What rare diseases does Sangamo Therapeutics focus on?

Sangamo Therapeutics has active programs across 7 rare diseases: Amyotrophic Lateral Sclerosis, Fabry Disease, Hemophilia B, Hunter Syndrome, Mucopolysaccharidosis Type I, Sickle Cell Disease, Thalassemia. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

Is Sangamo Therapeutics focused exclusively on rare diseases?

Yes. Sangamo Therapeutics is a rare disease specialist headquartered in Brisbane, United States, founded in 1995. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Amyotrophic Lateral Sclerosis, Fabry Disease, Hemophilia B among others.

How can I find Sangamo Therapeutics clinical trials for rare diseases?

This page shows all active and recruiting clinical trials sponsored by Sangamo Therapeutics, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.

How do I enroll in a Sangamo Therapeutics clinical trial?

To explore enrollment, find a recruiting trial on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.

What FDA-approved rare disease drugs does Sangamo Therapeutics have?

The FDA-Approved Drugs section on this page lists all Sangamo Therapeutics drugs with FDA approval that are relevant to rare diseases, including brand names, generic names, approval dates, and the rare conditions each drug treats. This data comes from the FDA's openFDA database and is updated automatically.

When was Sangamo Therapeutics founded and where is it based?

Sangamo Therapeutics was founded in 1995 and is headquartered in Brisbane, United States. The company has been active for 31 years. It trades publicly under the ticker symbol SGMO. As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.

Sangamo Therapeutics

Sangamo Therapeutics works on 7 rare diseases tracked on Trial Friend, including Amyotrophic Lateral Sclerosis, Fabry Disease, Hemophilia B and 4 more, with 5 recruiting clinical trials.

Sangamo Therapeutics develops gene therapies using its zinc finger nuclease (ZFN) platform. On June 23, 2026, Sangamo filed for Chapter 11 bankruptcy protection (District of Delaware) and entered stalking-horse asset-purchase agreements. Following a court-supervised auction, Sangamo completed the sale of its platform technologies to Eli Lilly on September 4, 2026 for $50 million in cash, covering its AAV capsid engineering platform (including the STAC-BBB capsid), its zinc finger protein platform, its Modular Integrase genome editing platform, and its ST-506 prion disease program. Its Fabry disease program ST-920 (isaralgagene civaparvovec) was sold to PTC Therapeutics, which outbid stalking-horse bidder Astellas at the court-supervised auction and completed the acquisition on September 21, 2026 for $111 million in cash plus up to $100 million in FDA-approval milestones. PTC expects to complete the rolling BLA (accelerated approval pathway) in Q4 2026. Sangamo's common stock was suspended from Nasdaq on May 5, 2026 and now trades on the OTCID Basic Market under the symbol SGMOQ.

Type

Rare Disease Specialist

Ticker

SGMO

Headquarters

Brisbane, United States

Founded

1995

Website

sangamo.com

Active Rare Disease Trials

Rare Diseases in Portfolio

Years Active

Sangamo Therapeutics Drug Pipeline

Sangamo Therapeutics has 5 active clinical trials across 3 development stages, with 5 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.

Note: This pipeline includes all of Sangamo Therapeutics's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.

Understand Sangamo Therapeutics's pipeline

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Early Phase / Phase 11 trial

HIV

Recruiting

Phase 21 trial

Chronic Neuropathic Pain

Recruiting

Other3 trials

Fabry Disease+1 more

Recruiting

Kidney Transplant Rejection+1 more

Recruiting

Blood and Lymphatic Diseases

Recruiting

Sangamo Therapeutics Clinical Trials (5)

Active and recruiting clinical trials sponsored by Sangamo Therapeutics, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.

Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.

Ask about Sangamo Therapeutics's trials

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ACTIVE NOT RECRUITINGRecently updatedNCT05039866

Long-Term Follow-up of Subjects Who Were Treated With ST-920

Intervention: ST-920

Fabry Disease Fabry Disease, Cardiac Variant

Long-term follow-up of subjects who received ST-920 in a previous trial (ST-920-201) and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 5 years following ST-920 infusion.

Ages 18 Years+13 locations

View full study

RECRUITINGPHASE1, PHASE2Updated a few months agoNCT06980948

Safety and Tolerability Study of ST-503 for Refractory Pain Due to Peripheral Neuropathy (Small Fiber Predominant, SFN)

Intervention: ST-503, Sham (No Treatment)

Chronic Neuropathic Pain

This research is being done to study a possible treatment for refractory pain due to small fiber neuropathy (SFN). ST-503 is intended to deliver a modified copy of the gene which will ideally repress Nav1.7 tissue-related pain signals reaching the brain, which should reduce the refractory pain due to small fiber neuropathy (SFN).

Ages 18 Years+11 locations

View full study

ACTIVE NOT RECRUITINGNo updates in a whileNCT05987527

Long-Term Follow-Up of TX200-TR101 (STEADFAST Long Term)

Intervention: Blood sample

Kidney Transplant RejectionEnd Stage Renal Disease

This long-term follow-up study is being conducted to collect long-term (up to 15 years post-infusion) safety and tolerability data from subjects enrolled in studies evaluating TX200-TR101.

Ages 18 Years - 72 Years5 locations

View full study

ACTIVE NOT RECRUITINGNo updates in a whileNCT05145062

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

Intervention: BIVV003, ST-400

Blood and Lymphatic Diseases

Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: * Long-term efficacy of the biological treatment effect of BIVV003 in SCD * Long-term efficacy of the clinical treatment effect of BIVV003 on SCD-related clinical events * Long-term efficacy of the biological treatment effect of ST-400 in TDT * Long-term efficacy of the clinical treatment effect of ST-400 in TDT

Ages 18 Years - 45 Years7 locations

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ACTIVE NOT RECRUITINGPHASE1No updates in a whileNCT02500849

Safety Study of Zinc Finger Nuclease CCR5-modified Hematopoietic Stem/Progenitor Cells in HIV-1 Infected Patients

Intervention: SB-728mR-HSPC Infusion 3 days following busulfan conditioning

HIV

The purpose of the study is to evaluate the safety and feasibility of administering SB-728mR-HSPC after conditioning with busulfan.

Ages 18 Years - 75 Years5 locations

View full study

FDA-Approved Drugs

No FDA-approved drugs found for this company at this time.

Sangamo Therapeutics Trial Locations

Sangamo Therapeutics clinical trials are running at 41 sites in 7 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.

United States

31▼

United Kingdom

3▼

Netherlands

3▼

Australia

1▼

Canada

1▼

Germany

1▼

Belgium

1▼

Rare Disease Focus Areas (7)

Diseases targeted by Sangamo Therapeutics's clinical trial and drug development programs

Amyotrophic Lateral SclerosisNeurological & Neuromuscular

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord. This causes progressive weakness and paralysis of voluntary muscles whil...

Prevalence: About 5,000 new cases per year in the U.S.; approximately 16,000 Americans living with ALS at any given time

Fabry DiseaseMetabolic & Lysosomal

Fabry disease is a rare inherited lysosomal storage disorder where a missing enzyme causes fatty substances called globotriaosylceramide to accumulate in cells throughout the body. This buildup damage...

Prevalence: 1 in 40,000 to 60,000 males; higher in females when accounting for carrier status

Hemophilia BBlood & Immune

Hemophilia B is an X-linked bleeding disorder caused by deficiency or dysfunction of clotting factor IX. Clinically indistinguishable from Hemophilia A, the key difference is factor type. Modern facto...

Prevalence: 1 in 25,000 to 33,000 males; very rare in females

Hunter SyndromeMetabolic & Lysosomal

Hunter syndrome is an X-linked lysosomal storage disorder caused by deficiency of iduronate-2-sulfatase enzyme, leading to accumulation of glycosaminoglycans in cells and tissues. This multisystem dis...

Prevalence: 1 in 100,000 to 150,000 males; females with disease are rare

Mucopolysaccharidosis Type IMetabolic & Lysosomal

Mucopolysaccharidosis Type I (MPS I) is a lysosomal storage disorder caused by deficiency of the enzyme alpha-L-iduronidase, which breaks down glycosaminoglycans (GAGs). This results in accumulation o...

Prevalence: Approximately 1 per 100,000 live births; Hurler form is the most common phenotype

Sickle Cell DiseaseBlood & Immune

Sickle cell disease is an inherited blood disorder where hemoglobin polymerizes under low oxygen, causing red blood cells to become rigid, sickle-shaped, and prone to hemolysis and vaso-occlusion. Thi...

Prevalence: About 100,000 Americans with SCD; 1 in 13 African-American births; 1 in 36 Hispanic-American births