Monday, April 16, 2018

Kiwi Credibility

My colleague and coauthor, Bill Tomlinson, a professor at the Victoria University of Wellington Business School/Orauariki, New Zealand/Aotearoa, alerted me to the fact that our 2009 article, Patents and the Regress of Useful Arts, has been tabled by the Commerce Committee of the New Zealand Parliament/Pāremata Aotearoa (see here) and featured as an informational resource on the "Forum" page of The Pirate Party of New Zealand (see here).

Thursday, January 11, 2018

The Cas Against CRISPR

The realization that some diseases have genetic etiologies long inspired the hope that "correcting" mutant DNA sequences in patients could cure their diseases.  Recent progress in gene therapy has made this tantalizing possibility seem achievable, and precise and efficient gene editing methods using tools like CRISPR-Cas9 have accelerated progress considerably.  However, an important preprint article, entitled "Identification of Pre-Existing Adaptive Immunity to Cas9 Proteins in Humans", published by Charlesworth et al. on bioRχiv, has now cast a shadow on the efficacy of CRISPR-Cas9 in humans.  The Matt Porteus' research team, based at Stanford Medical School, appear to have discovered an immune response to CRISPR-Cas9:
The most widely used homologs of the Cas9 protein are derived from the bacteria Staphylococcus aureus (S. aureus) and Streptococcus pyogenes (S. pyogenes). Based on the fact that these two bacterial species cause infections in the human population at high frequencies, we looked for the presence of pre-existing adaptive immune responses to their respective Cas9 homologs, SaCas9 (S. aureus homolog of Cas9) and SpCas9 (S. pyogenes homolog of Cas9)...[Our results] demonstrate[s] that there are pre-existing humoral and cell-mediated adaptive immune responses to Cas9 in humans, a factor which must be taken into account as the CRISPR-Cas9 system moves forward into clinical trials. [Italics added for Linnaean emphasis.]
If humans, and, presumably, other mammals, possess potent immune responses to the Cas protein component of this powerful gene editing system, the therapeutic prospects for CRISPR-Cas9 may dim considerably beyond applications in isolated cells or tissues or patients having immature or suppressed immune responses.  After many more than two steps forward, therapeutic gene editing may be about to take at least one substantial step backward.

Sunday, December 31, 2017

A New Gene Therapy Vision

Vertebrates see by converting photons into electrical signals in the retina, which, in turn are interpreted by the brain into visual depictions of the environment.  Part of this phototransduction system relies on an enzyme called retinoid isomerohydrolase (or retinal pigment epithelium-specific 65 kDa protein ("RPE65")) to convert all-trans-retinyl esters into 11-cis-retinol in the smooth endoplasmic reticulum.  Healthy functioning of retinal pigment epithelium ("RPE"), a thin layer of cells that nurtures the retina, can become impaired when both RPE65 genes contain mutations that prevent normal enzymatic function.  In patients with Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy, this can lead to poor vision or even blindness.

The Food and Drug Adminstration ("FDA") announced, on December 19, 2017, its approval of a gene therapy method developed to improve, or even restore, healthy RPE65 enzyme function by introducing wild-type copies of RPE65 genes directly into RPE tissue.  The method involves surgical delivery of RPE65 genes incorporated into adeno-associated viruses.  Spark Therapeutics, the company marketing this gene therapy, calls its "drug" voretigene neparvovec-rzyl (brand-named LUXTURNA).

As the FDA announced, LUXTURNA "is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene."  In the FDA press release, FDA Commissioner, Scott Gottlieb, waxed enthusiastic, suggesting that
this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases. The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses[.]
After previous fatal missteps (e.g., see here), gene therapy is finally seeing the light of day.

Thursday, October 26, 2017

The True North Strong IP?

On October 26, 2017, Television Ontario ("TVO") broadcast a fascinating, wide-ranging discussion about intellectual property in Canada and other countries.  Here it is.

Wednesday, September 27, 2017

Amaizing Syngenta Settlement

I appeared on Bloomberg Radio on September 27, 2017, to discuss the ~$1,400,000,000 settlement reached between Syngenta and farmers allegedly harmed by the agrobiotechnology firm's decision to sell genetically-modified corn in the United States, which subsequently caused China to ban U.S. corn imports.  You may listen to the story here.

Monday, September 25, 2017

CRISPR Becomes Clearer

Recently, The Hastings Center invited me to write an article discussing current legal, policy, and ethical issues surrounding the gene editing revolution triggered by CRISPR-Cas9.  The result, "CRISPR Becomes Clearer", was published on September 20, 2017, in The Hastings Center Report, Volume 47, Issue 5 (September/October 2017).