Oct 11, 2016: Allan B. Haberman, Ph.D. was quoted in an article in the October 2016 issue of Nature Biotechnology, entitled “Early clinical data raise the bar for hemophilia gene therapies”, by Elie Dolgin.

The article is an update on progress in development of gene therapies for hemophilia A and B, based in part on presentations at the World Federation of Hemophilia 2016 World Congress (Orlando, FL, July 24-28).

Notably, the article included discussions of progress in development of gene therapies for hemophilia A, especially by BioMarin Pharmaceuticals (San Rafael, CA). Commercial development of gene therapies for hemophilia B is far ahead of that for hemophilia A. This is because the gene coding for factor IX (mutated in hemophilia B) is smaller and easier to insert into commonly used gene-therapy vectors than the factor VIII gene mutated in hemophilia A. Nevertheless, BioMarin’s reported promising results from the Phase 1/2 trial of its hemophilia A gene therapy at the July 2016 meeting.

Haberman Associates has produced two publications that address gene therapy for hemophilia:

Because progress in gene therapy for hemophilia B is far ahead of that for hemophilia A, these materials by necessity emphasize hemophilia B. However, Chapter 5 of our book-length report includes strategies by which researchers and companies are now developing gene therapies for hemophilia A.

The Nature Biotechnology article also includes a discussion of a novel experimental treatment for hemophilia A that is not a gene therapy: Roche/Chugai’s emicizumab, a bispecific factor VIII mimic. This promising, but early-stage product (Phase 1/2) must be given to patients periodically—perhaps monthly. In contrast, a successful gene therapy may be active over the life of the patient, or at least over many years. Nevertheless, the half-life of emicizumab is much longer than that of current treatments.

As we say in the article, the data on these therapies is early-stage, and it is not known how long the therapeutic effects are going to last. However, the results are promising, and at least some commentators believe that hemophilia could prove to be the most competitive gene therapy race to date.

Read the full Nature Biotechnology article. (Subscription required).

Nov. 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.

This report looks at how researchers have been working to overcome critical barriers to development of safe and efficacious gene therapy, from 1990 to 2015. It then focuses on clinical-stage gene therapy programs that are aimed at commercialization, and the companies that are carrying out these programs. A major theme of the report is whether gene therapy can attain commercial success by the early-to-mid 2020s, which types of gene therapy programs have the greatest likelihood of success, and what hurdles might stand in the way of clinical and commercial success of gene therapy development.

Topics covered in the report:

  • Development of improved vectors (integrating and non-integrating vectors)
  • Gene therapy for ophthalmological diseases
  • Gene therapy for hemophilias and other rare diseases
  • Gene therapy for more common diseases (e.g., Parkinson’s disease, osteoarthritis, and heart failure)
  • Companies whose central technology platform involves ex vivo gene therapy
  • Gene editing technology
  • Outlook for gene therapy
  • Outlook for eight gene therapy products expected to reach the market before 2020

The report also includes:

  • An expert interview with Sam Wadsworth, Ph.D., the Chief Scientific Officer of Dimension Therapeutics and former Head of Gene Therapy R&D at Genzyme
  • Survey data from 88 researchers involved in gene therapy
  • Companies profiled: uniQure, Spark Therapeutics, GenSight, Dimension Therapeutics, Voyager Therapeutics, Oxford BioMedica, GeneQuine, bluebird, Juno Therapeutics, Kite Pharma, Editas, and others.

For more information on the report, or to order it, see the CHI Insight Pharma Reports website.

Sept. 9, 2014: Cambridge Healthtech Institute (CHI) announced the publication of a new book-length report, Cancer Immunotherapy: Immune Checkpoint Inhibitors, Cancer Vaccines, and Adoptive T-cell Therapies, by Allan B. Haberman, Ph.D.

This report focuses on the three principal types of therapeutics that have become the major focuses of research and development in immuno-oncology in recent years:

  • Checkpoint inhibitors
  • Therapeutic anticancer vaccines
  • Adoptive cellular immunotherapy

The discussions of these three types of therapeutics are coupled with an in-depth introduction and history as well as data for market outlook.

Also featured are three expert interviews and a survey of individuals working in immuno-oncology R&D, which focuses on market outlook, and portrays industry opinions and perspectives.

The report is designed to enable readers to understand current and future developments in immuno-oncology. It is also designed to inform the decisions of leaders in companies and in academic groups that are working in areas that relate to cancer R&D and treatment.

For more information on the report, or to order it, see the CHI Insight Pharma Reports website.

Mar. 11, 2013: Allan B. Haberman, Ph.D. was quoted in an article in The Pink Sheet entitled “Cystic Fibrosis Market Snapshot: Disease-Modifying Drugs Elusive 24 Years After Discovery Of Root Cause”. The article focused on the newly-approved disease modifying drug ivacaftor (Vertex’ Kalydeco), as well as drug discovery and development programs in cystic fibrosis at Vertex, PTC Therapeutics, Proteostasis Therapeutics, Pfizer, and Genzyme. It also discussed pipeline products aimed at treating or preventing life-threatening infections in cystic fibrosis patients at such companies as KaloBios, Insmed, and Savara.

Read full article.  (subscription required)