In The Spotlight: Sangamo Therapeutics

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(RTTNews.com) - Shares of Sangamo Therapeutics Inc. (SGMO) are down nearly 33% from their 52-week high to $18. Given the fact that the Company has potential clinical data readouts from 7 studies beginning in late summer of this year to 2019, it is worth keeping an eye on this stock.

Sangamo is a clinical stage biotechnology company focused on developing genomic therapies. A leader in the research and development of zinc finger proteins, or ZFPs, the Company uses its platform technologies in genome editing, gene therapy, gene regulation and cell therapy.

ZFP technology is designed to edit DNA, say by turning off or turning on genes for treating a disease. Zinc finger proteins are a class of naturally-occurring DNA binding proteins, and they are so named because they resemble a finger-like shape and include a zinc ion.

The Company's pipeline covers inherited metabolic diseases, hematology, CNS diseases, oncology and immunology.

Inherited metabolic diseases

There are three drug candidates in the inherited metabolic diseases space - SB-318 to treat Mucopolysaccharidosis Type I, or MPS I; SB-913 for the treatment of Mucopolysaccharidosis Type II, or MPS II, and ST-920 for Fabry disease.

-- SB-318 is under a Phase 1/2 clinical trial in adult subjects with MPS I. Initial data from this trial are expected this year.

Mucopolysaccharidosis refers to a group of inherited conditions in which the body is unable to properly breakdown the sugars due to a lack a specific protein called alpha-L iduronidase. Based on the signs, symptoms and the severity of the condition, Mucopolysaccharidosis is classified into various types like MPS I, MPS II, MPS III, MPS IV, MPS VI, MPS VII and MPS IX.

-- SB-913 is also under a Phase 1/2 clinical trial in subjects with MPS II, dubbed CHAMPIONS. Initial data from this study are expected in late summer of 2018. -- The Company expects to file an Investigational New Drug Application for ST-920 for Fabry disease this year.


The hematology assets include SB-525 for Hemophilia A; SB-FIX for Hemophilia B; ST-400 for Beta-thalassemia and BIVV-003 for sickle cell disease.

-- SB-525 is under a phase 1/2 clinical trial in patients with Hemophilia A, dubbed Alta. This investigational gene therapy is being developed as part of a global collaboration between Sangamo and Pfizer. Initial data from this trial are expected in late summer 2018.
-- SB-FIX is under a phase 1/2 clinical trial in patients with hemophilia B in the U.S. A phase I/2 clinical trial in the U.K. is expected to be initiated later this year.
-- ST-400 is under Phase 1/2 development in subjects with transfusion-dependent β-thalassemia (TDT).
-- BIVV-003, being developed for sickle cell disease, is expected to advance into clinical trials.

ST-400 and BIVV-003 are being developed as part of an exclusive worldwide collaboration between Sangamo and Bioverativ, a Sanofi Company.

CNS diseases

The drug candidates targeting the CNS diseases include ZFP-TFs and C9ORF72.

-- ZFP-TFs to downregulate the expression of tau, a protein associated with Alzheimer's disease and frontotemporal dementia (FTD), which are under preclinical testing. -- A gene therapy to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene. Sangamo and Pfizer are collaborating for the development of this potential gene therapy, which is under research stage. -- ZFP Therapeutic for patients with Huntington's disease, under research stage. Sangamo is collaborating with Shire AG to develop this therapeutic for Huntington's disease.


Sangamo has an agreement in place with Kite Pharma Inc., a wholly-owned subsidiary of Gilead Sciences Inc., for the research, development and commercialization of potential engineered cell therapies for cancer. The agreement became effective as recently as April 5, 2018.

Kite is responsible for all clinical development and commercialization of any resulting products out of the collaboration.


Undisclosed autoimmune disease targets.

Balance Sheet

In the first quarter ended March 31, 2018, Sangamo reported a consolidated net loss of $20.2 million or $0.23 per share compared to a net loss of $16.6 million, or $0.23 per share for the same period in 2017.

Revenues for the recent first quarter grew to $12.6 million from $3.4 million in the year-ago quarter. The Company generates revenue from its collaborations.

Sangamo ended March 31, 2018, with cash of $234.9 million. This amount does not include the $150 million upfront payment from the collaboration agreement with Kite, effective April 5th, or the approximately $216 million in net proceeds from the recent public offering of Sangamo's common stock, which closed on April 30th.

SGMO has traded in a range of $8.00 to $27.50 in the last 1 year. The stock closed Wednesday's (June 20) trading at $18.05, up 9.73%.

Read the original article on RTTNews (http://www.rttnews.com/2907162/in-the-spotlight-sangamo-therapeutics.aspx)

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