Therapeutic areas of focus

Our strategy is to retain and develop product candidates ourselves in certain therapeutic areas that meet key criteria including having breakthrough potential for unmet needs; where development and commercialization are financially and operationally feasible; and where we see synergies across our product candidates and therapeutic approaches.

We are focusing our proprietary pipeline on research and development of product candidates for inherited metabolic diseases, central nervous system diseases, and immunology.

Collaboration is critical to maximizing the value of our platform technology and has brought important funding and expertise to our research. We will continue to collaborate, especially in therapeutic areas that are highly competitive or that require special disease area expertise or resources.

Legend

Genome Editing

Gene Therapy

Cell Therapy

Gene Regulation

Inherited Metabolic Diseases

Approach:
Genome Editing

Program:
SB-318

Research

Preclinical

Phase 1/2

Approach:
Genome Editing

Program:
SB-913

Research

Preclinical

Phase 1/2

Approach:
Gene Therapy

Program:
ST-920

Research

Preclinical

Hematology

Approach:
Genome Editing

Program:
SB-FIX

Research

Preclinical

Phase 1/2

CNS Diseases

Approach:
Gene Regulation

Research

Preclinical

Immunology

Approach:
Cell Therapy

Program:
TX-200

Research

Preclinical