Issues to Watch

Designer Babies, Heritable Diseases, and Patent Applications: Legal Issues Surrounding CRISPR Gene Editing Technology


Designer Babies, Heritable Diseases, and Patent Applications: Legal Issues Surrounding CRISPR Gene Editing Technology

Adrianne C. Blake*

            The science fiction movie GATTACA first brought the concept of eugenics and its surrounding ethical concerns to the big screen.  GATTACA (Columbia Pictures 1997).  When the movie was released in 1997, human gene editing was not yet possible.  Only twenty years later, the idea of a designer baby is no longer a matter of science fiction, but of reality.  

Scientists in Portland, Oregon recently “succeeded in creating the first genetically modified human embryo in the United States.”  Vivek Wadhwa, If You Could ‘Design’ Your Own Child, Would You?, Wash. Post (July 27, 2017), https://www.washingtonpost.com/news/innovations/wp/2017/07/27/human-editing-has-just-become-possible-are-we-ready-for-the-consequences/?utm_term=.3853cc453d7d.  A scientific team led by study co-author Shoukhrat Mitalipov of Oregon Health & Science University used a technology known as CRISPR-Cas9, or “CRISPR,” to successfully correct a genetic mutation known to cause hypertrophic cardiomyopathy (HCM).  Amy Dockser Marcus, In Gene-Editing Advance, Scientists Correct Defect in Human Embryos, Wall Street J. (Aug. 2, 2017, 11:18 PM), https://www.wsj.com/articles/in-gene-editing-advance-scientists-correct-defect-in-human-embryos-1501693349.  HCM is an inheritable condition and “a common cause of sudden cardiac death in young athletes.”  Id.

CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, has the potential to make permanent changes to the human germline, affecting not only an individual’s genes, but those of future generations.  Suevon Lee, PTAB Sees No Overlap in Rival Schools’ Gene-Editing Patents, Law360 (Feb. 15, 2017, 9:17 PM), https://www.law360.com/articles/892549/ptab-sees-no-overlap-in-rival-schools-gene-editing-patents; Marcus, supra.  While the embryos used in the study were created for research and were not implanted in a woman, this scientific advancement brings ethical and regulatory questions to the forefront of quickly advancing technology.  Marcus, supra.

Specifically, CRISPR has been described as “a type of molecular scissors” that can target a specific part of a genome sequence and cut out the unwanted portion.  Julie Steenhuysen, U.S. Scientists Urge ‘Serious Consideration’ of Gene Editing in Human Embryos, HuffPost (Feb. 15, 2017, 12:30 PM), http://www.huffingtonpost.com/entry/us-scientists-urge-serious-consideration-of-gene-editing-in-human-embryos_us_58a48664e4b03df370dc6fcf.  New, healthy hereditary information is then used by repair enzymes to fill and seal the cut in the genome sequence.  Marcus, supra.  Ultimately, this information changes the underlying genetic code of the entire embryo.  See id.  

Supporters of gene-editing techniques, including an advisory panel from the National Academy of Sciences (NAS), celebrate the success of the HCM study.  Wadhwa, supra.  Proponents cite the potential for future advances, including the ability to eliminate hereditary diseases, produce stronger plants, withstand epidemics, and revive extinct species.  Id.  However, the potential for the technique’s misuse has raised ethical questions among genetic researchers because “editing the germ-line . . . not only would alter the individual[,] but [would] also be passed to future generations.”  Marcus, supra.  Additionally, while CRISPR is considered among experts to be an extremely precise editing tool, it is not free from editing errors.  Id.  For instance, while Oregon Health & Science University researchers were able to correct the HCM mutation in forty-two out of fifty-eight embryos, the study also cited a 27.6% failure rate.  Id.

The use of genetic engineering in the United States has increased in prevalence since the 1990s, when genetically engineered (GE) crops, including corn and soy, were first approved and introduced to the food supply.  Consumer Info About Food from Genetically Engineered Plants, U.S. Food & Drug Admin., https://www.fda.gov/food/ingredientspackaginglabeling/geplants/ucm461805.htm (last updated Oct. 19, 2015).  In addition to crops, the United States Food and Drug Administration (FDA) has taken steps to monitor and regulate intentionally altered genomic DNA in animals.  Q&A on FDA Regulation of Intentionally Altered Genomic DNA in Animals, U.S. Food & Drug Admin., https://www.fda.gov/animalveterinary/developmentapprovalprocess/geneticengineering/geneticallyengineeredanimals/ucm113605.htm (last updated Oct. 4, 2017).

However, the FDA has been banned from using federal funds to regulate the modification of human embryos since the 2016 Omnibus Spending Bill was signed into law on December 18, 2015.  Consolidated Appropriations Act, 2016, Pub. L. No. 114-113, § 749, 129 Stat. 2242, 2283 (2015); Tanya Lewis, Congress Just Put a Massive Roadblock in the Way of Genetically Editing Human Embryos, Bus. Insider (Dec. 16, 2015,  2:45 PM), http://www.businessinsider.com/congress-bans-funding-for-embryo-gene-editing-2015-12.  Writers of the bill believe that “researchers do not yet fully understand all the possible side effects of editing the genes of a human embryo.”  H.R. Rep. No. 114-205, at 69 (2015).  As such, a rider to the bill provides:

None of the funds made available by this Act may be used to notify a sponsor or otherwise acknowledge receipt of a submission for an exemption for investigational use of a drug or biological product . . . in research in which a human embryo is intentionally created or modified to include a heritable genetic modification.

Consolidated Appropriations Act, 2016 § 749.  This provision has since been renewed and integrated into the House and Senate appropriation bills providing funding through the end of the 2017 federal fiscal year.  I. Glenn Cohen & Eli Y. Adashi, The FDA is Prohibited from Going Germline, 353 Sci. 545, 545 (2016).

A congressionally-directed FDA ban has not stopped editing of human embryos; it has merely shifted genetic studies into the private market.  Lewis, supra.  This is concerning to bioethicists because allowing this market to go unregulated may ultimately result in only the wealthiest of citizens benefitting from what some consider to be extraordinarily useful medical advances.  Id.; see Wadhwa, supra.  In addition to the uncertain provisions of the upcoming fiscal year 2018 congressional budget, another uncertain legal issue surrounding CRISPR technology involves a United States Patent and Trademark Office (USPTO) patent dispute.  Niv Elis, House Heads into August Recess with Uncertain Path on Budget, Hill (July 30, 2017, 6:31 PM), http://thehill.com/policy/finance/budget/344377-house-heads-into-august-recess-with-uncertain-path-on-budget; Lee, supra.

The patent issue arises from two scientific teams, one led by Broad Institute, and the other led by the University of California, Berkeley, who both claim to be the first to invent CRISPR technology.  Lee, supra.  Although the UC Berkeley team was first to apply for a CRISPR patent in May 2012, the USPTO did not switch to a “first-to-file” patent system until March 2013.  Id.  Thus, the Broad Institute team was able to file expedited patent applications several months after the UC Berkeley team, and was issued twelve CRISPR patents while the Berkeley team still awaited review of their initial patent application.  Id.  When the USPTO’s Patent Trial and Appeal Board (PTAB) realized the similarities in the patent applications from the two teams, it ordered an interference hearing, placing a pause on the Broad Institute’s previously approved patents.  Id.  PTAB’s February 15, 2017 per curiam ruling terminated the interference proceedings after finding that the patents submitted by each team contained “patentably distinct subject matter.”  Id.; Broad Inst., Inc., v. Regents of the Univ. of Cal., No. 106,048 (DK), 2017 WL 657415, at *2 (Patent Tr. & App. Bd. Feb. 15, 2017).

This ruling, considered “significant given the potential multibillions of dollars at stake for these academic institutions,” prompted the University of California to recently file an appeal in the United States Court of Appeals for the Federal Circuit, the only appellate-level court with the jurisdiction to hear patent case appeals.  Lee, supra; Ryan Davis, UC Urges Fed. Circ. to Nix ‘Unjust’ Gene Editing Patent Ruling, Law360 (July 26, 2017, 6:39 PM), https://www.law360.com/articles/948313/uc-urges-fed-circ-to-nix-unjust-gene-editing-patent-ruling; 35 U.S.C. § 141(a) (2015).  The appellants cited “[f]undamental errors of law” requiring reversal, but it will be up to the appellate court to determine whether “PTAB committed significant errors of law or lacked substantial evidence to reach its decision.”  See Brief for Appellants at 21, Regents of the Univ. of Cal. v. Broad Inst., Inc., No. 2017-1907, 2017 WL 3331894, at *21; Davis, supra.

Longer-term legal and ethical dilemmas, including when and how to regulate this exciting technology are still to be determined.  Wadhwa, supra.  Based on current scientific trends, it will not be long before additional advances in human genome modification research are brought forward.  See Marcus, supra.  Ideally, CRISPR technology will be used “in a responsible way that will benefit the planet and its people,” but our own country’s inability to agree on a way is concerning and is likely the first issue to tackle.  Wadhwa, supra.  Hopefully, the United States government will heed experts’ recommendations to implement a regulatory plan formed by legal experts, scientists and bioethicists, and legislative experts sooner rather than later (if one is to be implemented at all), particularly before human genome issues grow in complexity.  David Baltimore et al., A Prudent Path Forward for Genomic Engineering and Germline Gene Modification, 348 Sci. 36, 36–37 (2015).

 

*Adrianne C. Blake is a second-year evening law student at the University of Baltimore School of Law, where she currently serves as a staff editor for Law Review, the secretary of the Student Bar Association, and a teaching assistant to Professor Michael J. Hayes (Introduction to Lawyering Skills/Torts) and Professor John A. Lynch, Jr. (Introduction to Lawyering Skills/Civil Procedure).

 

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