Thursday, December 14, 2017

$5 Billion Bond Measure: California Stem Cell Agency Sets a Course to Secure Its Financial Future

CIRM spending plan for next year and beyond. Educ refers to educational
 grants. Discovery refers to basic research. Tran refers to translational, and
 Clin refers to clinical trial related awards.
  CIRM spent $213 million this
year on Clin awards. 
OAKLAND, Ca. -- Directors of the California stem cell agency today virtually endorsed a plan to stave off its financial death, pinning their hopes on a possible $5 billion bond measure and a private fundraising effort to bring in an additional $222 million.

The California Institute for Regenerative Medicine (CIRM), as the agency is formally known, expects to run out of cash for new awards in late 2019 because of limits in the ballot measure that created it in 2004.

Robert Klein, who led the 2004 initiative campaign, appeared before the governing board meeting and touted the new bond proposal. The Palo Alto real estate investment banker told directors that California had a "moral imperative" to continue its stem cell work. He said,
"This is California’s contribution to the future of medicine....This is the bridge to the future of health care."
No vote was taken on endorsement of a ballot measure that has yet to be written or qualified for the ballot. But no objection was raised by board members to moving forward on what Thomas called the best option.

Under that scenario, Klein would lead a new, $5 billion initiative that would require hundreds of thousands of voter signature to qualify for the November 2020 ballot. Then it would require raising roughly $50 million to run a successful campaign, Klein told the California Stem Cell Report last month.

The $222 million fundraising effort is already underway and would continue until the end of 2019, with the expectation that it would provide bridge funding until voter approval of the new, multibillion dollar bond measure.

Klein told directors that 70 percent of California voters favor continuing funding of the agency. However, last month he declined a request by the California Stem Cell Report for a copy of the poll or the name of the firm that conducted it.

The 2004 campaign led voters to believe that miraculous stem cell cures were right around the corner. The agency has yet to help finance a treatment that is in widespread use. But it has currently invested $300 million in 26 clinical trials, the last stage before a proposed therapy can be certified for general use. Trials, however, can take years.

The agency is funded by money that the state borrows -- bonds. The authority to issue those bonds is expiring, and the agency is down to its last $269 million.

A fallback funding scenario would involve asking the legislature to place a bond measure on the ballot. There was no discussion of that today but it would involve negotiating with lawmakers and possibly making major changes in the way CIRM operates.

Prior to discussion of the bond measure, directors approved $68 million in cuts to the size of awards so that more awards could be made over the next two years. They also approved spending $270 million on awards next year, including $130 million on awards related to clinical trials, $30 million for research bridging the gap between the clinic and basic research and $10 million for basic research. Sphere: Related Content

$68 Million Saving: California Stem Cell Agency Cuts Size of Clinical Trial Awards

CIRM's new caps on its clinical trial programs

OAKLAND, Ca. -- Directors of the California stem cell agency this morning cut the size of its key awards, saving $68 million that will allow it to support more "shots on goal" as it pursues development of a stem cell therapy available for widespread use. 

The move was made in the clinical trials program of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. Clinical trials are the last stage of research prior to federal approval of a therapy for general use. 

The unanimous action will allow the agency to continue backing its non-clinical programs as well as generating support for 50 trials by the end of 2019. 

The agency is running out of cash for new awards because of limits in the ballot initiative that created it in 2004. The board will discuss funding for the more distant future at today's meeting. 

Sphere: Related Content

Wednesday, December 13, 2017

The California Stem Cell Agency, 'Lost' Opportunities and its Financial Lifeblood

A UC San Diego scientist has zeroed in on the declining finances of the $3 billion California stem cell agency, stressing that tomorrow may be the "last opportunity" for the agency to back a new approach to severe spinal injuries.

Mark Tuszynski, UCSD photo
Mark Tuszynski, director of the Translational Neuroscience Institute at UC San Diego, is
seeking $2.1 million to support his research to use neural stem cells to grow new connections through injured spinal cord. In a letter to the board, he said,
"As the present round of...funding winds down, this may be our last opportunity to develop this work to benefit the citizens of the state of California."
Tuszynski's comment could apply to almost any of the grant applications that will be submitted to the agency between now and late 2019. The California Institute for Regenerative Medicine (CIRM), as the agency is formally known, expects to run out of cash for new awards in 2019.

Tuszynski's comment was also unusual. The public silence from the California scientific stem cell community has been, as they say, "deafening" concerning the looming demise of CIRM. Researchers have not been heard from in any real way at the four public CIRM meetings in 2017 that have dealt with the issue.

The San Diego scientist's letter is part of the agenda at tomorrow's meeting of the CIRM governing board. At the forefront of the session is once again the question of whether the agency can find a way to continue its existence at the level at which it has operated since 2004.

CIRM's financial lifeblood is money that the state borrows -- state bonds. That source was provided by voters when they created the agency with a ballot initiative 13 years ago. However, the authority to issue those bonds is expiring, and the agency needs a major infusion.

Enter Bob Klein, a Palo Alto real estate investment banker and who headed the 2004 ballot campaign. He appeared before the board last month and talked about a $5 billion bond measure on the November 2020 ballot. He is expected to appear again tomorrow. 

Also on the table will be a strategy unveiled last month by CIRM Chairman Jonathan Thomas to raise privately $222 million between now and the beginning of 2020 to keep the agency sufficiently funded prior to a bond election. The board is also scheduled to act on a proposal to cut the size of awards so that more overall can be financed.

Then there are $21 million in applications for research into matters ranging from diabetes to dementia. That is were Tuszynski comes in. CIRM's all-important grant reviewers nixed his application (DISC2-10665), giving it a score of 80. The cutoff line for funding was 85.

He is asking the CIRM board to overturn the reviewers' decision, which would be a rare event. Another two researchers are also appealing negative decisions by reviewers, who meet behind closed doors and do not have to publicly disclose their professional or financial conflicts of interest.

The other scientists are Alice Tarantal of UC Davis and Gregorio Chazenbalk of UCLA.

Tarantal's $1.1 million application (DISC2-10599) involves, she said, "total-body positron emission tomography (PET) imaging technology, which currently is only available in California."
 Her application received a score of 84. In a letter to the board, she said her research, which also involves Simon Cherry of UC Davis, would "set the stage for stem cell applications and the transformative (imaging) tools developed, tested, and successfully applied in California."

In 2018, Tarantal said Cherry will place in operation "the world's first total-body PET scanner for humans that allows all tissues and organs to be imaged simultaneously."

Chazenbalk's $2.2 million application (DISC2-10473) involves a "new population of pluripotent stem cells" known as Muse cells, which he said can be used to treat acute myocardial infarction with a "high potential rate of success."  His score was not disclosed but appears to be below 65.

Scores on the applications can be found here. Summaries of reviewers' remarks on applications can be found  by scrolling more deeply into that document. CIRM withholds the names of applicants until after its board meeting. The three "appealing" scientists' names became public record when their letters were received by CIRM.

Eleven applications were approved by reviewers and 32 rejected.

The full agenda of the meeting with additional information can be found here. The meeting is based at the Oakland CIRM headquarters with teleconference locations where the public can participate in New York City, Sacramento, Santa Cruz, San Diego, Stanford and two in La Jolla.

The meeting is being audiocast as well. Full details on all locations and the audiocast can be found on the agenda. 

The California Stem Cell Report will be covering the meeting live from Oakland and filing stories as warranted.

Sphere: Related Content

Wednesday, December 06, 2017

California Stem Cell Board to Hash Over Cuts, Future Financing Plans Next Week

Directors of the $3 billion California stem cell agency, which is facing the loss of its funding, are scheduled for one of their more consequential meetings late next week -- a session that will deal with cutting the size of awards and planning for life after 2019.

The meeting Dec. 14 will continue a discussion that began last September and that now involves a possible $5 billion bond ballot measure, a private, $222 million fundraising campaign and major  reductions in the size of awards over the next two years.

The agency projects it will run out of cash for new awards in 2019. It is financed by California state bonds but that source is drying up under the terms of the ballot measure that created the effort in 2004.

Details of what exactly will be presented next week to the 29-member governing board were not available -- as of this writing -- on the web site of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. More information is expected to be posted in the next few days, but it is likely to resemble closely the matters discussed in late November by the directors. 

Also on tap are changes in the basic research and translational award programs and ratification of reviewer decisions on grant applications in the "Quest" program, which is aimed at development of technology that is uniquely enabled by stem cells. 

The meeting will be based in Oakland with teleconference locations where the public can participate in New York City, Stanford, Santa Cruz and San Diego. The meeting will be audiocast on a listen- only basis. More details can be found on the agenda.  Regarding the New York City site, from time to time teleconference sites have been set up out-of-state when a director is traveling.

The California Stem Cell Report will be covering the meeting live from CIRM's Oakland headquarters. Sphere: Related Content

California Stem Cell Research and a 'Heart-Tugging Media Blitz'

The critical, financial circumstances of the $3 billion California stem cell agency have drawn the attention of the San Francisco Business Times. 

The newspaper, which covers the biotech industry in the Bay Area, carried a piece last week that discussed the likelihood of a $5 billion stem cell bond measure on the November 2020 ballot supported by a powerful, emotional campaign. Also covered by the Times were plans to reduce the size of the awards next year along with a brief overview of the agency's progress.

The agency is on track to run out of money for new awards in 2019 and is down to its last $269 million. But it has made a big push into clinical trials and  anticipates many more in the next couple of years.

Reporter Ron Leuty wrote,
"The potential success of clinical trials would allow backers of a new bond measure to show real-world examples of how the 2004 measure (that created the agency) paid off in an intense, heart-tugging media blitz."
Leuty keyed off  Robert Klein's appearance before the agency's directors last month. Klein, sometimes known as the father of the stem cell agency, is touting the bond measure as a longer term financing solution, but did not respond to requests from the Business Times for additional comment.

Leuty continued with quotes from Maria Millan, CEO of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known:
"Because we're running so well and have so many potential projects — and we know there are more than what we can fund — the challenge is balancing the funding to as many as possible while making sure that resources are programmed well...We'd like to continue to deliver what we are currently delivering." 
"The next couple of years what we're doing is driving that mission because we're starting to take off. In the past, it was mainly hope; now that hope is based on something tangible."
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Sunday, December 03, 2017

Hope and the 'Helping Patients Part': The Uncertain Future of California's Stem Cell Program

One of the big selling points of the $3 billion California stem cell agency has always been hope. And it still is. 

It is a difficult matter to argue with. Hope underpins all our lives. But no more so than with persons suffering from terrible and incurable diseases. And those are precisely the targets of the 13-year-old stem cell research effort financed by the people of California. 

Currently the agency is on a course to run out of cash for new awards in 2019, ironically a fate dictated by Proposition 71, the ballot initiative that also created the agency in 2004. The measure provided $3 billion but no further stream of income. The agency is now wrestling with a variety of possibilities to extend its life for another decade or so, including mounting a $5 billion bond measure on the November 2020 ballot. 

A stem cell researcher at UC Davis, Paul Knoepfler, today addressed those critical matters -- and hope -- in a post on his blog, The Niche. He wrote, 
"The California Institute for Regenerative Medicine, more widely known as CIRM, has accomplished big things over the course of its history of about a decade and sparked a great deal of innovation, but what does the future hold for our stem cell agency?"
Knoepfler mentioned coverage of CIRM meeting last week by the California Stem Cell Report, noting that last week's item included the phrase "withering death." Knoepfler wrote,
"I don’t see things as so bleak and remain hopeful on the agency’s future. It’s unclear how California voters will be feeling about all this in 2020, but with our stem cell agency we are just now getting to the exciting part. The helping patients part. As a past grantee myself, I know how much the funding can make impact to advance science, whether basic, clinical, or somewhere in between. 
"At the recent brainstorming meeting on the agency’s future, there were apparently some upbeat reports of voter sentiment as described by (David) Jensen (producer of the California Stem Cell Report):

"'Robert Klein, a Palo Alto real estate investment banker who ran the 2004 campaign that created the agency, told the CIRM directors of a private poll that he said showed 70 percent of Californians supported stem cell research and continued funds for the stem cell agency.'
"What do you think about our stem cell agency’s future? 
"In terms of clinically relevant science, it is at the most promising point in its history with support for loads of clinical trials. Not all of them will work out, but I’m convinced that some will and other fresh trials that could be initiated with CIRM’s new funding in the future beyond 2020 would bring more hope."
The  California Stem Cell Report welcomes your thoughts on the agency's future and its strategy for getting there. You can submit them either by clicking on the word "comments" at the end of the item or by emailing them to djensen@californiastemcellreport.com.
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Thursday, November 30, 2017

California Researchers Score $16 Million Plus in State Stem Cell Awards

The California stem cell agency today handed out $16.4 million in research grants seeking therapies for afflictions ranging from gum disease and cancer to vision loss and Parkinson's Disease.

The award for Parkinson's was relatively tiny -- only $150,000 -- but represented a rare case in which the agency's governing board overturned its reviewers, who make the de facto decisions on awards.

The reversal came after one board member, David Higgins, of San Diego, who has Parkinson's, noted that the most common drug that Parkinson's patients take is 70 years old. He told the board.
David Higgins, CIRM photo
“I’m a fourth generation Parkinson’s patient and I’m taking the same medicine that my grandmother took. They work but not for everyone and not for long. People with Parkinson’s need new treatment options and we need them now. That’s why this project is worth supporting. It has the potential to identify some promising candidates that might one day lead to new treatments.”
The award went to Zenobia Therapeutics, Inc., of San Diego, whose president and co-founder, Vicki Nienaber, had filed an appeal on the reviewers' decision.  Another applicant rejected by reviewers, Toshio Miki of USC, also filed an appeal with the board. His appeal was not discussed. Miki's application sought $5.9 million for research involving metabolic disorders.

The largest award today, $5.6 million, went to Anthony Oro of Stanford, who will be testing a therapy to treat an affliction that creates wounds that will not heal. Dan Kaufman of UC San Diego received $5.5 million to produce "killer cells" to help people with a form of leukemia. Catriona Jamieson, also of UC San Diego, received $2.7 million for leukemia research.

Here is a link to summaries of reviewer remarks, including scores, on the three winners and the 11 other applications that were rejected in the translational awards category. (Scroll down on the page to see the reviews.)

In addition to the "discovery" award for Parkinson's, here are the names of the other winners in that category:
  •  DISC1-10603, Ngan F Huang, iPSC-Derived Smooth Muscle Progenitors for Treatment of Abdominal Aortic Aneurysm, Palo Alto Veterans Institute for Research, $172,621
  • DISC1-10475, Semil P Choksi, Generation of human airway stem cells by direct transcriptional reprogramming for disease modeling and regeneration, U.C. San Francisco, $238,408
  • DISC1-10643, Dmitriy Sheyn, IVD rejuvenation using iPSC-derived notochordal cells, Cedars-Sinai, $241,992 
  • DISC1-10598, Alice F. Tarantal, Enhanced Branching Morphogenesis and Pluripotent Cell Lineage Differentiation for Pediatric Regenerative Therapies, U.C. Davis, $235,80
  • DISC1-10583, John R Cashman, Human Pancreatic Cancer Stem Cells: Developing a Novel Drug for Cancer Eradication, Human BioMolecular Research Institute, $303,894 
  • DISC1-10555, Hiromitsu Nakauchi, Optimizing self-renewal signaling kinetics to stabilize ex vivo hematopoietic stem cell expansion, Stanford, $235,836 
  • DISC1-10620, David J. Baylink, Bone Marrow Targeting of Hematopoietic Stem Cells Engineered to Overexpress 25-OH-VD3 1-α- hydroxylase for Acute Myeloid Leukemia Therapy, Loma Linda University, $178,967 
  • DISC1-10513, Guillem Pratx, Novel metabolic labeling method for tracking stem cells to irradiated salivary glands using PET, Stanford, $235,613 
  • DISC1-10522, Gerald P Morris, Identification of antigenic neo-epitopes from in vitro reprogrammed human tissue precursors for regenerative therapy, U.C. San Diego, $193,500
  • DISC1-10588, Julia J. Unternaehrer-Hamm, Targeting cancer stem cells with nanoparticle RNAi delivery to prevent recurrence and metastasis of ovarian cancer, Loma Linda University, $172,870 
  • DISC1-10721, Karl J. Wahlin, An IPSC cell based model of macular degeneration for drug discovery, U.C. San Diego, $232,200 
  • DISC1-10516, Alyssa Panitch, Development of treatments to improve healing of ischemic wounds, U.C. Davis, $235,800 
  • DISC1-10718, Alireza Moshaverinia, Gingival mesenchymal stem cells as a novel treatment modality for periodontal tissue regeneration, U.C. Los Angeles, $194,483
Sphere: Related Content

Wednesday, November 29, 2017

Balloting on Stem Cell Person of the Year: Cast Your Vote Now

The countdown has begun for selection of the Stem Cell Person of the Year. You can vote online for your preference at The Niche, a blog operated by UC Davis researcher Paul Knoepfler.

stem cell person of the yearHe has put together a list of 20 nominees ranging from a California state legislative aide to the CEO of the New York Stem Cell Foundation, Susan Solomon. Also included is a host of researchers, including Shoukhrat Mitalipov and Alexey Bersenev. The full list can be found here.

Knoepfler is the sole judge of the winner and personally provides a prize of $2,000.

The current voting, which ends Dec. 12, will whittle the list down to 10 from which Knoepfler will pick the winner. Earlier this month, he had this to say about the award,
"Unlike some other science awards out there that shall remain nameless, the Stem Cell Person of the Year prize is not an insider kind of thing, but more of an anti-old boy’s club award. It’s not about who you know, but what you do to help science, medicine, and other people."
Personally, I think the team at the California stem cell agency should be on the list. The folks, past and present, at the agency have labored mightily since 2004 to advance stem cell research. Sometimes the circumstances have been difficult and challenging, but the work continued nonetheless. Maybe next year, as they say in the sports world. Sphere: Related Content

Monday, November 27, 2017

"Cuts," Private Fund-Raising, $5 Billion Bond Measure: Answers to the Critical State of California Stem Cell Finances?

Bob Klein explored a $5 billion bond measure at today's stem cell meeting. 
California Stem Cell Report photo

OAKLAND, Ca. -- Facing the likelihood of a slow and withering death, the California stem cell agency today edged gingerly forward on a path of "cuts" and risky fund-raising in hopes that its research results will soon generate voter support for more billions of dollars.

Two governing board committees of the $3 billion agency, formally known as the California Institute for Regenerative Medicine (CIRM), this afternoon recommended that the full board "entertain" the proposals at its Dec. 14 meeting.

The agency is down to its last $269 million and expects to halt new awards in 2019 unless additional funds are raised between now and the beginning of 2020. It has been running at a $300-million-a-year pace recently and has pumped out about $25,000 an hour, 24 hours a day, seven days a week since it started making awards in 2005, according to calculations by the California Stem Cell Report.

Today's meeting focused on both short and long term finances. The committees examined a proposal to cut its planned clinical awards by $68 million over the next two years by limiting their size. The committees also indicated support for an ambitious effort to raise $222 million privately between now and early 2020.

Longer term, directors staked their hopes on voter approval of a ballot initiative in November 2020 that could total $5 billion. Development of a stem cell therapy that would resonate with voters would be a major key to the success of that effort. The 2004 campaign raised expectations that stem cell cures were right around the corner, but so far the agency has not backed one for widespread use.

Robert Klein, a Palo Alto real estate investment banker who ran the 2004 campaign that created the agency, told the CIRM directors of a private poll that he said showed 70 percent of Californians supported stem cell research and continued funds for the stem cell agency.

Following the meeting, the California Stem Cell Report asked him for a copy of the poll. He declined to provide it or identify the firm that conducted the survey.

Klein told the CIRM board members that the agency has made "remarkable progress" and has created a "moral imperative" to continue the search for stem cell cures. He said,
"The bridge to the future is CIRM and stem cell therapies."
The now less-than-robust finances of CIRM are a product of the ballot initiative, Proposition 71, that created it. CIRM is not funded through the customary process used by most state agencies. The initiative  stipulated that CIRM would be supported by $3 billion in state bonds whose funds would flow directly to the agency and bypass the legislature and the governor. When that cash runs out, the agency is left with no other source of state funding.

Raising $222 million privately over the next two years is no small task, but deep pockets exist that may well be tapped. The Eli and Edythe Broad Foundation, for example, has donated nearly $80 million to stem cell research centers at UCLA, UC San Francisco and the University of Southern California. All three are linked to CIRM. In San Diego, the Sanford Stem Cell Consortium was given $100 million in 2013 by Denny Sanford, another billionaire. The consortium came into being after it was backed by $43 million from CIRM.

Nonetheless, competition for philanthropic cash is heated. Thomas and Klein, however, both indicated that they would collaborate on raising the $222 million, which would allow the agency to add eight new clinical awards in 2020, among other things.

The risk lies in the timetable for bringing in the cash. The agency has a relatively tight schedule for making awards --- a schedule that a major philanthropist may or may not be ready or willing to comply with.

Without more cash, the stem cell agency projected that it would wither away by 2023 as it managed a declining number of old research grants.

For more, recent articles on CIRM's current critical funding situation, see here, here, here and here. Sphere: Related Content

California Stem Cell Agency Examines its Financial Options Long-Term and Short-Term

OAKLAND, Ca. -- A key group of directors of the $3 billion California stem cell agency this afternoon opened a discussion dealing with its short and long-term financial future at 1:04 p.m. The meeting is scheduled to last until 4 p.m. The California Stem Cell Report will file stories as warranted. Sphere: Related Content

California Tightening Stem Cell Research Belt; Size of Research Awards Targeted

The stem cell agency outlined its assessment of its current position
 in this slide prepared for today's meeting. 

OAKLAND, Ca. -- California's $3 billion stem cell agency is preparing to put the squeeze on its research awards over the next two years, cutting the caps on new grants in some areas by 50 percent or more.

It is all part of a strategy to prolong the life of the 13-year-old research effort as it fights to fulfill the expectations of voters who created it when they approved a ballot initiative in 2004. Voters provided only $3 billion in funding, however, and the agency is now expected to run out of cash for awards in 2019 unless it changes its financial ways.

Meeting here today are two key groups, the Transition and Science subcommittees of the governing board of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

They are scheduled to wrestle with both the agency's short-term finances and the longer term effort to extend CIRM's life for perhaps an additional 10 years or more via another ballot initiative in 2020. As for next year, based on past discussions and practices it appears certain that the agency's directors will reduce the size of awards to the scientists and businesses who are often hard-pressed to find non-governmental support.

A proposal by the president of CIRM, Maria Millan, calls for reducing clinical program awards by a total of $68 million over the next two years. Here is how the cuts would work compared to the average award this year in each category.
  • Phase 1 and phase 1/2 clinical trial grants would see their caps reduced from $20 million to $12 million for nonprofits and $8 million for for-profit enterprises. The average award in 2017 in these categories was $10.3 million. 
  • Phase 2 trials would see their caps cut from also $20 million to $15 million for both non-profit and for-profit enterprises. The 2017 average award was $15.3 million. 
  • Phase 3 trial awards, which averaged $16.7 million in 2017, would have their caps cut from $20 million to $10 million for both types of enterprises.
Millan's presentation indicated that would her proposals would allow the agency to support 24 new trials and eight trial candidates over the next two years, in keeping with a strategy of taking "more shots on goal" as the agency tries to develop a stem cell therapy for widespread use. Clinical trials, which can take years, are the last stage before a therapy is approved by the federal government. 

Millan's plan would also allot only $10 million for what the agency calls "Discovery" awards (basic research) next year (10 projects) and $9.25 million in 2019 (nine projects). Discovery awards are expected to hit $46 million this year. Education awards are expected to total $1 million this year. Next year they  would receive $750,000 and nothing in 2019. 

Key to the spending plan is the assumption that the agency could raise an additional $220 million from a variety of sources between the end of next year and early 2020. The goal of the fund raising is to bring in $55 million by the final quarter of next year. The rest would be raised between then and the first quarter of  2020. 

CIRM did not release details of its strategy for raising the $220 million. CIRM Chairman Jonathan Thomas, however, has already been on the fundraising trail and reports that he has identified a number of potential donors.

The financial plans and any changes will go to the full board for ratification at a meeting Dec. 14 here at CIRM headquarters.

The California Stem Cell Report will cover today's meeting and file reports as warranted on this web site. Below is another presentation slide from the stem cell agency. 



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