programs developed in collaborations

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

Hematology

Approach:
Gene Therapy

Program:
SB-525

Research

Preclinical

Phase 1/2

Partner:
Pfizer

Approach:
Cell Therapy

Program:
ST-400

Research

Preclinical

Phase 1/2

Partner:
Bioverativ

Approach:
Cell Therapy

Program:
BIVV-003

Research

Preclinical

Partner:
Bioverativ

CNS Diseases

Approach:
Gene Regulation

Research

Partner:
Pfizer

Approach:
Gene Regulation

Research

Partner:
Shire

Oncology

Approach:
Cell Therapy

Program:
Multiple

Research

Preclinical

Partner:
Kite-Gilead

Investigator Sponsored Clinical Research

Approach:
Cell Therapy

Program:
SB-728-T

Research

Preclinical

Phase 1/2

Approach:
Cell Therapy

Program:
SB-728-HSPC

Research

Preclinical

Phase 1/2