H1N1 put a spotlight on Novartis's success with vaccines based on cells, a process that's faster and more reliable than the traditional method of growing viruses in chicken eggs. Novartis won EU approval to sell cell-based flu vaccines, and FDA approval is pending. Top 50 No. 8
2. Synthetic Genomics
Craig Venter's baby has teamed up with ExxonMobil to develop microbes that produce biofuel. Exxon's $600 million stake signals that algae fuel is going mainstream. Venter's business acumen, combined with Exxon's production and marketing power, positions Synthetic Genomics to dominate the market long-term. Top 50 No. 26
3. Cytori Therapeutics
Given the difficulties associated with embryonic stem cells, adult stem cells look like the therapeutic wave of the future, and Cytori is out in front. Its Celution system harvests adult stem cells with maximum efficiency, and its regenerative therapies have shown promise in trials.
Roche was the talk of the health-care world this year when it acquired biotech giant Genentech for $46.8 billion—a high-risk move that seems likely to be high reward. Genentech has three of the five best-selling biotech drugs.
5. Human Genome Sciences
As we noted in "The Gene Bubble" (November 2009), decoding the human genome has not produced miracle cures. But this year, Human Genome Sciences announced that its lupus drug, Benlysta, had produced substantial improvements in clinical-trial participants—a conspicuous success since so many lupus treatments have flopped.
6. Osiris Therapeutics
Like Cytori, Osiris is at the vanguard of the stem-cell revolution. According to Osiris, its marquee drug, Prochymal—a preparation of adult stem cells from connective tissue—may work for ailments as varied as diabetes and graft-versus-host disease.
7. Amyris Biotechnologies
Amyris is engineering yeast molecules to churn out hydrocarbon-based biofuels. It says it's on target to bring its fuels to market by 2011, giving it an initial edge over renewable-fuel stars like Synthetic Genomics.
8. Biogen Idec
Biogen shrewdly responded to concerns about side effects of its drug Tysabri, a leading treatment for people with relapsing multiple sclerosis, by launching a Web tutorial, teleconferences, and a mentor program for patients. Biogen boasts 20 drugs in Phase II clinical trials or beyond.
Novavax develops vaccines using proprietary virus-like particles (VLPs) rather than live viruses. The particles trigger the same immune response as a virus but cannot cause infection. In December, Novavax released the results of Phase II clinical trials of a VLP vaccine that proved effective against three kinds of flu, including H1N1.
10. Regeneron Pharmaceuticals
Big Pharma heavyweight Sanofi-Aventis has promised Regeneron an additional $1 billion over seven years to broaden its drug-development efforts. Regeneron's pipeline of human antibody products, now in clinical trials, treat rheumatoid arthritis, gout, chronic pain, and cancer.