Investigating future therapies

Our scientists are committed to developing new genomic medicines for patients with serious diseases that are not well managed with current treatments, where our suite of technologies may be able to make a difference.

Developing genomic medicines requires years of laboratory research and clinical testing to evaluate safety and efficacy. Volunteers who enroll in clinical trials help Sangamo researchers understand the potential benefits and risks of these investigational therapies in humans. Investigational therapies must pass through various phases of clinical research before they can be approved for medicinal use.

Sangamo is conducting several clinical trials to evaluate the safety, tolerability, and potential efficacy of its genomic medicines. Please visit the links below to learn more about our clinical trials.

CURRENTLY RECRUITING PARTICIPANTS

 

Hemophilia A: AFFINE Study (Pfizer-led trial)

Pfizer is conducting a Phase 3 study to evaluate the clinical efficacy and safety of giroctocogene fitelparvovec gene therapy in adult male participants with moderately severe or severe hemophilia A. For more information, please visit https://clinicaltrials.gov/ct2/show/NCT04370054.

Fabry Disease: STAAR Study

We are conducting a Phase 1/2 clinical trial to evaluate the safety and tolerability of ST-920 gene therapy in Fabry disease. Please visit our study website at https://staarclinicalstudy.com or https://clinicaltrials.gov/ct2/show/NCT04046224.

For more information, check out the STAAR Clinical Trial Factsheet to learn about Fabry disease, ST-920 and the study design.

Sickle Cell Disease: PRECIZN-1 (Sanofi-led trial)

Sanofi is conducting a Phase 1/2 clinical trial to evaluate the safety, tolerability, and efficacy of BIVV003 ex vivo gene-edited cell therapy in severe Sickle Cell Disease. For more information, please visit: https://clinicaltrials.gov/ct2/show/NCT03653247

ONGOING STUDIES, NOT CURRENTLY RECRUITING

 

Beta Thalassemia: Thales Study

A Phase 1/2 clinical trial evaluating the safety, tolerability, and efficacy of ST-400 ex vivo gene-edited cell therapy in transfusion-dependent beta thalassemia is ongoing and no longer recruiting. For more information, please visit https://clinicaltrials.gov/ct2/show/NCT03432364.

MPS II: CHAMPIONS Study

A Phase 1/2 clinical trial evaluating the safety, tolerability, and effect on IDS enzyme activity of SB-913 in vivo genome editing candidate is ongoing and no longer recruiting. For more information, please visit: https://clinicaltrials.gov/ct2/show/NCT03041324

MPS I: EMPOWERS Study

A Phase 1/2 clinical trial evaluating the safety and tolerability of SB-318 in vivo genome editing candidate is ongoing and no longer recruiting. For more information, please visit: https://clinicaltrials.gov/ct2/show/NCT02702115.

Hemophilia A: Alta Study

A Phase 1/2 clinical trial evaluating the safety, tolerability, and time-course profile of FVIII activity levels of SB-525 gene therapy in hemophilia A is ongoing and no longer recruiting. For more information, please visit: https://clinicaltrials.gov/ct2/show/NCT03061201

Hemophilia B: FIXTENDZ Study

A Phase 1/2 clinical trial evaluating the safety, tolerability, and effect on FIX antigen and activity levels of SB-FIX in vivo genome editing candidate is ongoing and no longer recruiting. For more information, please visit: https://clinicaltrials.gov/ct2/show/NCT02695160.

HIV/AIDS: SB-728mR-HSPC

An investigator sponsored Phase 1/2 clinical trial evaluating the safety and feasibility of administering SB-728mR-HSPC after conditioning with busulfan is ongoing and no longer recruiting. For more information, please visit: https://clinicaltrials.gov/ct2/show/NCT02500849