Timeline of key events in biotechnology

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The drug CTL019 (tisagenlecleucel) was developed by Novartis. Treatment involves removing T cells from the patient and genetically modifying them to increase their capacity to bind to tumour cells in order to get the immune sytem to attack the tumours. It is targeted at children and young adults from three to 25 years old who have not responded to traditional treatments.2017-07-12T00:00:00+0000M. Hong, N. Marti-Gutierrez, S-W Park, et al, 'Correction of a pathogenic gene mutation in human embryos', Nature, doi:10.1038/nature233052017-08-02T00:00:00+0000The drug Kymriah (tisagenlecleucel) is the first gene therapy to become available in the US. 2017-08-30T00:00:00+0000UK scientists modified 41 embryos shortly after fertilisation. N.M.E. Fogarty et al, 'Genome editing reveals a role for OCT4 in human embryogenesis', Nature, doi:10.1038/nature240332017-09-20T00:00:00+0000227 patient study showed drug from Alnylam Pharmaceuticals had benefits for rare hereditary disorder - ATTR amyloidosis with polyneuropathy, a rare disorder.2017-09-20T00:00:00+0000L. Xu, et al, 'Trispecific broadly neutralizing HIV antibodies mediate potent SHIV protection in macaques', Science, 20 September 2017, Science, 20 Sep 2017, eaan8630, DOI: 0.1126/science.aan86302017-09-20T00:00:00+0000The drug, a form of immunotherapy, is a PDL1 checkpoint inhibitor.2017-09-20T00:00:00+0000P. Liang, et al, 'Correction of beta-thalassemia mutant by base editor in human embryos', Protein and Cell (2017), doi.org/10.1007/s13238-017-0475-6.2017-09-23T00:00:00+0000Total of 17 boys treated in clinical trial, of which 15 showed marked improvement. Treatment used a modified form of HIV as the vector for infusing corrective genes to generate glial cells. F. Eichler, C. Duncan etl al, 'Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy', NEJM, DOI: 10.1056/NEJMoa17005542017-10-04T00:00:00+0000D.B.T. Cox, J.S. Gootenberg, O.O. Abudayyeh, B.Franklin, M.J. Kellner, et al, 'RNA editing with CRISPR-Cas13', Science (25 Oct 2017), eaaq0180, DOI: 10.1126/science.aaq01802017-10-25T00:00:00+0000N.M. Gaudelli, A. Komor, H. A. Rees, M. S. Packer, A. H. Badran, D. I. Bryson, D. R. Liu, 'Programmable base editing of A-T to G-C in genomic DNA without DNA cleavage', Nature (2017), doi:10.1038/nature24644. 2017-10-25T00:00:00+0000Research conducted by team led by Laurence Zitvogel at the Gustave Roussy Cancer Institute. B.Routy, 'Gut microbiome influences efficacy of PD-1–based immunotherapy against epithelial tumors', Science, eaan3706 DOI: 10.1126/science.aan37062017-11-02T00:00:00+0000Collaborative study led by Jennifer Wargo and Vancheswaran Gopalakrishnan. V. Gopalakrishnan et al, 'Gut microbiome modulates response to anti–PD-1 immunotherapy in melanoma patients', Science, 2 Nov 2017, eaan4236, DOI: 10.1126/science.aan42362017-11-02T00:00:00+0000Treatment was given to Leopold Fotso, a 33 year old man at King's College London using natalizumab, a drug licensed for multiple sclerosis. The trial is being led by Oliver Howe. 2017-11-03T00:00:00+0000Test developed to detect tumour DNA. Study investigated blood samples from 161 patients with stage 2 and 3 melanoma who had received surgery. Results showed that skin cancer was much more likely to return within a year of surgery in patients with faults in either BRAF or NRAS genes. R J Lee et al, 'Circulating tumor DNA predicts survival in patients with resected high-risk stage II/III melanoma', Annals of Oncology, mdx717, https://doi.org/10.1093/annonc/mdx7172017-11-03T00:00:00+0000CTJ van Velthoven, A de Morree, I M Enger, J O Brett, T A Rando, 'Transcriptional Profiling of Quiescent Muscle Stem Cells In Vivo', Cell Reports, 21/1 (2017), 1994-2004. 2017-11-14T00:00:00+0000Discovery made as a result of study of 177 members of the Old Order of Amish community in Indiana. S. Khan, et al, 'A null mutation in SERPINE1 protects against biological aging in humans', Science Advances, 3/11 (2017), DOI: 10.1126/sciadv.aao16172017-11-15T00:00:00+0000Treatment involved editing the patient's DNA using zinc finger nucleases technique. it was carried out on Brian Madeux, 44 year old man suffering from Hunter syndome, a metabolic disorder. Treatment was carried out by Paul Harmatz and his team at UCSF Benioff Children's Hospital. 2017-11-16T00:00:00+0000Patients with severe haemophilia A received a single infusion of a copy of a missing gene that allows their cells to produce Factor VIII, a protein needed to stop bleeding. This was delivered using an adeno-associated virus vector. The patients were enrolled between September 2015 and April 2016 into one of three dose cohorts at five sites across the UK. The five UK trial sites included: The Royal London, Guys and St Thomas', Birmingham, Cambridge and Hampshire hospitals. At 54 week follow-up 85% of the patients were found to have normal or near normal Factor VIII levels. Thirteen of the patients no longer needed their previously regular treatment. S. Rangarajan, et al, 'AAV5–Factor VIII Gene Transfer in Severe Hemophilia A', New England Journal of Medicine (9 Dec 2017), DOI: 10.1056/NEJMoa1708483.2017-12-09T00:00:00+0000
Date Event People Places Sciences
12 Jul 2017US FDA Oncologic Drugs Advisory Committee recommended the approval of the first adoptive cell therapy (CAR-T cell therapy) for B cell acute leukaemia Novartis, University of PennsylvaniaCancer immunotherapy, Oncology, Adoptive cell therapy, Gene therapy
2 Aug 2017Research published demonstrating possibility of editing gene defect in pre-implanted human embryos for preventing inherited heart diseaseHong, Marti-Gutierrez, Park, Mitalipov, Kaul, Kim, Amato, BelmonteOregon Health & Science University, Salk Institute, Center for Genome Engineering, Seoul National University, China National GeneBank,CRISPR-Cas9, Reproduction, Cardiovascular, Gene editing
30 Aug 2017USA FDA approved CAR-T therapy for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia Novartis, University of PennsylvaniaCancer immunotherapy, Oncology, Adoptive cell therapy, Gene therapy
Sep 2017DNA of human embryos edited using CRISPR-Cas9 to study cause of infertilityFogarty, McCarthy, Snijders, Powell, Kubikova, Blakeley, Lea, Elder, Wamaitha, Kim, Maciulyte, Kleinjung, Kim, Wells, Vallier, Bertero, Turner, NiakanFrancis Crick Instiitute, Cambridge University, Oxford University, Seoul National UniversityCRISPR-Cas9, Gene editing, Reproduction
20 Sep 2017Positive results reported from clinical trial with RNAi drug AlynlamRNA interference
20 Sep 2017Combination of three monoclonal antibodies reported to protect monkeys from HIV Xu, Pegu, Rao, Doria-Rose, Beninga, McKee, Lord, Wei, Deng, Louder, Schmidt, Mankoff, Wu, Asokan, Beil, Lange, Leuschner, Kruip, Sendak, Kwon, Zhou, Chen, Bailer, Wang, Choe, Tartaglia, Barouch, O’Dell, Todd, Burton, Roederer, Connors, Koup, Kwong, YangSanhofi, National Institute of Allergy and Infectious Disease, Harvard University, Massachusetts Institute of Technology, Scripps Research Institute, Monoclonal antibodies, Infectious diseases
20 Sep 2017Nivolumab (Opdivo®) made available for NHS patients with advanced lung cancer Honko, FreemanMedarex, Bristol-Myers Squibb, Ono PharmaceuticalImmune checkpoint inhibitors, Immunotherapy, Monoclonal antibodies
23 Sep 2017Chinese researchers report correction of gene linked to beta thalassaemia, inherited blood disorder, in human embryos using base editing techniqueLiang, Ching, Sun, Xie, Xu, Zhang, Xhiong, Ma, Liu, Wang, Fang, Songyang, Zhou, HuangSun Yat-sen University, Baylor College of MedicineCRISPR-Cas9, Gene editing, Reproduction
4 Oct 2017Gene therapy shown in clinical trials to halt progression of adrenoleukodystrophy, a fatal brain disease inherited by boys Eichler, Duncan, WilliamsHarvard University, Bluebird Bio, Boston Children’s Hospital Stem cells, Gene therapy
25 Oct 2017New CRISPR technique published for editing RNA providing means to fix genetic mutations without tampering with the genomeZhang, Cox, Gootenberg, Abudayyeh, Franklin, Kellner, Essletzbichler, Verdine, Joung, Lander, Belanto, Voytas, RegevMassachusetts Institute of Technology, University of MinnesotaCRISPR-Cas9, Gene editing, RNA
25 Oct 2017Base editing improvements announced for CRISPR technique, providing means to change individual chemical letters of DNA without need to cleave DNAGaudelli, Komor, Rees, Packer, Badran, Bryson, LiuMassachusetts Institute of Technology, Harvard UniversityCRISPR-Cas9, Gene editing
2 Nov 2017Cancer patients taking routine antibiotics before or soon after given PD-1 checkpoint inhibitor found to relapse quicker and have shorter survival timeZitvogel, KroemerInstitut Gustave RoussyCancer immunotherapy, Immune checkpoint inhibitors, Oncology, Microbiome
2 Nov 2017Experiments with mice show tumour growth can be reduced by giving faecal transplants from patients who positively responded to cancer immunotherapyWargo, GopalakrishnanMD Anderson Cancer Center, Institute Gustave-RoussyCancer immunotherapy, Immune checkpoint inhibitors, Oncology, Microbiome
3 Nov 2017First patient treated in first clinical trial using monoclonal antibody drug to treat schizophreniaHoweKing's College LondonMonoclonal antibodies
3 Nov 2017Research published demonstrating simple blood test can identify which patients are at most risk of skin cancer returningLee, Gremel, Marshall, Myers, Fisher, Dunn, Dhomen, Corrie, Middleton, Lorigan, MaraisUniversity of ManchesterOncology, DNA sequencing
14 Nov 2017Stem cells in the body found to have significantly different gene-expression profile than stem cells isolated and cultivated in the laboratory van Velthoven, de Morree, Enger, Brett, Rando, Stanford UniversityStem cells
15 Nov 2017Rare mutation of gene called Serpine 1 discovered to protect against biological ageing processKhan, Shah, Klyachko, Baldridge, Eren, Place, Aviv, Puterman, Lloyd-Jones, Heiman, Miyata, Gupta, Shapiro, VaughanNorthwestern University, University of British Columbia, New Jersey Medical School, Tohoku University, DNA sequencing, Genetics, Genomics
16 Nov 2017First patient receives therapy involving gene editing inside the bodyHarmatz, MadeuxUniversity of California San FranciscoGene therapy, Gene editing, Zinc fingers
9 Dec 2017Gene therapy shown to be safe and efficacious treatment for haemophilia A in British trialsRangarajan, Walsh, Lester, Perry, Madan, Laffan, Hua Yu, Vettermann, Pierce, Wong, PasiBarts Health NHS Trust, Queen Mary University, BioMarin PharmaceuticalGene therapy

12 Jul 2017

US FDA Oncologic Drugs Advisory Committee recommended the approval of the first adoptive cell therapy (CAR-T cell therapy) for B cell acute leukaemia

2 Aug 2017

Research published demonstrating possibility of editing gene defect in pre-implanted human embryos for preventing inherited heart disease

30 Aug 2017

USA FDA approved CAR-T therapy for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia

Sep 2017

DNA of human embryos edited using CRISPR-Cas9 to study cause of infertility

20 Sep 2017

Positive results reported from clinical trial with RNAi drug

20 Sep 2017

Combination of three monoclonal antibodies reported to protect monkeys from HIV

20 Sep 2017

Nivolumab (Opdivo®) made available for NHS patients with advanced lung cancer

23 Sep 2017

Chinese researchers report correction of gene linked to beta thalassaemia, inherited blood disorder, in human embryos using base editing technique

4 Oct 2017

Gene therapy shown in clinical trials to halt progression of adrenoleukodystrophy, a fatal brain disease inherited by boys

25 Oct 2017

New CRISPR technique published for editing RNA providing means to fix genetic mutations without tampering with the genome

25 Oct 2017

Base editing improvements announced for CRISPR technique, providing means to change individual chemical letters of DNA without need to cleave DNA

2 Nov 2017

Cancer patients taking routine antibiotics before or soon after given PD-1 checkpoint inhibitor found to relapse quicker and have shorter survival time

2 Nov 2017

Experiments with mice show tumour growth can be reduced by giving faecal transplants from patients who positively responded to cancer immunotherapy

3 Nov 2017

First patient treated in first clinical trial using monoclonal antibody drug to treat schizophrenia

3 Nov 2017

Research published demonstrating simple blood test can identify which patients are at most risk of skin cancer returning

14 Nov 2017

Stem cells in the body found to have significantly different gene-expression profile than stem cells isolated and cultivated in the laboratory

15 Nov 2017

Rare mutation of gene called Serpine 1 discovered to protect against biological ageing process

16 Nov 2017

First patient receives therapy involving gene editing inside the body

9 Dec 2017

Gene therapy shown to be safe and efficacious treatment for haemophilia A in British trials