Frequently asked questions

23andMe Therapeutics is conducting drug discovery and development from target identification through clinical stages, using  genetic insights from 23andMe’s proprietary database as a foundation; we believe this approach - starting with human genetic evidence - is a far more efficient way to develop novel therapeutics.

The Therapeutics group is helmed by an accomplished leadership team with a wealth of experience in target discovery, data analytics, biostatistics, biology, and clinical development.
By studying genetics data at scale, we can derive new insights into human biology, including how our genetics impact disease.

Our scientists study the aggregate, de-identified genetics alongside more than 4 billion health survey answers from millions of consented research participants.

Analyzing both genetic data and health survey responses, 23andMe identifies unique, disease-specific genetic “signatures” to pinpoint areas of the genome that may be promising for therapeutic intervention.

These signatures are more likely to be recognized by 23andMe due to the enormous size of the company’s genomic and health survey databases.

Studies have shown that therapeutic programs with a human genetic foundation are more than twice as likely to succeed*.

*Nelson et al., 2015 (Nature Genetics), King et al., 2019 (PLOS Genetics)

23ME-00610 (or “23ME’610”) is a high-affinity humanized monoclonal antibody that is designed to interfere with the ability of CD200R1 to interact with CD200 found on cancer cells. Preclinical data indicate that this mechanism has the potential to reinvigorate tumor-exhausted T-cells and myeloid cells to restore their ability to kill cancer cells.

23ME'610 is 23andMe’s first wholly-owned immuno-oncology antibody to enter the clinic and is part of the “P006” program.

Many cancers are able to escape the immune system, allowing them to grow in the body unchecked. Therefore, drugs that enhance immune-cell activity, or the ability of immune cells to recognize cancer cells, have the potential to be powerful anti-cancer therapies. CD200R1 is a cell surface receptor protein that is mostly expressed on human immune cells, specifically cancer-fighting T cells and myeloid cells. Tumor cells can express CD200, the only known binding partner for CD200R1, and use this regulatory protein to turn off the activity of T cells. A drug that blocks the ability of CD200 to bind to CD200R1 may activate T cells and enhance their ability to kill cancer cells.

23andMe analyzed its proprietary genetic and health survey database to identify an immuno-oncology genetic signature, which defines areas of the genome that have opposing associations with cancer and autoimmune diseases. Using this approach, 23andMe scientists discovered that three components of the CD200R1 pathway exhibit an immuno-oncology genetic signature, including the CD200R1 receptor, the CD200 ligand, and DOK2, a mediator of downstream signaling from CD200R1. Following this genetic insight, 23andMe subsequently generated data consistent with CD200R1’s role in inhibiting anti-cancer responses in immune cells.

The Phase 2a portion of 23andMe’s Phase 1/2a study of 23ME-00610, an investigational antibody targeting CD200R1 in patients with advanced solid malignancies, is currently underway. 23andMe announced the first patient dosed in the Phase 2a portion of the study in February 2023. (NCT05199272)

The Phase 2a portion of the study will evaluate the anti-cancer activity of 23ME-00610 in specific tumor indications, which include clear cell renal cell carcinoma; epithelial ovarian, fallopian tube or primary peritoneal carcinoma; neuroendocrine cancers including small cell lung cancer; and microsatellite instability-high (MSI-H) or tumor mutational burden-high (TMB-H) cancers that have progressed on standard therapies. A cohort of adolescents with locally advanced unresectable, or metastatic solid malignancies will also be enrolled.

The Phase 2a component will include assessment of objective response rate (ORR), progression-free survival (PFS) and overall survival (OS) in the expansion cohorts and will further characterize the safety, tolerability, pharmacokinetic and pharmacodynamic profile of 23ME-00610.

23andMe announced a poster presentation at the American Association of Cancer Research Annual Meeting 2023 that will disclose safety and pharmacokinetic data for 23ME-00610 for the phase 1 portion of the study.

The 23ME-00610 first-in-human dose escalation phase successfully identified a dose level and schedule for the Phase 2a portion of this study.