The Pharma Data

JUNE 7, 2023

Acuitas’ LNP technology will support Bayer’s in vivo gene editing and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver. LNPs are spheric drug delivery bodies which can be equipped with therapeutic payloads for intracellular delivery. Source link: [link]

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

XTalks

AUGUST 5, 2020

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level.

Gene Expression 112

Gene Expression Development RNA Drugs 112

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. . — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105

Drug Discovery World

MARCH 9, 2023

The US Food and Drug Administration (FDA) has cleared Intellia Therapeutics’ Investigational New Drug (IND) application for NTLA-2002 for the treatment of hereditary angioedema (HAE). This includes the measurement of plasma kallikrein protein levels and clinical activity as determined by HAE attack rate measures.

In-Vivo 52

In-Vivo Gene Editing Gene Trials 52

Drug Discovery World

APRIL 25, 2024

Cell line development is an essential underpinning of the drug development process, enabling teams to test, optimise and manufacture therapeutics at a commercial scale. For drug discovery and development, cell lines allow researchers to test the efficacy of therapeutics before moving into in vivo studies.

Development 59

Development Protein In-Vivo Life Science 59

Drug Discovery World

JANUARY 12, 2023

Oksana Sirenko looks at the advantages and limitations of using organoids from human cell sources in drug development and drug screening. . The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. What are organoids? .

Gene Editing 98

Gene Editing Gene Drugs In-Vivo 98

The Pharma Data

DECEMBER 10, 2020

The vaccine targeted both the inner nucleocapsid (N) and the outer spike (S) proteins of the virus to maximize the immune response. The vaccine targeted both the inner nucleocapsid (N) and the outer spike (S) proteins of the virus to maximize the immune response.

Vaccination 52

Vaccination Vaccine Immune Response Protein 52

Drug Discovery World

NOVEMBER 15, 2022

Anita Ramanathan outlines four synthetic biology approaches that are improving and accelerating drug discovery. . It has made its way into almost every aspect of drug discovery, whether it’s exploring basic biology, modelling diseases, identifying and validating targets, or performing drug screens. .

DNA 98

DNA Drugs Antibody Engineer 98

Drug Discovery World

JANUARY 23, 2023

Strand’s mRNA therapeutics enable precise control of the location, timing, intensity and duration of therapeutic protein expression for improved efficacy and lower toxicity. Our team has been focused on engineering mRNA so that it’s only used to express cargo protein in certain cells regardless of where the molecule ends up in the body.

RNA 98

RNA Genome Genomics Engineer 98

Drug Discovery World

SEPTEMBER 6, 2022

There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. Among these include Zolgensma, Novartis’ gene therapy drug for spinal muscular atrophy (SMA), which made headlines in the UK for its £1.79 billion, compared to $19.9 million price point per dose. Cancer is king .

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

Drug Discovery World

DECEMBER 15, 2022

Inducing double strand breaks at unintended locations in the genome can lead to off-target genetic modifications that are difficult to predict and potentially deleterious. Complicating matters even more, researchers currently lack effective and reliable methods for detecting and measuring off-target genomic events.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

NOVEMBER 8, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting.

Antibody 64

Antibody Engineer Protein Scientist 64

Drug Discovery World

JANUARY 17, 2024

Challenges in the Oncology Clinical Trials Space The oncology market is a key growth driver for the pharma industry, with global drug revenues expected to reach US$390 billion by 2027 and the annual number of new cancer cases set to grow from 18 to 27 million by 2040 1,2.

Medicine 59

Medicine DNA Genome Genomics 59

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. Years of development and fast-paced research have continued to unlock its potential, expanding how CRISPR can be used to treat, detect, and prevent common diseases such as cancer and Covid-19.

DNA 98

DNA Genome Genomics RNA 98

The Pharma Data

JANUARY 10, 2021

Nasdaq: EDIT), a leading genome editing company, today announced the U.S. Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. CAMBRIDGE, Mass., 11, 2021 (GLOBE NEWSWIRE) — Editas Medicine, Inc.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

The Pharma Data

OCTOBER 14, 2020

The AOP Orphan Group manufactures 80% of its drugs in Europe and has more than 20 subsidiaries and representative offices across the whole of Europe as well as in the United Arab Emirates and Israel. Food and Drug Administration granted Breakthrough Therapy designation to France-based Inventiva’s experimental NASH treatment lanifibranor.

Clinical Trials 52

Clinical Trials Clinical Trials Manufacturing Trials 52