Drug Discovery World

FEBRUARY 16, 2023

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. Aera’s technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes.

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Genetics Medicine Gene Editing In-Vivo 52

STAT News

MARCH 9, 2023

LONDON — Scientists at this year’s genome editing summit spent Tuesday showing the world just how far CRISPR -based medicines for treating human diseases have come in a decade. Continue to STAT+ to read the full story…

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Drug Discovery World

NOVEMBER 7, 2022

Genomic healthcare data is critical to identify disease risk, ancestry, traits and response to medicines and aids in the development of new targeted therapies – precision medicines. In April 2003, after its launch in October 1990, the project was completed, generating the first sequence of the human genome.

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Genome Genomics Development Genetics 98

Scienmag

MAY 26, 2022

— CRISPR has ushered in the era of genomic medicine. A line of powerful tools has been developed from the popular CRISPR-Cas9 to cure genetic diseases. ITHACA, N.Y.

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Gene Editing Genetic Disease Gene Genome 63

STAT News

SEPTEMBER 20, 2022

A new CRISPR startup — backed by some big names in venture capital — is planning to develop gene-editing treatments that can insert a genetic sequence of any length, at any location in the DNA strand, according to industry insiders and documents.

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Drug Discovery World

OCTOBER 10, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Gene Editing Genome Genomics Genetics 57

pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. At the moment, most CRISPR-based medicines rely on the modification of cells outside the body, that are then used as therapies. Jennifer Doudna. Benjamin Oakes.

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Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs 52

Drugs Gene Editing Genome Genomics 52

Drug Discovery World

OCTOBER 10, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Drug Discovery World

NOVEMBER 14, 2023

FH is one of the most common genetic conditions, affecting around one in 300 people globally. It is caused by a single gene mutation that impairs the body’s ability to mediate LDL cholesterol. Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments.

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XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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In-Vivo Gene Editing Trials Gene 52

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

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Drug Discovery World

JUNE 22, 2023

“This is part of our long-term business strategy of leveraging exosome-mediated delivery to create and enable genetic medicines such as gene therapy and genome editors”.

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Drug Discovery World

MARCH 21, 2023

CRISPR activation (CRISPRa) is a type of CRISPR gene editing that is used to overexpress certain genes. Although this technique is broadly used, predicting its efficiency when aimed at certain points in the genome can be challenging, making it hard to reliably overexpress certain genes.

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Drug Discovery World

DECEMBER 12, 2022

The announcement followed the signing of deals to make new medicines available across the country. . According to the publication, “A small clinical trial has shown that researchers can use CRISPR gene editing to alter immune cells so that they will recognise mutated proteins specific to a person’s tumours. Analysis .

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Drugs Gene Editing Genome Genomics 52

XTalks

MARCH 28, 2024

For CRC, treatment options that are targeting genetic mutations such as BRAF , KRAS and HER2 have been developed. Gene Editing and CAR T-Cell Therapy Genetic engineering technologies, such as CRISPR-Cas9, are providing newer potential avenues for cancer treatment.

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Gene Editing Life Science Clinical Trials Clinical Trials 59

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., 14, 2020 10:00 UTC. BEIJING & CAMBRIDGE, Mass.–( –( BUSINESS WIRE )– EdiGene, Inc. as Head of Business Development. “Our

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Business Development Development Gene Editing In-Vivo 40

Cloudbyz

MARCH 6, 2024

Personalized Medicine: Biologics offer the potential for personalized treatment approaches by taking into account individual variations in genetics, immune responses, and disease characteristics. Looking ahead, the future of pharmaceutical therapies is undeniably intertwined with the continued advancement of biologics.

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Gene Immune Response Manufacturing Gene Therapy 71

Drug Discovery World

JANUARY 12, 2023

The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. Altering one base pair can drastically change how the gene’s protein product functions, while mutations can lead to the development of a range of genetic disorders. . Limitations of organoids.

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Gene Editing Gene Drugs In-Vivo 98

XTalks

JULY 14, 2022

A downstream approach has traditionally been used by scientists to map networks, wherein a gene for a protein is removed one at a time and the impact on the immune cell’s function is observed. CRISPR-Cas9 gene editing was used to simultaneously perturb thousands of genes by targeting specific points of genetic code.

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Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

DECEMBER 15, 2022

Vor Bio, a clinical-stage cell and genome engineering company, has announced initial clinical data from VBP101, its Phase I/IIa multicentre, open-label, first-in-human study of tremtelectogene empogeditemcel or “trem-cel” (formerly VOR33) in patients with acute myeloid leukemia (AML). . Official comments .

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Drugs Genome Genomics Engineer 52

Delveinsight

FEBRUARY 25, 2021

Beam Therapeutics has taken over a startup whose technology could proffer the biotech’s genetic medicines to more tissues in the body, widening the potential to approach more diseases. Cambridge, Massachusetts-based Beam aims to make more precise edits with genetic medicines, which employ base-editing.

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Drug Discovery World

APRIL 14, 2023

Carl H June, MD, FAACR – Lifetime Achievement in Cancer Research June, a Fellow of the AACR Academy, is the Richard W Vague Professor in Immunotherapy, Director of the Center for Cellular Immunotherapies, and Director of the Parker Institute for Cancer Immunotherapy in the Perelman School of Medicine at the University of Pennsylvania.

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Genetics Research Medicine Clinical Research 52

The Pharma Data

SEPTEMBER 1, 2021

With a goal of developing rheumatoid arthritis therapies with minimal side effects, researchers at Washington University School of Medicine in St. Louis have genetically engineered cells that, when implanted in mice, will deliver a biologic drug in response to inflammation. ” The new findings are published online Sept.

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Drugs Engineer Drug Delivery Gene 52

The Pharma Data

NOVEMBER 3, 2020

Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. LogicBio Therapeutics is dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LEXINGTON, Mass.,

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Clinical Trials Clinical Trials Gene Editing Genome 52

Drug Discovery World

OCTOBER 16, 2023

Indeed, this is bolstered by top tier universities like Newcastle University, which specialises in structure-based approaches for drug discovery, developing innovative technologies that support the identification of new medicines, and is well-established in cancer drug discovery. Government investment of £1.3

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Drugs Life Science Gene Therapy Genome 73

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Gene Therapy Gene Production Clinical Trials 52

pharmaphorum

FEBRUARY 3, 2021

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

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XTalks

FEBRUARY 11, 2021

The Human Genome Project recently marked 20 years since the publication of the first full sets of human genomic sequences, an endeavor that spanned well over a decade. Today, new next-generation sequencing technologies allow for the sequencing of complex genomes within just a day or two. Rosalind Franklin.

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Genetics DNA Genome Genomics 119

Roots Analysis

FEBRUARY 15, 2022

Presently, there are several companies and universities, which are exploring the potential of different gene editing technologies beyond CRISPR for basic research, and the development of gene editing solutions. Genome Editing is a way of making changes in the DNA.

Genome 52

Genome Genomics Gene Editing DNA 52

XTalks

DECEMBER 20, 2023

CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason. Given its ability to precisely edit genes, there is tremendous promise for the revolutionary technology to offer cures for genetic disorders and to treat various diseases.

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The Pharma Data

JANUARY 10, 2021

11, 2021 (GLOBE NEWSWIRE) — Editas Medicine, Inc. 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.,

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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.

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The Pharma Data

JANUARY 19, 2021

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. In bacteria with unaltered tracr-L, levels of CRISPR-related genes were low. The authors found that tracr-L redirects Cas9 in S.

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Bacteria Gene Editing Research RNA 52

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. These efforts, along with many others, are opening doors for new possibilities in translational medicine.

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Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. In addition, iPSC lines, despite their pluripotency, have skewed suitability towards certain cell types 12,13.

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Gene Editing Gene Engineer DNA 52