Business Concentrates GENE THERAPY
Moderna mRNA therapy passes safety test
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Biotech firm and AstraZeneca release first clinical data testing mRNA for therapeutic-protein production Moderna has published its first peer-reviewed study testing the safety of its messenger RNA molecules for therapeutic-protein production in humans. The small, Phase I trial, conducted in collaboration with AstraZeneca, showed that an experimental mRNA therapy called
A scientist in Moderna’s research labs in Cambridge, Massachusetts AZD8601 led to localized and temporary production of a therapeutic protein without severe side effects after it was injected into the skin of 33 men (Nat. Commun. 2019, DOI: 10.1038/s41467-019-08852-4). Moderna has raised more than $2.6 billion in private funds to create its mRNA platform and fill its pipeline with 21 experimental therapies. In December, the company raised some $600 million more in its initial public offering—the largest debut for a biotech company in Wall Street his-
tory—and today has a stock market value of close to $7 billion. Moderna previously published studies testing its mRNA vaccines in humans. The new paper demonstrates that naked mRNA injected into human skin can produce a therapeutic protein called vascular endothelial growth factor (VEGF), which stimulates new blood vessel growth. VEGF has been eyed as a potential regenerative medicine that could help cardiac tissue heal after a heart attack, but researchers have had a hard time getting VEGF levels to be neither too low nor too high. “mRNA is uniquely suited to do this,” says Tal Zaks, Moderna’s chief medical officer. VEGF levels rose just hours after the injection and returned to normal within a day, indicating that the treatment was temporary, as expected. “This is a big step forward in the right direction for mRNA therapies,” says Lior Zangi, an assistant professor of cardiology at the Icahn School of Medicine at Mount Sinai. Zangi helped develop an experimental VEGF mRNA therapy in mice as a postdoctoral researcher at Harvard Medical School in 2013. That work laid the foundation for Moderna’s VEGF programs. AstraZeneca and Moderna are currently running a Phase IIa study testing their VEGF therapy in people undergoing open-heart surgery for a coronary artery bypass.—RYAN CROSS
BY THE NUMBERS
$6.9 billion
The value of Roche’s brand, the most valuable one in the pharmaceutical industry, according to Brand Finance. Roche enjoyed an 8% increase in brand value compared with last year, driven by strong performance in the US. The gap between Roche and secondplace Bayer, valued at $6.2 billion, narrowed slightly, the consulting firm notes. Pfizer came in third at $4.8 billion. Abbott and Merck & Co. rounded out the top five. 12
C&EN | CEN.ACS.ORG | FEBRUARY 25, 2019
James Wilson launches Passage Bio Passage Bio, a new start-up cofounded by University of Pennsylvania professor James Wilson, has raised $115.5 million in series A financing to develop gene therapies for rare genetic diseases of the central nervous system. Wilson’s research on adeno-associated viruses (AAVs), which are used to deliver DNA into cells, has been instrumental in catalyzing a gene-therapy boom. In 2009, Wilson helped found the AAV-based company Regenxbio. It now licenses AAVs to 11 other companies, including Novartis, which expects its experimental therapy Zolgensma to soon become the second gene therapy ever approved in the US. Wilson wanted to start a new company that he could be intimately involved with, explains Stephen Squinto, a partner at the venture capital firm OrbiMed Advisors and current CEO of Passage. After visiting Wilson’s lab at Penn, Squinto devised a unique arrangement for the new start-up. “He runs his gene-therapy center like you’d run a very efficient company,” Squinto says. “Why not use the world’s most proficient AAV lab to do your preclinical work?” Wilson, Squinto, and Tachi Yamada—a partner at Frazier Healthcare Partners—decided that Passage would pay Wilson’s lab to create AAVs and run experiments in monkeys. Passage will take over once it’s time for experiments in humans. “It will be a drug development organization without a research and discovery group because we are leveraging the expertise of Penn for that purpose,” Squinto says. Passage is focusing on five rare diseases that Wilson’s lab has already been studying, including a disease called GM1 gangliosidosis that affects and often kills infants and a disease called frontotemporal dementia that affects adults.—RYAN
CROSS
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