Once focused on basic science, CIRM is now writing checks to propel companies from the lab to the clinic.
In the world of medical research, the journey from a brilliant idea in a lab to an actual treatment in a clinic is notoriously difficult and expensive—so difficult that promising therapies often languish. The California Institute for Regenerative Medicine (CIRM), one of the world's largest institutions dedicated to regenerative medicine, is radically reshaping this pathway by strategically partnering with the biotechnology industry 2 .
With nearly $100 million awarded to various projects in early 2025 and a new $73 million investment in discovery research, the state agency is actively courting commercial partners to bridge the gap between academic discovery and real-world cures 2 5 . This shift marks a deliberate strategy to accelerate the delivery of transformative stem cell and gene therapies to patients.
CIRM was established by California voters in 2004 with a mission to accelerate stem cell and gene therapy research 5 . For years, its focus was heavily weighted toward foundational academic science. Today, its leadership recognizes that fulfilling its mission requires the unique skills of industry: scale, efficiency, and regulatory expertise.
By funding companies at critical stages of development, CIRM de-risks projects and enables the high-cost preclinical and clinical work that often deter private investment, especially for rare diseases.
This alignment with industry is not a mere side project; it is embedded in the structure of CIRM's new funding programs. The agency's Preclinical Development (PDEV) awards, which offer up to $13 million per project, are explicitly designed to help applicants—including biotech firms—navigate key milestones on the path to FDA approval 8 .
The table below illustrates the scope of CIRM's recent commitments to commercial entities, showcasing a focus on specific, unmet medical needs.
| Company/Institution | Project Focus | Award Amount | Development Stage |
|---|---|---|---|
| Amplo Biotechnology 2 | DOK7 Gene Therapy for Congenital Myasthenic Syndromes | $2,894,305 | Preclinical (CLIN1) |
| Tenaya Therapeutics 2 | TN-401 Gene Therapy for a Genetic Heart Condition (ARVC) | $8,000,000 | Clinical (CLIN2) |
| Navega Therapeutics 2 | Non-opioid Gene Therapy for Chronic Pain | $3,982,633 | Translational (TRAN1) |
| Iris Medicine, Inc. 5 9 | miRNA-based Genetic Medicine | $2,997,574 | Discovery (DISC0) |
| NysnoBio 2 | Parkin Gene Therapy for Parkinson's Disease | $2,450,510 | Translational (TRAN1) |
The story of Navega Therapeutics exemplifies the pipeline CIRM is building. The company first caught the agency's attention with promising early-stage research. This initial work was supported by a CIRM Discovery (DISC2) award, which funds early translational projects 2 .
With promising data in hand, Navega recently graduated to a larger $4 million Translational (TRAN1) award to advance its groundbreaking therapy for chronic pain 2 .
Chronic pain is a pervasive condition, often managed with opioids that carry a high risk of addiction. Navega's team sought a cure by targeting the root cause of certain inherited pain disorders: a overactive sodium channel gene called SCN9A 2 .
Researchers focused on the SCN9A gene, which encodes the Nav1.7 sodium channel, a key protein responsible for transmitting pain signals in the body 2 .
They developed a genetic therapy using an Adeno-associated virus (AAV) as a delivery vehicle. This virus was engineered not to edit the gene itself, but to carry a tool that would "silence" the SCN9A gene at the DNA level—an approach known as epigenetic editing 2 .
The potential therapy was tested in laboratory models that mimic human chronic pain conditions, such as Primary Erythromelalgia and Small Fiber Neuropathy 2 .
The experimental therapy demonstrated success in precisely targeting the Nav1.7 sodium channel, effectively reducing the pain signals 2 . This breakthrough is significant because it offers a potential one-time treatment for debilitating pain conditions for which current treatments are often ineffective. Most importantly, it provides a potential alternative to opioid-based pain management, which could revolutionize treatment for millions and reduce dependency on addictive pharmaceuticals 2 .
Key characteristics of Navega's epigenetic therapy for chronic pain based on CIRM-funded research. 2
| Therapeutic Candidate | Epigenetic gene therapy |
|---|---|
| Target Gene | SCN9A (encodes Nav1.7 sodium channel) |
| Mechanism of Action | AAV-delivered epigenetic silencer to regulate pain signal transmission |
| Target Indications | Primary Erythromelalgia, Small Fiber Neuropathy |
| Key Advantage | Potential one-time treatment; non-opioid, non-addictive |
| Development Stage | Translational Research |
The work at companies like Navega, Amplo, and NysnoBio relies on a suite of specialized tools. The table below outlines key reagents that power this research.
| Research Tool | Function in Therapy Development |
|---|---|
| Adeno-associated Virus (AAV) | A widely used viral vector engineered to safely deliver therapeutic genes to human cells without causing disease. 2 |
| Induced Pluripotent Stem Cells (iPSCs) | Adult cells reprogrammed to an embryonic-like state. They can be generated from patients and differentiated into various cell types (e.g., neurons, heart cells) for disease modeling and drug testing. 5 6 |
| Antisense Oligonucleotides | Short, synthetic nucleic acid molecules designed to bind to RNA and modify gene expression, used in therapies for diseases like spinocerebellar ataxia. 2 |
| Lentiviral Vectors | A type of virus used as a vehicle to introduce genetic material into cells, often used in ex vivo gene therapies where a patient's cells are modified outside the body. 2 |
| Human Cell Lines & Biobanks | Well-characterized human cells, derived from both healthy donors and patients with specific diseases, providing essential models for testing therapies. 6 |
CIRM's courtship of industry extends beyond direct grants. The agency is building a comprehensive ecosystem to make California the most attractive place for regenerative medicine companies to thrive.
CIRM has invested millions in building a "California Cell and Gene Therapy Manufacturing Network," with hubs at institutions like UCSF, UCLA, and Stanford 9 . This provides companies with critical infrastructure to produce clinical-grade therapies, a major bottleneck in the field.
The agency's new PDEV program explicitly prioritizes projects that have already engaged with the FDA and aims to guide them directly to an Investigational New Drug (IND) application—the key to starting human trials 8 .
The strategic direction of CIRM signals a maturation of the entire field of regenerative medicine. The agency's bet is that by acting as a strategic, deep-pocketed partner for biotech, it can dramatically accelerate the pace at which scientific discoveries become life-changing treatments.
"CIRM is a uniquely steadfast supporter of cell and gene therapies, removing roadblocks and creating efficiencies to ensure integration of new therapeutic modalities into the healthcare arsenal against human disease and suffering"
The hope for millions of patients with conditions from Parkinson's to chronic pain is that this targeted courtship of industry will finally deliver the cures that decades of basic science have promised.