Gene Therapy Experiencing a Revival

    By James DeGiulio --

After over a decade of suspicion following the death of an 18-year-old patient, gene therapy is experiencing a revival among scientists and drugmakers, according to a recent Bloomberg report ("'Dead as Doornail' Gene Therapy Revival Spurs Genzyme").  Since the patient's death in 1999, scientists have linked many of the safety problems with gene therapy to the modified adenovirus vectors used to deliver the gene.  Current trials utilize a new generation of gene delivery vectors, such as adeno-associated virus (AAV), which trigger a milder immune response than the adenovirus used in previous studies and are consequently much safer.

Recent successes have rejuvenated the gene therapy field.  The Bloomberg report highlights Genzyme, which has invested more than $200 million over the past 10 years on gene therapy technology.  Genzyme's recent Parkinson's disease trial showed a dopamine gene injected into patients' brains effectively reached the targeted nerve cells in the region of the brain controlling movement.  This is an important discovery because efficient gene delivery is a major limitation of gene therapy.  Genzyme is preparing to test the therapy in a larger trial using AAV that could begin next year.

Other recent successes have been reported in high impact factor journals.  The November 6 issue of Science contained a report that showed brain damage in two 7-year-olds with an inherited condition called adrenoleukodystrophy was halted or reversed using gene therapy ("Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy").  In this study, doctors removed the patients' blood-forming stem cells, inserted gene-bearing viruses into them, and reinfused the patients with the modified cells.

The November 7 issue of The Lancet reported that delivery of a therapeutic gene to patients with Leber's congenital amaurosis resulted in significant vision recovery, with the most improvement noted in children ("Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial").  In this study, genes were delivered directly to target cells using AAV virus injected into patients.

Large drug companies are forming partnerships to further gene therapy development.  In January, Novartis licensed gene treatments for hearing loss and balance disorders from GenVec in a deal worth up to $213 million.  In February, startup Tacere Therapeutics announced that Pfizer would license its hepatitis C treatment in an agreement valued at as much as $145 million.

Other trends show the industry's renewed interest in gene therapy.  According to the FDA, there are currently 354 U.S. gene therapy studies under way, up from only 116 worldwide in 2008.  A Jain Pharmabiotech report released this month indicates that a total of 189 companies are currently developing gene therapies, up from only 44 in 1995.

However, despite the recent scientific successes, gene therapy is unlikely to produce revenue for any U.S. company for several years, as no gene therapies have been approved for actual medical use.  Only China has approved gene therapies for two types of cancers.  Nonetheless, there is no shortage of economic optimism.  An October 2008 report from Global Industry Analysts predicts gene therapy products will have revenue of $465 million in 2015.

James DeGiulio has a doctorate in molecular biology and genetics from Northwestern University and is a third-year law student at the Northwestern University School of Law.  Dr. DeGiulio was a member of MBHB's 2009 class of summer associates, and he can be contacted at [email protected].

Comments

[Gena777]

Despite its early setbacks, I predict that gene therapy is an area that will soon yield highly valuable patents. Thank you for this item; I hope you'll keep us up-to-date on future developments.
http://www.industryweek.com/articles/patent_enforcement_21538.aspx?SectionID=2

[Susan London]

I have a rudimentry knowledge of spelling and medical. I believe that gene therapy is possible thru various methods including training the mind

Patents are interesting would like to know more. I have had this in my thoughts for some time.

Susan London
512-696-3619

[Rob]

Gene therapy is one of the areas i see a massive growth of patents happening over the next 10 years.