MassBio News

An Update from MBC President Bob Coughlin

Wed, 27 Aug 2008 00:00:00 EDT

August 27, 2008—It has been one year since Bob Coughlin took the reins at MBC. In this letter, he shares information on what the council has been doing for members this past year—and where the organization is going next.

Congratulations to Dyax

Wed, 20 Aug 2008 00:00:00 EDT

MBC congratulates Dyax for Phase 3 Trial success!

MBC Swings to Support the MassBioEd Foundation

Mon, 11 Aug 2008 00:00:00 EDT

Join us for a day of golf - and support our efforts to educate the next generation!

On Friday, Sept. 5, the MBC will hold its 14th Annual Golf Classic Fundraiser, Tee Off for Biotechnology Education, to benefit the Massachusetts Biotechnology Education Foundation.

MBC member companies can participate in a number of ways. You are invited to compete in the tournament in teams of four, sponsor the event, or make a donation to attend the reception following the tournament. With 350 athletes/biotech industry leaders in attendance, networking opportunities during and after the tournament abound.

For each of the past two years, Tee Off for Biotech Education has raised more than $100,000 for MassBioEd and its programs. MBC's ongoing commitment to the MassBioEd Foundation is based on the belief that the industry must do more to prepare the workers of tomorrow by providing students the best biotechnology educational opportunities today. The work of MassBioEd contributes to the state's cutting-edge, competitive workforce that attracts employers from around the world.

Here are some highlights of the Foundation's work:

The BioTeach program empowers teachers and inspires students through grants that provide teachers with professional development and schools with equipment and supplies needed to teach biotechnology labs in their classrooms. To date, BioTeach has reached 162 public high schools in the Commonwealth - 45 percent of all public high schools - and provided professional development for 150 teachers this summer alone.

The Biotech Learning Center offers courses, seminars, and hands-on training targeted towards professionals already in the life sciences industry. They can expand their skills and gain the necessary knowledge to pursue more advanced career options in the biotech sector.

Over the past 18 months, MassBioEd has been a leader in advocating for a statewide system of mobile labs to provide biotechnology to students who do not have access to needed scientific resources. The Massachusetts Life Sciences Initiative appointed the Foundation as the lead agency to purchase and convert buses into functioning mobile labs. Over the course of the next year, MassBioEd will develop collaborations and partnerships with the Department of Higher Education, the Massachusetts Life Sciences Center, and institutions of higher learning to make this program a reality and expand biotechnology education to the Commonwealth's middle and high schools.

PRESIDENT SIGNS BILL THAT BOOSTS BIOTECH

Mon, 04 Aug 2008 13:30:00 EDT

Measure Will Allow Companies to Redeem Earned but Unusable Tax Credits for Cash
President Bush has signed multi-billion dollar legislation addressing the nation's mortgage and housing crises that also contains an economic stimulus jolt for the biotechnology industry.

H.R. 3221 - inked by the President in an Oval Office ceremony and meant to ease foreclosure fears spurred by collapse of mortgage-related sectors - contains an MBC and BIO supported tax provision which will allow companies to expedite use of their R&D and AMT credits in lieu of the bonus depreciation utilized by other industries with different business models.

Specifically, the bill allows biotechnology companies making new capital investments during much of 2008 in qualifying laboratory equipment and software, or those gearing up for commercialization, to be able to receive from the government a "refundable" tax credit (i.e., the amount of the credit will be refunded to the company, even if it is not paying taxes as it is in a "loss" position). The bill's passage comes at a time when a severe capital crunch in the financial markets is threatening to put the brakes on biotechnology innovation. (Description of bill's provisions can be found on sidebar; final regulations to be issued by Dept. of Treasury.)

"This is a bonus and a benefit for the Massachusetts biotechnology industry on multiple fronts," MBC President Robert Coughlin said. "First and foremost, it rewards the good economic behavior of biotech companies which earn significant tax credits for their investments but have no taxable income against which to apply these credits." Coughlin continued, "Additionally, for the first time Congress has acknowledged that R&D-intensive biotechnology companies should be able to redeem hard earned but unused tax credits for working capital so that they may accelerate the discovery and development of new innovative products."

The ability to use R&D credits is doubly beneficial for biotechnology because the tax code generally requires a company to use its accumulated Net Operating Losses (NOLs) before its tax credits can be claimed. As a result, R&D credits will sometimes expire before they can be utilized. The provision not only allows companies to accelerate the date when they can claim their R&D credits, it also allows them to claim credits that might otherwise expire under existing tax law.

Numerous Massachusetts biotechnology companies are expected to take advantage of what will be for now a one-time cash benefit and to reinvest that capital into the Massachusetts economy. MBC President Coughlin noted that Massachusetts Senator John Kerry and Congressman Richard Neal (D - Springfield) are key players on the budget writing committees that preserved the biotechnology tax benefit measure within the larger housing measured as it moved through the legislative process. "We want to thank Senator Kerry and Congressman Neal for their work on this bill, and of course we thank the entire delegation for voting in its favor," said Coughlin.

CLICK HERE FOR NEW 2008 REFUNDABLE AMT AND R&D CREDIT ELECTION EXAMPLES

WITH SENATE ACTION, MBC MEMBERS WITH VC BACKING EDGE CLOSER TO QUALIFYING FOR SBIR GRANTS AGAIN

Wed, 30 Jul 2008 00:00:00 EDT

After five years of frustration in Congress, biotechnology companies with significant venture capital backing are on the verge of once again qualifying for SBIR (Small Business Innovation Research) grants, thanks in large measure to the persistent advocacy of MBC and its members.

On July 30, the Senate Small Business and Entrepreneurship Committee - chaired by John Kerry of Massachusetts - approved legislation that will allow companies with majority venture capital fund ownership to compete for a portion of SBIR dollars, including NIH funding. Such companies have been totally barred from SBIR participation since 2003.

The Senate action follows House passage of language even more favorable to biotechnology companies. Senate and House versions will now have to be reconciled.

MBC President Robert Coughlin said that although MBC will continue to push for less stringent SBIR limits on its venture backed members, MBC was generally pleased with the progress made on the issue in Congress thus far this year.

"These votes represent a big win for MBC member companies in need of capital for development of innovative new medicines," said Coughlin. "This is a clear indication that our Congressional leaders recognize that the SBIR program is perfectly suited to help MBC members in their pursuit of solutions for unmet medical needs, and that our members deserve to compete for SBIR funding."

SBIR grants had been an essential source of early stage funding until 2003, when a rules changed prohibited companies with 51% or more venture fund ownership from program participation. Knowing that most early stage biotechnology companies rely heavily on venture funding, MBC has been working to overturn that ruling ever since, advocating for the industry position on Capitol Hill, discussing the issue in depth with members of the state's Congressional delegation, and undertaking an intensive member-driven informational outreach campaign.

Under the Senate Small Business Committee version approved July 30, majority venture backed companies would be eligible to compete for a pool of dollars equivalent to 18% of NIH's total SBIR budget, 8% at all other federal agencies. The House set no such limits, and MBC will work to increase the venture company carve-out in the final legislation.

The Senate bill also proposes an increase in Phase II grants to $150,000 (up from $100,000), with Phase II going to $1 million from $750,000. The rules change is part of overall SBIR reauthorization legislation, which has to be approved before Congress adjourns in late September or else the program will be unfunded next year. The Senate version proposes a 14 year reauthorization, compared to the House bill which only reauthorizes the program for 2 years.

Members with questions about the pending SBIR legislation should contact the MBC Policy and Public Affairs Department.

GOVERNOR PATRICK SIGNS BLOCKBUSTER LIFE SCIENCES INITIATIVE

Wed, 25 Jun 2008 12:00:00 EDT

On June 16, at Boston's Joslin Diabetes Center, Massachusetts Governor Deval Patrick signed into law the state's landmark Life Sciences Initiative (LSI), then immediately flew to San Diego with legislative leadership to tout the plan in front of the world's biotechnology industry at the BIO 2008 International Convention.

Unprecedented in scope in the annals of Massachusetts politics and policy, the LSI - through which the state will invest at least $1 billion in its surging life sciences sector over the next decade - is expected to strengthen the Commonwealth's global leadership position in such fields as biotechnology and medical devices.

Just as important, the law seeks and is expected to spur bold advances in novel areas of scientific discovery, such as stem cell and RNAi research.

The Massachusetts LSI was a hot topic at San Diego's BIO 2008, held in the backyard of one of the Commonwealth's foremost competitors for life sciences business. Joined by Senate President Therese Murray and House Speaker Salvatore DiMasi, Governor Patrick talked up the LSI at a number of BIO 2008-related venues, including a jam-packed event at the Massachusetts Pavilion on the BIO exhibit hall floor (see photo), a "Town Hall" informational session at a nearby hotel, and a reception for industry leaders at PETCO Park, home of the San Diego Padres.

While there has been considerable enthusiasm about the LSI generally, it is clear that fleshing out LSI program particulars has now become the next big challenge for both government and the life sciences sector. Questions still abound about program eligibility and access. The MBC, which played a major role in the LSI's formation and passage, is dedicated to the LSI's final fitting, roll-out and implementation. "There is a lot of hard work on the LSI behind us, and a lot of hard work ahead," commented John Heffernan, MBC's Vice President of Policy and External Affairs. "The best news is that our state government has remained consistent in its commitment to the life sciences sector, and we look forward to working with leadership to justify this expenditure of taxpayer dollars, which we are confident will lead directly to a stronger economy and better health care."

The LSI calls for spending hundreds of millions of dollars on infrastructure, including construction of a facility at the University of Massachusetts Medical Center in Worcester dedicated to RNAi research pioneered by the University's Nobel laureate, Dr. Craig Mello. That stream of funding will also create what is expected to be the world's foremost stem cell bank and registry, established as a collaboration among UMass, Harvard and MIT.

Industry is most excited about nine tax benefits and incentives that will be made available to qualifying companies showing a demonstrable job-growth profile (for handy one-page digest of LSI highlights, click here). Benefits include an increase in the investment tax credit (ITC, to 10%) and an extension of the net operating loss carryforward (from five to fifteen years). For the first time qualifying life sciences companies can opt to redeem and monetize state tax credits (R&D, ITC) for up to 90% of face value, and the LSI establishes a 100% tax credit for FDA User Fees, also redeemable for cash if the company so chooses.

Small companies and research institutions are carefully eyeing $250 million in LSI grants and loans. One fund (named after the aforementioned Nobel Prize winner Craig Mello) will provide equity investments of up to $250,000 to small life sciences companies. Other features include an SBIR matching fund and grants to help financially strapped graduate students so that they may continue to live and do their great work in the Commonwealth.

As is often the case, the Legislature wrote LSI language specifically in some instances, more broadly in others. The MBC is committed to working with the administration and with the Life Sciences Center - the LSI's lead authority - to fine tune programmatic language and details.

Most important, the MBC plans to produce a guide - revisable as necessary - that will help its members navigate through the LSI's various programs and opportunities. A first edition of this guide should be available by mid-summer. Interested parties should contact the MBC with questions or comments.

MBC Applauds Passage of SBIR Reauthorization in US House of Representatives

Wed, 30 Apr 2008 00:00:00 EDT

MBC Played Advocacy Role to Help Biotech Companies Become Eligible for Federal Grants Once Again

Download the complete text of the letter MBC sent to MA Congressional delegation on SBIR issue by clicking here.

On Wednesday, April 23, the U.S House of Representatives voted overwhelmingly in favor of legislation that would once again let MBC member companies with venture capital funding compete for federal small business grants. The final tally was 369 - 43 in favor of Small Business Innovation Research (SBIR) Reauthorization. SBIR grants had been an essential source of early stage funding until 2003, when a Small Business Administration rules change barred some companies with venture backing from program participation. In last week's decision, eight of the ten House members from Massachusetts voted in favor of the MBC position rescinding that rules change and prohibition against venture capital backed firms.

"This is a big win for MBC member companies that are in need of capital for development of new innovative medicines," commented MBC President Robert Coughlin. "This is a clear indication that our congressional leaders recognize that the SBIR program is perfectly suited to help MBC members in their pursuit of solutions for unmet medical needs, and that our members deserve to compete for SBIR funding."

The MBC has been working on SBIR since the 2003 rules change, but the issue gained fresh momentum in the last few weeks when Congresswoman Nydia Velazquez (D - NY), House Chair of the House Small Business Committee, introduced a bill that would modify the eligibility rules affecting venture capital backed small companies. If passed, HR 5819 will increase access to critical, early-stage sources of funding for small businesses, including small biotechnology firms with venture backing, thus facilitating economic growth, job creation, new breakthrough therapies for patients in need, and American economic competitiveness in the global economy.

Introduction of the Velazquez bill was well aligned with the MBC's reinvigorated federal action plan, spearheaded by Coughlin. Last month, as he and MBC staff advocated for industry positions on Capitol Hill, they were able to discuss the SBIR issue in depth with members of the Massachusetts Congressional delegation. The MBC also engaged in an intensive letter writing campaign. (See sidebar, copy of letter MBC sent to MA Congressional delegation on SBIR issue.)

Tom Mathers, President and CEO of Peptimmune, Inc., in Cambridge, noted that promising therapies for hemophilia and Alzheimer's disease developed at two companies he has headed languished because of lack of SBIR funding. Mathers, who recently joined with MBC in meeting with members of the Massachusetts Congressional delegation, hailed the vote as a boost both for companies and for patients.

"Given the established infrastructure of VC backed companies, the SBIR grants would translate immediately into job creation," said Mathers. "A Phase I SBIR grant of $400,000 to my company would provide an opportunity for us to hire 2-3 new employees immediately." Mathers continued, "The irony about this limitation is that foundations that sponsor some research will only provide grants that are in VC backed companies…this VC investment serves as validation of the technology or management within the company!"

In 2003, the Small Business Administration ruled that companies more than 51% controlled by venture capital firms were ineligible for SBIR grants under an arcane interpretation of SBA ownership rules. The rule also impacted companies whose venture capital supporters had portfolio companies whose employees combined surpassed 500, another interpretation of the "affiliate" rule that has impacted our small member companies.

Specifically, HR 5819 would allow an SBIR applicant to be majority owned by VCs, with some conditions: a VC could not own greater than 51% of the company; no single VC has majority control of the board; and, the biotech could have ownership by no more than 2 corporate VC's (with a total of 20% ownership collectively). The last condition was developed due to concerns by House Members about "large companies" participating in the program.

MBC will now shift focus to the Senate, where the Small Business Committee has yet to mark up its SBIR reauthorization bill. Senator John Kerry of Massachusetts is chair of the Senate's Small Business Committee, and MBC will continue our efforts, and work with Senator Kerry and his able staff to advocate that venture backed companies from Massachusetts need and deserve to compete for SBIR funding.

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MBC Launches New Quarterly Newsletter

Tue, 29 Apr 2008 00:00:00 EDT

Click here to download the PDF version.

EMD Serono: Message of Hope

Fri, 04 Apr 2008 14:40:00 EDT

Listen to EMD Serono's Message of Hope

EMD Serono, Inc. is a leader in the US biopharmaceutical arena, integrating cutting-edge science with unparalleled patient support systems to improve people's lives. With fully integrated commercial, clinical and research operations at its headquarters in Rockland, MA, and research and production operations in Billerica, MA, EMD Serono's US footprint continues to grow with more than 850 employees around the country.

Through its focus on specialized therapeutic areas, including neurology, reproductive health, and metabolic endocrinology, as well as oncology and autoimmune diseases as emerging areas of expertise, EMD Serono is committed to discovering and developing innovative products that address unmet medical needs. With unique delivery systems and access support for many of its products, as well as educational resources across all of its therapeutic areas, including MS LifeLines®, Fertility LifeLines(TM), Connections for Growth® and SeroCare®, EMD Serono continues to improve the patient experience and help people live full and healthy lives.

Oncology

With several trials currently underway in the US in the area of cancer, EMD Serono is building a strong oncology pipeline that demonstrates a long-term commitment to developing new therapies for this unmet need. From early to later stage, the organization's cancer pipeline targets a range of cancers, including breast, prostate, colon, lung, multiple myeloma, b-cell malignancies, and more. Here are two examples of ongoing trials in the US:

Stimuvax
In Phase III, Stimuvax is being studied as a potential therapeutic vaccine for non-small cell lung cancer.

Atacicept
Atacicept is a soluble fusion protein in Phase I and is being investigated as a potential treatment of multiple myeloma and B-cell malignancies. It is thought to bind and neutralize cytokines that may be implicated in B-cell differentiation and survival as well as antibody production.

Alnylam: Message of Hope

Fri, 04 Apr 2008 00:00:00 EDT

Listen to Alnylam's Message of Hope

Alnylam Pharmaceuticals was founded in 2002 and now has approximately 110 employees. Based in Cambridge, Mass, the company is a leader in the development of RNAi therapeutics. Discovered in 1998, RNAi is a naturally occurring tool the body uses to turn off a gene's function when it isn't needed. This property makes RNAi a fantastic weapon against human disease. Using RNAi, Alnylam is developing a deep pipeline of drug products to treat a wide array of important diseases.

DISEASE PROGRAMS USING RNAi

Respiratory Syncytial Virus (RSV)

Alnylam is working to create a therapy for RSV, the most common cause of bronchiolitis and pneumonia among infants and children under 1 year of age. Using a direct RNAi approach, Alnylam will deliver the drug directly to the lungs in order to silence the gene the virus uses to infect the lung cells, neutralizing the virus and preventing further spread and infection. In June 2007, Alnylam initiated a second clinical trial to evaluate the safety, tolerability, and anti-viral activity for its lead therapy in experimentally infected subjects. Data are expected in the the first quarter of 2008.

Hypercholesterolemia

About 105 million U.S. adults have cholesterol levels of 200 mg/dL or higher (CDC, May 2007)
Familial hypercholesterolemia occurs in approximately 1 in 500 people in the U.S. (~500k people)
High cholesterol is a leading cause of heart disease, the #1 cause of death in the United States (CDC, May 2007)

Alnylam is collaborating with researchers at UT Southwestern Medical Center to evaluate new approaches for reducing LDL-cholesterol ( or "bad" cholesterol) levels using RNAi therapeutics directed to a disease target. In a pre-clinical study, this team has shown that their targeted RNAi approach can result in an approximately 50% lowering of LDL cholesterol.

Liver Cancers

Primary liver cancer is one of the most common cancers worldwide, with more than 600,000 people diagnosed each year. Secondary liver cancer, also known as metastatic liver cancer, is cancer that spreads to the liver from another part of the body. Worldwide, more than 500,000 people are diagnosed with secondary liver cancer each year. Alnylam is now in testing of its therapy that enables the efficient silencing of genes associated with liver cancer. In pre-clinical studies, Alnylam has demonstrated the ability to silence two important genes associated with liver cancer, stopping the cancer cells from growing.

Huntington's Disease (HD)

According to the Huntington's Disease Society of America (HDSA), one out of every 10,000 Americans has HD
Each child of a parent with HD has a 50/50 chance of inheriting the gene that causes HD
There are approximately 200,000 Americans "at-risk" of developing the disease

Alnylam and Medtronic are focusing on developing a drug-device combination for the treatment of Huntington's disease. The product is expected to consist of an RNAi therapeutic targeting the Huntington's disease gene that will be delivered by Medtronic's implantable infusion pump.

Genocea: Message of Hope

Fri, 04 Apr 2008 00:00:00 EDT

Listen to Genocea's Message of Hope

Genocea is a vaccine development company based in Cambridge, MA. Based on nine years of research at University of California at Berkeley and Harvard Medical School, Genocea's rapid screening technology will help the U.S. lead in vaccine discovery and preparedness capabilities for infectious disease outbreaks. Genocea is currently in pre-clinical development for Chlamydia, and many other disease areas could benefit from the company's technology, such as Strep, Malaria, Hepatitis, HIV, and other complex diseases to vaccinate against.

Current Vaccine Production Environment

Methods for developing new vaccines are slow and imprecise. The common way of discovering new vaccines normally takes months, if not years, of brute force trial and error. Scientists spend months examining each of the thousands of genes that are present in a pathogen. One by one, they test every conceivable component of a pathogen to see if it causes an immune system response. Scientists have lacked the ability to precisely mimic the human immune system in the laboratory and simultaneously analyze all possible combinations of genes. Genocea's technology changes the discovery paradigm by reconstituting the human immune system and allows scientists to screen every possible vaccine candidate within hours to find the most effective formula.

Approach

Genocea and its academic colleagues have developed an approach for rapid vaccine discovery that can be optimized for infectious disease, bioterrorism agents, cancer, and autoimmune conditions. The technology has the following advantages over conventional discovery methods: 1) rapid discovery of new vaccines (hours vs. months or years using conventional technology) 2) wide disease applicability 3) improved protection from diseases and bioterrorism agents by removing the guesswork of what turns on the immune system.

Background

Vaccination was first developed in the 18th century, when it was observed that exposure to cow pox could protect individuals from contracting small pox. Immunization was originally practiced by arm-to-arm transfer of the cow pox infection, but was replaced in the early 19th century with inoculations. Vaccination works by priming the immune system with various forms of pathogen (bacteria or virus), so that when the live pathogen is encountered, a much more powerful immune response is launched which effectively kills the virus or bacteria without causing disease. Using a whole bacteria or virus to prime the immune system is not a good approach for certain high risk patient populations like children, the elderly or immunocompromised patients. Giving just the small piece of the bacteria or virus that stimulates the immune system (Genocea's method) is a far safer, and often cheaper, approach.

There are two major types of pathogens. Extracelluar pathogens such as bacteria, fungi, worms, and protozoa affect the surface of cells. When these organisms attack, the body's immune system releases cells called antibodies, also called B-cells, to clear the infection. Antibodies, however, are ineffective against eliminating more complicated intracellular pathogens such as viruses, intracellular bacteria, and intracellular protozoa. Many of the world's leading infectious diseases such as TB, AIDS, malaria, and chlamydia are caused by intracellular pathogens To clear intracellular infections, specialized cells are required that can either directly kill infected cells, or can induce the infected cells to mobilize mechanisms for killing the intracellular pathogens . These specialized cells are called T cells.

When an intracellular pathogen invades a cell it is broken into pieces. These pieces are protein fragments called antigens. Antigens are brought to the surface of the cell and act as flags that T-cells recognize. When a T-cell latches onto one of these flags it invokes an immune response to kill the disease. These specific antigens are called "immunogenic antigens", meaning they stimulate an immune response.

Genocea's technology rapidly identifies every immunogenic antigen from a pathogen that all types of T-cells recognize, including new antigens that may have emerged from new strains of a disease. These are highly valued by pharmaceutical companies and governments, and can immediately be formulated into vaccines for clinical trials.

Beth Israel Deaconess Medical Center: Message of Hope

Fri, 04 Apr 2008 00:00:00 EDT

Listen to Beth Israel Deaconess Medical Center's Message of Hope

A major teaching hospital of Harvard Medical School, Beth Israel Deaconess Medical Center was created in 1996 through the merger of two prominent institutions, Beth Israel Hospital and New England Deaconess Hospital. Renowned for excellence in patient care, teaching and community service, BIDMC is also home to a preeminent academic research program, widely recognized for its translational "bench to bedside" research.

BIDMC's 264 principal investigators, all of whom are Harvard Medical School faculty, lead more than 850 active sponsored projects and more than 500 funded and non-funded clinical trials. Today, research at BIDMC is a $200 million enterprise, consistently ranking in the top four in National Institutes of Health (NIH) funding among independent hospitals nationwide.

Studies by BIDMC researchers are routinely published in the world's leading scientific journals including Nature, Science and The New England Journal of Medicine. Vascular biology - the study of the structure and function of the body's blood vessels - is a key area of research at BIDMC, as are molecular imaging, transplantation, signal transduction, cancer biology, metabolic disease and obesity, neurobiology, AIDS, inflammation and cardiology/cardiac surgery.

Technology Ventures Office

Since its creation in 1998, BIDMC's Technology Ventures Office has helped to translate hundreds of scientific discoveries from the labs of BIDMC scientists into commercial ventures that improve human health. BIDMC revenue from new and existing TVO deals totaled more than $10 million in 2006.

TVO-initiated strategic alliances with global health care companies including Japan's Takeda Pharmaceuticals, Merck, AGA Linde, and GE Health Care provide BIDMC researchers with many years and millions of dollars of research support, and provide efficient avenues for the translation of important new discoveries into new drugs, diagnostics, and imaging agents for diabetes, obesity, cancer, and heart disease.
Recent patents to emerge from the TVO office include a new method for diagnosing and treating sepsis, a device for the treatment of congestive heart failure, and several new compounds that hold promise for the treatment of cancer, diabetes, colitis, cystic fibrosis, and numerous other diseases.
The TVO team's successful partnerships with industry have led to the development of new biomedical products that are helping patients worldwide. Some examples include of products that have been derived from inventions made at BIDMC: Zenapax (sold by Roche) and Simulect (sold by Novartis) drugs that help patients tolerate newly transplanted organs; Impact (sold by Novartis), Optimental (sold by Ross), and Immnuo-Aid (sold by McGraw), nutritional supplements that are used in chronic care and post-surgical settings; the Cohn Stabilizer, a medical device sold by Teleflex that assists surgeons in the performance of off-pump open-heart surgery; T-Wave Alternans, an algorithm recently approved by the FDA, sold by GE and Medtronic, that predicts sudden heart failure; and SMASH, a technique developed at BIDMC to improve the resolution of magnetic resonance imaging, (sold by Siemens, GE, and Bruker.)

In addition, the TVO office has catalyzed the formation of over 20 new biomedical startup companies, including DeCode Genetics, Elixir Pharmaceuticals, Synta Pharmaceuticals, Cequent, and Tolerance. Many of these companies are developing leading-edge technologies that could lead to be the next generation of new medicines.

Massachusetts Biotechnology Council: Sustaining the Biotechnology Ecosystem

Mon, 31 Mar 2008 00:00:00 EDT



S. A. Karumanchi, MD	Jessica Flechtner, Ph.D.	Rachel Meyers, Ph.D.	Lee Ferrande
Division of Molecular and Vascular Medicine	Senior Scientist and Group Leader	Senior Director, RNAI Lead Development	Clinical Trials Manager
Beth Israel Deaconess Medical Center	Genocea Biosciences, Inc.	Alnylam Pharmaceuticals	EMD Serono
Research Hospital	Small Biotech	Mid-Sized Biotech	Large Bio/Pharma

Message of Hope: Advancing Science to Solve Unmet Medical Needs

Massachusetts Biotechnology Council: Sustaining the Biotechnology Ecosystem

In the Message of Hope radio series, you hear real stories of three Massachusetts researchers and a clinical trials manager who work each day to produce cures for unmet medial needs.

These talented individuals come from a cross-section of the Massachusetts Biotechnology Council Membership (MBC): an academic research institution, a small biotechnology company with 15 employees, a medium sized, public biotechnology company pushing for their first product, and a large biopharmaceutical company working on its next promising therapy.

Their stories could be from any of the biotechnology, pharmaceutical, or major research institutions that make up the membership of the MBC, the oldest biotechnology trade association in the United States. Supporting these research institutions are the MBC members the industry needs to sustain growth.

Short History of Biotechnology in Massachusetts

While the pharmaceutical industry is greater than 100 years old, the biotechnology industry is far less mature - roughly 30 years old. The first biotechnology company in the U.S. was San Francisco's Genentech, founded in 1976. But Cambridge's Biogen, founded in 1978 (now Biogen Idec), and Genzyme, founded in 1981, soon followed.

After nearly three decades of growth, the Commonwealth ranks as one of the most significant biotechnology centers in the world. Today, there are over 400 biotechnology companies located in the state, of which 235 are developing therapeutic drugs. Roughly 42,917 biotechnology employees in Massachusetts are responsible for over $5 billion of in-state payroll. Biotech employment grew by 25.6 percent between 2001 and 2005, adding over 8,000 jobs. It is estimated that for each new biotech position, five positions are created in other supporting industries.

Over this short span of time, Massachusetts-based biopharmaceutical companies have already developed 50 large molecule drugs covering a range of illnesses, including areas such as cancer, infectious and neurological diseases. Today, there are nearly 1,769 drugs being developed in Massachusetts, representing over 7 percent of the global drug pipeline.

The Commonwealth's biotech industry continues to grow. Twenty-one of the top 50 biopharmaceutical companies by revenue have a Massachusetts presence. These companies average $21 billion each in revenue over the past year.

How does a biotech drug differ from a traditional drug?

Traditional pharmaceuticals are small molecules made through chemistry. Biotechnology therapies are made using living cells. These therapies are 100 to 1000 times larger than pharmaceutical pills, and are typically injected or delivered through another special delivery system.

A few milestones mark the development of this field. The first breakthrough was the decoding to DNA's structure in 1953. Then in 1973, scientists learned how to create artificial, or recombinant DNA. With recombinant DNA, researchers developed a way to take DNA from one organism and put it in the DNA of another. This process was used to develop human insulin for diabetics. Human insulin DNA was placed into the DNA of a bacterium, which then became an insulin-producing factory.

In 1982, the Food and Drug Administration approved the first biotech therapy - bacterially produced human insulin. Today, 200 biotechnology companies have at least one commercial product, covering diseases such as diabetes, hepatitis B (vaccine), anemias, hemophilia, bone growth, rheumatoid arthritis, pneumococcal disease (vaccine), and cancers.

How did Massachusetts become the Biotech Hub?

In the 1930s, the United States Congress formed a new agency, the National Institute of Health. Over time, "Institute" became "Institutes" as appropriations and mandates grew.

Massachusetts research hospitals and academic medial centers soon became the epicenter for discovery. Today, the Commonwealth is home to the top five National Institutes of Health funded hospitals, including Beth Israel Deaconess Medical Center, Brigham and Women's Hospital, Children's Hospital Boston, Dana-Farber Cancer Institute, and Massachusetts General Hospital.

With federal and private funding, researchers at these hospitals as well as Harvard University, the Massachusetts Institute of Technology, University of Massachusetts, and other MBC member higher education institutions have produced novel, paradigm shifting research - knowledge that can be used to build new therapeutics.

Novel research, or "technology", produced by academic researchers is then often licensed to entrepreneurs who believe they can take this knowledge and develop it further into cures.

But to build new drugs from fundamental research you need further investment. Fortunately, Massachusetts has strong support from venture capital funds. Last year, eighteen percent of U.S. biotechnology venture capital was invested in Massachusetts.

The Biotech Landscape: Massachusetts is not alone

Many states in the U.S., as well as foreign countries, are seeking to build economies around biotechnology. There are roughly 4,500 public and private biotechnology companies worldwide (compared to 12,000 pharmaceutical companies). In the U.S., there are approximately 1,452 public and private companies accounting for 180,800 jobs.

In terms of public companies, in 2006 the U.S. had 336 public companies, Europe had 156, the Asia-Pacific region had 136, and Canada had 82. In total, the U.S. had 43 percent of the public biotechnology companies worldwide.

Massachusetts and the U.S. in general are seeing growing international competition around the biotechnology sector. Already, Sweden, Israel, Finland, and Japan spend more public funds on research and development as a percent of gross national product than the U.S. Following the U.S. on this list is South Korea, Switzerland, Germany, Iceland, France, Denmark, Netherlands, Belgium, United Kingdom, and Singapore.

In the U.S., a sign of biotechnology growth in individual states is the amount of venture capital support they receive. In 2005, the top ten states in biotech venture capital were California ($1,819 mil); Massachusetts ($562 mil); New Jersey ($192 mil); North Carolina ($191 mil); Maryland ($148 mil); Pennsylvania ($133 mil); Washington ($131 mil); Connecticut ($57 mil); Texas ($53 mil); and Michigan ($31 mil).

State Incentives: Building Biotech Economies

To nurture biotech growth, many U.S. states are developing innovative financing strategies. California's $3 billion stem cell initiative is perhaps the best known, but many other states have passed life sciences incentive legislation.

In 2005, as part of a tobacco settlement, the state of Washington passed legislation allocating $35 million per year for bioscience research;
Maryland passed a bill in 2005 that provided a tax credit for individuals, corporations, and qualified venture capital firms that invest in a state-based biotech with less than 50 employees and is less than 10 years old;
In 2004, Kansas passed legislation that created a BioAuthority to administer funds and incentive programs. The BioAuthority will distribute $500 million over the next 10 to 12 years;
Pennsylvania passed legislation in 2001 providing $100 million for regional bioresearch centers as part of their tobacco settlement. This was part of Pennsylvania's larger $2 billion Biosciences Enterprise Commitment to fuel research and development, venture capital, and other early stage company investment.

Several other states have passed bio-friendly legislation, including Arizona, Arkansas, Colorado, Delaware, Georgia, Illinois, Kentucky, Michigan, Missouri, New Jersey, North Carolina, Ohio, Oklahoma, and Texas. Legislative initiatives typically support Bioscience Workforce Initiatives; Facilities Development; Business Climate Incentives; Tech Transfer Funding; Stem Cell Funding; and Venture Capital and Discovery Funds.

Dangers to the Biotech Ecosystem

Massachusetts has long had a strong life sciences ecosystem, which has contributed to the growth of the state's biotechnology industry.

Basic science researchers in the state's academic medical centers and universities attract strong federal research support. From this research, these investigators produce "disruptive" discoveries, i.e. knowledge that can change the therapeutic landscape.

The state has many talented entrepreneurs who have built biotechnology companies from licensed technology, and their expertise in developing products is respected by New England's venture capital community.

The state also enjoys strong state and federal leadership who recognize that the industry is still maturing and that policy has the ability to strengthen or weaken any part of the ecosystem, and therefore benefit or hurt all of its collective parts.

But dangers are threatening the ecosystem, particularly at the federal level. The National Institutes of Health budget doubled between 1998 and 2003. However, since 2003, increases have not kept up with inflation, which in effect has caused the budget to fall each year. The funding success rate of NIH applications has therefore fallen from 31 percent to 19 percent overall. It is not uncommon for proposals scored in the top 10 percent to go unfunded. This environment puts at risk a whole generation of young scientists seeking their first federal grant. It also puts the U.S. at a disadvantage, allowing other countries to potentially move ahead quickly in areas such as stem cell and genomic research knowledge.

Beyond the need to increase the NIH budget, two other areas threaten the ecosystem. The first is patent legislation that would weaken the value of licensed technology from academic researchers and hospitals to the biotech community. The strength of any biotech company is its patent protected technology. Venture capital funding is given to biotechnology companies in part based on the strength of their patented technology. Legislation passed in the House of Representatives earlier this year seriously weakens biotechnology patent protections, and the U.S. Senate is now considering patent legislation.

The second equally worrisome legislation surrounds follow-on biologics. The U.S. Senate has produced strong legislation that would give biotechnology companies 12 years of protection before a company's therapy could be produced by another company. The biotechnology community is seeking similar protections in legislation being drafted in the House. It takes 14 to 16 years for a company to recapture the $1.5 billion investment made in producing a new biotechnology therapy. If legislation is passed that would allow secondary companies to copy a drug sooner than 12 years, then the future of biotechnology as an industry will be weakened.