California's $3 Billion Stem Cell Program Partners with BlueRock and Vivo Capital, Looks for More Industry Collaborators

California's drive to produce a stem cell therapy is ratcheting up a notch with announcement of  two new, global industry partners along with a plan to engage more companies and give them "direct access" to hundreds of millions of dollars in state-funded research.

The California Institute for Regenerative Medicine (CIRM), as the $3 billion state stem cell agency is formally known, said the program represents an opportunity "to bring the most promising stem cell, gene therapy and regenerative medicine programs to market where they can help people with unmet medical needs."

The first two participants are BlueRock Therapeutics of  Cambridge, Ma., with offices in Toronto and New York City, and  Vivo Capital of  Palo Alto, Ca., which has offices in Bejing, Shanghai and Tapei. BlueRock was founded in 2016 with $225 million in backing from Versant Ventures and Bayer AG. Vivo has more than $1.7 billion under management, according to the firm's web site.

In addition to venture capital firms, the agency said its Industry Alliance Program (IAP) is looking for pharmaceutical and biotech partners to give them "direct access to CIRM’s growing stem cell portfolio."

Maria Millan, CEO and president of CIRM, said in a news release,

Maria Millan

"The goal of the IAP is to secure industry partnerships and funding for CIRM’s translational and clinical-stage projects. Our agency provides researchers the initial funding to advance promising projects towards the clinic. Now, we’re going a step further by offering a program that facilitates connections between industry partners and our grantees. These companies can offer the support or additional funding needed to give these promising projects the best chance for success and the best chance of helping patients.”

The stem cell agency is nearing its final days and is looking to fulfill promises to California voters who created it 13 years ago through a ballot initiative. The measure provided $3 billion in state bond funding, but no more. The campaign also generated expectations that stem cell cures were just around the corner. The agency has yet to back a therapy that is widely available.

The agency expects to run out of cash for new awards by the end of next year. A $220 million private fundraising effort is being waged to help the agency along until November 2020. That's when CIRM backers hope that the agency's efforts will excite California voters enough for them to approve $5 billion more in funding via another citizen-based initiative.

Deeper involvement with industry is one way to produce quicker results. Venture capital firms are willing to move fast and bet big on research that they deem likely to produce handsome profits.

BlueRock focuses on cell therapies that target severe brain and heart conditions.  According to Biospace, the company expects its most advanced lead therapeutic, a compound for Parkinson’s disease, to begin clinical trials this year.

Vivo focuses on high quality companies in the United States and China but includes building companies from scratch. 

California also brings something to the game.  Karen Ring, the agency's Internet majordomo, noted last week on the agency's blog,

"CIRM is the world’s largest stem cell research funding institution dedicated to helping patients by accelerating the development of quality stem cell treatments. We’re currently funding 244 active stem cell research programs including 39 ongoing clinical trials."

Neil Litman, the agency's director of business development, said CIRM has a "unique vantage point" because of its broad scope. He said the new program is "essentially a built-in concierge service for the stem cell space."

Media Coverage of Stem Cell Therapy for Blindness Loses Sight of California's $36 Million in Support

Dennis Clegg of UC Santa Barbara, one of the leaders in developing a new stem cell treatment for AMD, speaks broadly about the approach in this 2016 video.

The news this week was good for the $3 billion California stem cell agency, which is facing its possible demise in less than two years.

The stories demonstrated what the CEO of the agency, Maria Millan, calls the "value proposition" of the agency's work for the people of California. But only if the agency is mentioned in the news coverage.

Public perceptions are no small matter for supporters of the research agency, formally known as the California Institute for Regenerative Medicine (CIRM).  They wonder as does Millan: How does the agency get real and robust credit for its work, a likely life-or-death question given the agency's hopes to win voter approval of $5 billion more in funding in 2020?

CIRM says it is on track to run out of cash for new awards by the end of 2019 unless its ambitious fundraising plans are successful. And those are only a partial solution until billions more are provided by California citizens.

This week's "good news" stories generated national attention, albeit relatively modest, about CIRM-funded research that has led to to the first-in-human clinical trial for dry, age-related macular degeneration. The treatment uses technology that was described as "very exciting" by one researcher not connected with trial.

The headline on the New Scientist story on Wednesday said,

"Eye implant improves vision in people with age-related blindness"

Reporter Andy Coghlan wrote, 

"A patch implanted at the back of the eye has improved or stabilised sight in four people with severe age-related macular degeneration. The treatment enabled one 69-year-old woman to read 24 letters on a standard eye chart, when she could previously manage only seven.
"The patch is made of eye cells made from human embryonic stem cells, and it has been designed for treating the 'dry' form of macular degeneration, which accounts for 90 per cent of all cases, and affects 1.7 million people in the US."

Nowhere in the New Scientist story was it mentioned, however, that the state of California, through its stem cell agency, has pumped $36 million into the work. 

Likewise for the Los Angeles Times, whose story also did not mention the agency.  And likewise for articles on MIT Review, MedicalXpress, HealthDay, WebMD, US News and World Report, Futurism and FierceBiotech. The list could go on, but you get the idea.

Even the press release from Regenerative Patch Technologies LLC of Portola Valley, Ca., which holds the exclusive license for the implant, buried the Golden State's contribution in the next-to-last paragraph of a nine-paragraph press release.   Plus the company failed to note that the funding was a not insignificant $36 million.

FierceBiotech did mention some private money that was involved in other AMD research, but ignored California's cash.

Readers not familiar with the traditions surrounding news stories about medical research might wonder why the California support received almost no attention. Generally speaking, the amount of cash and the funding source are all but ignored in such stories with some notable exceptions. How that has come about is not clear, but money talks in most circles. The scientist who has lost his or her appointment by failing to bring in sufficient grants can speak to that.

For the California stem cell agency, it is also likely to be a matter of survival. If it fails to receive the widespread public credit for what it believes is a strong and important body of work, it is likely to wither away in very short order. 

$30 Million Cash Infusion for San Diego Firm, On Top of $20 Million from California Stem Cell Agency

A recent recipient of nearly $20 million from the California stem cell agency is on a financial roll this week as it pursues its efforts to translate what it says are "best-in-class gene therapy technologies into lifesaving cell therapies."

The firm is Poseida Therapeutics, Inc., of San Diego. Last October, it was awarded $19.8 million by the California Institute of Regenerative Medicine (CIRM), as the stem cell agency is formally known.

Poseida said yesterday it has hauled in $30.5 million more. The firm said in a news release that the cash will go to "advance a pipeline of autologous and allogeneic CAR-T immunotherapies, as well as gene therapies, using Poseida’s suite of gene engineering technologies."

The firm said its CIRM-backed "P-BCMA-101(product) is a CAR-T therapy currently in Phase 1 clinical development for relapsed/refractory multiple myeloma, with initial clinical data accepted for presentation at the upcoming AACR (American Association for Cancer Research) Annual Meeting." The firm is also tackling prostate cancer.

Jack Murtha of Health Care Analytics News wrote,

"It’s a good day for Poseida Therapeutics, one of the trailblazing precision medicine ventures that aims to transform cancer treatment through cutting-edge gene-editing technologies. But just how good of a day is it? Try $30.5 million good."

Murtha said the company has now raised more than $80 million for its endeavors, including the CIRM award, venture capital of $11.2 million last August and $23 million in 2015. The bulk of today's funding came from Longitude Capital of Menlo Park.

At the time of the CIRM award last fall, Maria Millan, president of CIRM, described the nature of the problem that Poseida was addressing, 

“Multiple myeloma disproportionately affects people over the age of 65 and African Americans, and it leads to progressive bone destruction, severe anemia, infectious complications and kidney and heart damage from abnormal proteins produced by the malignant plasma cells. Less than half of patients with multiple myeloma live beyond 5 years.”

Last fall, CIRM reviewers, meeting behind closed doors, unanimously approved, 10-0, Poseida Therapeutics application (CLIN2-10395), which included Poseida's commitment to provide $8.6 million in co-funding. The agency's governing board later ratified the decision with no discussion.

A public, CIRM-prepared summary of the reviewers' comments said, 

"Reviewers thought that the proposed product has the potential to provide a very high rate of durable response in myeloma patients. There is strong scientific and clinical rationale for targeting BCMA on myeloma cells. Reviewers thought that the proposed improvements to the CAR T cell platform technology are highly innovative and could enhance the efficacy and durability of the treatment. Reviewers unanimously recommended the application for funding."

Job Openings at California's Stem Cell Agency

The California stem cell agency, which is set to basically expire in less than two years, is looking for a few good folks to continue to help it out.

The California Institute for Regenerative Medicine (CIRM), as the agency is formally known, has posted openings for an associate director of marketing communications and interns in the communications and human resources areas.

The associate director's responsibilities include oversight of the agency's Web site plus additional task. The person will replace Karen Ring, who is leaving to join Weber Shandwick, a major public relations firm. Here is the job information for for the marketing position.

The internships are unpaid and part-time. Here are links to the CIRM information on those openings: Communications,which also includes Web work, and human resources.

Currently the Oakland-based agency has a little more than 50 employees. It predicts it will run out of cash for new awards by the end of 2019 unless ambitious private fund-raising plans are successful.

New Tool for Wading Through California's $2.5 Billion in Stem Cell Spending

The $3 billion California stem cell agency this week added a valuable feature that is aimed at giving better access to information concerning its active research portfolio, which currently runs to more than 160 projects.

The feature is called the Active Awards Portfolio Dashboard. Its development was overseen by Karen Ring, the agency's social media honcho. Writing on the agency's blog, she said,

"This interactive tool makes it easy to search through the active research projects that we’re currently funding, and filter these projects by disease focus, technology type or stage of research."

The new dashboard is a companion to the clinical awards dashboard, also engineered by Ring. The agency's website contains vast quantities of information. The dashboards provide tools that help readers wade through it and ferret out what is most relevant to their individual needs.

The California Institute for Regenerative Medicine(CIRM), as the agency is formally known, has funded 969 projects costing nearly $2.5 billion during its 13-year history . It is slated to run out of cash in 2019 unless ambitious fundraising plans fulfill their promise.