Stem Cell and Gene Therapy Agency Funds Clinical Trial for a Functional Cure for HIV

South San Francisco, CA – The use of antiretroviral drugs has turned HIV/AIDS from a fatal disease to one that can, in many cases in the US, be controlled. But these drugs are not a cure. Today the governing Board of the California Institute for Regenerative Medicine (CIRM) invested $6.85 million in a therapy that aims to cure the disease.

This is the 82nd clinical trial funded by CIRM.

There are approximately 38 million people worldwide living with HIV/AIDS. And each year there are an estimated 1.5 million new cases. The vast majority of those living with HIV do not have access to the life-saving antiretroviral medications that can keep the virus under control. People who do have access to the medications face long-term complications from them including heart disease, bone, liver and kidney problems, and changes in metabolism.

The antiretroviral medications are effective at reducing the viral load in people with HIV, but they don’t eliminate it. That’s because the virus that causes AIDS can integrate its DNA into long-living cells in the body and remain dormant. When people stop taking their medications the virus is able to rekindle and spread throughout the body.

Dr. William Kennedy and the team at Excision Bio Therapeutics have developed a therapeutic candidate called EBT-101. This is the first clinical study using the CRISPR-based platform for genome editing and excision of the latent form of HIV-1, the most common form of the virus that causes AIDS in the US and Europe. The goal is to eliminate or sufficiently reduce the hidden reservoirs of virus in the body to the point where the individual is effectively cured.

“To date only a handful of people have been cured of HIV/AIDS, so this proposal of using gene editing to eliminate the virus could be transformative,” says Maria T. Millan, MD, President and CEO of CIRM. “In California alone there are almost 140,000 people living with HIV. HIV infection continues to disproportionately impact marginalized populations, many of whom are unable to access the medications that keep the virus under control. A functional cure for HIV would have an enormous impact on these communities, and others around the world.”

The Excision Bio Therapeutics team also scored high on their plan for Diversity, Equity and Inclusion. Reviewers praised them for adding on a partnering organization to provide commitments to serve underserved populations, and to engaging a community advisory board to help guide their patient recruitment.

The CIRM Board also approved $15.6 million in funding for 11 awards under its DISC-0 program. The goal of the program is to:
• Broadly re-initiate funding of basic stem/progenitor (“stem”) cell science and genetic research.
• Support rigorous studies addressing critical basic knowledge gaps or bottlenecks in regenerative medicine research.
• Advance the development of stem cell-based tools for innovation.

The awards are for three years and up to $1 million in direct costs and they will be offered twice a year.

The successful applicants are:

APPLICATION

TITLE

P.I/INSTITUTION

AMOUNT

DISC0-13901

Drug Discovery for Dilated Cardiomyopathy using
Patient-Derived Human iPSCs

 

Syed Mukhtar Ahmed — Greenstone Biosciences

 

$1,350,000

DISC0-13757

Drivers of trophoblast stem/progenitor cell identity
in human placenta

 

Francesca Soncin — University of California, San Diego

 

$993,881

 

DISC0-13801

Control of OCT4 abundance and function in human
stem cells

 

Michael P. Rape — University of California, Berkeley

 

$1,415,301

 

DISC0-13914

Developing a Human Model of Sporadic ALS Using
Machine Learning and Robotic Microscopy

 

Julia Kaye — The J. David Gladstone Institutes

 

$1,406,622

 

DISC0-13765

Engineering AAV capsids for transduction of neural
and muscle stem cells

 

Melissa Jan Spencer — University of California, Los Angeles

 

$999,999

DISC0-13788

Modulation of human alveolar stem cells to promote
lung regeneration and avoid pulmonary fibrosis

 

Harold A. Chapman — University of California, San Francisco

 

$1,626,001

 

DISC0-13875

Developing a microglia replacement therapy

 

Marius Wernig — Stanford University

 

$1,577,979

 

DISC0-13750

Generation of cortical organoids with tunable areal
identities by spatial engineering of morphogens

 

Momoko Watanabe — University of California, Irvine

 

$1,497,032

 

DISC0-13816

Towards a trophectoderm stem-cell model
representing human blastocysts of the highest
implantation potential

 

Heidi Cook-Andersen — University of California, San Diego

 

$1,584,000

 

DISC0-13806

Development of universal off-the-shelf iPSC
derived dendritic cells for use as patient specific
anti-tumor vaccine

 

Robert Blelloch — University of California, San Francisco

 

$1,625,998

 

DISC0-13808

Development of a stem-cell based approach to
interpret global effects of genetic variants
contributing to neurodevelopmental disease risk

 

Alexis Komor — University of California, San Diego

 

$1,518,982

 

 

 

About CIRM

At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission.

To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies.

With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.

For more information go to www.cirm.ca.gov