Sandy Macrae at Rare Disease Day at NIH 2018 (cropped)

Sangamo Therapeutics, Inc. (previously known as Sangamo Biosciences, Inc.) is an American biotechnology company based in

Brisbane Brisbane ( ) is the capital and most populous city of the Australian state of Queensland, and the third-most populous city in Australia and Oceania, with a population of approximately 2.6 million. Brisbane lies at the centre of the Sou ...

, California. It applies cell and gene therapy to combat haemophilia and other genetic diseases.

History

The company was founded in 1995 in Richmond, California. It was originally known as Sangamo Biosciences, Inc. before changing names in 2017. In September 2018, it had 182 employees. Sandy Macrae is the president. In 2018, Edward Rebar became the senior vice president and chief technology officer of Sangamo.

Research

Sangamo applies technology to treat haemophilia B and

lysosomal storage disease Lysosomal storage diseases (LSDs; ) are a group of over 70 rare inherited metabolic disorders that result from defects in lysosomal function. Lysosomes are sacs of enzymes within cells that digest large molecules and pass the fragments on to other ...

s including mucopolysaccharidosis type I (Hurler Syndrome) and mucopolysaccharidosis type II (Hunter Syndrome). The

FDA The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food s ...

granted Sangamo

fast track designation Fast track is a designation by the United States Food and Drug Administration (FDA) of an investigational drug for expedited review to facilitate development of drugs that treat a serious or life-threatening condition and fill an unmet medical ne ...

for SB-525, a gene therapy candidate for haemophilia A. In its partnership with Pfizer in 2017, Sangamo uses

Bioverativ Bioverativ Inc. is an American multinational biotechnology company that specializes in the discovery, development, and delivery of therapies for the treatment of haemophilia. Bioverativ competes with Baxalta (acquired by Shire Plc in 2016), Pfiz ...

in hemoglobinopathies such as

beta thalassemia Beta thalassemias (β thalassemias) are a group of inherited blood disorders. They are forms of thalassemia caused by reduced or absent synthesis of the beta chains of hemoglobin that result in variable outcomes ranging from severe anemia to cl ...

and

sickle cell disease Sickle cell disease (SCD) is a group of blood disorders typically inherited from a person's parents. The most common type is known as sickle cell anaemia. It results in an abnormality in the oxygen-carrying protein haemoglobin found in red blo ...

. It is also developing

zinc finger A zinc finger is a small protein structural motif that is characterized by the coordination of one or more zinc ions (Zn2+) in order to stabilize the fold. It was originally coined to describe the finger-like appearance of a hypothesized struc ...

gene editing technology. In February 2019, medical scientists, working with Sangamo Therapeutics, announced the first ever "in body" human gene editing therapy to permanently alter DNA - in a patient with

Hunter syndrome Hunter syndrome, or mucopolysaccharidosis type II (MPS II), is a rare genetic disorder in which large sugar molecules called glycosaminoglycans (or GAGs or mucopolysaccharides) build up in body tissues. It is a form of lysosomal storage disease. ...

. clinical trials by Sangamo involving gene editing using

zinc finger nuclease Zinc-finger nucleases (ZFNs) are artificial restriction enzymes generated by fusing a zinc finger DNA-binding domain to a DNA-cleavage domain. Zinc finger domains can be engineered to target specific desired DNA sequences and this enables zin ...

were ongoing.

Clinical trials

Sangamo's programs are a mix of wholly owned and partnered; major partners include Pfizer,

Biogen Biogen Inc. is an American multinational biotechnology company based in Cambridge, Massachusetts, specializing in the discovery, development, and delivery of therapies for the treatment of neurological diseases to patients worldwide. History ...

Sanofi Sanofi S.A. is a French multinational pharmaceutical and healthcare company headquartered in Paris, France. Originally, the corporation was established in 1973 and merged with Synthélabo in 1999 to form Sanofi-Synthélabo. In 2004, Sanofi-Sy ...

, Takeda, and

Kite A kite is a tethered heavier than air flight, heavier-than-air or lighter-than-air craft with wing surfaces that react against the air to create Lift (force), lift and Drag (physics), drag forces. A kite consists of wings, tethers and anchors. ...

Gilead Gilead or Gilad (; he, גִּלְעָד ''Gīləʿāḏ'', ar, جلعاد, Ǧalʻād, Jalaad) is the ancient, historic, biblical name of the mountainous northern part of the region of Transjordan.''Easton's Bible Dictionary'Galeed''/ref> ...

company). As of 2020 Sangamo had the following clinical trials underway in the US: * Phase 1/2 Thales study in

* Phase 1/2 STAAR study in

Fabry disease Fabry disease, also known as Anderson–Fabry disease, is a rare genetic disease that can affect many parts of the body, including the kidneys, heart, and skin. Fabry disease is one of a group of conditions known as lysosomal storage diseases. T ...

* Phase 1/2 PRECIZN-1 study in

* Phase 1/2 CHAMPIONS study in MPS II * Phase 1/2 EMPOWERS study in MPS I * Phase 1/2 Alta study in hemophilia A * Phase 1 FIXTENDZ study in

hemophilia B Haemophilia B, also spelled hemophilia B, is a blood clotting disorder causing easy bruising and bleeding due to an inherited mutation of the gene for factor IX, and resulting in a deficiency of factor IX. It is less common than factor VIII def ...

In February 2023, Sangamo Therapeutics announced that it would stop the late-stage development for the Phase 1/2 PRECIZN-1 study of BIVV003 its sickle cell drug.

References

External links

* {{DEFAULTSORT:Sangamo Therapeutics Companies based in San Mateo County, California Biotechnology companies of the United States 1995 establishments in California Orphan drug companies Biotechnology companies established in 1995 Health care companies based in California

History

Research

Clinical trials

See also

References

External links