Timeline of key events in biotechnology

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Developed by Spark Therapeutics and Novartis, the drug (Voretigene neparvovec/Luxturna) aims to correct restore vision in patients born with Leber congenital amaurosis, a rare genetic retinal disease. Those with the condition have mutations in both copies of the RPE65 gene which cause sight loss from an early age and eventually causes blindness. The treatment aims to provide a working copy of the RPE gene. 2018-11-23T00:00:00+0000He Jiankui, a genome-editing researcher at Southern University of Science and Technology of China, reported transplanting embryos into a woman that he had edited with CRISPR-Cas9 to disable a gene called CCR5, to disable the genetic pathway HIV uses to infect cells. More than 100 Chinese biomerical researchers condemned the experiment and called on Chinese authorities to investigate the case and introduce strict regulations. 2018-11-24T00:00:00+0000The project, led by Genomics England in partnership with the NHS, sequenced the DNA of both cancer patients and those with rare disorders. Overall 15,000 cancer patients had their DNA analysed, half of whom went on to take part in a clinical trial or receive targeted treatment. One in four participants with rare diseases who had their genomes sequenced received a diagnosis for the first time, thereby paving the way to getting effective treatment. All the sequencing was carried out by the Wellcome Sanger Institute, near Cambridge, in laboratories run by Illumina, a Californian biotechnology company. 2018-12-05T00:00:00+0000A team of managed to reprogramme the skin cells into dendritic cells. These immune cells search the body's tissues for foreign particles, such as bacteria, viruses or cancer cells, and devour them. The team identified three essential proteins (PU.1, IRF8 and BATF3) that are required to transform the the skin cells into dendritic cells. The process takes only 9 days and opens the possibility of developing novel dendritic cell-based immunotherapies against cancer. The research was published in FF Rosa et al, 'Direct reprogramming of fibroblasts into antigen-presenting dendritic cells', Science Immunology, 3/30 (2018) eaau4292, DOI: 10.1126/sciimmunol.aau42922018-12-07T00:00:00+0000The efficacy of the technique was tested in mice genetically modified to have only one working copy of genes known to regulate satiety (Sim1) and hunger (MC4R). CRISPRa was shown to increase the expression of the genes in the mice. Mice that had missing one copy of the Sim1 gene that received CRISPRa injections were found to maintain a healthy body weight like normal mice, whereas those that did not get the injections could not stop eating and developed severe obesity on a normal diet. The research was published in N Matharu et al, 'CRISPR-mediated activation of a promoter or enhancer rescues obesity caused by haploinsufficiency', Science 362/6420 (2018) eaau0629, DOI: 10.1126/science.aau06292018-12-14T00:00:00+0000Scientists found that it was possible to stop tumour growth in both tissue culture and in a mouse by disabling the tumour gene, NRF2, that helps the tumours develop resistance to drugs. They achieved this by using the CRISPR-Cas9 gene editing system. The work was led by Eric Kmiec at the Gene Editing Institute of the Helen F Graham Cancer Center and Research Institute, USA. It was published in P Bialk, Y Wang, K Banas, E B Kmiec, ' Functional Gene Knockout of NRF2 Increases Chemosensitivity of Human Lung Cancer A549 Cells In Vitro and in a Xenograft Mouse Model', Molecular Therapy Oncolytics', 11 (Dec 21 2018), DOI:https://doi.org/10.1016/j.omto.2018.10.0022018-12-21T00:00:00+0000A team of scientists managed to engineer mice to express Cas9 and a DNA sequence needed for the gene drive, called a cassette, which encoded a guide RNA that targets a sequence in the TYR gene which affects the mouse coat colour. This provided a means of tracking the frequency of the genetic modification over several generations of mice. The work was published in HA Grunwald et al. 'Super-Mendelian inheritance mediated by CRISPR–Cas9 in the female mouse germline', Nature, January 23, 2019.2019-01-23T00:00:00+0000The patient received a stem-cell transplant that replaced their white blood cells with HIV-resistant versions. The cells were taken from a donor who had two copies of a mutation in the CCR5 gene that confers resistance to HIV infection. The CCR5 gene codes for a receptor on white blood cells involved in the body's immune response. HIV normally binds to these receptors and attacks the cell. By removing the gene the receptors stop working normally. The patient was given the treatment as part of therapy for blood cancer. He was able to stop taking antiretroviral drugs after 16 months, and 18 months later had no sign of the virus. The research was published in RK Gupta, et al, Nature (2019), DOI 10.1038/s41586-019-1027-42019-03-05T00:00:00+0000Born in South Africa, Brenner was a geneticist and biologist who shared the 2002 Nobel Prize for Medicine for helping to discover how genes regulate tissue and organ development. Using the roundworm Caenorhabditis elegans as a model organism, Brenner demonstrated that triplets of nucleotides within RNA encode the individual amino acids of a protein, and signals when protein manufacture should stop. 2019-04-05T00:00:00+0000Developed by a team of researchers led by Brian Sorrentino at St. Jude Children’s Research Hospital, the gene therapy was given to eight infants. The treatment involved collecting bone marrow from the patients and then using the lentiviral vector to insert a correct copy of the IL2RG gene into the genome of patients’ blood stem cells. The research was published in E Momcartz, et al, 'Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1', New England Journal of Medicine, 380: (2019): 1525-34, 2019-04-19T00:00:00+0000The recommendation was based on advice from WHO's 18 member expert advisory committee on human genome editing. 2019-07-30T00:00:00+0000Known as 'whole exome sequencing', the test makes it possible to scan for around 20,000 human genes in just 27 hours rather than 10 days as was the case previously. The test was developed by South West Genomic Laboratory Hub and enable quick diagnoses of approximately 5,000 rare conditions like cystic fibrosis. 2019-10-01T00:00:00+0000The advantage of the technique is it enables scientists to disable specific genes and correct harmful mutations by providing cells with fresh DNA strands with to repair the cut. Unlike Crispr-Cas9, which chops target DNA in half, prime editing nicks it and then writes a new section of DNA into the specified region. The new procedure was published in AW Anzalone et al, 'Search-and-replace genome editing without double-strand breaks or donor DNA', Nature (2019), https://doi.org/10.1038/s41586-019-1711-42019-10-21T00:00:00+0000Developed by Biogen, the drug, aducanumab, targets a protein called amyloid that forms abnormal deposits in the brains of people with Alzheimer's. The company originally discontinued work on the drug in March 2019 after disappointing trial results. 2019-10-22T00:00:00+0000He Jiankui, who used the gene-editing technology to make twin babies immune to HIV by altering gene CCR5, was sentenced to three years in prison by a Chinese court and fined around $430,000. 2019-12-30T00:00:00+0000The experiments were carried out by a team at the US Army Medical Research Institute of Chemical Defense. The treatment used a gene, delivered via a virus, that codes for an enzyme made in the liver called PON1 which enables the body to find and break down toxic nerve agents in the body to make them harmless. All mice injected with the gene therapy developed immunity to nerve agents for five month. Should the treatment prove successful in humans it could help provide immunity to common nerve agents like tabun, sarin, cyclosarin and soman. The research was published in V Betapudi et al, 'Gene therapy delivering a paraoxonase 1 variant offers long-term prophylactic protection against nerve agents in mice', Science Translational Medicine, 12/527, eaay0356, DOI: 10.1126/scitranslmed.aay0356.2020-01-22T00:00:00+0000
Date Event People Places Sciences
23 Nov 2018Gene therapy approved in Europe for treatment of patients with vision loss linked to genetic mutation Novartis, Spark TherapeuticsGene therapy, Ophthalmology
24 Nov 2018First gene-edited babies announced by Chinese scientistJiankuiSouthern University of Science and Technology of ChinaCRISPR-Cas9, Reproduction
5 Dec 2018Genomics England completed sequencing 100,000 whole genomesCaulfieldSanger Institute, IlluminaDNA sequencing, Genomics
7 Dec 2018Mouse and human skin cells reprogrammed into immune cells to fight cancerRosa, Pires, Kurtochkin, Ferreira, Gomes, Palma, Shaiv, Solanas, Azenha, Papatsenko, Schulz, Reis e Sousa, Carlos-Filipe Lund University, Skolkovo Institute of Science and Technology, Francis Crick InstituteCancer immunotherapy, Stem cells
14 Dec 2018New gene modification technique (CRISPRa) makes it possible to increase expression of its target geneMatharu, Rattanasopha, Tamura, Maliskova, Wang, Bernard, Hardin, Eckalbar, Vaisse, AhituvUniversity of California San FranciscoCRISPR-Cas9
21 Dec 2018CRISPR-Cas9 editing helped restore effectiveness of first-line chemotherapies for lung cancerKmiec, Bialk, Wang, Hanas Helen F Graham Cancer Center and Research InstituteCRISPR-Cas9, Oncology
23 Jan 2019CRISPR-Cas9 used to control genetic inheritance in miceGrunwald, Gntz, Poplawski, Xu, Bier, CooperUniversity of California San DiegoCRISPR-Cas9, Genetics, Transgenic animals
5 Mar 2019Second patient reported free of HIV after receiving stem-cell therapyGuptaUniversity of CambridgeStem cells, Gene therapy
5 Apr 2019Sydney Brenner diedBrennerLaboratory of Molecular BiologyGenetics
19 Apr 2019Gene therapy shown to be promising in treating infants born with X-linked severe combined immunodeficiency (SCID-X1)Mamcarz, Zhou, Lockey, Abdelsamed, Cross, Kang, Ma, Condori, Dowdy, Triplett, Maron St. Jude Children’s Research HospitalGene therapy, Stem cells
30 Jul 2019World Health Organisation called on countries to ban experiments that would lead to more gene-edited babies  CRISPR-Cas 9, Genetic editing, Reproduction
Oct 2019NHS introduced new fast-track DNA test to scan for rare diseases in babies and children South West Genomic Laboratory HubDNA Sequencing, Genomics, Genetics
21 Oct 2019New DNA editing technique called 'prime editing' publishedAnzalone, Randolph, Davis, Sousa, Koblan, Levy, Chen, Wilson, Newby, Ranguram, LiuMassachusetts Institute of Technology, Harvard UniversityCrispr-Cas9, Gene editing,
22 Oct 2019Monoclonal drug originally dismissed for treating Alzheimers got new green light from FDA for testing for the disease BiogenMonoclonal antibodies
30 Dec 2019Chinese scientist convicted for using CRISPR-Cas9 in human babiesJiankuiSouthern University of Science and Technology of ChinaCrispr-Cas9, Gene editing,
22 Jan 2020Mice experiments indicate gene therapy could provide long-lasting protection against different chemical nerve agentsBetapudi, Goswami, Silayeva, Doctor, ChilukuriUS Army Medical Research Institute of Chemical DefenseGene therapy

23 Nov 2018

Gene therapy approved in Europe for treatment of patients with vision loss linked to genetic mutation

24 Nov 2018

First gene-edited babies announced by Chinese scientist

5 Dec 2018

Genomics England completed sequencing 100,000 whole genomes

7 Dec 2018

Mouse and human skin cells reprogrammed into immune cells to fight cancer

14 Dec 2018

New gene modification technique (CRISPRa) makes it possible to increase expression of its target gene

21 Dec 2018

CRISPR-Cas9 editing helped restore effectiveness of first-line chemotherapies for lung cancer

23 Jan 2019

CRISPR-Cas9 used to control genetic inheritance in mice

5 Mar 2019

Second patient reported free of HIV after receiving stem-cell therapy

5 Apr 2019

Sydney Brenner died

19 Apr 2019

Gene therapy shown to be promising in treating infants born with X-linked severe combined immunodeficiency (SCID-X1)

30 Jul 2019

World Health Organisation called on countries to ban experiments that would lead to more gene-edited babies

Oct 2019

NHS introduced new fast-track DNA test to scan for rare diseases in babies and children

21 Oct 2019

New DNA editing technique called 'prime editing' published

22 Oct 2019

Monoclonal drug originally dismissed for treating Alzheimers got new green light from FDA for testing for the disease

30 Dec 2019

Chinese scientist convicted for using CRISPR-Cas9 in human babies

22 Jan 2020

Mice experiments indicate gene therapy could provide long-lasting protection against different chemical nerve agents

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