Our Top Pick for speculative investors has a powerful and broad, second-generation gene therapy technology platform that is supported by very strong intellectual property, explains John McCamant, editor of The Medical Technology Stock Letter.
The ability to either turn on or turn off individually selected genes is unique and provides significant potential for sound efficacy that is also very safe.
2015 is shaping up to be a watershed year for Sangamo Biosciences (SGMO); they should have proof-of-concept Phase II data mid-year from their HIV program that would position the company for a major partnership.
Additionally, they will file the first Investigational New Drug (IND) for their In Vivo Protein Replacement Platform (IVPRP) program, a cutting edge technology, which, in our view, is the crown jewel in SGMO’s platform.
IVPRP has the potential to elevate the valuation to a higher biotech tier as investors’ realize the company’s ability to potentially transform medicine with functional cures in previously thought of impossible-to-cure diseases.
Sangamo also had a very strong ASH (hematology) as they presented exciting preclinical data from IVPRP demonstrating the efficient production, secretion, and tissue uptake of enzymes that are deficient in the lysosomal storage disorders (LSDs), Hunter, and Hurler's disease.
In our view, IVPRP has the potential to be a truly disruptive technology as it can eliminate the requirement for repeated infusions of protein or enzyme replacement therapy (ERT) throughout the patient's life, which is the current standard of care for both hemophilia and LSDs.
SGMO’s stock has begun to attract more investors following the big move in Blue Bird (BLUE), which has a competing beta-thal program. SGMO is also developing a drug candidate for beta-thal with its collaborator Biogen (BIIB) and expects to file an IND in Q1/15.
Sangamo also presented some interesting preclinical data on a second generation CART program for cancer. The CART IPOs were the hottest class in 2014; SGMO has yet to be mentioned in the CART field, but in our view, it is only a matter of time. 2015 will be a busy year for SGMO.
