Scienmag

JANUARY 12, 2021

Martins Scientists have used gene-editing advances to achieve a tenfold increase in the production of super-bug targeting formicamycin antibiotics. Credit: Dr Dino J. Discovered within the last ten years, formicamycins have great […].

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Gene Editing Gene Bacteria Scientist 53

Worldwide Clinical Trials

OCTOBER 24, 2023

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. Roughly 80% of rare diseases are due to a known genetic driver. The conference was sponsored by several rare disease drug developers, with dozens more in attendance.

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Gene Gene Editing Gene Therapy Clinical Trials 130

Scienmag

NOVEMBER 19, 2020

Media contacts: Jennifer Kuzma, jkuzma@ncsu.edu Khara Grieger, kdgriege@ncsu.edu Mick Kulikowski, News Services, 919.218.5937 or mick_kulikowski@ncsu.edu Researchers at North Carolina State University call for a coalition of biotech industry, government and non-government organizations, trade organizations, and academic experts to work together to (..)

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Gene Editing Gene Research Production 44

pharmaphorum

JUNE 28, 2021

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.

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In-Vivo Gene Editing Trials Drugs 105

pharmaphorum

MAY 7, 2021

Gene editing firm CRISPR Therapeutics has announced a collaboration with US biotech Nkarta to develop natural killer (NK) cell therapies for cancer. It’s at the forefront of gene editing although the technology has spawned rivals including Intellia Therapeutics, Caribou Biosciences, Sangamo Therapeutics and Mammoth Biosciences.

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Gene Editing Gene Genetic Engineering DNA 52

pharmaphorum

OCTOBER 21, 2020

There is currently no treatment approved for RP patients independently of their genetic background. The financing will support manufacturing of a first clinical batch of the product, regulatory activities and a first human study scheduled to start next year.

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Gene Therapy Gene Gene Editing Genetics 98

XTalks

MAY 22, 2023

Pairwise , a startup based in Durham, North Carolina, has launched its first CRISPR-developed product in the US: Conscious Greens. Touted as a mix of superfood leafy greens, the product claims to offer twice the nutrition of traditional romaine lettuce and comes with an appealing fresh flavor.

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Branding Gene Editing DNA Production 98

pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting. billion product if it gets approved for both indications.

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Gene Editing Gene In-Vivo Genetics 52

Pharmaceutical Technology

MARCH 2, 2023

The research using ImmunoGen’s technology will enable the discovery of the agents for use with gene editing. ImmunoGen has announced an agreement granting rights to Vertex Pharmaceuticals to carry out research using antibody-drug conjugate (ADC) technology for the discovery of new targeted conditioning agents.

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Gene Editing Gene Development Research 130

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.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

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

The Pharma Data

NOVEMBER 22, 2020

Scientists in Israel have used the CRISPR Cas-9 gene editing system to destroy cancerous cells in mice without damaging other cells. Peer went onto say that he believes that, in the near future, there will be many personalized treatments based on genetic messengers. Photo courtesy of Science Advances. ” Source link.

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Gene Editing DNA Genomics Genome 52

WCG Clinical

NOVEMBER 17, 2023

Although the NIH Guidelines were originally written with non-clinical laboratory research in mind, they also apply to human gene transfer (HGT) research, wherein rsNA or rsNA-containing products are administered to research participants. Since then, however, certain genetic engineering technologies (e.g.,

Genomics 52

Genomics Genome Gene Editing Genetic Engineering 52

pharmaphorum

APRIL 20, 2022

The potential to develop treatments with CRISPR gene editing is hinged on its ability to add, delete or replace elements within a target DNA sequence. Cancer is a genetic disease, caused by certain changes in the way that genes control cell function, such as how they grow and divide. Why cancer? The pipeline.

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Gene Editing DNA Gene Genetics 52

Delveinsight

JANUARY 14, 2021

Both companies retain full rights to their product candidates and discuss the potential path forward for any future combination studies based on the outcome of the Phase 2 trial. Eligible members will get an integrated product experience, comprising the Livongo for Diabetes program, Dexcom CGM technology, and CGM-powered insights at no cost.

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Gene Editing Gene Genetics DNA 40

XTalks

NOVEMBER 3, 2023

CRISPR works as genetic scissors to edit parts of the genome. The CRISPR-Cas9 gene editing system was first discovered to be endogenous in bacteria. Given the system’s powerful ability to make cuts in genes in a precise manner, the system is being leveraged in gene therapies.

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Gene Therapy Gene Gene Editing In-Vivo 59

The Pharma Data

DECEMBER 13, 2020

“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Dr. Zhang completed his postdoctoral fellowship at Harvard Medical School/Boston Children’s Hospital.

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Business Development Development Gene Editing In-Vivo 40

The Pharma Data

FEBRUARY 22, 2021

Gene editing investments advance agriculture with improved food quality, higher yield, and improved crop nutrition. Gene editing is the key to better solutions and greater choices for farmers and consumers around the world.

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Gene Editing Genomics Genome Protein 40

pharmaphorum

SEPTEMBER 14, 2022

The French biotech said the cash injection – which follows a €45 million Series A in 2020 – will also help it bring forward the first development candidate in a collaboration signed with gene-editing specialist Intellia Therapeutics last year, currently in the early-discovery stage.

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Gene Therapy Gene Gene Sequencing Gene Editing 52

Advarra

NOVEMBER 29, 2022

Recent Problems in Cell and Gene Therapy Development. Dealing with confusing regulatory guidelines is one of the top obstacles researchers face as they develop CGT products 1. In the last two years, there haven’t been any CGT product approvals, primarily because of the COVID-19 pandemic.

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Gene Therapy Gene Production Clinical Trials 52

pharmaphorum

NOVEMBER 2, 2021

Though their bispecific nature complicates large-scale production and purification workflows, with challenges such as antibody chain mispairing, bsAbs have come a long way since first developed. This lack of complexity also promotes simpler production workflows, offering the advantage of reduced costs and high yields.

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Antibody Immune Response Production Engineer 52

The Pharma Data

MARCH 13, 2021

Three biotech trait projects advance to launch phase / Advanced eight new small molecule crop protection actives / Leading soybean pipeline to offer the greatest weed control flexibility, premium genetics. Delivering an industry-leading R&D pipeline in scale, productivity, and value.

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Production Development Gene Editing Marketing 40

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. The FDA’s Fast Track designation is a process designed to expedite or facilitate the review of product candidates to treat serious conditions and fill an unmet medical need.

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Clinical Trials Clinical Trials Gene Editing Genomics 40

Camargo

JULY 27, 2021

It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy. Additionally, gene editing allows us either to remove or to modify harmful genes.

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Gene Therapy Gene Gene Editing Genetics 238

Pharmaceutical Technology

FEBRUARY 13, 2023

Gene therapies are an attractive way of targeting the underlying genetic mutations, but traditional approaches cannot be used while targeting mutations for Dravet Syndrome, forcing researchers to develop new ways. Meanwhile, others are researching the link between gene variation and different responses to treatments.

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Research Gene Gene Therapy Genomics 264

Pharmaceutical Technology

AUGUST 24, 2022

A lead product candidate of the company, GTP-506 is a single-dose gene editing therapy. The ARCUS nuclease vector (GTP-506A) targets gene editing in the PCSK9 gene locus that is well characterised, while the therapeutic donor vector (GTP-506D) introduces the OTC gene to provide the required genetic correction.

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Gene Editing Gene Therapy Gene Genetics 130

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.

Gene 99

Gene Immune Response Manufacturing Gene Therapy 99

XTalks

DECEMBER 13, 2023

While Lyfgenia delivers a functional copy of hemoglobin A to replace the abnormal one in hematopoietic (blood) stem cells via a lentiviral vector, Casgevy edits the HBB gene using CRISPR technology to increase the production of fetal hemoglobin in blood stem cells. Both therapies will be available in early 2024.

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Gene Therapy Gene FDA Approval In-Vivo 98

XTalks

JANUARY 4, 2024

Hemophilia B, also known as congenital Factor IX (FIX) deficiency or Christmas disease, is a rare genetic bleeding disorder caused by a deficiency or defect in the FIX clotting protein. The gene therapy was shown to reduce treatment burden, offering persistent production of FIX after a single infusion​​.

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Gene Therapy Gene Gene Editing Sales 111

Pharmaceutical Technology

MAY 26, 2023

Enzymatic DNA production company Touchlight have augmented its DNA production capabilities with a newly announced expansion to its London facilities. Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines.

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DNA Manufacturing Gene Gene Therapy 130

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Bluebird’s method “uses a virus to drop an entire gene encoding a new hemoglobin. SATURDAY, Dec. Source link.

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Gene Therapy Gene Gene Editing Genetics 52

The Pharma Data

JANUARY 10, 2021

EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. Previously, the Company received Rare Pediatric Disease designation from the FDA for EDIT-301. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing.

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The Pharma Data

JANUARY 17, 2021

–( BUSINESS WIRE )– Genus plc (LSE: GNS), a global biotechnology leader in animal genetics, today reported continued strong performance across its operations for the six-months period ending December 31, 2020. 18, 2021 07:00 UTC. BASINGSTOKE, England & MADISON, Wis.–( and £49.0m

Containment 52

Containment Genetics Gene Editing Production 52

XTalks

SEPTEMBER 4, 2024

Compared to current treatments, vutrisiran’s focus on reducing abnormal protein production could really improve patient outcomes. The HELIOS-B study’s findings are timely as new gene-editing therapies, like the one being studied in MedStar Health’s Phase III MAGNITUDE trial for ATTR-CM, are also being explored.

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Trials RNA Protein Gene Editing 52

The Pharma Data

NOVEMBER 24, 2020

–( BUSINESS WIRE )– Genus plc (LSE: GNS), a global biotechnology leader in animal genetics, today reported continued strong performance across its operations for the period from July 1, 2020 through November 24, 2020, coinciding with its Annual General Meeting today at 11:00 am GMT. 25, 2020 07:00 UTC.

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Genetics Containment Sales Production 40

XTalks

NOVEMBER 30, 2023

More Investigational Therapies for HIV Are on the Way American Gene Technologies, a pioneering biotech company headquartered in Rockville, Maryland, has released promising results from its Phase I trial of AGT103-T, a gene therapy designed for individuals with chronic HIV disease.

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XTalks

DECEMBER 29, 2022

In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light. Given their precise targeting, gene therapies have largely been focused on oncology indications followed by rare genetic diseases.

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Life Science Gene Therapy Gene Manufacturing 52