Timeline of key events in biotechnology

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P. 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+0000C T Charlesworth et al, 'Identification of Pre-Existing Adaptive Immunity to Cas9 Proteins in Humans', bioRXiv (2018), https://doi.org/10.1101/2433452018-01-05T00:00:00+0000The test hunts for 16 gene that regularly arise in cancer and 8 proteins released by tumours. It was trialled in 1,005 patients with cancers of the ovary, liver, stomach, pancreas, oesophagus, colon, lung or breast that had not yet spread to other tissues. J.D. Cohen et al, Detection and localization of surgically resectable cancers with a multi-analyte blood test', Science, 18 Jan 2018, eaar3247, DOI: 10.1126/science.aar3247 2018-01-17T00:00:00+0000Z. Liu et al, 'Cloning of macaque monkeys by somatic cell nuclear transfer', Cell (2018), http://dx.doi.org/10.1016/j.cell.2018.01.020 2018-01-24T00:00:00+0000Sulston was a biologist. He played a central role in sequencing the genome of the Caenorhabditis elegans, a transparent nematode (roundworm). It was the first animal to have its genome sequenced. Based on his work with the nematode Sulston helped set up the project to sequence the human genome which he did as director of the Sanger Centre. The first draft of the human genome sequence was completed in 2000. In 2002 he shared the Nobel Prize for identifying how genes regulate the life cycle of cells through apoptosis. 2018-03-09T00:00:00+0000Twenty-two patients were treated in the trial over 42 months in six centres around the world. The treatment consisted of taking immature stem cells from the patient's bone marrow and using a harmless virus to infect the cells with a copy of the normal globin gene. The new genetically altered cells were then reintroduced into the patient's bloodstream after their marrow had been cleared of diseased cells using chemotherapy. Nine out of the twenty-two patients severe beta thalassemia were able to cut down on the number of transfusions they needed by 74%, with three of them no longer needing any transfusions at all. The same was true of the twelve out of thirteen patients with less severe thalassemia. A A Thompson et al, 'Gene therapy in patients with transfusion-dependent beta thalassemia', New England Journal of Medicine, 378 (2018), 1479-93.2018-04-19T00:00:00+0000
Date Event People Places Sciences
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
5 Jan 2018Researchers identify pre-existing antibodies targeting CAS9 proteins raising possibility of immune responses undermining utility of CRISPR-Cas9 for gene therapyCharlesworth, Deshpande, Dever, Dejene,Gomez-Ospina, Mantri, Pavel-Dinu, Camarena, Weinberg, PorteusStanford UniversityGene therapy, Gene editing, CRISPR-Cas9
17 Jan 2018Blood test detecting mutated DNA and proteins released by tumours shown to pick up early signs of 8 common cancersCohen, Yuxuan Wang, Thoburn, Afsari, Danilova. Douville, Javed, Wong, Mattox, Hruban, Wolfgang, Goggins, Molin, Wang, Roden, Klein, Ptak, Dobbyn, Schaefer, Silliman, Popoli, Vogelstein, Browne, Schoen, Brand, Tie, Gibbs, Wong, Mansfield, Jen, Hanash, FalcJohns Hopkins UniversityDiagnostics, Oncology
24 Jan 2018Chinese scientists cloned the first monkeys by somatic cell nuclear transferLiu, Cai, Wang, Nie, Zhang, Xu, Xiatong, Zhang, Lu, Poo, SunChinese Academy of SciencesCloning
9 Mar 2018John E Sulson diedSulstonLaboratory of Molecular Biology, Sanger InstituteCell, Genetics, DNA sequencing
19 Apr 2018Gene therapy shown to be promising treatment in clinical trials for beta thalassemiaThompson, Walters, Kwiatkowski, Rasko, Ribeil, Hongeng, Magrin, Schiller, Payen, Smeraro, Moshous, LefrerNorth Western University, University of California San Francisco, University of California Los Angeles, University of Sydney, University of Paris, Harvard University, Mahidol University, German Cancer Research CentreGene therapy

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

5 Jan 2018

Researchers identify pre-existing antibodies targeting CAS9 proteins raising possibility of immune responses undermining utility of CRISPR-Cas9 for gene therapy

17 Jan 2018

Blood test detecting mutated DNA and proteins released by tumours shown to pick up early signs of 8 common cancers

24 Jan 2018

Chinese scientists cloned the first monkeys by somatic cell nuclear transfer

9 Mar 2018

John E Sulson died

19 Apr 2018

Gene therapy shown to be promising treatment in clinical trials for beta thalassemia