How Massachusetts' Bluebird Bio tapped California's stem cell agency for cash, clinical trial help

Bluebird Bio's David Davidson.

California’s stem cell research funding agency took another step toward putting more potential therapies into human clinical trials.

The California Institute for Regenerative Medicine awarded $9.3 million Thursday to Cambridge, Mass.-based Bluebird Bio and $10.1 million to ViaCyte Inc. of San Diego.
Bluebird is using stem cells and gene therapy to target young patients with the blood disorder beta-thalassemia. ViaCyte is working on an embryonic stem cell-based therapy for patients with insulin-dependent diabetes.
The awards are important because San Francisco-based CIRM, which is funded by California state bonds after voters in 2004 approved Proposition 71, to date has spent hundreds of millions of dollars on basic science and supporting the construction of new stem cell science buildings at places like the University of California, San Francisco, Stanford University and theBuck Institute for Age Research in Novato.
I spoke recently with David Davidson, chief medical officer of Bluebird Bio, about the company’s experimental stem cell treatment, called LentiGlobin, how a Massachusetts company came to win money from a California taxpayer-funded initiative (the answer might surprise you) and Bluebird’s upcoming clinical trial.

One-on-One with Roman Reed, spinal cord injury patient advocate

Roman Reed.

The community of patient advocates is full of the most passionate, goal-oriented people you'll ever meet, and Roman Reed is near the top of the list. Reed, paralyzed in 1994 from a tackle during a Chabot College football game, has been one of the most visible advocates for stem cell research fundingthrough the San Francisco-basedCalifornia Institute for Regenerative Medicine, or CIRM. He also was a poster child for a state law passed a law in 2000 to fund spinal injury paralysis research through the state's general fund. But in the course of California's budget meltdown, desperate state officials took away the spinal cord research funds. Reed launched a new effort, resulting in Assembly Bill 1657, that would add just $1 from every moving traffic violation to fund spinal cord injury research in California. That bill, authored by Assemblyman Bob Wieckowski, D-Fremont, passed the Assembly on a 46-26 vote and the state Senate is expected to vote Monday on the measure.

CIRM awards $151 million to push stem cell treatments toward human trials

California's stem cell research funding agency doled out $151 million in new funding to move potential therapies closer to human testing, but awarded nearly $100 million less than it had planned to give. Only one of the seven so-called "disease team" awards from the California Institute for Regenerative Medicine, or CIRM, went to industry. That was a $20 million grant for Newark-based Stem Cells Inc. for a spinal cord injury treatment.

Buck Institute researchers fix Huntington's Disease mutation in mouse study

Researchers at the Buck Institute fixed the genetic mutation that causes Huntington’s Disease, raising hopes of eventually reversing and curing the inherited brain-wasting disease in humans.

The work, using induced pluripotent stem cells in the lab of Lisa Ellerby at the Buck Institute for Research on Aging in Novato, is early stage and currently is using mice. The therapy has not been tested in humans, and the lab continues to see if mice transplanted with corrected cells have functional improvements.

But in a paper published online Thursday in the journal Cell Stem Cell, Ellerby’s lab details how it used reverse-engineered cells from a Huntington’s patient, made a genetic correction and generated fresh neural stem cells. When the disease-free cells were transplanted into mice, the animals generated normal neurons in the area of the brain affected by Huntington’s.

“The thing that’s really neat about this in any disease (is) you really are correcting the mutation,” Ellerby said. “You’re fixing the problem.”

CIRM awards $69 million in stem cell research grants targeting 'bubble boy' syndrome, other diseases

Bay Area stem cell researchers looking to cure a "bubble boy" syndrome, fix damaged heart muscle and take on a host of other diseases grabbed more than $25 million in funding from California's stem cell research funding agency.

The San Francisco-based California Institute for Regenerative Medicine, or CIRM, said it awarded a total of $69.4 million to California stem cell scientists. Those projects include the first CIRM-funded collaboration in China and the first project with the Australian federal government.

Seven awards went to three Bay Area institutions: Stanford University, theUniversity of California, San Francisco, and the Gladstone Institutes.

Stem cell agency CIRM earmarks $30M to woo biopharma, VCs

A $30 million effort from California’s stem cell research funding agency will dole out awards to help biotech or pharmaceutical companies or venture capital firms take research into early clinical trials.
The program, announced Tuesday, represents the closest collaboration to date between the San Francisco-based California Institute for Regenerative Medicine and the biopharma industry and venture capital.
The three or more Strategic Partnership Awards would provide up to $10 million each. Successful applicants must match those funds, either dollar-for-dollar or by providing in-kind services like manufacturing and product development.
CIRM's vision is to tap the product development expertise of the companies and venture capitalists to move therapies or cures for a wide range of diseases closer to Food and Drug Administration approval.

CIRM's $3 billion question: Will stem cell bets pay off?

(San Francisco Business Times subscription required for full article.)
In the Buck Institute for Age Research’s gleaming $36.5 million stem cell research facility, which opens April 14 in Novato, scientists will seek ways to use stem cells to treat Parkinson’s disease and regenerate cells to restore sight to glaucoma patients.
It is the type of work that many California voters envisioned in 2004 when they approved Proposition 71, the $3 billion bond issue that supported the creation of the California Institute for Regenerative Medicine.
But even as the San Francisco-based stem cell research funding agency nears the halfway point of its life, and the 11th of 12 CIRM-backed major facilities comes on line, the future of the agency itself isn’t quite as clear.
Without stem cell treatments or cures for cancers, diabetes or some other high-profile malady, voters are unlikely to approve a follow-on bond measure, if CIRM seeks one. CIRM leaders haven’t yet settled on how to carry on the agency’s mission after the last of Prop. 71’s funds are turned over to CIRM in five years. Proposals have included a venture philanthropy fund or corporate support that would take potential treatments into mid-stage clinical trials.

Executive Profile: Jonathan Thomas, chairman of CIRM

San Francisco Business Times subscription required.
Jonathan Thomas — or J.T., as he’s known around the California Institute for Regenerative Medicine — came to the state’s stem cell research funding agency in June with a unique blend of experience. He’s a lawyer, investment banker and biologist who’s adept at maneuvering around the halls of government and the nonprofit world. He founded Saybrook Capital, an investment bank that did early-round financing in 2000 for stem cell company Advanced Cell Technology, and as the chairman of CIRM’s oversight board is responsible for overseeing an agency ultimately backed by $3 billion in state bonds.

Stem cell agency inks pact with Scotland

California’s stem cell research funding agency signed a memorandum of understanding with Scotland, it said Thursday.
It is one of a number of such agreements with government agencies by the San Francisco-based California Institute for Regenerative Medicine. The deals don’t lay out specific financial terms but are meant more to develop closer ties between CIRM-funded researchers and others worldwide.

CIRM makes $30M deposit toward stem cell bank

California’s stem cell research funding agency wants to make a $30 million investment in the banking business.
The California Institute for Regenerative Medicine’s three-part strategy ultimately will create a stem cell bank for so-called pluripotent stem cells that can be manipulated to form skin, heart or other types of cells. It would allow researchers to more quickly and efficiently create “disease-in-a-dish” models of genetically complex diseases like diabetes, asthma, autism or heart disease.
Although the bank would house some embryonic stem cell lines, the project’s emphasis is on the fast-moving field of induced pluripotent stem cells, or IPS cells.

CIRM puts up $5.6M to recruit stem cell researcher to UC Berkeley

UC Berkeley’s attempt to woo stem cell researcher Zhigang He from Children’s Hospital Boston got a $5.6 million boost from California’s stem cell research funding agency.
The six-year award, approved Thursday by the board overseeing the San Francisco-based California Institute for Regenerative Medicine, will help the University of California recruit He, whose research has focused on regenerating nerve projections that carry signals along the spine.

Stem cell board approves two research-centered programs totaling $27 million

California’s stem cell research funding agency will pump $27 million into various projects, including up to $12 million for disease-focused research teams, programs looking to move academic work into patients and for clinical trials.
The oversight board of the San Francisco-based California Institute for Regenerative Medicine voted Thursday to approve the programs, which are designed to accelerate stem cell research projects in the state.

CIRM names former iPierian exec as first CFO

The former chief financial officer of stem cell company iPierian Inc. was hired for the same job at California’s stem cell research funding agency.
Matthew Plunkett, who also has worked as a biopharma investment banker at Oppenheimer & Co. and predecessor CIBC World Markets, was named the California Institute for Regenerative Medicine’s first chief financial officer. He worked at iPierian in South San Francisco from August 2009 to this past April, when he, along with much of the management team and a couple of board members, exited the company.

What Geron's stem cells exit means. (It ain't good)

Geron Corp.’s dismantling of its stem cell research on Monday puts the pressure on companies trying to discover or develop drugs by tapping into the cells that make us what we are.
Geron may have been trying to do too much too soon, but the real issue is that human embryonic stem cell drug development clearly was costing too much money for a publicly held company (NASDAQ: GERN).
That’s a shame in many ways. After all, Menlo Park-based Geron was in stem cells before stem cells were cool from a commercial point of view — not that they are more hip now. By being one of the first to harness embryonic stem cells as a potential treatment, it blazed a trail, enduring a Food and Drug Administration hold on its trial in acute spinal cord injuries.

Stem cell agency CIRM, NIH to work together in pilot project

California’s stem cell research funding agency and the National Institutes of Health signed a memorandum of understanding for a pilot project to help medical researchers prevent, diagnosis and treat a wide range of diseases.
The two groups also are looking at ways that researchers funded by the San Francisco-based agency, the California Institute for Regenerative Medicine, and those supported by the NIH can collaborate in earlier translational or basic biology projects.

Video: UC Berkeley dedicates Li Ka Shing biomedical research center

Philanthropist Li Ka Shing, UC Berkeley Chancellor Robert Birgeneau and a standing-room-only crowd on Friday dedicated the campus’ new Li Ka Shing Center for Biomedical and Health Sciences.
The $267 million, six-level, 200,000-square-foot facility, which will feature the University of California, Berkeley’s stem cell program on the third and fourth floors, was funded in large part with a $40 million gift in 2005 from Hong Kong businessman Li. It also received $20 million from California’s taxpayer-supported stem cell research funding agency.
“I believe that investments in higher quality education are the best investments for improving the human condition,” Li said through an interpreter.

Geron, StemCells trials tackle different approaches to spinal cord injuries

Two groundbreaking spinal cord trials led by Bay Area companies, both of which hit milestones in September, could make or break commercial stem cell therapies.
Although the trials are different in many ways, both are closely watched by stem cell advocates and opponents and could have implications for California’s stem cell research funding agency.

CIRM targets patients, advocates to carry its message

True believers in stem cell research — patients and patient advocacy groups — are being recruited by California’s stem cell research funding agency to spread the gospel as officials consider asking voters to support another multibillion-dollar bond issue.
Whether the campaign by the California Institute for Regenerative Medicine results in a new bond measure to supplement the $3 billion guarantee provided by Proposition 71’s passage in 2004 is an open question. Leaders of the San Francisco-based agency say they have not decided whether to ask voters to extend funding past 2017, when Prop. 71 money stops.
One thing, however, is sure: Many patients, advocacy groups and scientific researchers are eager to become both preacher and choir.

For CIRM, there's no place like home

California’s stem cell research funding agency may be unpacking its bags. At last some of them.
When Gov. Jerry Brown called for state agencies to cancel unnecessary out-of-state travel in light of the state’s ongoing fiscal problems, the California Institute for Regenerative Medicine told Brown that all its impending trips — including to Australia, Japan, Paris and Amsterdam — were “mission critical.”
In fact they were, in CIRM’s view, virtually a matter of life and death.

Federal judge dismisses suit over stem cell funding

A federal judge dismissed a lawsuit Wednesday against the Obama administration’s funding of embryonic stem cell research, a victory for stem cell researchers in California and elsewhere whose work has been under a cloud.
The suit, brought by two adult stem cell researchers who said funding violated the 1996 Dickey-Wicker law against taxpayer funding of projects that harm embryos, put a pall on stem cell research even as new facilities were built in California with state and private support.