Broad Institute

OCTOBER 23, 2024

The DNA change underlying Emma’s disorder is now known, thanks to years of work by an international team of scientists and physicians at the Broad Institute of MIT and Harvard, Northwestern University, University of Nantes, the Weizmann Institute of Science, and the Baylor College of Medicine.

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New Drug Approvals

MAY 17, 2025

Liu A (1 May 2019). Jump up to: a b New Drug Therapy Approvals 2023 (PDF). Food and Drug Administration (FDA) (Report). January 2024. Archived from the original on 10 January 2024. Retrieved 9 January 2024. ^ “Biogen’s antisense ALS drug shows promise in early clinical trial” FierceBiotech. Retrieved 25 April 2023.

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The Premier Consulting Blog

APRIL 13, 2025

In 2019, we published a detailed peer-review article outlining this process based on our long history of leveraging existing animal and human data to streamline drug development programs and reduce or eliminate animal testing under the 505(b)(2) pathway.[ 3] Salminen et al. link] [7] H.R.2565

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PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). It aims to correct the faulty instructions at the DNA level, straightforward, at least conceptually, because mutations are deletions, of part or all of the gene. That’s not the case for ALS.

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The Pharma Data

DECEMBER 23, 2020

” Dr. Stanley Plotkin , Professor Emeritus at The Wistar Institute, said, “INOVIO’s DNA vaccine appeared to be quite safe with few significant reactions but yet induced both antibody and T cell responses to SARS-CoV-2.” About INOVIO’s DNA Medicines Platform. mg and 2.0

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Broad Institute

JULY 10, 2023

At the Broad, Martin is also helping lead data analysis in two large international studies that are sequencing the DNA of people from Africa and Latin America to learn about the genetics of severe mental illness. That paper changed hearts and minds,” Neale, a co-author, said of the 2019 paper in Nature Genetics.

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Drug Target Review

OCTOBER 12, 2023

Prime editing, a pioneering genome editing technique, was applied, enabling the scientists to introduce a single DNA base substitution at a time and to assess individual variant function at the TSPAN14 AD risk locus. 2019 August 30 [cited 2023 October 6]; 11(233):20. References Hemonnot AL, Hirbec H, Hua J, Ulmann L.

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Drug Target Review

JUNE 9, 2023

Adaptive Biotechnologies and Genentech entered a partnership in 2019 to develop, manufacture and commercialise novel neoantigen directed T-cell therapies for the treatment of a broad range of cancers. While tumour cells share a majority of their DNA with healthy cells, they also carry numerous unique mutations.

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Drug Target Review

DECEMBER 4, 2023

2019 May 29;20(1). Upstart Element ratchets up race for cheaper DNA sequencing with a $200 genome [Internet]. Available from: [link] Navarro FCP, Mohsen H, Yan C, et al. Genomics and data science: an application within an umbrella. Genome Biology [Internet]. Available from: [link] Wosen J. 2023 [cited 2023 Nov 15].

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PLOS: DNA Science

MARCH 7, 2024

The researchers searched the DNA of humans and other primates for clues to how we lost our tails. They discovered that a bit of repetitious DNA, called an Alu sequence, inserted into a gene associated with tail development early in the ape lineage. ” The post How the Human Lost Its Tail appeared first on DNA Science.

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PLOS: DNA Science

APRIL 18, 2024

Gene #1: The Famous Case of Aliria from the Colombian Family In 2019. ” The post Mutations in Three Genes Protect Against Alzheimer’s appeared first on DNA Science. Rare variants of three well-studied genes appear to delay inherited forms of Alzheimer’s disease – by decades.

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The Pharma Data

OCTOBER 18, 2020

(NASDAQ:CDXC) today highlighted a new study published in The European Molecular Biology Organization Journal looking at the effect of nicotinamide riboside (NR) on maintaining telomeres, the protective regions at the end of DNA strands. Telomeres are “caps” at the end of chromosomes that protect DNA from getting worn away as cells replicate.

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The Pharma Data

AUGUST 27, 2020

Cancer is a disease of the genome, driven by genetic mutations within a tumour’s DNA. FoundationOne Liquid CDx analyses circulating cell-free DNA from a patient’s blood sample and uses massively parallel sequencing to detect the four main classes of genomic alterations. In 2019, Roche invested CHF 11.7

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Codon

OCTOBER 13, 2024

The chefs at Farma know that by embracing food technologies such as recombinant DNA, precision fermentation, and cell culture, food can become more nutritious and better for the planet. Since its doors opened to the public a few weeks ago, Farma has crusaded against the apocryphal purity of organic foods.

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The Pharma Data

JANUARY 5, 2021

So therapeutics that focus on RNA are designed to stop the DNA from coding for disease-causing proteins. On September 30, 2019, Ribometrix signed a strategic collaboration deal with Boston’s Vertex Pharmaceuticals. Targeting RNA is believed to be a way to develop therapeutics for so-called undruggable proteins.

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The Pharma Data

AUGUST 6, 2020

In addition, Inovio previously teamed up with Beijing Advaccine Biotechnology for work on its DNA vaccine candidate, INO-4800. Clover Biopharmaceuticals has an S-Trimer subunit vaccine candidate, SCB-2019 , which is being evaluated with two adjuvant systems by GlaxoSmithKline and Dynavax in phase 1 study, with results expected this month.

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The Pharma Data

OCTOBER 21, 2020

While the majority of participants (93%, 1,615) maintained their routine dosing schedule from December 2019 through the analysis end point of September 15, 2020, 129 participants (7%) in the ongoing clinical trials had a planned injection visit that was impacted by COVID-19. Source: GSK . Posted: October 2020.

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Agency IQ

OCTOBER 12, 2023

These 2020 bans stemmed from an April 2019 meeting , held as part of the standard renewal process for pesticide active substances, between experts from EU member states and EFSA. These statements emboldened member states to vote in favor of draft implementing acts denying renewal for both active substances in December 2019.

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The Pharma Data

JANUARY 24, 2021

Pamer joined the University of Chicago from Memorial Sloan Kettering Cancer Center in New York City, where he served as head of the Division of Subspecialty Medicine from 2011 to 2018 and director of the Center for Microbes, Inflammation and Cancer from 2010 to 2019.

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The Pharma Data

NOVEMBER 23, 2020

24, 2020 /PRNewswire/ — United Therapeutics Corporation (Nasdaq: UTHR) today announced the release of its initial Corporate Responsibility Report with primarily 2019 data and selected 2020 highlights, providing stakeholders important information regarding the company’s commitment to environmental, social, and governance (ESG) priorities.

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DrugBank

JUNE 27, 2023

In contrast, non-coding mutations have effects on gene expression, transcript stability, and the physical state of the DNA itself (e.g., 20 , 467–484 (2019). Coding mutations have a more direct effect through direct alteration of a gene product. by altering its accessibility to transcriptional machinery).  Tam, V.

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The Pharma Data

SEPTEMBER 1, 2020

In general, antigen tests have a high specificity, though are not as sensitive as molecular tests that amplify the target viral DNA or RNA sequence in order to generate a quantifiable signal to indicate the presence of the virus in a sample. In 2019, Roche invested CHF 11.7 billion in R&D and posted sales of CHF 61.5

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The Pharma Data

OCTOBER 14, 2020

Assistant Professor of Medical Sciences at Columbia University Vagelos College of Physicians and Surgeons, is designed to generate DNA aptamer-based anti-idiotypes to selected monoclonal antibodies identified in Dr. Ilya Trakht’s study. The study led by Dr. Sergei Rudchenko, Ph.D.,

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Codon

JUNE 5, 2025

” Four years earlier, in 2019, Gray had become the first patient with sickle cell anemia — a genetic disorder that causes red blood cells to become sticky and rigid — to receive an experimental treatment using CRISPR genome editing. While crossing Trafalgar Square, Gray paused briefly to reflect on her experience.

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Elrig

APRIL 29, 2024

Srinivasan has led the development of multiple computational pipelines to process data from different next generation sequencing techniques with applications in oncology, genome editing systems including CRISPR-Cas mediated DNA editing, and ADAR-mediated RNA editing.

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Alta Sciences

APRIL 17, 2024

Ketamine is a dissociative anesthetic approved in 2019 by the U.S. Several viral vectors are being used currently, in addition to non-viral vectors, such as oligonucleotides, naked DNA, and lipoplexes and polyplexes. Food and Drug Administration (FDA) as a nasal spray called esketamine, for treatment-resistant depression.

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The Pharma Data

JULY 11, 2021

The complete results were based on data collected between May 2016 and October 2019 and included 109 rollover patients who were originally evaluated in the HELP Study, and 103 eligible non-rollover patients who did not participate in the initial study but had experienced at least one HAE attack within the previous 12 weeks.

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Agency IQ

AUGUST 11, 2023

Assessment of one of the impurities in glyphosate could not be undertaken without more literature regarding its clastogenic potential, that is its capacity to cause DNA breakages. In fact, EU law was altered in 2019 explicitly to allow multiple member states to serve as rapporteurs on this risk assessment.

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Agency IQ

AUGUST 11, 2023

By early 2019, it became apparent that NDMA was not the only nitrosamine forming in API synthesis. This latter category is “not predicted to be metabolically activated via an α-hydroxylation pathway,” or would not be predicted to react with DNA. To ensure the safety of the U.S. Read AgencyIQ’s analysis of the nitrosamine guidance here.]

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The Pharma Data

DECEMBER 27, 2020

Following changes to our leadership team in late 2018 and early 2019, we have sought to transform Lineage into the preeminent allogeneic cell transplant company. Notably, we never modify the DNA of these cells, which avoids some of the safety concerns that have been reported with certain gene-editing technologies.

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Agency IQ

JULY 26, 2024

in equipment) for products that are more complex or aren’t as well-characterized, such as antibody drug conjugates or recombinant DNA products, will be considered higher risk than they would if the change was made to the production of standard, small molecule products. Any CMC changes (e.g.,

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The Pharma Data

APRIL 30, 2021

Under the multi-year agreement, AbbVie will utilize Caribou’s next-generation Cas12a CRISPR hybrid RNA-DNA genome editing and cell therapy technologies to research and develop two new CAR-T cell therapies directed to targets specified by AbbVie. Refer to the Key Product Revenues schedules for further details. Full-Year 2021 Outlook.

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PLOS: DNA Science

MAY 18, 2023

Perhaps more telling, I recently discovered a previously unknown branch of my own family tree that includes a dozen half-siblings that I know of, from a DNA test that I didn’t even remember taking. It was eerily like forensic scientists using familial DNA to track down serial killers. Which one was our biodad?

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Drug Target Review

JULY 9, 2024

Bowel cancer is the fourth most common cancer in the UK, accounting for 11 percent of all new cancer cases from 2017 to 2019 1 , and is the second most common cause of cancer death in the UK, accounting for 10 percent of all cancer deaths in the same time period.

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Drug Target Review

SEPTEMBER 4, 2023

5 Cytology-based profiling can facilitate antibiotic discovery efforts and, these assays assess cell cycle progression, nuclear and mitochondrial DNA content, mitochondrial DNA replication, nuclear DNA damage, mitochondrial membrane potential, and lysosome structure and function. PE 740.238 Bryant, M. Sebastian, B., Adriana, T.,

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PLOS: DNA Science

OCTOBER 5, 2023

Amy in June 2019. Bess’s mom died of BC in October, 2019. And Tobie was just diagnosed, late 2019. The post How Breast Cancer Reunited Six High School Friends appeared first on DNA Science. For what it’s worth, our school’s history wasn’t always something to be proud of. Wendy in late 2018.

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Broad Institute

DECEMBER 13, 2023

One cell at a time Slide-seq, reported in 2019 , involves transferring slices of tissue onto arrays of special beads, each one tagged with a DNA barcode that identifies its location in the array.

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