For those of you who are not familiar with RNA interference, here’s what it is and how it works: DNA is, in a sense, the operating system software for our cells. As such, DNA does not directly interact with genes. It’s too important to risk corruption through unnecessary exposure. Instead, DNA operates by sending out chemical instructions. These instructions are in the form of complex RNA molecules. They are similar to double-stranded DNA, but are usually single stranded.

Basically, these extraordinarily complex RNA molecules control gene activity or expression. This is important because nearly all diseases are either caused or cured by the proteins produced by genes. You can, therefore, think of the ability to increase or decrease the production of these proteins as an on/off switch for diseases.

The remarkably young science of RNA interference is based on the accidental discovery that it is possible to flip these switches. The remote control, so to speak, for these switches consists of portions of RNA molecules. Because these portions are recognized as invaders by the body, they provoke the rejection of larger disease-causing RNA molecules. The other side of the coin is “RNA activation.” This is the process that increases gene expression.

The birthday of the science, according to many, was in 1998. That was the year when scientists Craig Mello and Andrew Fire published their paper on RNA interference in nematode worms. The paper earned them the Nobel Prize in physiology or medicine in 2006.

RNAi companies, unlike stem cell firms, have grown very rapidly. Many have already been gobbled up by Big Pharma companies, positioning for a foothold in this promising new field.

Unlike regenerative medicine (think: stem cells), RNAi scientists and companies sidestepped the legal and ethical scrutiny that has hobbled some stem cell companies. Now the legal situation has been clarified and embryonic stem cells have been replaced for therapeutic uses by iPS and parthenogenetic cells. As a result, we can expect important stem cell companies to make similar deals with Big Pharma companies.

Regardless, many RNAi companies already have significant capitalization and Big Pharma partnerships. Even at that stage of their development, however, they still have profound transformational potential. For example, I would have added RNAi pioneer Sirna Therapeutics to our portfolio. Sirna, however, was acquired in 2006 by Merck & Co. Inc. in a deal worth $1.1 billion.

That deal, the largest in the RNAi space so far, was followed by others:

Anglo-Swedish pharm firm, AstraZeneca Intl., made a $400 million deal with a European RNAi firm, Silence Therapeutics. Alnylam formed a $1 billion partnership with Swiss giant, Roche. The high-water mark for RNAi stockholders, however, is still the Sirna Therapeutics acquisition by Merck in October 2006.

As I’ve said repeatedly, RNA interfering molecules work. There is no question that they flip the switches they’re supposed to flip. The challenge, however, is getting them to their target genes before they are recognized and destroyed by the body’s immune system. Various companies are homing in on specific delivery solutions now. There are, however, many different solutions to the delivery problem. Each gene switch has its own special considerations and there is no “one size fits all” solution.

Most of the new RNAi companies have been founded specifically to develop RNAi therapies. Opportunity abounds in this sector. But beware; the failures will be as spectacular as the successes.

Source: RNAi’s Buyout Deals: Who Will Benefit?

This from Today’s Financial News:

Investors expect President-Elect Obama to make good on his campaign promise to lift restrictions on federally funding human embryonic stem cell research. House Speaker Nancy Pelosi has vowed to introduce legislation for a regulatory framework necessary to enable expanded stem cell research.

Biotechnology experts consider both the regulatory framework (to underpin clinical trials) and the additional funding to be crucial to the future of the American bio-pharma industry. In fact, it could ring in a new era for American stem cell companies. Many foreign stem cell researchers already fear that even private and institutional U.S. funding may be “repatriated” to U.S. companies.

No matter how long it may take for federal dollars to have a measurable effect on companies’ actual bottom lines, stem cell research will make headlines in the weeks ahead.

This is just the kind of opportunity we at TFN… and our premium service Hot Stock Confidential… are looking for. Right now, we’re keeping our eyes on literally dozens of similar opportunities.

As a general rule, the equity recommendations we select for Hot Stock Confidential Members are stocks we’d buy ourselves, even in a “normal” market… and would have no problem recommending to our relatives. They’re companies whose insiders are buying… that have easy-to-understand business concepts, established markets, growing revenues and unique products. Some are making great progress in the fields of biotechnology… genetics… medical diagnostics. Long-term, they will make good investments.

But in the meantime, every small breakthrough… every successful step in the approval process… holds the potential for amazing short-term profits. Especially in volatile markets like this!

In many instances the daily trading volume of these companies is so light, a single story in the mainstream media… an Internet rumor… a line dropped on a bulletin board… could double or triple their average daily trading activity and push stock prices out of our advisable buying range.

Use the current market slump to position yourself for publicity-driven price spikes in some of the best-situated stem cell stocks.

We’ve found three of the hottest stem cell stocks around. Each has winning potential… and is currently selling at bargain rates! Invest now… and you could see gains of 50%, 100%, even 200% within the first two quarters of 2009.

These stocks are priced below $5 each. Even a moderate investment may pay off handsomely if the reinstatement of federal funding triggers the wave of investor interest we expect it to!

HOT STEM CELL STOCK #1: Aastrom Biosciences, Inc. (NASDAQ:ASTM)

Over 5 million people in the U.S. suffer from cardiomyopathy (heart muscle disease). The condition is a contributing factor in at least 250,000 deaths each year.

Dilated cardiomyopathy (DCM) is the most common form of the disease. More men suffer from it than women. It usually occurs in those aged between 20 and 60 years — though even children can have it. In fact, my own brother is a sufferer and I lost a grandmother and uncle to DCM.

Patient numbers are bound to increase as the U.S. population ages.

But one company is hoping to reduce the mortality rate through revolutionary treatments.

Aastrom Biosciences, Inc. develops products for the repair or regeneration of human tissue. Aastrom’s proprietary Tissue Repair Cell (TRC) technology involves autologous, mixed-cell products containing stem and early progenitor cells to treat cardiac and vascular tissue regeneration.

Mending the Ailing Heart

The company has just launched Phase II IMPACT-DCM clinical trials for the evaluation Aastrom’s Cardiac Repair Cells (CRCs) in the treatment of dilated cardiomyopathy (DCM) or congestive heart failure.

The CRCs are delivered surgically, directly into the heart muscle.

The company intends to enroll patients with end-stage DCM. Half will have ischemic DCM (often caused by repeated heart attacks) and the other half, non-ischemic DCM (where the damage is due to hypertension, viral infection or alcoholism).

Orphan Drug Designation

Early indications from a “compassionate use” case in Germany are promising. (“Compassionate use” means an experimental therapy that is not yet officially approved but permission has been granted to treat a patients who has run out of other options)

As a result, the U.S. Food and Drug Administration gave Aastrom’s CRCs the Orphan Drug Designation. That allows for an expedited FDA review, a reduction of filing fees, possible tax credits, and the right to seven years of marketing exclusivity once the product receives FDA approval.

The current market has battered down stocks like this micro cap. As a matter of fact, the Nasdaq has given Aastrom until March to bring their stock price above $1 for 10 consecutive days. Otherwise, Aastrom will lose their listing.

Of course, they aren’t the only company in this situation. But with the increased interest in stem cell research, potential federal grant money coming, and a treatment that — if it works — could change the lives of millions, I recommend you buy shares of Aastrom Biosciences, Inc. at or under 85 cents.

I expect this stock to go up at least 20% in the next 6 months.

PS. This stock pick is part of a free special report for Hot Stock Confidential subscribers, which offers two more hot stem cell stock picks and much, much more. Click here for more information.

Source: The 3 Hottest Stem Cell Stocks Under $5

This from The Rude Awakening:

For the first time in history, we are seeing a rapidly emerging medical technology with the power to dramatically extend life spans. As a result, no technology on Earth has greater potential for investors.

To quickly review, stem cells are unique in human biology. Unlike all other cells, they are immortal and can be programmed, or potentiated, to replace any aging or damaged cell. Whether you want new skin, a new heart, new knee cartilage or new eyes, all these things are theoretically possible using stem cells therapies.

There were, however, two serious barriers to this exciting technology. The most obvious was that the only source of stem cells was embryos. This not only raised ethical questions, but it raised the possibility that stem cell therapies would require immune system repression. Just a year ago, many scientists believed these barriers were insurmountable. Stem cell therapies were in the same class as jet packs and flying cars. As a result, Big Pharma shied away from the partnerships that new scientific fields usually enjoy.

Then, last year, the entire picture changed. In rapid succession, five groups of scientists proved that adult cells could be reprogrammed to become stem cells using four transformative genes. The mechanism for introducing these genes into the adult cells was viruses.

Let me explain.

Viruses can’t reproduce on their own. They invade host cells and hijack their genetic mechanisms. In effect, they inject their own genetic code into host cells to duplicate themselves. By attaching these four transformative genes to the viruses, scientist tricked them into genetically reprogramming adult cells into stem cells.

These revolutionary new cells are called induced pluripotent stem, or iPS, cells. They are identical to embryonic stem cells. Mouse skin cells were transformed to iPS cells. They were then allowed to continue developing into living mice. Still, however, skeptics doubted that the almost alchemical power of stem cells had finally been released. The reason was that the virus used for producing these iPS cells was a retrovirus.

Retroviruses are associated with cancers and remnants lingered in the iPS cells. Obviously, therefore, the FDA would not allow their use in human therapies.

At the time, I predicted that the problem was temporary and would be worked out in a few years. I was wrong: It took months. A few weeks ago, Harvard researchers announced they had transformed adult cells into iPS cells using the adenovirus. The virus used was referred to in the press as a cold virus because it produces symptoms similar to those of the common rhinovirus. After a few cell divisions, it is completely deactivated.

This is amazing, astonishing news. I don’t have room here to go over even a few of the implications of this momentous breakthrough. I will remind you, however, that stem cells can be programmed to replace any cells in your body with perfect, youthful versions. Think about what that means. The financial implications for investors are staggering.

Harvard’s stem cell biologist Konrad Hochedlinger said, “I have never seen a field move forward as fast as this one.” That’s an understatement. In practical terms, this breakthrough means that right now, we could take a little of your blood and clone you. At this very moment, scientists have the ability to rejuvenate your heart and vascular tree. Not only that, but the telomere length of these replacement cells will have a longer life span than your heart and arteries had on the day you were born.

Few people know this and, consequently, few understand the virtually unlimited potential for both our species, in general, and early investors in this space, in particular.

There is another reason to expect stem cell stocks to increase in value. The cold virus breakthrough prompted pharma giant Pfizer (NYSE:PFE) to announce a major new emphasis on stem cell science. Pfizer’s previous timidity was caused by ethical concerns that arose from using stem cells lines derived from human embryos. Now that this issue is moot, Pfizer has jumped into the field with both feet, fully clothed.

Pfizer’s executive director of global research and development John McNeish said, “These cells will be tremendous in drug discovery. They will help us understand personalized medicine, genetic variation, ethnic populations, what biomarkers to follow.” Later on, McNeish says, Pfizer will market stem cells to rejuvenate aging and damaged organs and tissue. Insiders consider this the tipping point the industry has been waiting for.

We can expect that, finally, the holders of important stem cell patents will form partnerships with big pharmaceuticals. This has always been the pattern with biotech.

Adding to the excitement, another remarkable event has just taken place that will benefit companies in the RNAi space, though the benefits are a few years out. That’s the price drop on a complete genetic blueprint I talked about above. The company, Complete Genomics, says it will map your DNA for $5,000 next year. Moreover, I expect the price tag will drop to $1,000 within another two years.

At this price, gene mapping makes sense for the individual, the industry and insurers. For individuals, gene mapping could enable preventative therapies for genetic diseases. Insurers will also see cost savings here and Pharma will accelerate genetic research as part of drug development. The more DNA maps there are, the faster researchers will be able to identify the genetic causes of both diseases and resistance.

Because RNAi gives us the ability to switch individual genes on or off, RNAi companies stand to benefit massively from an increased understanding of our genome. The more maps correlated to medical histories that exist, the more we will know – and, unbelievably, the faster progress will be made.

Investors looking to go “long life” might like to consider this the investment of a lifetime.

Source: Going “Long Life”

This from Today’s Financial News:

The theoretical benefits of stem cell therapy could have a revolutionary effect on biotechnology and medicine.

Stem cell technology could create a renewable source of specifically differentiated cells to replace and regenerate cells and tissues damaged by conditions such as heart disease, Alzheimer’s, diabetes, spinal cord injury, Parkinson’s etc…

It could provide tools for the identification and (hopefully) prevention of the causes of abnormal cell division that lead to birth defects and cancer.

And it could change the way we test new medications entirely.

Adult vs. embryonic stem cells

As a general rule, adult stem cells can only be relied upon to divide and replenish into cell types of their original tissue. This is fine in situations where a patient’s own cells can be used and such treatment thereby avoids immune rejection.

Embryonic stem cells, on the other hand, can develop into any and all cell types. And they are much easier to grow in culture as compared to adult stem cells.

What does this mean for stem cell biotech companies?

Most of the smaller American companies engaged in stem cell research have had to focus on a specific adult stem cell for narrow applications because of limitations to Federal funding for new stem cell cultures.

For example…

StemCells, Inc. (NASDAQ:STEM), is currently focused on human neural stem cell and human liver engrafting cells.

Stem Cell Therapeutics Corp. (CVE:SSS) and BrainStorm Cell Therapeutics (OTC:BCLI) take cells from patients’ own bone marrow in order to treat, Parkinson’s, ALS, spinal cord injury, etc.

Transitions Therapeutics Inc. (NASDAQ:TTHI) and Ixion Biotechnology, Inc. focus on using islet beta cells in the pancreas to treat diabetes.

The addition of embryonic stem cells to genetic therapy has the potential to revolutionize the revolutionary.

Imagine… rather than focusing all their money and time on one specific type of cell, they could apply their science to cells affecting areas throughout the body.

These unspecified embryonic cells can (again, in theory) be specialized to fix whatever ails you, once the science catches up.

The tiny biotech firms would no longer have to rely on qualified adult donors (think of all the restrictions the Red Cross now has regarding acceptable blood donors!).

And with the relative ease of embryonic stem cell culture proliferation, experimentation can reach new levels.

Obama Administration to support stem cell research

President-elect Barrack Obama has clearly stated his opinion, “ … we must all work together to expand federal funding of stem cell research and continue moving forward in our fight against disease by advancing our knowledge through science and medicine.”

And that could mean lower costs and higher ROI for these small biotech companies (many of which are trading under $2 today!).

Source: New administration could mean advances — and profits — for U.S. stem cell biotech companies