Sangamo Therapeutics Inc. (NASDAQ:SGMO) initiated the trial phases for SB-913 ZFN and SB-318 ZFN last year. In particular, the studies were to determine the effectiveness of the therapies in treating Hunter Syndrome and Hurler Syndrome respectively. In January this year, the firm revealed that data from the studies is ready for presentation.

CHAMPIONS and EMPOWERS are in advanced stages

Notably, the first study, called CHAMPIONS, aimed to trial the clinical effectiveness of the “dose escalation of SB-913 ZFN-mediated in vivo human genome editing.” In particular, the study targeted patients suffering from MPS II otherwise called Hunter Syndrome. CHAMPIONS is a Phase 1/2 trial which, depending on the data collected, will transition into Phase 2.

The second study is also a Phase 1/2 trial that will test under, clinical conditions, the viability of SB-318 ZFN therapy. In particular, the therapy targets patients suffering from MPS I otherwise called Hurler Syndrome. Interestingly, Sangamo christened this study as “EMPOWERS.”

Notably, ZFN is a proprietary therapy where it edits genomes and inserts corrective genes. Interestingly, this technology targets the exact location in the patient’s DNA where there is a faulty gene. In particular, the procedure targets liver cells since they are responsible for the supply of iduronate 2-sulfatase (IDS) for MPS II. Further, the same cells produce alpha-L-iduronidase (IDUA) which is an enzyme that affects MPS I.

Acquisition of TxCell now complete

Even as Sangamo’s investors anticipate positive results from the trials, they can take heart regarding the firm’s growth strategy. Notably, Sangamo Therapeutics is not only developing drugs in the pipeline but is also acquiring businesses. Late last year, the firm revealed that the acquisition plans for TxCell were successful. As such, this marks the conclusion to a process that began in July 2018.

At the time, Sangamo agreed to buy a majority stake of the company in an all-cash deal. Notably, the agreement was that the acquisition would happen at a price of €2.58 for every single share purchased. As such, the whole transaction was to amount to about €72 million. After the transaction completed in December, Sandy Macrae, CEO of Sangamo said, “The acquisition of TxCell immediately positions Sangamo as the leader in CAR-Treg development.”

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