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Sickle Cell Clinical Trial Aims to Cure Disease by Correcting Patient’s Mutated Gene

Sickle cell disease is a genetic blood disorder that affects the structure and function of hemoglobin, reduces the ability of red blood cells to transport oxygen efficiently, and progresses to a chronic vascular disease that affects approximately 100,000 Americans and millions worldwide, with a disproportionate effect on the Black community.

Published

3 years ago

February 15, 2022

Oakland Post

Brooklynn Haynes is participating in a research trial that aims to cure sickle cell anemia, a disease that affects 100,000 people in the U.S. Photo courtesy of UCSF.

Benioff Children’s Oakland Receives $17M in Grants to Launch 4-Year Study Using CRISPR to Eradicate Sickle Cell Mutation

By Lorna Fernandes

UCSF Benioff Children’s Hospital Oakland has received the largest research trial grant in its history to launch an innovative clinical trial that aims to cure sickle cell disease.

The study, which plans to enroll its first patient by the middle of 2022, will use CRISPR-Cas9 gene-editing technology on patients’ own blood stem cells to correct the mutated gene that causes the debilitating disorder.

Benioff Oakland researchers have received $17 million to support the 4-year trial, in two grants of $8.4 million from the California Institute for Regenerative Medicine (CIRM) and $8.6 million from the NIH Cure Sickle Cell Initiative, supported by the National Heart, Lung, and Blood Institute (NHLBI).

This trial is the first to apply CRISPR-Cas9 technology in humans using a non-viral therapy to correct the mutation that causes sickle cell disease and is part of a UC research consortium led by UCSF with UCLA and UC Berkeley.

Researchers are recruiting patients for treatment in Oakland and Los Angeles, beginning with up to six adults with sickle cell disease. If found to be safe and effective, it will expand to enroll three adolescents aged 12 to 17 years old. Seven patients are expected to be treated in Oakland and two at UCLA.

It is the first-time clinical researchers have attempted to correct a harmful beta-globin gene mutation in a patient’s own cells with non-virally delivered CRISPR gene-correction tools, according to Mark Walters, MD, a professor of pediatrics at UCSF and principal investigator of the clinical trial and gene editing project.

“This therapy has the potential to transform sickle cell disease care by producing an accessible, curative treatment that is safer than the current therapy of stem cell transplant from a healthy bone marrow donor,” Walters said. “If this is successfully applied in young patients, it has the potential to prevent irreversible complications of the disease.”

This clinical study continues BCH Oakland’s commitment to treating and finding a cure for sickle cell disease. The Comprehensive Sickle Cell Disease Center was created at the hospital in 1973 to provide expert diagnosis, treatment and long-term health management for children and adults with sickle cell disease.

Now known as the BCH Oakland Sickle Cell Center of Excellence, it is recognized as having one of the largest and most comprehensive sickle cell programs in the Western United States.

This trial will combine CRISPR technology developed at Innovative Genomics Institute (IGI) – a joint UC Berkeley-UCSF initiative founded by Nobel Laureate Jennifer Doudna – with UCLA’s expertise in genetic analysis and cell manufacturing, as well as its clinical excellence in the field, and nearly 50 years of expertise at Benioff Children’s Oakland in sickle cell care, including cord blood and marrow transplantation, and gene therapy.

“The use of CRISPR gene editing to fix the mutation causing sickle cell disease in each patient’s own blood-forming stem cells required the development of new methods to produce more than 100 million cells per patient that are gene-corrected, healthy and pure,” said Donald Kohn, MD, medical director of the UCLA Human Gene and Cell Therapy Facility. “Previous research studies were limited to producing ½ to 2 million genetically corrected cells, so this represents a significant increase in scale.”

The disease is caused by a single-letter change, or mutation, in human DNA. This trial will use CRISPR genome editing to correct this mutation directly. The project team from UCSF, the IGI, and UCLA have developed CRISPR_SCD001, a patient-specific blood stem cell therapy that has been modified by a CRISPR-Cas9 nuclease to stimulate repair of the sickle mutation.

In the current trial, the patient’s blood stem cells will be extracted and sent to UCLA’s gene manufacturing lab to be processed using electrical pulses that create temporary pores in their membranes. These pores allow the CRISPR-Cas9 platform to enter the cells and travel to the nucleus, where it corrects the sickle cell mutation before the cells are reintroduced to the patient.

“The goal is to develop a cure that is not just safe and effective, but one that is affordable by to those who need it most,” Doudna said. “There have been a lot of promising developments in recent years with CRISPR-based therapies for sickle cell and other genetic diseases, but it’s essential that we have studies like this that can help us create truly accessible cures.”

As part of its legacy, BCH Oakland was recognized for establishing the first not-for-profit sibling donor cord blood bank, leading to children being cured of sickle cell disease and other blood diseases.

Among its many accomplishments in treating sickle cell disease, BCH Oakland scientists studied and received FDA approval for the drug Voxelotor to improve hemoglobin levels and reduce anemia and hemolysis in sickle cell patients.

Going forward, the UCSF research team is hoping to use expanded resources under development in the UCSF Living Therapeutics Initiative as this new treatment is expanded, including the potential to manufacture the investigational cell therapy product at UCSF.

Lorna Fernandes is the senior public information representative at the UCSF Office of Communications and Public Affairs.

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Oakland Post: Week of May 21 – 27, 2025

The printed Weekly Edition of the Oakland Post: Week of May 21 – 27, 2025

Published

4 days ago

May 25, 2025

Oakland Post

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Chevron Richmond Installs Baker Hughes Flare.IQ, Real-time Flare Monitoring, Control and Reduction System

While the sight of flaring can cause concern in the community, flares are essential safety systems that burn pollutants to prevent them from being released directly into the atmosphere. They activate during startup and shut-down of facility units or during upsets or equipment malfunctions. The typical flare stack is about 200 feet high so that vapors are well above street levels.

Published

1 week ago

May 19, 2025

Oakland Post

Image courtesy The Richmond Standard.

The Richmond Standard

Chevron Richmond recently installed flare.IQ, a real-time, automated system that will improve the facility’s flaring performance.

The technology, developed by Panametrics, a Baker Hughes business, uses sensors to monitor, reduce and control flaring in real time. It collects and assesses data on refinery processes, such as temperature, pressure, gas flow and gas composition, and adjusts accordingly to ensure flares burn more efficiently and cleanly, leading to fewer emissions.

“The cleaner the flare, the brighter the flame can look,” said Duy Nguyen, a Chevron Richmond flaring specialist. “If you see a brighter flame than usual on a flare, that actually means flare.IQ is operating as intended.”

“A key element in Baker Hughes’ emissions abatement portfolio, flare.IQ has a proven track record in optimizing flare operations and significantly reducing emissions,” said Colin Hehir, vice president of Panametrics, a Baker Hughes business. “By partnering with Chevron Richmond, one of the first operators in North America to adopt flare.IQ, we are looking forward to enhancing the plant’s flaring operations.”

The installation of flare.IQ is part of a broader and ongoing effort by Chevron Richmond to improve flare performance, particularly in response to increased events after the new, more efficient hydrogen plant was brought online in 2019.

Since then, the company has invested $25 million — and counting — into flare minimization. As part of the effort, a multidisciplinary refinery team was formed to find and implement ways to improve operational reliability and ultimately reduce flaring. Operators and other employees involved in management of flares and flare gas recovery systems undergo new training.

“It is important to me that the community knows we are working hard to lower emissions and improve our flaring performance,” Nguyen said.

Also evolving is the process by which community members are notified of flaring incidents. The Community Warning System (CWS), operated by Contra Costa County is an “all-hazard” public warning system.

Residents can opt-in to receive alerts via text, e-mail and landline. The CWS was recently expanded to enable residents to receive notifications for “Level 1” incidents, which are considered informational as they do not require any community action.

For more information related to these topics, check out the resources included on the Chevron Richmond, CAER and Contra Costa Health websites. Residents are also encouraged to follow @chevronrichmond and @RFDCAOnline on Facebook and X (formerly Twitter), where additional information may be posted during an incident.

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Oakland Hosts Town Hall Addressing Lead Hazards in City Housing

According to the city, there are 22,000 households in need of services for lead issues, most in predominantly low-income or Black and Latino neighborhoods, but only 550 to 600 homes are addressed every year. The city is hoping to use part of the multimillion-dollar settlement to increase the number of households served each year.

Published

2 weeks ago

May 18, 2025

Oakland Post

iStock.

By Magaly Muñoz

The City of Oakland’s Housing and Community Development Department hosted a town hall in the Fruitvale to discuss the efforts being undertaken to remove lead primarily found in housing in East and West Oakland.

In 2021, the city was awarded $14 million out of a $24 million legal settlement from a lawsuit against paint distributors for selling lead-based paint that has affected hundreds of families in Oakland and Alameda County. The funding is intended to be used for lead poisoning reduction and prevention services in paint only, not water or other sources as has been found recently in schools across the city.

The settlement can be used for developing or enhancing programs that abate lead-based paint, providing services to individuals, particularly exposed children, educating the public about hazards caused by lead paint, and covering attorney’s fees incurred in pursuing litigation.

Most of the homes affected were built prior to 1978, and 12,000 of these homes are considered to be at high risk for lead poisoning.

City councilmember Noel Gallo, who represents a few of the lead-affected Census tracts, said the majority of the poisoned kids and families are coming directly from neighborhoods like the Fruitvale.

“When you look at the [kids being admitted] at the children’s hospital, they’re coming from this community,” Gallo said at the town hall.

In order to eventually rid the highest impacted homes of lead poisoning, the city intends to create programs and activities such as lead-based paint inspections and assessments, full abatement designed to permanently eliminate lead-based paint, or partial abatement for repairs, painting, and specialized cleaning meant for temporary reduction of hazards.

In feedback for what the city could implement in their programming, residents in attendance of the event said they want more accessibility to resources, like blood testing, and information from officials about lead poisoning symptoms, hotlines for assistance, and updates on the reduction of lead in their communities.

Attendees also asked how they’d know where they are on the prioritization list and what would be done to address lead in the water found at several school sites in Oakland last year.

City staff said there will be a follow-up event to gather more community input for programming in August, with finalizations happening in the fall and a pilot launch in early 2026.