A legally required affordability plan for a cure for California children with a deadly, ultra-rare disease suffers from an “embarrassing” lack of information as the several-million-dollar therapy moves into the marketplace.

The genetic treatment was developed by Rocket Pharmaceuticals of New Jersey. The California Institute for Regenerative Medicine (CIRM) awarded the firm $5.9 million in 2019 to help finance a clinical trial at UCLA, which successfully tested the one-time treatment for LAD-1, whose price tag is expected to run as high as $4 million.

LAD-1 is a genetic affliction that leaves children vulnerable to dangerous, recurrent bacterial and fungal infections. “Survival beyond childhood is rare without treatment,” according to UCLA. It affects only about 25 children annually.

Rocket’s affordability and access plan is officially open for public comment until next Tuesday, Sept. 8. CIRM released the plan after agreeing to the redactions proposed by Rocket. The blacked-out information involves “confidential intellectual property, commercially sensitive information, and/or protected work product” that can be legally withheld, CIRM said.

The concealed information includes sections dealing with the proposal’s scope, eligibility, insurance, documentation, patient and provider access, financial assistance, and governance and monitoring.

Its broad outlines are clear, however. Treatment would be free for legal residents of California who have “no other means” of obtaining the therapy. Unsubsidized would be “administration, facility fees or other non-drug medical services,” which can be costly. One family treated in the UCLA clinical trial had to stay months in the area, which is one of the more expensive in California.

CIRM and Rocket should be applauded for putting together an assistance plan, said Hank Greely, a Stanford University law professor and director of its Center for Law and the Biosciences, in an interview this week. He also said some of the redactions are “embarrassing” and “uninformative.”

“It is in CIRM’s interest, as well as in the public interest, that the public knows what’s going on….What’s proprietary about the documentation needed to establish the right residency?” Greely said.

The affordability plan, CIRM’s first, stemmed from the 2020 ballot initiative that refinanced CIRM with $5.5 billion. The initiative required CIRM to “establish and oversee” programs to help make its commercialized stem cell and gene therapies “available and affordable” for all Californians. An access and affordability plan from grant recipients is required as a condition of a clinical stage award.

A California Stem Cell Report review of the six-page plan showed omission of key information that would be useful for patients and their families. The context of some redactions indicated that the withheld information could be valuable for patients and for taxpayers concerned about how the voters’ affordability mandate is being fulfilled. Here are some of the issues that emerged from the review:

• No definition of what is meant by legal resident of California is offered. Different residency definitions are used in California depending on the purpose, such as for taxes or for attendance at the University of California. Can patients pick and choose their own definition?

• A section on residency (section 3.3) mentions “acceptable proof,” which is also undefined. Fifty percent of the lines on the subject are blacked out.

• In an insurance status portion (section 3.4), seven lines have been blacked out on that topic, which may rule out a patient. Are patients being deprived of important information? What does this redaction have to do with proprietary or other non-disclosable information?

• A section dealing with eligibility (section 3.7) contains 10 lines of information. Six are blacked out.

• The documentation section (3.8) contains 15 lines. Only about three lines remain visible to patients and the public.

• Information (section 4) on patient support and “navigation” contains 15 lines. Thirteen are blacked out. The program scope section ( section 5) contains 11 lines. Seven are blacked out.

The California Stem Cell Report queried CIRM on the redactions and other matters Monday morning (Aug. 31), but has not received a response.

The Rocket gene therapy is the first commercial treatment to emerge in CIRM’s 21-year history. It is a “one-and-done,” handcrafted, revolutionary therapy. It avoids the expense of a lifetime of medical care and hospitalization, not to mention the accompanying pain and suffering. Rocket plans to launch it commercially before the end of the year.

“Shadowing the excitement about the transformational potential of many gene therapies has been widespread concern about the combination of uncertainty in the durability of their benefits over the long term and the short-term financial shock of high prices, which are now cresting above $3 million dollars,” a 2024 article in the scientific journal Nature said.

“Although the aggregate costs of gene therapies at these prices have not proven yet to be unmanageable to large payers given the small number of patients currently treated, growth in the approval of gene therapies for larger populations is on the horizon.

“All told, 85 new gene therapies across more than 12 therapeutic areas are expected to receive regulatory approval by 2032, with an estimated ten-year list price spend of $35 to $40 billion,” the article said.

Comments on the Rocket plan may be emailed to legal@cirm.ca.gov. Oral comments may be delivered during a 30-minute online meeting on Sept. 8 at 9 a.m. PDT.

Editor’s note: The quote from Hank Greely was mangled in an earlier of this article. It has been clarified.

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In a landmark move, California’s stem cell/gene therapy program has opened the door to public comment on an affordability plan for its first approved treatment, developed after spending billions over the last 21 years.

The six-page, heavily redacted proposal is for patients with an ultra-rare genetic affliction known as LAD-1. The California Institute for Regenerative Medicine (CIRM) backed a clinical trial for the treatment, kresladi, at UCLA with $5.9 million. CIRM is supported by $12 billion in state taxpayer funds and has made nearly 1,600 awards since it was created by voters in 2004.

The agency is the only research program of that magnitude in the nation that is required by law to make its state-funded treatments affordable for its residents.

The New Jersey company behind kresladi is Rocket Pharmaceuticals, Inc, a publicly traded firm. Rocket has not announced a price for the therapy yet, but it is expected to run as high as $4 million. That is at the top end of the range for other gene therapies internationally.

“While the price per patient is staggering to the public, the ‘ground truth’ in 2026 remains that these therapies are priced as a front-loaded investment to eliminate decades of catastrophic medical expenses and, more importantly, to save lives that were previously considered untreatable,” said one health outcomes consultant, Michael A. S. Guth, on his website.

Key elements of Rocket’s affordability plan were blacked out in the CIRM internet posting. CIRM said they involved proprietary information that can be withheld under state law. But redacted material also appears to be critical to understanding whether the plan actually provides affordability.

Other elements of the plan would apply a federal poverty measure to California residents, who live in one of the nation's most unaffordable states. That could make potential patients appear less impoverished and result in the denial of assistance.

The plan states that treatment would be free for qualified applicants. Their numbers would be very small. Estimates are that only 25 new cases occur each year. Most would die young without treatment. Kresladi is approved for use only in patients who don’t have a matched sibling who can provide a stem cell transplant.

While the treatment would be free, Rocket’s plan specifically says, “The company will not provide coverage for administration, facility fees, or other non-drug medical services under this access plan.” The next 4.5 lines in the same paragraph are blacked out.

Nine patients were treated in the clinical trial that supported the treatment’s efficacy. Three were in Spain and London and six at UCLA, where the CIRM funding went.

The nine ranged in age from five months to nine years. “The small cohort reflects the rarity of the condition,” said UCLA.

Three children with the affliction, however, emerged in one Ohio family, the Langenhops. They were successfully treated with the curative therapy in Los Angeles by UCLA’s Donald Kohn and his team.

The treatments were lengthy and required the Langenhops to take up temporary residence near UCLA, one of the more expensive areas in the state, where they spent eight months. Zillow reports that a two-bedroom apartment currently runs in the $4,000 to $6,000-a-month range. CIRM’s affordability plan would not have applied to the family, however, because they were not legal residents of the Golden State.

CIRM’s affordability mandate is unique in the country. But the agency is also unique in other ways. It is the only state program that will need a vote of the people to continue its work. Its funding will run out in a few years, and it will need to ask voters for more billions or close its doors. Fulfilling the promise of affordability will be one of the keys to success at the ballot box.

(Written comments should be sent to legal@cirm.ca.gov by 5 p.m. Sept. 8, the deadline for submission. Oral comments are scheduled during a 30-minute public meeting Sept. 8 at 9 a.m. PDT.)

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A video from a family with a Duchenne muscular dystrophy child.

An estimated 15,000 boys and young men and their families are waiting this week for the latest development involving a possible therapy that would help ease some of the symptoms of a deadly disease that progressively destroys their muscular structure, including their hearts.

The treatment was developed by a San Diego firm backed with millions from the state of California. The therapy is under review by federal regulators, who are considering whether to approve it for use outside of clinical trials.

The firm is Capricor Therapeutics, which has tussled with the Food and Drug Administration (FDA) for more than a year over approval of its deramiocel cell therapy for the genetic affliction Duchenne muscular dystrophy.

Still up in the air is the FDA “action date” of Aug. 22, which was last Saturday. The company submitted new information regarding the treatment’s efficacy and hoped that the date would be extended.

However, Capricor has not announced any change in the date as of this writing yesterday, nor has the FDA. However, without FDA action, the review of the therapy presumably continues.

Capricor has seen its stock price decline sharply over the past year, down considerably from its 52-week high of $40.37. Last week, the STAT news service carried a column that said it was “near-certain” that the Capricor therapy would be rejected by regulators.

Capricor’s stock price dropped 14 percent following publication of the column. It closed at $6.29 on Friday, but was still higher than its 52-week low of $2.96.

Adam Feuerstein wrote that Capricor’s latest effort to win approval was a “stall tactic. Nothing more.”

“The only option left for Capricor to secure approval of its treatment, called deramiocel, is to conduct an entirely new, randomized clinical trial,” Feuerstein said.

“We’re now 10 years into a downward spiral of ineffective and unproven medicines for the progressive, muscle-wasting disease approved by the FDA under the guise of regulatory flexibility. Billions of dollars have been spent on treatments for Duchenne without knowing if they truly help patients. It’s madness, and it needs to stop.”

The California Institute for Regenerative Medicine has invested $18 million in Capricor, including $3.4 million specifically for deramiocel.

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California’s $12 billion effort to finance stem cell and gene therapy research is looking for someone who will help it use AI to bring “the promise of regenerative medicine closer to reality.”

The posting for the new position at the California Institute for Regenerative Medicine (CIRM) says the person will be the agency’s “technical expert for artificial intelligence, data engineering, and business intelligence initiatives.”

“Improved organizational effectiveness and data driven decision-making” are part of the responsibility of the job.

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An infectious video involving the transparency of a California state biomedical research organization, whose 17,000-word genome rarely inspires influencers, caught my attention recently.

It popped up on LinkedIn, an online site dedicated to those seeking to work and companies seeking to hire. At least origin…

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Why hasn’t the California state agency that has invested $18 million in taxpayer dollars in Capricor Therapeutics spoken up to help the company in its struggles with federal regulators?

That is a question that the California Stem Cell Report posed recently to the $12 billion California Institute for Regenerative Medicine (CIRM). After all, its directors unanimously approved awarding the $18 million to the San Diego firm.

CIRM’s explanation emerged yesterday. Here is the full text.

“CIRM does not comment on the finances or business strategy of organizations we have supported. However, we know that Duchenne muscular dystrophy is a devastating disease for both the person with the disease and their families.

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The largest scientific research measure on the California ballot since 2020 -- an ambitious $8.4 billion initiative -- received some significant media attention during the past few days, including a brief look at its sponsor, a Honda-driving billionaire.

He is Gary…

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Despite unfavorable action by a key federal committee and another steep drop in its stock price, Capricor …

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A proposed treatment for a fatal affliction that slowly destroys the muscles of boys and young men hit a major roadblock today in its developer’s quest to win federal approval of the new therapy.

…

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Young men facing death from Duchenne muscular dystrophy spoke about the affliction today at a federal hearing on proposed, new treatment under development by a California firm, Capricor Therapeutics.

The condition leads to a slow wasting …

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