Sangamo Biosciences, Inc. (NASDAQ:SGMO)

Sangamo is a gene therapy and human gene editing pioneer.

Founded in 1995, it’s one of the oldest biotechs in the gene editing business.

This means the company has spent a long time working out the kinks and fine-tuning its own unique gene editing platform, known as zinc finger nuclease (ZFN).

Last year, the company made history by launching the world’s first in vivo gene editing clinical trial.

Unlike other kinds of gene therapy, in vivo gene editing means actually fixing bad genes inside the body by adding corrections to existing nuclear DNA.

But SGMO has also become a gene therapy company — where a small piece of DNA is inserted into a cell in addition to existing material so that the cell can carry out its instructions.

This week, Sangamo announced promising news regarding one of its gene therapy programs for hemophilia A.

Many gene therapy companies are working on this bleeding disorder as well as its closely related cousin, hemophilia B.

In both cases, a miscoded gene blocks the production of a blood clotting protein necessary for life.

In the case of hemophilia A, the deficient protein is known as Factor VIII (antihemophilic factor).

Most gene therapy companies are working on hemophilia because it represents a “low-hanging fruit,” one of the easier targets for this new biotechnology — and one that would be worth many billions in annual sales.

But that doesn’t mean it’s been easy…

Getting a working Factor VIII gene into cells is tricky, requiring a careful design of viral vectors to do the job.

Sangamo may be on the right path with its hemophilia A gene therapy, SB-525.

A few days ago, the company and its Big Pharma partner Pfizer Inc. (PFE) presented some early data at a scientific conference.

Patients receiving the highest tested dose of SB-525 showed rapid, positive change.

Enough of their cells received the gene therapy to raise production of Factor VIII to normal levels and didn’t suffer the typical symptoms of hemophilia A, which involves bleeding.

This success is more than just a validation for Sangamo’s hemophilia program.

The high gene delivery validates the company’s gene delivery platform, which spans multiple indications in several different classes of gene-related disease.

Sangamo is still in early stages, but is well on its way to one day becoming a gene therapy powerhouse — if that’s allowed to happen…

Big Pharma and Big Biotech have recently shown a strong desire for acquiring gene therapy companies in order to take advantage of what this emerging biotechnology has to offer.