Member Companies Driving Innovation in Immunology

Autoimmune diseases occur when the immune system attacks healthy cells, organs and tissues, mistaking them for harmful intruders such as viruses and bacteria. More than 80 autoimmune diseases are currently known to exist, with Type 1 diabetes, Crohn’s disease and lupus being the most common, and the National Institutes of Health notes it’s the third-most prevalent disease category. Research and development in immunology today spans vaccinations, finding treatments for immunodeficiencies and allergies, and immunotherapy—using the body’s own immune system to fight diseases such as cancer. Biocom California members have achieved several milestones in immunology and immunotherapy over the past year, including the development of new treatment for ulcerative colitis, plans for an expansive research facility in Southern California dedicated to the field and the discovery of additional promising findings for an established treatment for large B-cell lymphoma.

From the Ground Up

The site of a former shopping mall in Los Angeles is on its way to being transformed into a 700,000-square-foot research center devoted to advancing immunology and immunotherapy. UCLA announced earlier this year that it acquired the space with plans to develop the UCLA Research Park, which will also house the new California Institute for Immunology and Immunotherapy as well as the UCLA Center for Quantum Science and Engineering. The new institute will be located just two miles from the university’s campus. “The goal is to build the immunology equivalent of Silicon Valley in Los Angeles,” UCLA Health CEO John Mazziotta said in a press release. “This is probably the most exciting project I’ve worked on during my UCLA career.” In addition, Mazziotta said the institute will “leverage emerging discoveries in immunology and immunotherapy to develop paradigm-shifting treatments for a range of diseases.”

Landmark Treatment for Ulcerative Colitis, Gearing Up for Immunotherapy Expansion

Ulcerative colitis is an autoimmune-related disease that causes inflammation in the digestive system. Many patients report experiencing unpleasant symptoms, such as an unpredictable sense of urgency to use the restroom, pain and even rectal bleeding. Nour, who was diagnosed with ulcerative colitis when she was 21, shared her story with Eli Lilly on how the condition disrupted her life—a single sip of water could trigger a flare up, and she canceled taking the MCAT because she was not confident she could sit through the duration of the exam.

Last fall, Eli Lilly received FDA approval for Omvoh, a first-in-class treatment for patients who have moderate to severe ulcerative colitis. Administered via injection, it’s the first and only interleukin-23p19 (IL-23p19) antagonist—an antibody therapy—for the condition. The company is currently investigating Omvah’s effectiveness in possibly treating Crohn’s disease, a chronic inflammatory bowel disease.

In a quest to further its immunology work, earlier this month Eli Lilly announced its acquisition of Morphic for $3.2 billion and to advance its lead candidate, a small molecule inhibitor in development to treat inflammatory bowel disease.

Historic Institution

The La Jolla Institute for Immunology is a historic San Diego-based organization devoted to researching the immune system to develop therapies and cures. Founded in the 1980s by a group of scientists and industry leaders, including Kimishige Ishizaka and Teruko Ishizaka—two pioneering immunologists who discovered the IgE (immunoglobulin E) protein, which plays a critical role in the body’s immune response—the institute continues to innovate today. In a recent paper published in Genome Biology, the institute’s scientists outlined a new method they developed to match genes to their molecular switches using machine learning and computational tools, called a neural network, that is modeled after how neurons in the brain work. The research examined how a DNA modification, 5hmC, is related to gene expression, and the institute says the study can help better understand the link between gene expression and the development of diseases such as cancer.

Disrupting Cancer

The Parker Institute for Cancer Immunotherapy is a network of several leading cancer centers across the U.S., including UCLA and University of California, San Francisco, and its scientists are devoted to researching and developing immune therapies to treat cancer. The institute recently announced a new $125 million investment to expand its research for cancer immunotherapy, and says the funds will be distributed as grants over the next five years to researchers in its network. The institute notes its scientists have published nearly 4,000 research papers.

The institute was founded in 2016 with a $250 million gift by Sean Parker, a Silicon Valley entrepreneur who founded Napster and served as Facebook’s first president. Parker has shared his personal experiences living with allergies, and over the years he has won several notable awards for his philanthropic efforts.

New Ways of Thinking

Capstan Therapeutics has been attracting attention for its focus on developing in vivo RNA-based therapies that engineer immune cells and target pathogenic cells using its proprietary targeted lipid nanoparticles (tLNPs). The San Diego-based company’s lead candidate, CPTX2309, is an in vivo CAR-T cell aimed at treating autoimmune disorders. A company statement said the goal of this approach is to reset the immune system through rapid deep B-cell depletion in both blood and lymphoid tissues, without the challenges of conventional ex vivo CAR-T. Capstan was named one of BioSpace’s Top Life Sciences Startups to Watch in 2023, and this spring the company announced a $175 million oversubscribed Series B.

Going Natural

NK cells, or natural killer cells, are part of a group of white blood cells in our immune system that can destroy infected cells and cancer cells. Artiva Biotherapeutics is a San Diego-based company that focuses on developing therapies based on NK cells, which have been called a “cellular Swiss Army knife” as they are not tailored to specific antigens. NK cells are derived from donors, and these cells can be given to multiple patients without the need for gene editing. Artiva’s lead candidate, AlloNK, is an allogeneic and cryopreserved NK cell-based treatment that is currently undergoing a clinical trial as a possible treatment for lupus nephritis—a disease where the immune system attacks the kidneys—and the first subject was dosed with AlloNK this spring. In July, the company upsized its IPO to $167 million and was listed on the Nasdaq.

New Solutions for a Skin Condition

Our skin is an organ that can be affected by autoimmune disorders, with psoriasis being a common one—several celebrities have shared their diagnoses and experiences on living with psoriasis. The body’s immune system mistakenly targets its own skin cells, leading to chronic inflammation and skin abnormalities. One rare form of the disease, Generalized Pustular Psoriasis (GPP), causes widespread pustules and scaling and could even be potentially life-threatening if not treated. Anaptys Bio, a San Diego-based company, developed imsidolimab, a fully humanized IgG4 antibody, to treat GPP. It recently announced promising findings from its Phase 3 clinical trial in patients who receive an initial intravenous dose of imsidolimab followed by monthly subcutaneous doses.

“The success of the GEMINI-1 and GEMINI-2 Phase 3 trials highlights Anaptys’ ability to internally discover and develop differentiated antibodies that deliver meaningful outcomes for patients,” Daniel Faga, president and CEO of Anaptys, said in a release. “We remain intent on out-licensing imsidolimab to bring this therapy to patients living with this highly morbid condition.”

Promising New Findings

First co-founded in Los Angeles by renowned cancer researcher and entrepreneur Arie Belldegrun, Kite, a Gilead Company, has been at the forefront of developing CAR-T and cell therapies to treat blood cancers. This summer, the company presented new findings about Yescarta, its therapy for large B-cell lymphoma, at the 2024 European Hematology Association Annual Congress in Madrid. Findings included that Yescarta showed higher success rates in manufacturing and in the performance of T-cells when used for patients in the earlier stages of treatment (second-line) compared to later stages (third-line or beyond). The company also said it is studying the administration of Yescarta in outpatient settings, which could make the treatment more accessible for patients.