Our Science

We are developing platforms that can repair and control genes in cells or replace any cell in the body.

Our technologies are being developed in collaboration with leading academic laboratories and industry partners.

Scalable manufacturing with a focus on its cost and working to align with key stakeholders will enable broader patient access to our therapies.

Scientists have made tremendous progress over the past decade in understanding how to modulate genes and use them now to make transformative medicines. The first wave of these transformative medicines has recently launched or is on pace to reach patients soon.  However, the field is still in its infancy.

Assembling the right technologies is key to opening up therapeutic opportunities ranging from rare diseases caused by a single genetic mutation to acquired diseases that affect millions.

In our work, three aspirations guide us:

  1. Developing the ability to repair any cell in the body

  2. Developing the ability to replace any cell in the body

  3. Developing the technologies and processes to enable access to our therapies

Next

Pipeline


In Vivo Cell Engineering

Fusogen Technology

Product Candidate Cell Type Potential Indications

SG295 (CD8/CD19)

T cells

Oncology
• Non-Hodgkin lymphoma (NHL)
• Acute lymphoblastic leukemia (ALL)
• Chronic lymphocytic leukemia (CLL)


Pre-Clinical

SG239 (CD8/BCMA)

T cells

Oncology
Multiple myeloma


Pre-Clinical

SG242 (CD4/CD19)

T cells

Oncology
• Non-Hodgkin lymphoma (NHL)
• Acute lymphoblastic leukemia (ALL)
• Chronic lymphocytic leukemia (CLL)


Pre-Clinical

SG221 (CD4/BCMA)

T cells

Oncology
Multiple myeloma


Pre-Clinical

SG328

Hepatocytes

Liver-related genetic disorders
Ornithine transcarbamylase deficiency (OTC)


Pre-Clinical

SG418

Hematopoietic stem cells

Hemoglobinopathies
• Sickle cell disease
• Beta-thalassemia


Pre-Clinical

Ex Vivo Cell Engineering

Hypoimmune Technology: Donor-derived

Product Candidate Cell Type Potential Indications

SC291 (CD19)

T cells

Oncology
• Non-Hodgkin lymphoma (NHL)
• Acute lymphoblastic leukemia (ALL)
• Chronic lymphocytic leukemia (CLL)


Pre-Clinical

SC255 (BCMA)

T cells

Oncology
Multiple myeloma


Pre-Clinical

Hypoimmune Technology: Stem cell-derived

Product Candidate Cell Type Potential Indications

SC451

Beta cells

Diabetes
Type 1 diabetes


Pre-Clinical

Stem cell-derived (to migrate to Hypoimmune Technology)

Product Candidate Cell Type Potential Indications

SC379

Glial progenitor cells

Central nervous system (CNS)
• Huntington’s disease
• Pelizaeus-Merzbacher disease
• Secondary progressive multiple sclerosis


Pre-Clinical

SC187

Cardiomyocytes

Cardiovascular
Heart failure


Pre-Clinical