UNITED STATES PATENT AND TRADEMARK OFFICE
`____________
`
`BEFORE THE PATENT TRIAL AND APPEAL BOARD
`____________
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`BLUEBIRD BIO, INC.,
`Petitioner,
`
`v.
`
`SLOAN KETTERING INSTITUTE FOR CANCER RESEARCH,
`Patent Owner.
`____________
`Case No. IPR2023-00074
`Patent No. 8,058,061
`____________
`
`DECLARATION OF MICHEL SADELAIN
`IN SUPPORT OF PATENT OWNER’S PRELIMINARY RESPONSE
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`SKI Exhibit 2006
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`____________
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`BEFORE THE PATENT TRIAL AND APPEAL BOARD
`____________
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`BLUEBIRD BIO, INC.,
`Petitioner,
`
`v.
`
`SLOAN KETTERING INSTITUTE FOR CANCER RESEARCH,
`Patent Owner.
`____________
`Case No. IPR2023-00074
`Patent No. 8,058,061
`____________
`
`DECLARATION OF MICHEL SADELAIN
`IN SUPPORT OF PATENT OWNER’S PRELIMINARY RESPONSE
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`I, Michel Sadelain, declare as follows:
`1.
`I am over the age of twenty-one years and am fully competent to make
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`this Declaration. I make the following statements based on personal knowledge and,
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`if called to testify to them, could and would do so.
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`Education
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`2.
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`I am the founding director for the Center for Cell Engineering and head
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`of the Gene Transfer and Gene Expression Laboratory at Memorial Sloan-Kettering
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`Cancer Center (“MSKCC”), where I hold the Stephen and Barbara Friedman Chair.
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`I am also a member of the departments of medicine at Memorial Hospital for Cancer
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`and Allied Diseases (“Memorial Hospital”) and the immunology program of the
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`Sloan-Kettering Institute for Cancer Research (“Sloan-Kettering Institute”).
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`3.
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`The Center for Cell Engineering at MSKCC was established to foster
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`research on cellular therapies. The Center brings together researchers who
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`investigate adoptive T-cell therapies, bone marrow and cord blood transplantation,
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`human stem cell biology, and transfer regulation and repair of genes in human cells.
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`This unique physician-scientist partnership comprises over twenty faculty members
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`from both Memorial Hospital and Sloan-Kettering Institute, all of whom have
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`scientific or clinical interests in cancer or monogenic blood disorders and strive to
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`devise and implement breakthrough therapies for those diseases. As the director of
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`the Center, I provide scientific advice and organize meetings, annual retreats, and
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`peer reviews of scientific data. The Center has led to the establishment of two other
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`MSKCC facilities that are critical for the clinical implementation of cell therapies:
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`(1) the Michael G. Harris Cell Therapy and Cell Engineering Facility led by Dr.
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`Isabelle Rivière; and (2) the Cellular Therapeutics Center led until recently by Dr.
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`Renier Brentjens.
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`4. My laboratory, the Gene Transfer and Gene Expression Laboratory
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`investigates, under my leadership, ways to insert genes into hematopoietic stem cells
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`using viral vectors and to control how those genes are expressed, as well as ways to
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`improve immune responses against tumor cells. My laboratory currently comprises
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`about twenty-five members who conduct research on T cells and stem cell
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`engineering. I am responsible for the scientific direction, experimental planning,
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`review and analysis of data from, publication, and funding of those research
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`activities.
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`5.
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`I earned Doctor of Medicine (M.D.) degree and Master of Science
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`(M.S.) degree in physiology from the University of Paris, France, in 1984, and a
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`Doctor of Philosophy (Ph.D.) degree in immunology from the University of Alberta,
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`Canada, in 1989. Following a clinical residency at the Centre Hospitalier
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`Universitaire Saint-Antoine in Paris, I completed a postdoctoral fellowship at the
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`Whitehead Institute for Biomedical Research, Massachusetts Institute of
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`Technology (“MIT”), Cambridge, Massachusetts, before joining MSKCC in 1994
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`as an Assistant Member in the Sloan-Kettering Immunology Program.
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`6.
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`I am a member of the American Society of Hematology, the American
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`Association for Cancer Research, and the American Society of Cell and Gene
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`Therapy, where I served on the board of directors from 2004 to 2007, and as
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`president from 2015 to 2016. I am an elected member of the American Society for
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`Clinical Investigation. I have authored more than 200 scientific papers and book
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`chapters. Among other awards, I received the 2012 William B. Coley Award for
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`Distinguished Research in Tumor Immunology, the 2013 Sultan Bin Khalifa
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`International Thalassemia Award, the 2017 Passano Laureate and Physician
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`Scientist Award, the 2018 Pasteur-Weizmann/Servier International Prize, the 2019
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`Jacob and Louise Gabbay Award in Biotechnology and Medicine, the 2019
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`INSERM International Prize, and the 2020 Leopold Griffuel Award.
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`7.
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`I have been working for approximately three decades on a gene therapy-
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`based treatment for hemoglobinopathies, which are blood disorders caused by
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`mutations in or near globin genes. My focus has been on using stem cells for the
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`blood disease beta-thalassemia. Beta-thalassemia is a genetic disorder caused by
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`mutations in or near the beta-globin gene, which provides instructions for making
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`the beta-chain of hemoglobin (also referred to as beta-globin), that is produced in
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`red blood cells and carries oxygen to cells throughout the body. In people with beta-
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`thalassemia, low levels of hemoglobin lead to a lack of oxygen in parts of the body.
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`Patients with two deficient beta-globin genes have a more severe form of the disease
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`called beta-thalassemia major. Persons with beta-thalassemia major require regular
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`blood transfusions. For most patients, the disease is incurable because they lack
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`matched donors for bone marrow or stem cell transplants to restore the production
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`of red blood cells with a normal hemoglobin content. In addition to the logistical
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`difficulties associated with lifelong blood transfusions, a side-effect of blood
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`transfusions is a build-up of iron in the body of the transfusion recipient. Patients
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`with beta-thalassemia major experience this condition, known as iron-overload, as a
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`result of requiring regular blood transfusions. This side effect can lead to other health
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`problems requiring additional therapies.
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`8. Whereas beta-thalassemia major affects only a few thousand people in
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`the United States, beta-thalassemia (in all of its forms) is one of the two most
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`common monogenic blood disorders worldwide, with the other being sickle cell
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`anemia. Beta-thalassemia is commonly found in people of Mediterranean origin.
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`Worldwide, approximately 68,000 people are born with beta-thalassemia each year.
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`9.
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`A copy of my current curriculum vitae is attached as Appendix A.
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`The TNS9 Vector’s Conception and Reduction to Practice
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`10.
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`In the late 1990s and into the 2000s, which was the time I was doing
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`the work described below, gene therapy was regarded as a nascent, cutting-edge, but
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`potentially dangerous technique. Indeed, some well-publicized gene therapy failures
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`during that time caused some scientists and others to be very wary of this field in
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`general. For example, in 1999, Jesse Gelsinger, a patient in a gene-therapy clinical
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`trial for a liver disorder run by the University of Pennsylvania died from an adverse
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`event. His death received widespread media coverage. In addition, in 2000, gene
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`therapy trials were conducted in France to treat children with Severe Combined
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`Immune Deficiency. The treatment in those trials, similar in concept to the treatment
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`we had invented for thalassemia, used retroviral vectors for inserting genes into
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`HSCs. While the results of this trial were initially promising, in the years following
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`the application of the gene therapy, several patients developed a leukemia-like
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`cancer that was traced to the retroviral vectors. That prompted researchers,
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`institutions, and regulators to proceed very cautiously in the gene-therapy area.
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`Indeed, in 2003, in direct response to the trial in France, the FDA temporarily halted
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`all trials that used retroviral vectors for inserting genes into bone marrow stem cells.
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`11. My research in gene therapy began in 1989. During my postdoctoral
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`fellowship at MIT, the head of the laboratory in which I was working suggested that
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`I explore thalassemia (in addition to the research I was conducting on T-cells). My
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`research on both T-cells and thalassemia continued after I joined MSKCC in
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`September 1994. From 1989 to 1994, I conducted the research myself while at MIT;
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`then, from 1994 to 2000, I pursued this research in my laboratory with graduate
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`students and postdoctoral fellows, devoting approximately 50% of my time to
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`thalassemia research and the other 50% to CAR-T cell research. I worked on
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`thalassemia research with a number of individuals, including postdoctoral fellows
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`Chad May and Stefano Rivella and Joseph Bertino, who was Chair of the Molecular
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`Pharmacology and Therapeutics Program at MSKCC. It is my understanding that
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`Dr. Bertino passed away in October 2021.
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`12. As background, the body produces red blood cells through a
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`complicated series of events that begins with cells called hematopoietic (i.e., “blood
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`producing”) stem cells, or HSCs. HSCs are normally found in the bone marrow. Our
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`potentially curative treatment involves (1) extracting a patient’s hematopoietic stems
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`cells (HSCs), (2) utilizing a genetically-modified virus, known as a lentiviral vector,
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`to stably insert a functional beta-globin gene into the extracted HSCs—this process
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`is called transduction, and then (3) infusing the patient with the “genetically
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`restored” HSCs that should now be capable of producing beta-globin in the
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`derivative red blood cells. To achieve this, we designed what we have termed the
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`“TNS9 vector.”
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`13. The first step in our research that led to the TNS9 vector—and the most
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`innovative—was designing the genetic material that would be inserted into the
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`patient’s cells. The genetic material needed to include a functional beta-globin gene,
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`but the beta-globin gene alone was not enough to cause the patient’s cells to produce
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`enough beta-globin. Genes are typically accompanied by other DNA sequences that
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`allow the cell to control when the gene is turned on or off. These DNA sequences,
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`called regulatory elements, are necessary for the cell to make beta-globin from the
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`beta-globin gene. By the late 1990s, scientists were just beginning to understand the
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`role that these regulatory elements played in gene expression, both in general and in
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`the specific case of hemoglobin. At the time, and as discussed below, the
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`understanding was to try and use as small of regulatory elements as possible. In fact,
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`for several years, scientists had been working to reduce the LCR regulatory elements
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`to very small pieces in the hopes of trimming them down to manageable sizes for
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`vectors that would still provide expression. However, no one had yet succeeded in
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`this endeavor.
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`14.
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`I designed a sequence of genetic material that included both the beta-
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`globin gene and the regulatory elements that were necessary for cells to express it
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`(i.e., to make beta-globin from the beta-globin gene). We produced and tested
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`several iterations of a line of such vectors we referred to as RNS, QNS, and TNS
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`vectors. For example, we produced vectors designated TNS1, TNS2, TNS3, etc.
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`These TNS vectors included varying lengths of the fragments surrounding the HS
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`core elements. I remember one vector included one long fragment and two short
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`fragments, another vector included two longer fragments and one short fragment,
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`and others that included fragments of approximately the same length. Each vector
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`contained different LCR regulatory elements.
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`15. When designing this genetic material, we still wanted the LCR
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`regulatory elements to be as short as possible. We knew the longer it gets, the better
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`expression. In addition to the HS core elements, we were aware of other putative
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`sites where transcription factors might bind. As these sites were still poorly
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`understood, our goal was to try and preserve these sites in the fragments we were
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`using in systematic studies. However, we also understood that the longer the
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`fragment, the less likely the transfer would be successful. The longer the fragment
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`was also more expensive to manufacture. It really was a reconciliation process
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`between competing concerns.
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`16. Another unique aspect of our design was to place the HS2 core element
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`at one end of the fragment encompassing that core element. The common approach
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`at the time was to place the core elements in roughly the center of any fragment.
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`17. When designing the LCR fragments, we initially used restriction
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`enzymes to cut the DNA. There were several different companies that sold
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`restriction enzymes at the time and each had their own catalog of the different
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`enzymes that they sold (with several being sold by each company). At the time, we
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`bought restriction enzymes from multiple companies for different projects.
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`18. However, other approaches could be used to create the LCR fragments.
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`For example, the LCR fragments could have also been constructed using polymerase
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`chain reactions (or PCR), which many laboratories used at the time.
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`19. After we created the LCR fragments, we used junctions to connect the
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`fragments, which simplified assembly for testing purposes. This included one
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`junction between the promoter and the HS2 fragment, another junction between HS2
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`and HS3 fragments, and another between the HS3 and HS4 fragments. These
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`junctions varied in length but would have been as small as possible for the same
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`reasons stated above, to keep the amount of material being transferred to a minimum.
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`These fragments would have increased the length of the fragment. However, once a
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`desired fragment was selected, one would have wanted to preserve each base pair of
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`that fragment. It was known that transcription factors can be comprised of a small
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`number of base pairs and removal of transcription factors could affect expression of
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`the globin gene. Therefore, we would have worked to prevent the shortening of the
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`fragments, beyond what we had originally selected, to preserve as many
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`transcription factor binding sites as possible.
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`20. Designing this genetic material, however, was only the first step toward
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`getting defective cells to make hemoglobin. The next challenge was getting this
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`genetic material into the DNA, or genome, of defective cells. Genetic material that
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`is directly injected into a cell typically is not incorporated into the cell’s genome, so
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`this step required further experimentation.
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`21. Certain viruses are known to infect cells by inserting their own genes
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`(i.e., the viral genes) into the cell’s genome. They do this through a complicated
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`series of biochemical processes. Beginning in the mid-1980s, scientists discovered
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`that they could modify these viruses to insert genetically engineered DNA into the
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`genome of living cells. These modified viruses are called vectors.
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`22. Early vectors could only hold short DNA sequences. For example, we
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`initially attempted to incorporate larger LCR designs into retroviral vectors based on
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`the Moloney Murine Leukemia Virus (MMLV) but were unable to stably transfer
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`the retroviral genomes due to RNA splicing that resulted from the addition of the
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`larger LCR sequences. This is one of the reasons that the understanding at the time
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`was to use as small regulatory fragments. In fact, I remember one of my
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`contemporaries was using a vector capable of holding a maximum of about 4.5 kb,
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`leaving little space for the incorporation of LCR elements.
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`23.
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`In or around 1996, we became aware of other viruses that could afford
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`gene transfer into human cells. These are called lentiviruses, from which lentiviral
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`vectors are derived. Lentiviruses are a type of retrovirus. The genetic material that I
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`assembled to express the beta-globin gene was too large for early vectors, but we
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`hypothesized that they potentially could be stably transferred using a suitable
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`lentiviral vector. To make such a lentiviral vector, we combined the human genetic
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`material that we had assembled to control the beta-globin gene with the viral genetic
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`material known to be necessary to produce lentiviral vectors from so-called
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`“packaging cells” (called a 293T cell). When these components were combined, the
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`293T cell produced lentiviral vector particles containing the therapeutic genetic
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`material that we had designed. From 1996 on, we designed multiple globin vectors
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`(and actually tested at least three in vivo no later than 1999, possibly starting in
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`1998). One of these, which I called the TNS9 vector, was a success.
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`24. For the TNS9 vector to have a therapeutic effect, it would need to cause
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`a large number of red blood cells in the patient or animal’s body to contain adequate
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`amounts of normal hemoglobin. Prior to the TNS9 vector, I was not aware of anyone
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`designing a vector that was capable of achieving more than 1% of the normal level
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`of expression. To be therapeutically effective, the gene therapy would need to result
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`in 10-20% of the normal level of expression, and preferably over 15%. It is not
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`possible to achieve this effect by modifying red blood cells directly. Instead, it is
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`necessary to modify HSCs—i.e., the cells in the body that produce red blood cells.
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`25. My laboratory performed the first experiments with the TNS9 vector in
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`mice. The first step for these studies was to concentrate the TNS9 vector from the
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`293 T cells. As of the late 1990s, there were methods for producing vectors on a
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`small scale and to an acceptable level of purity for animal studies; however, it was
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`difficult to purify vectors on the scale or to the extent necessary for human studies.
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`26. To treat the thalassemic mice in our study, we extracted their bone
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`marrow, isolated the HSCs, treated the HSCs with the TNS9 vector, and reinjected
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`the treated HSCs into the mice. We found that these treated mice expressed
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`functional hemoglobin as the result of the TNS9 vector treatment. In other words,
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`we discovered that our technology could eventually work for treating human patients
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`with beta-thalassemia.
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`27.
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`In May 2000, my laboratory published an Abstract in Molecular
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`Therapy. At the time, many labs were working on the same problem, i.e., working
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`to create a vector design capable of successful transduction or incorporation of a
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`globin gene into a thalassemic cell that would express the globin gene at therapeutic
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`levels. We wanted to release a sort of teaser to let the world know we had designed
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`what appeared to be an effective vector. However, we also were very intentional
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`about what we disclosed and what we did not disclose. We purposefully did not
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`disclose much information about our vector design because we did not want our
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`competitors to be able to copy what we had done, i.e., throw a decade of research
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`out the window and let others capitalize on it. Based on the disclosures in this
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`Abstract, our competitors would not have understood how to recreate the TNS9
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`vector. For example, a vector that included just the HS2, HS3, and HS4 core
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`elements alone, you could assemble the LCR fragment in over a dozen different
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`ways. There would be hundreds to thousands of possible options (if not more) if
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`longer fragments (more than just the core elements) were used.
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`28.
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`In July 2000, my laboratory published a landmark paper in Nature
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`describing the technology we had developed to introduce globin genes into
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`hematopoietic stem cells (the “Nature Article”). While we used the human beta-
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`globin gene, our TNS9 vector design could be used
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`to
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`treat various
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`hemoglobinopathies and/or could incorporate a different globin gene. Indeed, when
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`my coworkers and I reported the results of our work in Nature, we noted that our
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`work was an “advancement
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`towards
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`the genetic
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`treatment of severe
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`haemogloinopathies.” (Ex. 1005 (the Nature Article) at 6.)
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`29. At a high level, the research in the Nature Article was a proof of concept
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`for using gene-therapy techniques to modify a patient’s cells to produce therapeutic
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`levels of hemoglobin. The work was the result of a complex series of experiments.
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`30. The Nature Article solved a conundrum of globin gene therapy that had
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`previously been unsolved by other researchers in the field. A major challenge in
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`designing an effective gene therapy was to design a vector that could efficiently
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`insert a functional globin gene in patient cells. This required two things. First, it
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`required high-efficiency transduction — i.e., gene transfer via vector into the
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`defective cell. Second, in order to be therapeutic, the vector would need to express
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`high levels of the encoded globin gene — or in lay terms, it would need to produce
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`a very substantial amount of, in the case of the TNS9 vector, beta-globin. Once you
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`solved two big issues: (1) levels of expression that are high enough to be therapeutic
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`and (2) stable transfer of genetic material and good efficiency, it would be apparent
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`that you can use this design as a recipe for other vectors. Instead of the beta-globin
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`gene, you could use an animal globin gene, a fetal globin gene, etc. and reasonably
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`expect expression of that gene.
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`31.
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`In the Nature Article, we provided more insight into our vector and the
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`results, and provided information on the size of the fragments surrounding the HS
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`fragments. However, we did not describe the start and end positions of these HS
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`fragments, such as the flanking restriction sites of each HS fragment. It should be
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`noted that size alone would be insufficient to identify those fragments. We also did
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`not disclose whether we constructed those fragments using PCR, by cutting the DNA
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`using restriction enzymes, or otherwise much less where each cut should be made.
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`In addition, even if a competitor tried to recreate our vector, the only way to test
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`whether it met the challenges identified above would be to conduct a therapeutic
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`model testing the vector, which would require in vivo testing and trial and error to
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`make such a determination. It would take a very long time — several years — to
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`create, test, and verify all the possible combinations.
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`32. For years, neither we nor any of the number of other research groups in
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`the United States or in Europe conducting this research had succeeded in designing
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`a vector that could both be transduced at high efficiency and express therapeutic
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`levels of the beta-globin protein. Throughout the 1990s, it was a mad race to find a
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`vector capable of stable transfer and that would cause expression of the transferred
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`gene. My understanding was that most who were conducting this research had
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`concluded that retroviral vectors could not serve this purpose. I was aware of
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`research teams, other than us, who had turned their efforts to alternative vector
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`systems (adeno-associated virus, alpha-virus, etc.). We, in contrast, perceived that
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`some unique biological attributes of lentiviral vectors (a type of retroviral vector)
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`may allow us to assemble inside such a vector a set of elements that we had carefully
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`studied and reduced to a size compatible with genetic stability and efficient
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`transduction. It took me eleven years — from 1989 to 2000 — to succeed in
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`designing a vector meeting those two fundamental criteria, and I continued to test
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`and develop that design thereafter. That design — as published in the Nature Article
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`— became the foundation for all present gene therapies for thalassemia and sickle
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`cell disease.
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`33.
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`I (and three co-inventors from MSKCC) filed for patent protection on
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`the discoveries reported in the Nature Article and ultimately were granted U.S.
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`Patent No. 7,541,179 (“the ‘179 Patent”) and No. 8,058,061 (“the ‘061 Patent”), both
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`entitled “Vector Encoding Human Globin Gene and Use Thereof in Treatment of
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`Hemoglobinopathies.”
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`34.
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`I have also located excerpts of our inventor notebooks from the 1990s
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`(particularly 1997-1998) that demonstrate we had conceived of and were actively
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`working on the vector design that is claimed in the ‘179 and ‘061 Patents. I
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`understand these are being filed as Exhibit 2033.
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`Inventorship
`35.
`I am an inventor of the subject matter disclosed and claimed in the ‘179
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`and ‘061 Patents. I am familiar with the specification and the claims of the ‘179 and
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`‘061 Patents.
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`36.
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`I understand that bluebird bio, Inc. (hereafter, “Petitioner”) has
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`challenged the validity of certain claims of the ‘179 and ‘061 Patents.
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`37.
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`I understand Petitioner has asserted May, et al., “Lentiviral-Mediated
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`Transfer of the Human β-Globin Gene and Large Locus Control Region Elements
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`Permit Sustained Production of Therapeutic Levels of β-Globin in Long-Term Bone
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`Marrow Chimeras,” Mol. Therapy, 1(5):S248-49 (2000) (the “May Abstract”)
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`against the ‘179 and ‘061 Patents. The May Abstract represents the work of the
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`inventors of the ‘179 and ‘061 Patents. In addition to the inventors of the ‘179 and
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`‘061 Patents, the May Abstract lists John Callegari and Karen Gaensler as authors.
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`Mr. Callegari was a laboratory technician, and Dr. Gaensler provided a mouse for
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`control purposes that we used in the study. These individuals are named as co-
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`authors because of these non-inventive contributions to the publication.
`
`38.
`
`I understand Petitioner has asserted May et al., “Therapeutic
`
`Haemoglobin Synthesis in β-Thalassaemic Mice Expressing Lentivirus-Encoded
`
`Human β-globin,” Nature, 406:82-86 (2000) (i.e., the Nature Article) against the
`
`‘179 and ‘061 Patents. The Nature Article represents the work of the inventors of
`
`the ‘179 and ‘061 Patents. In addition to the inventors of the ‘179 and ‘061 Patents,
`
`the Nature Article lists John Callegari, Glenn Heller, Karen Gaensler, and Lucio
`
`Luzzatto as authors. Mr. Callegari and Dr. Gaensler had the same non-inventive
`
`contributions identified above. Similarly, Dr. Heller was a statistician, and Dr.
`
`Luzzatto was my chairman; both provided support and consultation. They were
`
`named as co-authors for these non-inventive contributions to the publication.
`
`39. None of these individuals (Dr. Heller, Mr. Callegari, Dr. Gaensler, and
`
`Dr. Luzzatto) are inventors of the invention claimed in the ‘179 or ‘061 Patents.
`
`40.
`
`I understand that Petitioner has asserted “Therapeutic Hemoglobin
`
`Synthesis in Beta-Thalassemic Mice Expressing Lentivirus-Encoded Human Beta-
`
`Globin,” (the “May Thesis”), which I understand became publicly available on
`
`November 26, 2001. (See Pet. at 17.). The May Thesis reflects some of Dr. May’s
`
`contributions to the research underlying the ‘179 and ‘061 Patents.
`
`139471957.5
`
`17
`
`SKI Exhibit 2006
`Page 18 of 84
`
`
`
`
`
`41. Even though we did not provide these details in the May Abstract or the
`
`Nature Article, regarding the three restriction enzyme fragments of the LCR (a BstXI
`
`and SnaBI HS2-spanning nucleotide fragment, a BamHI and HindIII HS3-spanning
`
`nucleotide fragment, and a BamHI and BanII HS4-spanning nucleotide fragment)
`
`which are claimed in the ’179 patent, it is clear of course that we, as in the inventors,
`
`had knowledge and possession of these fragments since we had created the TNS9
`
`vector and were testing it in vivo no later than 1999.
`
`42. As discussed above, the Nature Article published in 2000. By the time
`
`of this publication, we (the inventors) had unquestionably conceived of the TNS9
`
`vector design, which I understand is an embodiment of the vector claimed in the
`
`’179 and ‘061 Patents, as well as reduced it to practice. Both the Nature Article and
`
`the May Abstract disclose our TNS9 vector and provides preliminary results from
`
`our testing. While neither the May Abstract nor the Nature Article identifies the
`
`claimed restriction enzyme fragments of the LCR, the very fact that these references
`
`show we possessed this vector demonstrates that I, along with my co-inventors,
`
`clearly recognized and knew of these claimed fragments.
`
`
`
`
`
`139471957.5
`
`18
`
`SKI Exhibit 2006
`Page 19 of 84
`
`
`
`I declare under penalty of perjury that the foregoing is true and correct.
`
`Executed on January 23, 2023.
`
`___________________________
`Michel Sadelain
`
`139471957.5
`
`19
`
`SKI Exhibit 2006
`Page 20 of 84
`
`
`
`CURRICULUM VITAE
`
`Name:
`Michel William Jeffrey Sadelain
`Place of Birth: Neuilly sur Seine, Paris, France
`Nationality:
`Canadian and French citizenships
`U.S. Permanent Resident (citizenship pending)
`Office Address: Center for Cell Engineering, S-1021A
`Memorial Sloan Kettering Cancer Center
`1275 York Avenue, Box 182
`New York, NY 10065
`Tel: (212) 639-6190
`E-mail: m-sadelain@ski.mskcc.org
`Fax: (917) 432-2340
`Home Address: 15 West 84 Street, Apt 2C
`New York, NY 10024
`Tel: (212) 423-0403
`Baccalaureat (Mathematiques), 1976
`Lycee Jehan Ango, Dieppe, France
`M.D., 1984
`University of Paris–Pierre et Marie Curie, Paris, France
`(Thesis Advisor: Gabriel Richet)
`M.S. (Physiology), 1984
`University of Paris–Rene Descartes, Paris, France
`Ph.D. (Immunology), 1989
`University of Alberta, Edmonton, Canada
`(Thesis Advisor: Thomas Wegmann)
`Postdoctoral Fellow 1989-94
`Whitehead Institute for Biomedical Research,
`Massachusetts Institute of Technology,
`Cambridge, MA (Laboratory of Richard Mulligan)
`Medical Training and Licensure:
`1983
`Examen clinique medical, chirurgical et obstetrique
`(European Medical Licensure)
`Resident
`Pulmonary Division/Internal Medicine and Clinical Immunology
`Centre Hospitalier Universitaire Saint-Antoine, Paris, France
`FMGEMS (ECFMG examination)
`FLEX certification.
`
`1984
`1993
`
`Education:
`
`1983-84
`
`SKI Exhibit 2006
`Page 21 of 84
`
`APPENDIX A
`
`
`
`Positions and Appointments:
`Research Appointments
`1994
`Assistant Member, Immunology Program, Sloan-Kettering Institute
`2000
`Associate Member, Immunology Program, Sloan-Kettering Institute
`2004
`Member, Immunology Program, Sloan-Kettering Institute
`2007
`Member, Molecular Pharmacology & Chemistry Program, Sloan-Kettering
`Institute
`Academic and Hospital Appointments
`1994
`Assistant-Attending Geneticist, Department of Human Genetics and
`Hematology/Oncology and Bone Marrow Transplant Services, Departments of
`Medicine and Pediatrics, Memorial Hospital
`Assistant Professor, Graduate School of Medical Sciences, Cornell University
`Medical College
`Director, Gene Transfer and Somatic Cell Engineering Facility, Memorial Sloan
`Kettering Cancer Center
`Assistant Professor, Department of Medicine, Weill College of Medicine at
`Cornell University
`Associate-Attending Geneticist, Department of Human Genetics and
`Hematology/Oncology and Bone Marrow Transplant Services, Departments of
`Medicine and Pediatrics, Memorial Hospital
`Associate Professor, Graduate School of Medical Sciences, Cornell University
`Attending Geneticist, Department of Medicine, Hematology/Oncology and
`Bone Marrow Transplant Services, Departments of Medicine and Pediatrics,
`Memorial Hospital
`Professor, Immunology Program and Microbial Pathogenesis, Weill Cornell
`Medical College and Graduate School of Medical Sciences, Cornell University
`Director, Center for Cell Engineering, Memorial Sloan Kettering Cancer Center
`
`1995
`
`1997
`
`1998
`
`2000
`
`2001
`2004
`
`2004
`
`2007
`
`Scientific and Medical Societies:
`American Association for Cancer Research (AACR)
`American Society for Clinical Investigation (ASCI)
`American Society of Clinical Oncology (ASCO)
`American Society of Gene and Cell Therapy (ASGCT)
`American Society of Hematology (ASH)
`International Society for Stem Cell Research (ISSCR)
`
`
`
`2
`
`SKI Exhibit 2006
`Page 22 of 84
`
`
`
`1984
`1989
`1995
`2003
`2004
`2005
`2007
`2012
`
`2013
`
`2013
`2013
`
`2013
`2014
`2014
`
`Honors and Awards:
`1976
`Baccalaureat Mathematiques, “Mention Tres Bien” (Top 1%, national).
`1984
`These d'Etat de Docteur en Medecine, awarded with thesis medal and mention
`“honorable”. Thesis President: Pr. Gabriel Richet.
`Co
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