Biopharmconsortium Blog

Expert commentary from Haberman Associates biotechnology and pharmaceutical consulting.

Posts filed under: Stem Cells

Strimvelis (GSK2696273), a gene therapy for a deadly immunodeficiency in children, expected to reach the European market in mid-2016

TweetOur recent book-length report, Gene Therapy: Moving Toward Commercialization was published by Cambridge Healthtech Institute in November 2015. As indicated by its title, the report focuses on clinical-stage gene therapy programs that are aimed at commercialization, and the companies that are carrying out these programs. Until recently, gene therapy was thought of as a scientifically-premature…

Our New Year’s 2015 article: Notable researchers and breakthrough research of 2014

Tweet  As is their customary practice, both Nature and Science ran end-of-year specials. The Nature special (in their 18 December issue) is entitled “365 days: Nature’s 10. Ten people who mattered this year.” The Science special (in their 19 December issue) is entitled, as usual “2014 Breakthrough of the Year.” As is also usual, there…

Chemokine receptor inhibitors for prevention of cancer metastasis

TweetIn our October 31, 2013 blog article, we discussed recent structural studies of the chemokine receptors CCR5 and CXCR4. We discussed the implications of these studies for the treatment of HIV/AIDS, especially using the CCR5 inhibitor maraviroc (Pfizer’s Selzentry/Celsentri). As discussed in the article, researchers are utilizing the structural studies of CCR5 and CXCR4 to…

Japanese group to take induced pluripotent stem (iPS) cell-derived retinal cells into the clinic

Tweet  As reported in Nature News on 27 February 2013 ophthalmologist Masayo Takahashi M.D., Ph.D. and her colleagues at the RIKEN Center for Developmental Biology (Kobe, Japan), plan to submit an application to the Japanese health ministry for a clinical study of induced pluripotent stem cell (iPS)-derived cells. The researchers planned to submit their application…

Is gene therapy emerging from technological prematurity?

Tweet  [For updated information on gene therapy, please see our articles on this blog dated November 16, 2015 and November 23, 2015.] The idea of gene therapy has been around since at least the early 1970s. In 1972, an article by Theodore Friedmann and Richard Roblin advanced the concept of treating genetic diseases by replacing…

Obesity therapeutics revisited

Tweet  The CNS-targeting “Class of 2010” drugs We have not had an article on obesity therapeutics on this blog since February 1, 2011. At that time, we had an article entitled “That’s all, folks!”, complete with the old Warner Brothers Porky Pig graphic. As of that date, all three of the obesity drug candidates that…

“It’s not junk”–RaNA Therapeutics emerges from stealth mode with $20.7 million in venture funding-Part 2

Tweet  This is Part 2 of the article on RaNA Therapeutics that we began on March 29, 2012. Jeannie Lee’s research and RaNA’s technology platform Jeannie Lee’s laboratory focuses on the study of the mechanism of X-chromosome inactivation in mammals. In X-chromosome inactivation, one of the two copies of the X chromosome present in the…

“It’s not junk”–RaNA Therapeutics emerges from stealth mode with $20.7 million in venture funding-Part 1

Tweet    On January 18, 2012, start-up company RaNA Therapeutics (Cambridge, MA) emerged from stealth mode with 20.7 million in cash. The Series A venture funding was co-led by Atlas Venture, SR One, and Monsanto, with participation of Partners Innovation Fund. RaNA will work on developing a technology platform that involves targeting long noncoding RNA…

Preclinical-stage biotech Verastem goes public. Really‽

Tweet  On November 3, 2011, Cambridge MA biotech firm Verastem announced that it was filing a prospectus for an initial public offering (IPO). At that time, the company was 15 months old. Verastem is led by Christoph Westphal, MD, PhD, a founder and the former CEO of Sirtris and a veteran entrepreneur and venture capitalist….

The Big Pharma retreat from RNAi therapeutics continues

Tweet  On July 29, 2011, Merck announced that It was shutting down the San Francisco research laboratory that it had acquired as part of its $1.1 billion acquisition of therapeutic RNAi specialist company Sirna Therapeutics. This announcement was covered in a July 29, 2011 article in Xconomy, and in a news brief in the 4…