Friday, August 13, 2021

Bluebird bio's $1.8 Million Setback on Gene Therapy: Is It a Growing Sign of Rejection of Reimbursement Profit Models?

"Some bad news about a $1.8 million gene therapy echoed across the Atlantic Ocean this week and carries implications for California’s state stem cell agency, which also is engaged in developing so-called “one-and-done” therapies. 

" The matter involves bluebird bio of Cambridge, Mass., and its proposed Zynteglo therapy for the rare blood disorder beta thalassemia. The European Union last week refused to go along with bluebird’s price for the treatment -- $1.8 million. 

"Bluebird, which spells its name in all lowercase, said it would fold up its European operations...." "This news (about bluebird) plus Orchard's departure demonstrates urgency around affordability that needs to be addressed, especially when likely sickle cell disease therapies come to fruition,” said an observer who has followed CIRM (the state stem cell agency)  for years, but declined to be identified publicly.

“The technology is similar to proven therapies in X-linked SCID and beta thalassemia that are languishing. Serious consideration needs to take place around utilizing ‘march-in’ rights built into CIRM's IP (intellectual property) policies to force the provision of these therapies in an affordable manner...."

Sunday, June 13, 2021

Subscribe Free Today to the California Stem Cell Report -- - The Most Comprehensive Source for the Ins and Outs of California's $12 Billion Stem Cell Program

Don't miss important fresh news and information about California's $12 billion stem cell research agency. It is charting a new course with new opportunities and significant changes in direction, including a greater emphasis on the fast-growing gene therapy field.  

The changes already mean a lot for patients, researchers and policymakers and will have an even greater impact in the next several years.  Tracking the agency's affairs is not easy, but you can find what you need on the The California Stem Cell Report

Dubbed "indispensable" and "authoritative" in the Los Angeles Times, the Report is the only regular, independent source of news and information dealing with the California Institute for Regenerative Medicine (CIRM).  

It is news that can be found nowhere else. Plus it is comprehensive. More than 5,000 items have been published since CIRM was created in 2004. 

You can subscribe free to the California Stem Cell Report on its new platform by clicking on the "Subscribe Today!" box on the upper right of this page. Or just go to one of the following recent stories and click on the subscribe button.

California Stem Cell Agency Withholds $5.8 Million from Orchard in Bubble-Baby FlapMoney to Go to New Effort Involving Compassionate Use

Monday, May 24, 2021

Read All About It: Bubble Babies Hit by Suspension of Trial for Genetic Cure; California Taxpayers Paying for Trial

 "Indispensable" is what the Los Angeles Times this morning called this blog, the California Stem Cell Report. 

The comment came in a story that was triggered by our coverage of the Orchard-CIRM-UCLA bubble baby clinical trial, which was shoved aside by Orchard Therapeutics for financial reasons. That leaves a bunch of children out in the cold as far as the genetic treatment is concerned. 

UCLA says the treatment has saved the lives of more than 50 persons. 

Below is a list of the Orchard stories that we have carried on our new platform since we unearthed the issue on May 11. You can subscribe free to articles on our new home by clicking on the subscribe button in stories on the new platform. Subscribe today.

Here is the list.

Wicked' Problems, Orchard Therapeutics and Bubble Babies: A California Stem Cell Story

CaliforniaStem Cell Agency Pledges to Do "Everything" to Salvage Cure for Bubble Baby Disease

Can a California 'March-In' Save Terminally Ill 'Bubble Babies?'

Orchard's Suspension of Bubble Baby Disease Trial Earns Sharp Rebuke

Bubble Baby Cancellation Story Draws Attention in California's Largest Newspaper

Sunday, April 04, 2021

Essential Reading on California's Ground-Breaking, $12 Billion, Stem Cell/Gene Therapy Research Program

 Don't miss out on news and information on the $12 billion California stem cell agency. 

Its activities affect the lives of thousands of people and have the potential to affect the lives of millions of more. Not to mention that it is venturing into new areas of health policy and research. 

Subscribe today to the California Stem Cell Report on its new platform. You can do so by clicking here and tapping the subscribe button.  Plus it's free. 

Cited as “indispensable” in the Los Angeles Times, the California Stem Cell Report is the only independent voice regularly reporting on the activities of the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is officially known. In 2012, the Institute of Medicine invited the publisher of this site to testify during its $700,000 evaluation of CIRM.

This report has covered CIRM since its inception in 2004 and has published more than 5,000 items and attracted more than 3 million page views. 

It is essential reading if you have something at stake in what CIRM does and how it spends its money.

Here are links to recent articles on the California Stem Cell Report's new platform. 

California's Sickle Cell Surge: Researchers Tackle Disease with First-Ever, Gene-Editing Clinical Trial

Friday, March 26, 2021

Photo: License Dashboard

The talk at the California stem cell agency this week was of ”boiling the ocean,” the meaning of “unlikely” and “DEI.” All of which involves how $5.5 billion in taxpayer dollars will be used over the next decade or so....

Read all about it on the new platform for the California Stem Cell Report. And please subscribe on the new site to keep seeing the most complete and independent source of information on the doings at the state's $12 billion therapy development enterprise. 

Thursday, March 18, 2021

The Latest: Deciding who Decides Who Gets California's Stem Cell Billions

CIRM directors tomorrow take up the matter of how the agency chooses the judges of applications for billions of California stem cell research dollars. You can read more about what's in store on the new platform of the California Stem Cell Report

You can also make sure you don't miss any of the news by subscribing free.  It's easy. Just click on the subscribe button when you go to the link below. 

Who Should be a Stem Cell Judge for California's $12 Billion Research Agency?

Looking at how scientists are chosen to score grant applications

The California stem cell agency has released its plans to revise the process of choosing the folks who make the de facto decisions on billions of dollars in research awards.

The revisions grew out of a meeting last month of the governing board of the $12 billion agency, officially called the California Institute for Regenerative Medicine (CIRM).

Some directors sought more diversity among as many as 300 scientists who are tapped to review applications for awards. One director sought assurances that the process would lead to the selection of the most talented scientists, ones who also could be counted on work positively with others during the review process....

Saturday, March 13, 2021

Subscribe Today to the Leading Source of Information on a $12 Billion Source of Stem Cell Research Funding

 It's time to subscribe -- free! -- to the California Stem Cell Report, which has moved to a new platform. 

Don't miss news and information concerning critical developments at the $12 billion California stem cell agency. It is the leading, independent and reliable source for all that happens at the California Institute for Regenerative Medicine (CIRM). 

With more than 3 million page views and more than 5,000 items during the last 16 years, the Report's readers range from researchers throughout California to across the nation. Not to mention policymakers and entrepreneurs in the burgeoning stem cell field. "Indispensable" is how the Los Angeles Times described the California Stem Cell Report.

You can subscribe on the blog's new platform while reading the latest CIRM news. Please do it today. Here are recent links and excerpts that will take you to the location where you can subscribe with just one click.  

Telling the California Stem Cell Story: A Matter of Billions of Dollars

Building a Base of Voter Support

Can the stork be relied on to bring stem cell cash?

The respected California news service Capitol Weekly this week brought the exploits of the state’s $12 billion stem cell agency to a much wider audience than normal -- one that includes policymakers, lobbyists, lawmakers and even some information-hungry folks in the general public. 

The case in point involves the $17 million provided by the stem cell agency to help finance the world’s first clinical trial to treat spina bifida in the womb. ....

Bulldogs and Babies: World's First Trial to Treat Spina Bifida in the Womb

UC Davis Research Began More than 10 Years Ago

Bulldog pups were tested earlier in the spina bifida research and came out lively. UCD video

Backed by $17 million in cash from California’s stem cell agency, researchers at UC Davis this month are launching “the world’s first clinical trial using stem cells to treat spina bifida before the child is born.”...


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