a collaboration under which Wave Biotech will provide process and equipment expertise for Lentigen's manufacturing platform based on its proprietary lentiviral vector technology.

The highly efficient gene delivery properties of lentiviral vectors significantly reduce the cost, time and risk involved in biologics manufacturing. Wave's key products feature disposable contact materials that eliminate cleaning and validation, thereby further reducing costs in operations and drastically reducing the time-to-market for biological products.

"This is one of the many partnerships that we are forging to develop our lentiviral vector platform. We're excited to have Wave Biotech as one of our partners," said Dr. Boro Dropulic, founder and CEO of Lentigen.

Daniella Kranjac, VP Sales & Marketing of Wave Biotech, LLC commented, "As the leader in disposable bioreactors and supporting instrumentation, the Wave Biotech product line offers Lentigen an ideal platform technology for their production and contract manufacturing needs."

About Lentiviral Vectors

Lentiviral vectors (LV) are vehicles that can deliver genes or RNAi into cells with up to 100% efficiency and stability. Previous viral vector systems such as non-viral, adenoviral and adeno-associated viral vectors could achieve high, but not stable gene delivery into cells. Other vectors such as murine retroviral vectors can deliver genes stably, but not efficiently.

Gene delivery is accomplished by the binding and fusing of the LV pseudotyped envelope protein to the target cell membrane. The LV RNA containing the gene or gene silencing sequence is then incorporated into the cell via reverse transcription creating a DNA complex. This complex enters the nucleus incorporating into the chromosomal DNA creating a stable molecule. The gene sequence is integrated in the chromosome and is copied along with the DNA during ongoing cell division.

About Lentigen Corporation

Lentigen Corporation is a privately owned biotechnology company focused on the manufacturing and development of lentiviral vectors using its proprietary gene delivery technology for a wide range of applications in biotechnology and medicine. Lentiviral vectors are highly adapted delivery vehicles that can transport genes or gene silencing sequences into cells with high efficiency and stability. Lentigen is positioning itself to become the leading provider of lentiviral vector products and services for academic, government, biotechnology and pharmaceutical researchers. For further information, visit www.lentigen.com.

About Wave Biotech

Wave Biotech is a research-based company that develops and manufactures innovative process equipment for the pharmaceutical and biotechnology industries. The company's focus is on developing disposable bioprocessing equipment. Key products, such as the Wave Bioreactor(R), WaveMixer(R), FlexMixer(R), and Sterile Tube Fuser, feature disposable contact materials that eliminate cleaning and validation, thereby reducing costs in operations ranging from cell culture, media preparation, and buffer dissolution and thawing process intermediates to patient specific cell therapy in hospitals. These unique, patented devices can be installed and commissioned rapidly, thereby drastically reducing the time-to-market for biological products. For additional information, visit: www.wavebiotech.com

Press Release: Blue Heron Biotechnology Names John Fess CEO

Maureen Martino — Mon, 04 Dec 2006 12:18:49 -0500

Blue Heron Biotechnology, Inc. Names John Fess Chief Executive Officer

BOTHELL, Wash, -- Blue Heron Biotechnology, Inc., a leader in gene synthesis, today announced that John Fess has been named Chief Executive Officer. Mr. Fess succeeds John Mulligan, Ph.D., Blue Heron’s founder who will become the company’s Board Chairman and Chief Scientific Officer. “This critical management change reflects Blue Heron’s commitment to continued growth and to meeting our customers’ increasingly complex requirements. John’s leadership is key to Blue Heron’s existing operations as well as its future success,” Dr. Mulligan remarked.

Previously, as Chief Operating Officer of Blue Heron Bio, Mr. Fess oversaw Sales, Marketing, Manufacturing, and Information Systems and directed ongoing redesign and deployment of critical business systems. Mr. Fess brings 25 years of experience to Blue Heron from assignments at companies ranging from startups to Fortune 5, in the oil, semiconductor and computer-related industries. He holds degrees in Math, Computer Science, and Operations Research from Stanford and an MBA from Harvard Business School.

“I’m very eager to take on this new challenge to guide Blue Heron to a new level of capability. Blue Heron has accomplished some amazing things in its five-year history and I see my primary task as freeing up some very talented people to continue to do what they do best,” Mr. Fess commented.

About Blue Heron

Blue Heron Biotechnology is a pioneer and leader in the gene synthesis market, producing nearly ten million base pairs of DNA since its inception in 1999. Blue Heron’s proprietary GeneMaker® technology provides customers access to complex synthesized genes that are difficult or impossible to synthesize with other companies’ standard processes. Blue Heron regularly produces both simple and complex genes from less than 100 base pairs to over 20,000 base pairs.

Press Release: Artemis to construct genetically engineered mouse models for Merck

Maureen Martino — Sun, 19 Nov 2006 11:52:42 -0500

Artemis Signs Agreement With Merck & Co., Inc. for Generation of Genetically Engineered In-Vivo RNAi Mouse Models

COLOGNE, Germany, November 20 /PRNewswire/ -- Artemis Pharmaceuticals GmbH, Cologne announced today that it has signed a research agreement with Merck & Co., Inc. to construct a large number of shRNA interference ("shRNAi ") - genetically engineered mouse models for the in vivo functional analysis of selected disease related genes. The agreement represents a significant initiative to undertake a large-scale approach to gene function analysis using shRNA knock down in genetically engineered mice as models for human biology.

Under the terms of the agreement, Artemis will generate genetically engineered shRNAi "knock down" mouse models for Merck using Artemis' proprietary, optimized and fully integrated vector construction, ES cell transfection and inducible RNAi technology. Merck will provide selected shRNA sequences that correspond to mouse genes that may also play a central role in human diseases. Artemis will use its technologies to achieve constitutive as well as inducible functional down-regulation of the expression of the gene targets provided by Merck.

Artemis is the first company to have developed a robust methodology for the controlled induction of shRNA based gene knock down in adult mice. This inducible system permits the down-regulation of a selected target gene to be turned on and off thereby more closely modeling the dosing of a pharmacologic inhibitor. The generation time for such a mouse model at Artemis is only four months.

"We are highly pleased to establish this research service agreement with Merck & Co. Inc.," said Prof. Peter Stadler, Managing Director of Artemis. "This agreement further expands our broad and successful ongoing relationship with Merck. It definitively provides evidence of the great interest in using our RNAi technology on a broad basis for drug discovery research. Disease related genes can be carefully analyzed in adult mouse models by combining shRNA technology with our methodologies to generate these mouse models for in vivo phenotypic characterization. For the first time ever it's now possible to generate large numbers of mouse models within a time frame previously thought unachievable. We at Artemis believe our technology platform is broadly applicable throughout the biotechnology and pharmaceutical industry."

About Artemis

Artemis Pharmaceuticals has developed the worldwide leading portfolio of scientific and technological tools in murine molecular genetics and functional genomics. It offers custom-made genetically engineered mouse models for use at various stages of the industrial drug discovery process or for academic research.

Artemis has formed a strategic alliance with Taconic, one of the world's leading breeders of laboratory rodents. This alliance enables the generation and delivery of genetically modified in vivo models to the customers in an unpreceeded speed and quality.

Artemis customers include the majority of the leading pharmaceutical companies including Merck & Co., Inc., Sanofi-Aventis, Bayer AG, Boehringer Ingelheim, Astra Zeneca, Glaxo Smith Kline, Schering AG, Bristol-Myers Squibb, Lilly, Merck KGaA, Biovitrum AB, and other pharma companies and biotech firms as well as various Academic Research Institutions worldwide.

Artemis is a wholly owned subsidiary of Exelixis, Inc., South San Francisco, CA. USA (Nasdaq:EXEL - News) since 2001.

DeCode IDs tablet problem, suspends pivotal trial

Thu, 05 Oct 2006 20:01:38 -0400

Iceland's deCode Genetics has suspended a late-stage trial of the heart attack prevention drug veliflapon after determining that the tablets being used weren't dissolving properly. Researchers were concerned that the faulty tablets would have skewed the data on the drug. The developer announced that it had notified the FDA and was looking at alternative manufacturing sources to resolve the issue. Veliflapon is directed at two genes which contain proteins linked to an elevated risk of heart attack. Another compound with a similar target is in early trials.

- read the report on deCode from Hemscott