Biopharmconsortium Blog

Expert commentary from Haberman Associates biotechnology and pharmaceutical consulting.

Posts filed under: Gene therapy

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…

Gene therapy for hemophilia—an update

Tweet  The boy pictured above is Tsarevich Alexei Nikolaevich of Russia, who lived between 1904 and 1918, and was the heir to the throne of Imperial Russia. He is arguably the most famous hemophiliac in history. Alexei suffered from hemophilia B, a form of hemophilia that was passed from Queen Victoria of Britain through two…

Can adoptive cellular immunotherapy successfully treat metastatic gastrointestinal cancers?

Tweet  On September 6, 2014, we published an article on this blog announcing the publication of our book-length report, Cancer Immunotherapy: Immune Checkpoint Inhibitors, Cancer Vaccines, and Adoptive T-cell Therapies, by Cambridge Healthtech Institute (CHI). In that article, we cited the example of the case of a woman with metastatic cholangiocarcinoma (bile-duct cancer), which typically…

Spark Therapeutics’ retinal disease gene therapy SPK-RPE65 may reach the U.S. market in 2017

Tweet  As we discussed in our November 16, 2015 article on this blog, Spark Therapeutics (Philadelphia, PA) recently announced positive top-line results from the Phase 3 pivotal trial of SPK-RPE65, a gene therapy for treatment of inherited retinal diseases (IRDs) caused by mutations in the gene for RPE65.  At a later scientific meeting, the company…

“Our Promise to Nicholas”, Batten disease, and gene therapy

Tweet  Russell’s Garden Center, on Route 20, a family-owned business established in 1876, is a unique Wayland MA institution. When you shop at Russell’s and approach the check-out counter with your plants, flowers, or other purchases, you will see a donation box for a rare-disease charity called “Our Promise to Nicholas Foundation”. This charity is…

Gene editing technology used to treat infant with leukemia

Tweet  In November 2015, the use of gene editing technology to treat an 11-month-old child with leukemia was reported in news articles in Nature and in Science. Because of the human-interest value of this story, it was also reported in Time magazine and in the New York Times. Data from this first-in-humans clinical use of…

Gene Therapy Report Published By CHI Insight Pharma Reports

Tweet  On November 6, 2015, Cambridge Healthtech Institute (CHI) announced the publication of a new book-length report, Gene Therapy: Moving Toward Commercialization, by Allan B. Haberman, Ph.D. As demonstrated by several late-breaking news items that appeared as our report was in the process of publication, gene therapy is a “hot”, fast-moving field. For example: On…

Breakthrough of the year 2013–Cancer Immunotherapy

TweetAs it does every year, Science published its “Breakthrough of the Year” for 2013 in the 20 December 2013 issue of the journal. Science chose cancer immunotherapy as its Breakthrough of the Year 2013. In its 20 December 2013 issue, Science published an editorial by its Editor-in-Chief, Marcia McNutt, Ph.D., entitled “Cancer Immunotherapy”. The same…

Bluebird Bio, Celgene, and Adoptive Immunotherapy for Cancer

TweetThe Biopharmconsortium Blog includes several articles that are–in whole or in part–about adoptive T-cell immunotherapy [or adoptive cell transfer (ACT)] for cancer. In particular, we have produced two blog articles that discuss the Novartis/University of Pennsylvania (Penn) collaboration, which is aimed at finally commercializing adoptive immunotherapy for cancer. “Is Novartis Building A Viable Business Model…

Determining the molecular cause of a disease does not necessarily enable researchers to develop disease-modifying drugs

Tweet  A major objective of research in genomics is to identify mutations that cause genetic diseases. However, doing so does not necessarily directly enable researchers to discover and develop drugs to treat these diseases. Two examples of genetic diseases whose causes were identified decades ago, without directly enabling the development of any disease-modifying drug, are…