`
`
`
`Exhibit No.
`2001
`
`2002
`2003
`2004
`
`2005
`2006
`2007
`2008
`2009
`2010
`
`2011
`
`2012
`2013
`
`2014
`
`2015
`
`2016
`
`2017
`2018
`
`Patent Owner’s Exhibit List
`IPR2023-00074
`Patent 8,058,061
`
`Description
`Exclusive Licensee Agreement Between Sloan Kettering
`Institute for Cancer Research and San Rocco Therapeutics, LLC
`Declaration of Dr. James Riley
`October 2020 Declaration of Dr. Michel Sadelain
`Petitioner’s October 2020 Letter Submitting Dr. Sadelain’s
`October 2020 Declaration in New York State Court
`Joint Defense Agreement
`January 2023 Declaration of Michel Sadelain
`Declaration of Chad May
`Declaration of Stefano Rivella
`Declaration of Lucio Luzzatto
`Sorrentino, “One step closer to gene therapy for
`hemoglobinopathies”
`Caterina et al., “Human beta-globin locus control region:
`analysis of the 5’ DNase I hypersensitive site HS2 in transgenic
`mice.” Proc Natl Acad Sci U S A. 1991 Mar 1;88(5):1626-30.
`doi: 10.1073/pnas.88.5.1626. PMID: 2000371; PMCID:
`PMC51077
`Judson, “Glimmering Promise of Gene Therapy”
`Jackson, et al., (1996), “Role of D N A sequences outside the
`cores of DNase hypersensitive sites (HSs) in functions of the p-
`globin locus control region.” Domain opening and synergism
`between HS2 and HS3. J Biol Chem. 271:11871-8
`Philipsen, et al. “The β-globin dominant control region:
`hypersensitive site 2.” EMBO J. (1990) 9:2159-67
`Hardison, et. al., “Locus control regions of mammalian β-globin
`gene clusters: combining phylogenetic analyses and experimental
`results to gain functional insights.” (1997) Gene. 205:73-94.
`Persons, D. A., A. W. Nienhuis. 2000. “Gene therapy for the
`hemoglobin disorders: past, present, and future.” Proc Natl Acad
`Sci U S APNAS. 97:5022-4
`Kafri, “Lentiviral Vectors: Regulated Gene Expression”
`Amado, “Lentiviral Vectors — the Promise of Gene Therapy
`within Reach?”
`
`
`
`
`
`Exhibit No.
`2019
`
`2020
`
`2021
`
`2022
`
`2023
`
`2024
`
`2025
`2026
`2027
`2028
`
`2029
`2030
`2031
`2032
`2033
`2034
`2035
`2036
`
`2037
`
`Description
`Chada, et al., “Specific expression of a foreign β-globin gene in
`erythroid cells of transgenic mice.” Nature. (1985) 314:377-80
`Townes, et al., “Expression of human β-globin genes in
`transgenic mice: effects of a flanking metallothionein-human
`growth hormone fusion gene.” (1985) Mol Cell Biol. 5:1977-83
`Dzierzak, et al., “Lineage-specific expression of a human β-
`globin gene in murine bone marrow transplant recipients
`reconstituted with retrovirus-transduced stem cells.” (1988)
`Nature. 331:35-41
`Bodine, et. al., “Combination of interleukins 3 and 6 preserves
`stem cell function in culture and enhances retrovirus-mediated
`gene transfer into hematopoietic stem cells.” (1989) Proc Natl
`Acad Sci USA. 86: 8897-901
`Bender, et al., “A majority of mice show long-term expression of
`a human β-globin gene after retrovirus transfer into
`hematopoietic stem cells.” (1989) Mol Cell Biol. 9:1426-34
`Sadelain et al., Proc. Nat’l Acad. Sci. (USA) 92:6728-6732
`(1995)
`GenBank Accession No. Z84721 (March 19, 1997)
`GenBank Accession No. NM_ 000517 (October 31, 2000)
`Hardison et al., J. Mol. Biol. (1991) 222(2):233-249
`A Syllabus of Human Hemoglobin Variants (1996), by Titus et
`al., published by The Sickle Cell Anemia Foundation in Augusta,
`Georgia (available online at http://globin.cse.psu.edu)
`GenBank Accession No. JOO179 (August 26, 1993)
`Tagle et al., Genomics (1992) 13(3):741-760
`Li et al., Blood (1999) 93(7):2208-2216
`Slightom et al., Cell (1980) 21(3):627-638
`Excerpts from Inventor Notebooks
`Excerpts from Inventor Notebooks
`October 2020 Declaration of Dr. Isabelle Rivière
`Verma et al., “Gene Therapy: Twenty-First Century Medicine,”
`Annu Rev. BioChem (2005). 74:711-38
`Blau, et al., “Moleular Medicine, Gene Therapy – A Novel Form
`of Drug Delivery,” The New England Journal of Medicine
`(1995).
`
`2
`
`
`
`
`
`Exhibit No.
`2038
`
`2039
`
`2040
`
`2041
`
`2042
`
`2043
`
`2044
`2045
`
`2046
`
`2047
`
`
`
`Description
`Morris et al., “MHC class II gene silencing in trophoblast cells is
`caused by inhibition of CIITA expression,” American Journal of
`Reproductive Immunology (1998)
`Physical mapping of the globin gene deletion in β-thalassemia,
`Benards (1979)
`Ryan, T. M., R. R. Behringer, N. C. Martin, T. M. Townes, R. D.
`Palmiter, R. L. Brinster. 1989. A single erythroid-specific DNase
`I super-hypersensitive site activates high levels of human beta-
`globin gene expression in transgenic mice. Genes Dev. 3: 314-23
`Pasceri, P., D. Pannell, X. W u, J. Ellis. 1998. Full activity from
`human beta-globin locus control region transgenes requires
`5'HSl, distal beta-globin promoter, and 3’ beta-globin sequences.
`Blood. 92:653-63
`Hardison, R., J. L. Slightom , D. L. Gumucio, M. Goodman, N .
`Stojanovic, W. Miller. 1997. Locus control regions of
`mammalian beta-globin gene clusters: combining phylogenetic
`analyses and experimental results to gain functional insights.
`Gene. 205: 73-94
`Hacein-Bey-Abina, et. al., “Vector mediated
`transformation,” (2003) N. Engl. J. Med. 348:255-256.
`[PubMed])
`Pfeifer, Gene Therapy: Promises and Problems
`Declaration of Wanda French-Brown in Support of Motion for
`Pro Hac Vice
`Declaration of James H. McConnell in Support of Motion for
`Pro Hac Vice
`Declaration of Howard S. Suh in Support of Motion for Pro Hac
`Vice
`
`3
`
`
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