Advancing Dynamic Cell and Gene Therapies for Difficult-to-Treat Diseases
| Program | Indication | Stage of Development | Market Opportunity |
|---|---|---|---|
| Regulator Dial™ | Rett Syndrome | Discovery | ~23,000 patients across United States, Japan, EU4 & UK1 |
| Regulator Dial™ | Solid Tumors Armored TIL Therapies | Discovery | ~1.9 million U.S. solid-tumor cases annually2 |
- DelveInsight. Rett Syndrome Epidemiology Forecast to 2034. Published 2025.
- American Cancer Society estimates for all cancers excluding lymphoma, myeloma and leukemia.
Regulator Dial
We engineer cell and gene therapies with our proprietary Regulator Dial technology, building a drug-responsive control system directly into the therapy. After administration, an oral medication provides the signal to regulate production of the therapeutic protein. This built-in control is designed to give clinicians greater flexibility to adjust the timing and level of therapeutic gene expression, with the potential to optimize treatment effects and manage toxicity.
Rett Syndrome
Rett syndrome is a severe genetic disorder that can impair communication, movement and other essential functions. Mutations in the MECP2 gene disrupt a protein critical to normal brain function. Gene therapy offers the potential to address this underlying cause, but controlling MeCP2 production is essential because excessive levels can also cause harm.
Through a research collaboration, we are exploring Regulator Dial-enabled gene therapy designed to restore functional MeCP2 while allowing its expression to be adjusted after administration. The goal is to support therapeutic levels of the protein while limiting excessive production.
Armored TIL Therapies for Solid Tumors
Tumor-infiltrating lymphocytes, or TILs, are immune cells collected from a patient’s tumor, expanded in a laboratory and returned to help fight the cancer. However, the environment inside solid tumors can suppress their activity. Engineering TILs to produce immune-stimulating proteins could strengthen their response, but excessive production of these proteins can cause toxicity.
Through a research collaboration, we are exploring armored TIL therapies engineered with Regulator Dial to control production of added immune-stimulating proteins. The goal is to support sustained cancer-fighting activity while giving clinicians a way to regulate protein production after administration.