CIRM Grants for AIDS Cure
$71,336,396.00 – Fact Sheet
as of 11/12/2015
List of Active Research at CIRM. Includes HIV/AIDS and all diseases.
Stem Cell Programming With Chimeric Antigen Receptors to Eradicate HIV Infection
GENE-MODIFIED HEMATOPOIETIC STEM/PROGENITOR CELL BASED THERAPY FOR HIV DISEASE
A Phase I, Open-Label Study to Assess the Safety, Feasibility and Engraftment of Zinc Finger Nucleases (ZFN) CCR5 Modified Autologous CD34+ Hematopoietic Stem/Progenitor Cells with Escalating Doses of Busulfan in HIV-1 (R5) Infected Subjects with Suboptim
- The following provides details of HIV/AIDS research by Calimmune, Inc., Gladstone Institute, and Sangamo Biosciences. (For research details at hospitals and universities visit our AIDS Research Institutions page.)
- We have only scratched the surface of biomedical corporations and institutions researching a cure for HIV/AIDS in California.
Calimmune is a clinical-stage HIV gene medicines company focused on developing innovative cell-based therapies for HIV.
We are developing a full product pipeline of therapeutic candidates to address the unique needs of individuals at different stages of HIV infection and with various treatment histories. Discovery-stage research is underway for other disease areas including cancer and immune deficiencies.
Our gene therapy process works by genetically modifying patients’ own cells to shield and strengthen their immune system.
The promise of this approach is to create enduring and perhaps life-long protection for individuals living with a broad range of debilitating and currently incurable diseases.
We have extensive expertise and more than 120 patents in viral vector and non-vector delivery technology, therapeutic product design and manufacturing, which we have developed into a potent gene therapy platform with the potential of broad applications in a wide variety of indications.
Our treatments seek to shield and strengthen patients’ immune system, rather than trying to modify the virus itself.
In HIV, our approach is designed to mimic the genetic immunity that naturally occurs in approximately 1 percent of the European population. People with this mutation do not produce CCR5, a protein on the surface of white blood cells that enables HIV to infect T cells. T cells play a key role in maintaining the human immune system and are HIV’s main target.
By infusing patients with their own blood stem cells that have been treated to reduce production of CCR5, as well as mature T cells that are modified to block CCR5 and prevent HIV entry, Calimmune aims to stop the progression of HIV to AIDS with a single treatment. After a decade of refinements to the early technology and promising pre-clinical results, the first-generation of Calimmune’s technology is in Phase I/II human trials.
Viral vectors play a fundamental role in gene therapy. They’re the vehicles for delivering new, beneficial genetic material into patients’ cells, and millions of them are used in a single procedure. They are most commonly based on naturally-occurring viruses that have been modified (stripped) of all of the components required to self-replicate and infect additional cells. These viral vectors are ideal for introducing gene therapies.
The traditional process for manufacturing these therapeutic vectors is expensive, labor-intensive and yields small batches. We have exclusively licensed and developed a proprietary cell-based vector manufacturing process that is both reproducible and scalable.
Calimmune’s Cytegrity™ platform is an important step toward advancing the field of gene therapy by realizing its full potential on a commercial scale. Not only does this technology enable gene therapy manufacturing for large patient populations, but also due to its streamlined automation capacity, will significantly lower the cost of production—and ultimately, the cost of treatment.
We are committed to making this technology broadly available to academic, government and corporate partners, and we believe that effective gene therapies should be accessible to all patients who can benefit.
Calimmune, Inc., which has received $8.3 million from the California stem cell agency, (CIRM) today announced it has rustled up another $15 million to help out with the work that the agency is backing.
Calimmune is currently engaged in a clinical trial in Los Angeles and San Francisco involving a treatment for HIV.
Other accounts state that Irvin Chen, director of the UCLA AIDS Institute, was a co-founder
Calimmune, which has been backed by California’s stem cell agency CIRM, has been working on Cal-1, looking to insert a gene that slashes the production of CCR5, a protein which is found on the surface of white blood cells. By re-engineering patient-derived CD34-positive stem cells and CD4-positive T cells, which are reinfused after they’re adapted, the company is looking to provide patients with a mix of short- and long-term protective treatments. And initially they’ll be focused on patients who can’t tolerate the current generation of cocktails used to keep HIV in check.
Calimmune hasn’t had much of a profile outside of the HIV world, but Breton is looking to change that. The company has a staff of 40 now, he says, and is looking to expand and possibly strike a partnership deal with a Big Pharma in the space. And unlike some of the leading gene therapy companies in the industry which are targeting tiny populations, Calimmune is tackling a treatment for a disease with a huge, global population of patients. Discussions about million-dollar therapies, he says, won’t work for something like HIV.
“Our mission,” says Breton, “is to provide broad capacity; democratize gene therapy for the masses.”
Gladstone Institute, San Francisco
27 CIRM Grants Total: $51,372,763
Gladstone uses game-changing stem cell biology to advance our mission to better understand, prevent, treat and ultimately cure some of the world’s most relentless diseases. Building on current stem cell research, our scientists envision a world in which:
- stem cells are used to create a human immune system in the laboratory, where it is used to test a vaccine that prevents HIV/AIDS.
We have ongoing Phase 2 clinical trials to evaluate the first therapeutic application of our ZFN technology, SB-728-T, a ZFN-modified T-cell product for the treatment of HIV/AIDS.
We are also developing a ZFN-modified hematopoietic stem cell product, SB-728mR-HSC, for HIV/AIDS. The IND for this program is active and the Phase 1 study is expected to begin at City of Hope in 2015, partially funded by a $5.6 million CIRM Strategic Partnership Award.
Encouraged by Belgium patient cured of AIDS via a bone marrow transplant. https://www.youtube.com/watch?v=lASLpZPu-k0
We are located in Richmond, California, in the Bay Area, the birthplace of biotechnology.