Member Companies Advancing Innovations in Precision Medicine

A “one-size-fits-all” method doesn’t always work when it comes to everyday things like fashion, and proponents of precision medicine say the same approach should not be applied to healthcare. Precision medicine tailors medical care and treatments to the needs of each patient and has the potential to transform how diseases are diagnosed and treated. Johnson & Johnson noted most future healthcare interventions will have a precision medicine component, and it’s a growing field that reached a market size of nearly $25 billion in the U.S. in 2023. Recently in California, Gov. Gavin Newsom’s office announced it will focus on pandemic preparedness through precision medicine research.

In this edition, we’re highlighting Biocom California member companies that are on the forefront of advancing innovations in precision medicine, including emerging startups and recent news of a large acquisition.

$1.5B Acquisition for Immunotherapy Advancement

Human Immunology Biosciences, or HI-Bio, is a Bay Area-based company that uses a precision medicine approach to develop new therapies for immune-mediated diseases (IMDs). The company says dysfunction in the cells that make up the immune system—such as plasma cells, neutrophils (a type of white blood cell) and mast cells—can lead to IMDs, and it has been developing an antibody candidate, felzartamab, to target the cellular causes of diseases. Felzartamab is currently in Phase 2 for the treatment of IMDs including: IgA nephropathy (IgAN), a chronic kidney condition; antibody-mediated rejection (AMR) in kidney transplant patients; and primary membranous nephropathy (PMN, an autoimmune disease affecting the kidneys that can cause nephrotic syndrome in adults who do not have diabetes. The company recently made headlines when Biogen announced it completed its acquisition of HI-BIO for $1.15 billion to diversity its scope of therapies. Biogen says it will advance felzartamab to Phase 3.

Engineering Better Cell and Gene Therapy Delivery

Basilard Biotech is a San Diego-based company focused on addressing what it says are current limitations of viral and non-viral gene delivery in cell-based therapies with its technology platform Celletto, which uses a novel approach to nanomechanical gene delivery. The company was founded by its CEO Brynley Lee at a technology incubator at UC Riverside, where he was CEO-in-Residence, and says Celletto addresses bottlenecks that are increasing in gene delivery in the development of cell and gene therapies with its proprietary mechanical delivery solution.

Developing ‘Smart mRNA’ for New Therapies

Radar Therapeutics is a Bay Area-based startup that just emerged from stealth mode this past May and made headlines for raising more than $13 million in seed funding, with companies such as Eli Lilly participating. Led by CEO Sophia Lugo and CSO and President Eerik Kasenitt, Radar’s technology was developed at Stanford University and focuses on developing mRNA-based therapies that are programmable, targeted and “smart.” The company says that by using therapies with RNA sensor, therapies can be delivered to the right cells at the right time, and notes its technology allows them to read the information in a cell in real-time. In the May, Radar Therapeutics was one of three companies selected to receive free lab space and facilities at Bakar Labs at UC Berkeley for one year.

Innovation in Targeted Cancer Therapies

Boundless Bio is a precision oncology company focused on developing treatments for patients with oncogene-amplified cancers—which it says makes up more 5 million new cancer diagnoses every year. The San Diego-based company’s niche is extrachromosomal DNA (ecDNA)-directed therapies, meeting an unmet treatment need. Boundless Bio launched a $100 million IPO this March to advance its cancer therapy candidates, including two candidates, BBI-355 and BBI-825, currently in the clinical stage. The company is also working on a discovery-phase ecDNA program.

Promising Outcomes

Researchers at City of Hope in Los Angeles recently announced that kidney cancer patients who are currently being treated with cabozantinib (a tyrosine kinase inhibitor developed by Exelixis) and nivolumab (an immunotherapy) and took a microbiome (live bacterial) supplement, Clostridium butyricum MIYAIRI 588 (CBM588), during treatment could see improved clinical outcomes. The single-center Phase 1 trial, patients’ microbiomes were tested after 13 weeks of treatment. The study and its findings were published in Nature Medicine.

A Landmark FDA Approval

Earlier this spring, the FDA announced its approval of Pfizer’s gene therapy for a rare genetic blood disorder, Beqvez, making it the company’s first gene therapy to receive clearance in the U.S. The drug is for adult patients who have moderate to severe hemophilia B, a genetic disorder where blood is not able to clot properly to control bleeding due to the lack of a protein, factor IX. Pfizer says Beqvez is a one-time treatment that will enable patients to produce sufficient amounts of the protein, versus having to get multiple protein infusions. “A one-time treatment with Beqvez has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term,” Adam Cuker, M.D., M.S., director of Penn Comprehensive and Hemophilia Thrombosis Program, said in a release.