Timeline of Gene and Cell Therapy


ASGCT was established in 1996 by Dr. George Stamatoyannopoulos, professor of medicine at the University of Washington's School of Medicine, and a group of the country's leading researchers in gene therapy. With more than 3,275 members in the United States and worldwide, today ASGCT is the largest association of individuals involved in gene and cell therapy research.

The concept of gene therapy was introduced in the late 1970s after the development of recombinant DNA technology. After the development of basic science and technology for gene transfer into patient's cells, the first gene therapy trial on humans was performed in 1990 by researchers at the National Institutes of Health. A four-year-old girl was treated for adenosine deaminase deficiency (ADA), a rare genetic disease in which children are born with severe immunodeficiency and are prone to repeated serious infections. Gene therapy has since been applied in clinical trials to treat a variety of diseases.

1920
1928
Transforming Principle First Described
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Transforming Principle First Described

J Hyg (Lond). 1928 Jan; 27(2): 113–159. The Significance of Pneumococcal Types.

Read the article in  The Journal of Hygiene

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1940
1944
Proof of Phenotypic Modification with Exogenous DNA
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Proof of Phenotypic Modification with Exogenous DNA

Avery, O.T., Mcleod, C.M.& McCarty, M. Studies on the chemical nature of the substance Inducing transformation of pneumococcal types. J.exp. Med. 79, 137-158 (1944).

Read the article in the Journal of Experimental Medicine

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1947
Gene Therapy: Mouse Breeding
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Gene Therapy: Mouse Breeding

Genes introduced into mouse strains by breeding can correct phenotypic abnormalities.

Clyde E. Keeler, PhD, Journal of Heredity, Volume 38, Issue 10, 1 October 1947, Pages 294–298, https://doi.org/10.1093/oxfordjournals.jhered.a105653

Read the article in the Journal of Heredity.

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1950
1953
James Watson, PhD and Francis Crick, PhD
Watson and Crick Describe the Structure of DNA
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Watson and Crick Describe the Structure of DNA

Watson, J. D., & Crick, F. H. C. A structure for deoxyribose nucleic acid. Nature 171, 737–738 (1953)

Read the article in Nature

Photo courtesy of Gonville and Caius College Cambridge 

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1956
Oncolytic Adenovirus for Cervical Cancer
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Oncolytic Adenovirus for Cervical Cancer

The use of a lytic virus to treat cervical carcinoma.

Huebner, R. J., W. P. Rowe, W. E. Schatten, R. R. Smith, and L. B. Thomas. 1956. Studies on the use of viruses in the treatment of carcinoma of the cervix. Cancer 9: 1211-1218.

Read the article in the British Medical Journal

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1957
First Allogeneic Hematopoietic Stem Cell Transplantation
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First Allogeneic Hematopoietic Stem Cell Transplantation

Foundational studies on hematopoietic stem cell transplanataion as a platform for gene therapy.

Thomas ED, Lochte HL, Lu WC, Ferrebee JW. Intravenous infusion of bone marrow in patients receiving radiation and chemotherapy. N Engl J Med. 1957;257:491–6.

Read the article in the New England Journal of Medicine

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1960
1960
Demonstration that viruses can convey heritable traits to cells
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Demonstration that viruses can convey heritable traits to cells

Mixed infection with two types of Rous sarcoma virus. Howard M. Temin. Division of Biology, California Institute of Technology, Pasadena, California USA. Accepted 14 October 1960, Available online 11 February 2004.

Read the article in Virology

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1968
Virus-based DNA transfer
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Virus-based DNA transfer

Demonstration that papova tumor viruses cause neoplastic transformation by introducing foreign transfforming genes stably and heritably into cells.

Sambrook, J., Westphal, H., Srivansan, P.R. & Dulbecco, R. The integrated state of viral DNA in SV40-transformed cells. Proc. natn. Acad. Sci. U.S.A. 118, 1288--1293 (1968).

Read the article from PNAS

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1968
Genetically modified viruses can be used to transmit genetic information
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Genetically modified viruses can be used to transmit genetic information

Early concept of engineering virus genomes to create vectors to transfer foreign genetic material into mammalian cells for gene therapy. Use of polynucleotide phosphorylase to extend poly(A) tails of the tobacco mosaic virus (TMV) RNA genome.

Use of Viruses as Carriers of Added Genetic Information. Stanfield Rogers & Peter Pfuderer. Nature 219, 749–751 (17 August 1968)

Read the article in Nature

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1970
1972
Foundational proposal and rationale for gene therapy
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Foundational proposal and rationale for gene therapy

T. Friedmann and R. Roblin. Gene Therapy for human Genetic Disease. Science 178, 648-649 (1972).

Read the article in Science

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1975-1976
NIH establishes the Recombinant DNA Advisory Committee (RAC)
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NIH establishes the Recombinant DNA Advisory Committee (RAC)

In response to call for federal guidelines for recombinant DNA research from the National Academy of Sciences and from the Asilomar Conference, NIH publishes Guidelines for Recombinant DNA research and establishes Recombinant DNA Advisory Committee (RAC).

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1977-1986
Development of adenovirus vectors
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Development of adenovirus vectors

Recombinant adenoviruses engineered as gene transfer vectors.

Graham, F.L., Smiley, et al. Characteristics of a human cell line transformed by DNA from human adenovirus type 5. J. Gen. Virol. 36,  59-72, 1977.

Read the article in the Journal of General Virology

Ballay A, Levrero M, et al. In vitro and in vivo synthesis of the hepatitis B virus surface antigen and of the receptor for polymerized human serum albumin from recombinant human adenoviruses.  EMBO J. 1985 Dec 30;4(13B):3861-5.

Read the article in The EMBO Journal

Haj-Ahmad Y, Graham FL. Development of a helper-independent human adenovirus vector and its use in the transfer of the herpes simplex virus thymidine kinase gene.  J Virol. 1986 Jan;57(1):267-74

Read the article in the Journal of Virology

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1978
CaPO4-mediated viral DNA transfection and application to human DNA
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CaPO4-mediated viral DNA transfection and application to human DNA

New chemical avenues to genetically modifying cells.

Graham, F and van der Eb, A. A new technique for the assay of infectivity of human adenovirus 5 DNA. Virology 52: 456-467 (1973) 

Read the article in Virology

Wigler, M., Pelllcer, A.. Silverstein, S. & Axel, R. Transfer of single-copy eucaryotic genes using total cellular DNA as donor. Cell 14, 72!>--731 (1978).

Read the article in Cell

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1979
First recombinant viral vector expressing a disease-related transgene
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First recombinant viral vector expressing a disease-related transgene

First demonstrations of effective virus vector-based genetic modification of mammalian cells.

Mulligan RC, Howard BH, Berg P. Synthesis of rabbit beta-globin in cultured monkey kidney cells following infection with a SV40 beta-globin recombinant genome. Nature. 1979 Jan 11;277(5692):108-14. PubMed PMID: 215915.

Read the article in Nature

Hamer DH, Leder P. Expression of the chromosomal mouse Beta maj-globin gene cloned in SV40. Nature. 1979 Sep 6;281(5726):35-40. PubMed PMID: 233122.

Read the article in Nature

Photo courtesy of Harvard Medical School

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1980
1980
A mis-step: Premature and unapproved human gene transfer experiment
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A mis-step: Premature and unapproved human gene transfer experiment

Martin Cline, a hematology investigator at UCLA, administered autologous bone marrow cells exposed to plasmid DNA carrying a hemoglobin gene to two patients with thalassemia. There was no clinical benefit, and these human experiments were performed without UCLA IRB/Ethics Committee approval, and in direct opposition to NIH guidelines. Technical and clinical results of this experiment were not fully reported.

Mercola, K.E., Stang, H.D., Browne, J., Salser, W. & Cline, M.J. Insertion of a new gene of viral origin Into bone marrow cells of mice. Science 208, 1033- 1035 (1980).

Read the article in Science

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1981-1982
Engineered retroviruses for gene transfer
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Engineered retroviruses for gene transfer

Shimotohno, K. & Temin, H.M. Formation of Infectious progeny virus after insertion of herpes simplex thymidlne kinase gene into DNA of an avian retrovirus. Cell 26, 67-77 (1981).

Read the article in Cell

Wei, C., Gibson, M., Spear, P.G. & Scolnick, E.M. Construction and isolation of a transmissible retrovirus containing the Sarc gene from Harvey Murine Sarcoma Virus and the thymldine kinase gene from herpes simplex virus type 1. J. Virol. 39, 93s-944 (1981).

Read the article in the Journal of Virology

Tabin, C.J., Hoffmann, J.W., Goff, S.P. & Weinberg, R.A. Adaptation of a retrovirus as a eucaryotlc vector transmitting the herpes simplex thymidine kinase gene. Molec. cell. Biol. 2, 426-436 (1982).

Read the article in Molecular and Cellular Biology

Photos courtesy of the Broad Institute and the Whitehead Institute

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1982
First International symposium on gene therapy
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First International symposium on gene therapy

Report of first inter-disciplinary gathering of molecular biologists, virologists, human geneticists and ethicists to discuss human gene therapy.  

Theodore Friedmann. Prospect for Gene Therapy: Fact and Fiction

Banbury Center of Cold Spring Harbor.

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1982, 1984
Development of AAV vector system
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Development of AAV vector system

RJ Samulski, KI Berns, M Tan, N Muzyczka. Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells. Proc Natl Acad Sci USA, 79 (1982), pp. 2077–2081

Read the article in PNAS

PL Hermonat, N Muzyczka. Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells. Proc Natl Acad Sci USA, 81 (1984), pp. 6466–6470

Read the article in PNAS

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1984
First demonstration of correction of disease phenotype with virus vector-delivered transgene
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First demonstration of correction of disease phenotype with virus vector-delivered transgene

Miller, A.D., Jolly, D.J., Friedmann, T. and Verma, I.M. A transmissible retrovirus expressing human hypoxanthlne phosphoribosyl transferese (HPRT: Gene transfer Into cells obtained from humans deficient In HPRT. Proc. natn. Acad. Sci. U.S.A. 80, 4709-4713 (1983).

Read the article in PNAS

Willis, R.C. et al. Partial phenotypic correction of human Lesch Nyhan (hypoxanthlne-guanlnephosphoribosyltransferese-deficient) lymphoblasts with a transmissible retrovlral vector. J. Biol. Chem. 250, 7842- 7849 (1984).

Read the article in the Journal of Biological Chemistry

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1984
RAC response to publication of President’s Commission on “Splicing Life”
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RAC response to publication of President’s Commission on “Splicing Life”

1. Creation of RAC working group to develop “points to consider” for gene therapy studies,
2. Institutional Biosafety Committees (IBCs) to become responsible for reviewing gene transfer research.
3. Broaden oversight role of RAC to include ethical, legal and social implications of research with recombinant DNA. 

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1984-1985
First gene transfer into pluripotent hematopoietic stem cells of a mouse
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First gene transfer into pluripotent hematopoietic stem cells of a mouse

Introduction of new genetic material into pluripotent haematopoietic stem cells of the mouse. Williams DA, Lemischka IR, Nathan DG, Mulligan RC. Nature. 1984 Aug 9-15;310(5977):476-80.

Read the article in Nature

Retroviral vector-mediated gene transfer into human hematopoietic progenitor cells. Gruber HE, Finley, et al. Science. 1985 Nov 29;230(4729):1057-61.

Read the article in Science

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1986
Vector developments – SIN retroviral vectors
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Vector developments – SIN retroviral vectors

This study led to important improvements in retrovirus vector design. 

Yu SF, von Rüden T, Kantoff PW, Garber C, Seiberg M, Rüther U, Anderson WF, Wagner EF, Gilboa E. Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc Natl Acad Sci USA. 1986 May; 83(10):3194-8.

Read the article in PNAS

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1986
Genetic Therapy Inc. established
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Genetic Therapy Inc. established

Genetic Therapy Inc. was the first biotechnology company devoted to gene therapy.  Its early clinical trials were disappointing. It was established with funds from venture capital and pharmaceutical (Sandoz) support. The company was later acquired by Cell Genesis Inc. Its gene therapy program discontinued in 1995.

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1987-1989
Lipid-mediated non viral gene delivery
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Lipid-mediated non viral gene delivery

These studies were important early advances toward lipid-based agents for gene transfer.

Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. P L Felgner, T R Gadek, M Holm, R Roman, H W Chan, M Wenz, J P Northrop, G M Ringold, and M Danielsen. Proc Natl Acad Sci U S A. 1987 Nov; 84(21): 7413–7417.

Read the article in PNAS

Efficient gene transfer into mammalian primary endocrine cells with lipopolyamine-coated DNA. Behr JP, Demeneix B, Loeffler JP, Perez-Mutul J. Proc Natl Acad Sci U S A. 1989 Sep;86(18):6982-6.

Read the article in PNAS

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1989
Concept of chimeric antigen receptor T cells
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Concept of chimeric antigen receptor T cells

This study lays the groundwork for the develoment of CAR-T technology for cancer gene/immuno therapy. 

Proc Natl Acad Sci U S A. 1989 Dec;86(24):10024-8. Expression of immunoglobulin-T-cell receptor chimeric molecules as functional receptors with antibody-type specificity. Gross G1, Waks T, Eshhar Z.

Read the article in PNAS

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1990
1990-91
NIH RAC finalizes “Points to Consider” for gene therapy studies
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NIH RAC finalizes “Points to Consider” for gene therapy studies

Appendix “M” added to NIH Guidelines, and describes requirements for submission to NIH of protocols for human gene transfer studies.

Human Gene Therapy Subcommittee: minutes of meeting, 1 Jun 1990. U.S. National Institutes of Health. Recombinant DNA Advisory Committee. Human Gene Therapy Subcommittee. Hum Gene Ther. 1991. Spring; 2(1):87-96. PMID: 11642891

Read the article in Human Gene Therapy

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1990
First human gene transfer study
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First human gene transfer study

This study describes the Infusion of autologous tumor infiltrating lymphocytes (“TIL”) genetically-marked with a retrovirus vector carrying a marker gene, in an attempt to determine if infused TIL home back to tumors.

Rosenberg SA, Aebersold P, Cornetta K, Kasid A, Morgan RA, Moen R, Karson EM, Lotze MT, Yang JC, Topalian SL, Merino MJ, Culver K, Miller AD, Blaese RM, Anderson WF. 1990. Gene transfer into humans - immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 323:570-578.

Read the article in the New England Journal of Medicine

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1990
Direct in vivo plasmid-based gene transfer
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Direct in vivo plasmid-based gene transfer

These studies document the efficacy of direct gene transfer into muscle without need for virus vectors.

Science. 1990 Mar 23;247(4949 Pt 1):1465-8. Direct gene transfer into mouse muscle in vivo. Wolff JA, Malone RW, Williams P, Chong W, Acsadi G, Jani A, Felgner PL, Phil L. Felgner

Read the article in Science

Photo courtesy of Genetic Support Foundation

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1991
Retrovirus vector pseudotyping with the G-protein of VSV
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Retrovirus vector pseudotyping with the G-protein of VSV

This work demonstrates that pseudotyping of retroviruses with the G-protein of VSV facilitates the preparation of high titer retrovirus vector and broadens host range of target cells.

Emi, N., Friedmann, T., and Yee, J.-K., “Pseudotype Formation of Murine Leukemia Virus with the G Protein of Vesicular Stomatitis Virus.” J. Virology 65, 1202-1207 (1991)

Read the article in the Jounral of Virology

Burns, J.C., Friedmann, T., Driever, W., Burrascano, M., and Yee, J.-K. “Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells.” Proc. Nat’l. Acad. Sci. USA 90, 8033-8037 (1993)

Read the article in PNAS

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1991
Gene gun gene transfer in vivo
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Gene gun gene transfer in vivo

This potentially important advance describes a physical approach toward a non-viral mechanism for gene transfer.

Williams, R. S., S. A. Johnston, M. Riedy, M. J. DeVit, S. G. McElligott, and J. C. Sanford. 1991. Introduction of foreign genes into tissues of living mice by DNA-coated microprojectiles. Proceedings of the National Academy of Sciences USA 88: 2726-2730.

Read the article in PNAS

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1991
Tumor inhibition and prolonged survival with genetically engineered herpes simplex virus
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Tumor inhibition and prolonged survival with genetically engineered herpes simplex virus

Demonstrates improved anti-tumor efficacy of thymidine kinase-deficient herpes simplex virus in mouse model of human glioma. 

Martuza RL1, Malick A, et al. Experimental therapy of human glioma by means of a genetically engineered virus mutant. Science 1991;252:854-6.

Read the article in Science

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1993
Development of DNA vaccines
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Development of DNA vaccines

These advances demonstrate the usefulness of genetic modification in the design of more efficient immunogens and vaccines.

Ulmer JB, Liu, MA, et al. Heterologous protection against influenza by injection of DNA encoding a viral protein. Science. 1993. 259:1745-9.

Read the article in Science

E F Fynan, R G Webster, et al. DNA vaccines: protective immunizations by parenteral, mucosal, and gene-gun inoculations. Proc Natl Acad Sci U S A. 1993. 90: 11478–11482.

Read the article in PNAS

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1995
Early retroviral gene therapy trials for immunodeficiency disorders
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Early retroviral gene therapy trials for immunodeficiency disorders

Initial attempts at treating a serious human immune disorder, adenosine deaminase deficiency, were carried out at the National Institutes of Health in the USA and San Rafaelle in Milan, targeting lymphocytes or hematopoietic stem and progenitor cells.  Without a selective advantage for corrected cells, given ongoing enzyme replacement therapy,  or conditioning therapy to allow engraftment, these early trials did not produce convincing evidence for efficacy.

T Lymphocyte-Directed Gene Therapy for ADA− SCID: Initial Trial Results After 4 Years R. Michael Blaese, Kenneth W. Culver, A. Dusty Miller, Charles S. Carter, Thomas Fleisher, Mario Clerici, Gene Shearer, Lauren Chang, Yawen Chiang, Paul Tolstoshev, Jay J. Greenblatt, Steven A. Rosenberg, Harvey Klein, Melvin Berger, Craig A. Mullen, W. Jay Ramsey, Linda Muul, Richard A. Morgan, W. French Anderson. Science 20 Oct 1995 : 475-480

Read the article in Science

Science. 1995 Oct 20;270(5235):470-5. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients. Bordignon C, Notarangelo LD, Nobili N, Ferrari G, Casorati G, Panina P, Mazzolari E, Maggioni D, Rossi C, Servida P, Ugazio AG, Mavilio F.

Read the article in Science

Photo courtesy of Comitato di Esperti per la Politica della Ricerca (CEPR)

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1995
Formation of the NIH National Gene Vector Laboratories
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Formation of the NIH National Gene Vector Laboratories

The NGVB (originally NGVL) established as a vital national resource for researchers to obtain adequate quantities of clinical-grade vectors for human gene transfer protocols. 

The NGVLs consist of three vector production centers:

  • Baylor College of Medicine
  • City of Hope National Medical Center Beckman Research Institute
  • Indiana University, which serves as the Coordinating Center for all the laboratories

Two additional laboratories conduct toxicology studies for NGVL-approved investigators:

  • Southern Research Institute
  • University of Florida
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1995
Report and Recommendations of the Panel to Assess the NIH Investment in Research on Gene Therapy
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Report and Recommendations of the Panel to Assess the NIH Investment in Research on Gene Therapy

Official NIH response to scientific and procedural deficiencies of early human clinical trials.

Co-Chairs: Stuart H. Orkin, MD and Arno Motulsky, MD

  • No evidence of clinical benefit to date despite 100 protocols.
  • Significant deficiencies in the basic science underpinning
  • Only a few clinical trials designed to yield useful basic information
  • Overselling of studies is rampant, which hinders confidence and progress

Read the report

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1996
American Society of Gene Therapy established
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American Society of Gene Therapy established

ASGT was established in 1996 by Dr. George Stamatoyannopoulos, professor of medicine at the University of Washington's School of Medicine, and a group of the country's leading researchers in gene therapy.

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1996
Development of Lentivirus vectors
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Development of Lentivirus vectors

In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector. Luigi Naldini, Ulrike Blömer, Philippe Gallay, Daniel Ory, Richard Mulligan, Fred H. Gage, Inder M. Verma, Didier Trono. Science  12 Apr 1996: Vol. 272, Issue 5259, pp. 263-267

Read the article in Science

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1996-1999
Multiple crucial steps in preclinical development of AAV2 as a gene therapy vector, including these landmark papers in mice and in a canine disease model
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Multiple crucial steps in preclinical development of AAV2 as a gene therapy vector, including these landmark papers in mice and in a canine disease model

Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. X Xiao, J Li and R J Samulski. J Virol 70: 8098, 1996

Read the article in the Jounral of Virology

Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Herzog RW1, Yang EY, Couto LB, Hagstrom JN, Elwell D, Fields PA, Burton M, Bellinger DA, Read MS, Brinkhous KM, Podsakoff GM, Nichols TC, Kurtzman GJ, High KA.Nat Med. 1999 Jan;5(1):56-63.

Read the article in Nature Medicine

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1997
Effective suicide gene therapy transfer to T cells allowing mitigation of graft-versus-host disease following donor lymphocyte infusion in allogeneic transplantation recipients
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Effective suicide gene therapy transfer to T cells allowing mitigation of graft-versus-host disease following donor lymphocyte infusion in allogeneic transplantation recipients

In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector.
Naldini L1, Blömer U, Gallay P, Ory D, Mulligan R, Gage FH, Verma IM, Trono D. Science 272: 263. 1996.

Read the article in Science

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1998
First ASGT Annual Meeting in Seattle
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First ASGT Annual Meeting in Seattle

George Stamatoyannopoulos, MD, Dr.Sci planned the first ever meeting of the American Society of Gene Therapy. Dr. Stamatoyannopoulos was the founding president of the Society. 

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1999
Patient Dies During a Trial Of Therapy Using Genes
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Patient Dies During a Trial Of Therapy Using Genes

Death of 18 year-old patient, Jesse Gelsinger following administration of an adenoviral vector carrying the ornithine decarboxylase gene in a gene therapy protocol. The death of J. Gelsinger undescored the need for greater rigor and administrative care in deigning and performing human gene therapy trials. The event was a major defining moment and vital turning point in the evolution of human gene therapy.

Read the article in the New York Times

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2000
2000
Launch of Molecular Therapy
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Launch of Molecular Therapy

Molecular Therapy is the official journal of ASGCT. 

Read the latest articles in Molecular Therapy

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2002
Development of novel serotypes of AAV with increased efficiency and extended tropism as gene therapy vectors
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Development of novel serotypes of AAV with increased efficiency and extended tropism as gene therapy vectors

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
Guang-Ping Gao, Mauricio R. Alvira, Lili Wang, Roberto Calcedo, Julie Johnston, and James M. Wilson
PNAS September 3, 2002. 99 (18) 11854-11859

Read the article in PNAS

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2002
Clinical benefit of hematopoietic stem cell retrovirus- mediated gene therapy for X-SCID
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Clinical benefit of hematopoietic stem cell retrovirus- mediated gene therapy for X-SCID

Retroviral transfer of IL2R gene to hematopoietic stem and progenitor cells of boys with X-linked severe combined immunodeficiency (SCID)

Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease. Marina Cavazzana-Calvo, Salima Hacein-Bey, Geneviève de Saint Basile, Fabian Gross, Eric Yvon, Patrick Nusbaum, Françoise Selz, Christophe Hue, Stéphanie Certain, Jean-Laurent Casanova, Philippe Bousso, Françoise Le Deist, Alain Fischer. Science  28 Apr 2000: Vol. 288, Issue 5466, pp. 669-672

Read the artcile in Science

Photo courtesy of Science

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2003
Insertional mutagenesis and proto-oncogenic activation-mechanism of geno toxicity
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Insertional mutagenesis and proto-oncogenic activation-mechanism of geno toxicity

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, de Saint Basile G, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, Romana S, Radford-Weiss I, Gross F, Valensi F, Delabesse E, Macintyre E, Sigaux F, Soulier J, Leiva LE, Wissler M, Prinz C, Rabbitts TH, Le Deist F, Fischer A, Cavazzana-Calvo M. Science 302: 415, 2003

Read the article in Science

Photo courtesy of Université Paris Descartes

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2003
First regulatory approval of a gene therapy product
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First regulatory approval of a gene therapy product

Gendicine, a cancer treatment consisting of a replication- incompetent adenovirus vector expressing wild-type p53 approved in China

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2003-2004
Elucidation of vector integration patterns
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Elucidation of vector integration patterns

These studies provided important clues for understanding and eventually averting the mechanisms of virus-based genotoxicity in gene therapy studies.

Transcription Start Regions in the Human Genome Are Favored Targets for MLV Integration. Xiaolin Wu1, Yuan Li2, Bruce Crise2, Shawn M. Burgess1. Science  13 Jun 2003: Vol. 300, Issue 5626, pp. 1749-1751

Read the article in Science

Distinct Genomic Integration of MLV and SIV Vectors in Primate Hematopoietic Stem and Progenitor Cells. Peiman Hematti, Bum-Kee Hong, Cole Ferguson, Rima Adler, Hideki Hanawa, Stephanie Sellers, Ingeborg E Holt, Craig E Eckfeldt, Yugal Sharma, Manfred Schmidt, Christof von Kalle, Derek A Persons, Eric M Billings, Boris Calmels. Published: November 23, 2004

Read the article in PLOS

Retroviral DNA Integration: ASLV, HIV, and MLV Show Distinct Target Site Preferences. Rick S Mitchell, Brett F Beitzel, Astrid R. W Schroder, Paul Shinn, Huaming Chen, Charles C Berry, Joseph R Ecker, Frederic D Bushman. Published: August 17, 2004

Read the article in PLOS

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2008
The first clinical trial utilizing electroproation
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The first clinical trial utilizing electroproation

The first clinical test of electroporation methods for gene transfer.

Phase I Trial of Interleukin-12 Plasmid Electroporation in Patients With Metastatic Melanoma Adil I. Daud, Ronald C. DeConti, Stephanie Andrews, Patricia Urbas, Adam I. Riker, Vernon K. Sondak, Pamela N. Munster, Daniel M. Sullivan, Kenneth E. Ugen, Jane L. Messina, and Richard Heller. Journal of Clinical Oncology 26, no. 36:5896-5903, 2008.

Read the article in the Journal of Clinical Oncology

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2008
Derivation of human induced pluripotent stem cells
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Derivation of human induced pluripotent stem cells

The Beginning of a vital development of induced pluripotent stem cells in understanding and treating human genetic disease.

Read the 2008 Time 100

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2008
Pioneering AAV gene therapy clinical trials for inherited form of blindness
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Pioneering AAV gene therapy clinical trials for inherited form of blindness

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis. Albert M. Maguire, M.D., Francesca Simonelli, M.D., Eric A. Pierce, M.D., Ph.D., Edward N. Pugh, Jr., Ph.D., Federico Mingozzi, Ph.D., Jeannette Bennicelli, Ph.D., Sandro Banfi, M.D., Kathleen A. Marshall, C.O.T., Francesco Testa, M.D., Enrico M. Surace, D.V.M., Settimio Rossi, M.D., Arkady Lyubarsky, Ph.D., et al. May 22, 2008 N Engl J Med 2008; 358:2240-2248

Read the article in the New England Jounral of Medicine

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis, James W.B. Bainbridge, Ph.D., F.R.C.Ophth., Alexander J. Smith, Ph.D., Susie S. Barker, Ph.D., Scott Robbie, M.R.C.Ophth., Robert Henderson, M.R.C.Ophth., Kamaljit Balaggan, M.R.C.Ophth., Ananth Viswanathan, M.D., F.R.C.Ophth., Graham E. Holder, Ph.D., Andrew Stockman, Ph.D., Nick Tyler, Ph.D., Simon Petersen-Jones, Ph.D., Shomi S. Bhattacharya, Ph.D., et al. May 22, 2008 N Engl J Med 2008; 358:2231-2239

Read the article in the New England Jounral of Medicine

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2009
Clinical benefit of hematopoietic stem cell retrovirus gene therapy for adenosine deamaninase-deficient SCID
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Clinical benefit of hematopoietic stem cell retrovirus gene therapy for adenosine deamaninase-deficient SCID

These studies demonstrate the therapeutic efficacy of virus-transferred transgene for therapy of ADA immunodeficiency model. 

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency. Alessandro Aiuti, M.D., Ph.D., Federica Cattaneo, M.D., Stefania Galimberti, Ph.D., Ulrike Benninghoff, M.D., Barbara Cassani, Ph.D., Luciano Callegaro, R.N., Samantha Scaramuzza, Ph.D., Grazia Andolfi, Massimiliano Mirolo, B.Sc., Immacolata Brigida, B.Sc., Antonella Tabucchi, Ph.D., Filippo Carlucci, Ph.D., et al. January 29, 2009. N Engl J Med 2009; 360:447-458

Read the article in the New England Journal of Medicine

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2009
ASGT changes name
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ASGT changes name

ASGT changes name to American Society of Gene & Cell Therapy to reflect the integration of cell therapies. 

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2009-2013
Positive clinical trial results using hematopoietic stem cell lentiviral gene therapy for central nervous system metabolic/storage disorders
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Positive clinical trial results using hematopoietic stem cell lentiviral gene therapy for central nervous system metabolic/storage disorders

First evidence of therapeutic efficacy of lentivirus-transferred transgene for treatment of neurodegenerative disease.

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Nathalie Cartier, Salima Hacein-Bey-Abina, Cynthia C. Bartholomae, Gabor Veres, Manfred Schmidt, Ina Kutschera, Michel Vidaud, Ulrich Abel, Liliane Dal-Cortivo, Laure Caccavelli, Nizar Mahlaoui, Véronique Kiermer, Denice Mittelstaedt, Céline Bellesme, Najiba Lahlou, François Lefrère, Stéphane Blanche, Muriel Audit, Emmanuel Payen, Philippe Leboulch, Bruno l’Homme, Pierre Bougnères, Christof Von Kalle, Alain Fischer, Marina Cavazzana-Calvo, Patrick Aubourg. Science  06 Nov 2009: Vol. 326, Issue 5954, pp. 818-823

Read the article in Science 

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
Alessandra Biffi, Eugenio Montini1, Laura Lorioli, Martina Cesani, Francesca Fumagalli, Tiziana Plati, Cristina Baldoli, Sabata Martino, Andrea Calabria, Sabrina Canale, Fabrizio Benedicenti, Giuliana Vallanti, Luca Biasco, Simone Leo, Nabil Kabbara, Gianluigi Zanetti, William B. Rizzo, Nalini A. L. Mehta, Maria Pia Cicalese, Miriam Casiraghi, Jaap J. Boelens, Ubaldo Del Carro, David J. Dow, Manfred Schmidt, Andrea Assanelli, Victor Neduva, Clelia Di Serio, Elia Stupka, Jason Gardner, Christof von Kalle, Claudio Bordignon, Fabio Ciceri, Attilio Rovelli, Maria Grazia Roncarolo, Alessandro Aiuti, Maria Sessa, Luigi Naldini. Science  23 Aug 2013: Vol. 341, Issue 6148, 1233158

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2010
2011
Successful AAV8 gene therapy for hemophilia B
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Successful AAV8 gene therapy for hemophilia B

Proof of clinical efficacy of AAV-delivered transgene for therapy of classical gene therapy model – hemophilia B. 

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B. Amit C. Nathwani, M.B., Ch.B., Ph.D., Edward G.D. Tuddenham, M.B., B.S., M.D., Savita Rangarajan, M.B., B.S., Cecilia Rosales, Ph.D., Jenny McIntosh, Ph.D., David C. Linch, M.B., B.Chir., Pratima Chowdary, M.B., B.S., Anne Riddell, B.Sc., Arnulfo Jaquilmac Pie, B.S.N., Chris Harrington, B.S.N., James O'Beirne, M.B., B.S., M.D., Keith Smith, M.Sc., et al. December 22, 2011 N Engl J Med 2011; 365:2357-2365

Read the article in the New England Journal of Medicine

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2011-2014
Dramatic clinical responses to chimeric antigen receptor engineered T Cells for CD19+ lymphoid malignancies
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Dramatic clinical responses to chimeric antigen receptor engineered T Cells for CD19+ lymphoid malignancies

Dramatic clinical proof of principle of use of genetically engineered T cells (CAR-T) to treat intractable hematological malignancies. 

Eradication of B-lineage cells and regression of lymphoma in a patient treated with autologous T cells genetically engineered to recognize CD19. James N. Kochenderfer, Wyndham H. Wilson, John E. Janik, Mark E. Dudley, Maryalice Stetler-Stevenson, Steven A. Feldman, Irina Maric, Mark Raffeld, Debbie-Ann N. Nathan, Brock J. Lanier, Richard A. Morgan and Steven A. Rosenberg. Blood 2010 116:4099-4102

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Chimeric Antigen Receptor–Modified T Cells in Chronic Lymphoid Leukemia. David L. Porter, M.D., Bruce L. Levine, Ph.D., Michael Kalos, Ph.D., Adam Bagg, M.D., and Carl H. June, M.D. August 25, 2011. N Engl J Med 2011; 365:725-733

Read the article in the New England Journal of Medicine

Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia. Shannon L. Maude, M.D., Ph.D., Noelle Frey, M.D., Pamela A. Shaw, Ph.D., Richard Aplenc, M.D., Ph.D., David M. Barrett, M.D., Ph.D., Nancy J. Bunin, M.D., Anne Chew, Ph.D., Vanessa E. Gonzalez, M.B.A., Zhaohui Zheng, M.S., Simon F. Lacey, Ph.D., Yolanda D. Mahnke, Ph.D., Jan J. Melenhorst, Ph.D., et al.October 16, 2014. N Engl J Med 2014; 371:1507-1517

Read the article in the New England Jounral of Medicine

Efficacy and Toxicity Management of 19-28z CAR T Cell Therapy in B Cell Acute Lymphoblastic Leukemia. Marco L. Davila, Isabelle Riviere, Xiuyan Wang, Shirley Bartido, Jae Park, Kevin Curran, Stephen S. Chung, Jolanta Stefanski, Oriana Borquez-Ojeda, Malgorzata Olszewska, Jinrong Qu, Teresa Wasielewska, Qing He, Mitsu Fink, Himaly Shinglot, Maher Youssif, Mark Satter, Yongzeng Wang, James Hosey, Hilda Quintanilla, Elizabeth Halton, Yvette Bernal, Diana C. G. Bouhassira, Maria E. Arcila, Mithat Gonen, Gail J. Roboz, Peter Maslak, Dan Douer, Mark G. Frattini, Sergio Giralt1, Michel Sadelain1, and Renier Brentjens. Science Translational Medicine  19 Feb 2014: Vol. 6, Issue 224, pp. 224ra25

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2012
Launch of Molecular Therapy Nucleic Acids
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Launch of Molecular Therapy Nucleic Acids

ASGCT launches the first open-access sibling journal in the Molecualr Therapy family of journals. This was a major expansion of publication efforts at ASGCT to enhance the reach of Molecular Therapy.

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2012
European Commission approves Glybera, the first approved gene therapy in Europe
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European Commission approves Glybera, the first approved gene therapy in Europe

AAV Vector delivering the lipoprotein lipase gene to patients with genetic hypercholesterolemia. This was the first approved gene therapy product in Europe for gene therapy of a rare metabolic disease with small target patient population. It was withdrawn from market in 2017 because of failure to secure approval in U.S. and unproven efficacy.

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2012-2013
Discovery of CRISPR/Cas9 and utilization of this system to perform targeted gene editing in mammalian cells
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Discovery of CRISPR/Cas9 and utilization of this system to perform targeted gene editing in mammalian cells

A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity. Martin Jinek, Krzysztof Chylinski, Ines Fonfara, Michael Hauer, Jennifer A. Doudna1, Emmanuelle Charpentier. Science 17 Aug 2012: Vol. 337, Issue 6096, pp. 816-821

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Multiplex Genome Engineering Using CRISPR/Cas Systems. Le Cong, F. Ann Ran, David Cox, Shuailiang Lin, Robert Barretto, Naomi Habib, Patrick D. Hsu, Xuebing Wu, Wenyan Jiang, Luciano A. Marraffini, Feng Zhang. Science  15 Feb 2013: Vol. 339, Issue 6121, pp. 819-823

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RNA-Guided Human Genome Engineering via Cas9. Prashant Mali, Luhan Yang, Kevin M. Esvelt, John Aach, Marc Guell, James E. DiCarlo, Julie E. Norville, and George M. Church. Science. 2013 Feb 15; 339(6121): 823–826.

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2013
FDA approval of Kynamro (mipomesen), an antisense oligonucleotide inhibitor of ApoB-100, for use in patients with hypercholesterolemia
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FDA approval of Kynamro (mipomesen), an antisense oligonucleotide inhibitor of ApoB-100, for use in patients with hypercholesterolemia

Development and first approval of a therapeutic anti-sense oligonucleotide. 

Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial. Prof Frederick J Raal, MD, Raul D Santos, MD, Dirk J Blom, MD, Prof A David Marais, MD, Min-Ji Charng, MD, William C Cromwell, MD, Robin H Lachmann, MRCP, Daniel Gaudet, MD, Ju L Tan, MB, BS, Scott Chasan-Taber, PhD, Diane L Tribble, PhD, JoAnn D Flaim, PhD, Stanley T Crooke, MD, Published: March 14, 2010.

Read the atricle in The Lancet

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2014
First clinical trial of genome editing
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First clinical trial of genome editing

First clinical trial of Zinc finger-edited cells and first demonstration of clinical efficacy of genome editing to treat HIV. 

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV. Pablo Tebas, M.D., David Stein, M.D., Winson W. Tang, M.D., Ian Frank, M.D., Shelley Q. Wang, M.D., Gary Lee, Ph.D., S. Kaye Spratt, Ph.D., Richard T. Surosky, Ph.D., Martin A. Giedlin, Ph.D., Geoff Nichol, M.D., Michael C. Holmes, Ph.D., Philip D. Gregory, Ph.D., et al. March 6, 2014 N Engl J Med 2014; 370:901-910

Read the article in the New England Journal of Medicine

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2014
Launches of Molecular Therapy Methods and Clinical Development and Molecular Therapy Oncolytics
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Launches of Molecular Therapy Methods and Clinical Development and Molecular Therapy Oncolytics

ASGCT launches the next two open-access sibling journals in the Molecualr Therapy family of journals. Further strengthening of ASGCT publication platforms with addition of clinically-oriented journal and emphasis on oncolytic agents. 

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2014
First clinical trial of iPSC-derived cells to regenerate tissue
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First clinical trial of iPSC-derived cells to regenerate tissue

First clinical use of iPSC-derived cells to treat major eye disease. 

Autologous Induced Stem-Cell–Derived Retinal Cells for Macular Degeneration. Michiko Mandai, M.D., Ph.D., Akira Watanabe, Ph.D., Yasuo Kurimoto, M.D., Ph.D., Yasuhiko Hirami, M.D., Ph.D., Chikako Morinaga, Ph.D., Takashi Daimon, Ph.D., Masashi Fujihara, M.D., Ph.D., Hiroshi Akimaru, Ph.D., Noriko Sakai, B.S., Yumiko Shibata, M.S., Motoki Terada, Yui Nomiya, M.S., et al. March 16, 2017. N Engl J Med 2017; 376:1038-1046

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2014-2016
iPSC-derived cardiomyocytes engraft following injection into large animal myocardial infarctions
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iPSC-derived cardiomyocytes engraft following injection into large animal myocardial infarctions

Demonstration of potential use of genetically-engineered iPSC-derived cardiac myocytes in regenerating damaged heart muscle.

James J. H. Chong, Charles E. Murry, et al. Human embryonic-stem-cell-derived cardiomocytes regenerate non-human primate hearts. Nature volume 510, pages 273–277 (12 June 2014). 

Read the article in Nature

Yuji Shiba, Uichi Ikeda, et al. Allogeneic transplantation of iPS cell-derived cardiomyocytes regenerates primate hearts.  Nature volume 538, pages 388–391 (20 October 2016).

Read the article in Nature

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2015
FDA approves first-of-its-kind product for the treatment of melanoma
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FDA approves first-of-its-kind product for the treatment of melanoma

FDA approved Imlygic (talimogene laherparepvec), the first FDA-approved oncolytic virus therapy, for the treatment of melanoma lesions in skin and lymph nodes. Imglygic is a genetically modified herpes simplex virus type 1 that selectively replicates in and lyses tumors by producing the immunostimulatory protein GM-CSF that produces an anti-tumor immune response.

Read the press release from Amgen

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2015
Japan Prize Winners
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Japan Prize Winners

Drs. Alain Fischer and Theodore Friedmann are awarded the prestigious Japan Prize for the proposal of the concept of gene therapy and its clinical applications. This is a major international prize that recognizes the emergence of gene therapy as a conceptually new approach to treatment of human disease.

Photo courtesy of Getty Images

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2016
CAR T cell immunotherapy effective against the most aggressive solid tumors
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CAR T cell immunotherapy effective against the most aggressive solid tumors

The first demonstration that CAR T cell immunotherapy can be effective effective not only against hematological malignancies but also against solid tumors, such as metastatic glioblastoma. The new IL-13Ra2-specific (and 4-1BB equipped) CARs used in this case study resulted in unprecedented regression of all lesions of multifocal glioma, in brain and in the spine.

Brown, C., Alizadeh, D et al. Regression of glioblastoma after chimeric Antigen Receptor T-Cell Therapy. N Engl J Med. 2016 375:2561-9.

Read the article in the New England Journal of Medicine

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2016
European Commission approves Strimvelis, the first ex vivo gene therapy product worldwide
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European Commission approves Strimvelis, the first ex vivo gene therapy product worldwide

Retrovirus vector expressing the adenosine deaminase gene to treat patients with ADA- deficient SCID

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2016
FDA approval of the oligonucleotide drug Spinraza for spinal muscle atrophy
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FDA approval of the oligonucleotide drug Spinraza for spinal muscle atrophy

FDA approval for anti-sense therapy of spinomuscular atrophy. Impressive clinical efficacy. 

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Richard S Finkel, MD, Claudia A Chiriboga, MD, Jiri Vajsar, MD, John W Day, MD, Jacqueline Montes, EdD, Darryl C De Vivo, MD, Mason Yamashita, MD, Frank Rigo, PhD, Gene Hung, MD, Eugene Schneider, MD, Daniel A Norris, PhD, Shuting Xia, MSC, Frank Bennett, PhD, Kathie M Bishop, PhD. Published: December 06, 2016

Read the article in The Lancet

Photo courtesy of SMA News Today

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2017
Use of “universal donor” TALEN-engineered CAR-T cells to achieve remission in a patient with refractory CD19+ acute leukemia
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Use of “universal donor” TALEN-engineered CAR-T cells to achieve remission in a patient with refractory CD19+ acute leukemia

Combination of TALEN genome editing with CAR-T gene/cell immunotherapy for successful treatment of refractory leukemia. 

Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells. Waseem Qasim, Hong Zhan, Sujith Samarasinghe, Stuart Adams, Persis Amrolia, Sian Stafford, Katie Butler, Christine Rivat, Gary Wright, Kathy Somana, Sara Ghorashian, Danielle Pinner, Gul Ahsan, Kimberly Gilmour, Giovanna Lucchini, Sarah Inglott, William Mifsud, Robert Chiesa, Karl S. Peggs, Lucas Chan, Farzin Farzaneh, Adrian J. Thrasher, Ajay Vora, Martin Pule, and Paul Veys. Science Translational Medicine  25 Jan 2017: Vol. 9, Issue 374, eaaj2013

Read the article in Science Translational Medicine

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2017
First report of efficacy of gene therapy for Sickle Cell Disease
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First report of efficacy of gene therapy for Sickle Cell Disease

Successful lentivirus-mediated transfer into autologous hematopoietic stem cells of an anti-sickling β-globin gene is therapeutic in Sickle Cell Anemia.

Ribeil JA, Hacein-Bey-Abina S, Cavazzana M, et al. N Engl J Med. 2017 Mar 2;376(9):848-855. doi: 10.1056/NEJMoa1609677.

Read the article in the New England Journal of Medicine

Photo courtesy of Université Paris Descartes

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2017
FDA Approval of Kymriah
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FDA Approval of Kymriah

FDA approval of Tisagenlecleucel (Kymriah®, Novartis Pharmaceuticals Corp.), an innovative gene therapy for certain pediatric and young adult patients with some forms of acute lymphoblastic leukemia.

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Read ASGCT's news story

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2017
FDA Approval of Yescarta
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FDA Approval of Yescarta

FDA approval of axicabtagene ciloleucel (Yescarta®, Kite Pharma, Inc/Gilead), the first CAR T cell therapy for adult patients for adults with relapsed or refractory large B-cell lymphoma and second-ever approved CAR T cell therapy.

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Read the ASGCT news story

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2017
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Effective lentivirus-mediated gene transfer for spinomuscular atrophy – important alternative to approved and effective anti-sense oligonucleotide therapy with Nusinersen (Spinraza).

Jerry R. Mendell, M.D., Sukumar Nagendran, M.D., et al. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. 2017, N Engl J Med 2017; 377:1713-1722.

Read the article in the New England Journal of Medicine

Photo courtesy of Nationwide Children's

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2017
LAMB3 gene replacement therapy for epidermolysis bullosa
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LAMB3 gene replacement therapy for epidermolysis bullosa

Retrovirus-mediated gene transfer for gene therapy of junctional epidermolysis bullosa, a rare disorder caused by mutations in genes encoding the basement membrane component laminin-332.  Grafted genetically modified skin keratinocytes regenerated normal skin at multiple sites in a single patient with epidermolysis bullosa. 

Tobias Hirsch, Tobias Rothoeft, et al. Regeneration of the entire human epidermis using transgenic stem cells. Nature 2017 551, 327–332 (2017).

Read the article in Nature

Photo courtesy of CMR Unimore, Stefania Bettinelli

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2017
FDA Approval of Luxturna
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FDA Approval of Luxturna

FDA approval of voretigene neparvovec-rzyl (Luxturna®,Spark Theraputics), the first gene therapy approved for an inherited disease; i.e. retinal dystrophies LCA2 and RP20.

Read the FDA press release

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Photo courtesy of Spark Therapeutics

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2017
Efficacy of AAV gene therapy in Hemophilia A
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Efficacy of AAV gene therapy in Hemophilia A

Single infusions into men with severe hemophilia A of an AAV vector expressing a B-domain-deleted human factor VIII (AAV5-hFVIII-SQ) in nine men with severe hemophilia A produced sustained correction of the factor VIII deficiency and marked reduction in bleeding episodes.

Savita Rangarajan, M.B., B.S., Liron Walsh, M.D., ……. K. John Pasi, M.B., Ch.B., Ph.D. AAV5–Factor VIII Gene Transfer in Severe Hemophilia AN Engl J Med 2017; 377:2519-2530.

Read the article in the New England Journal of Medicine

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2024

Breakthroughs in Muscular Dystrophy

November 19-20, 2024 | Chicago, IL

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