Analysts at JP Morgan just initiated coverage on this biotech with an “overweight” rating. And five of its scientists were also recognized by Thomson Reuters as being “Among Most Highly Cited Researchers Worldwide Of 2014”.
Sangamo Biosciences Inc. (SGMO)
from The Medical Technology Stock Letter
The conference call for Sangamo Biosciences Inc.’s (SGMO) second quarter results provided some updates on what to expect in the second half of 2014. SGMO will present immunological data from subjects who were treated in earlier studies of SB-728-T at the Conference on Cell and Gene Therapy for HIV Cure 2014 (Seattle, 8/26-27).
The company will also provide an update on previous Phase I/II trials at ICAAC in early September. SGMO’s cutting edge HIV program that represents a potential “functional cure” for the disease continues to provide solid proof-of-concept data that validates the company’s unique gene therapy/gene editing technology.
The HIV program is currently enrolling1 12 patients in a Phase II trial that will provide additional efficacy data and, importantly, will validate SGMO’s new delivery vehicle, mRNA, instead of the viral vector, AAV. The use of mRNA provides both process and cost saving advantages over viral delivery of the ZFNs, and will enable the potential for retreatment if needed. First-generation gene therapy approaches rely on random insertion of replacement genes and viral vectors to drive their expression, which if present in the wrong place, can drive the expression of oncogenes and conceivably cause cancer. In contrast, SGMO’s precise and highly targeted ZFNs are delivered using a non-viral method in which the targeted gene is specifically encoded as a messenger RNA. Thus, nothing is inserted into the genome which eliminates the risks of insertion and mutagenesis.
SGMO still uses AAV for delivery in its in vivo protein replacement platform, (IVPRP). This makes the recent appointment of Stewart Parker to its Board of Directors an important addition. She is the former CEO of Targeted Genetics, a pioneering gene therapy company focused on AAV. Stewart’s unique experience in gene therapy is directly applicable to many of the company’s programs that use AAV, including the aforementioned IVPRP. With gene therapy growing more in vogue as a biotech investment, in our view, it is only a matter of time until more investors gravitate towards SGMO’s second-generation gene therapy technology, which is strongly protected by IP and validated by major partners. SGMO is a BUY under 20 with a target price of 30.
John McCamant, The Medical Technology Stock Letter, www.bioinvest.com, August 7, 2014